Author Archives: nashpatientsclinicaltrialexchange

Dina Halegoua-Demarzio, MD (Investigator)

I wrote quite long post about my experience in clinical trial with Dina Halegoua-Demarzio. And if you read it you may say maybe it is one off maybe she got her guard down in Covid or something like this. How is doctor with such angelic face can do all these horrible things? She works tirelessly presenting about fatty liver and NASH on local TV, she has 5 star Google reviews, etc. I will present you with one fact first that could leave no doubt about her intentions because her signature is on both documents. I will start with a simple example: So lets say you bought car for $10,000 and financed it with your bank. Bank would not let you drive that car out of dealer lot unless certificate of insurance is presented, bank needs to protect that $10,000 loan. Common practice that happens million times every day. Now look at page 21 of amended informed consent Dina Halegoua-Demarzio approved and signed (bottom of this post and initial consent was prepared long before Covid). It mentions sponsor, Galmed research and development, carrying insurance to pay for damages to participants occurred in clinical trial she was conducting.  Now look at letter stating her response to PA attorney general about clinical trial insurance.  It is 100% clear she is stating her contract with Galmed requires Galmed to maintain clinical trial insurance but she does not know if such insurance actually exists. IF THIS DOES NOT TELL YOU VALUE SHE PUTS ON LIFE AND HEALTH  OF PEOPLE SHE RECRUITING FOR HER TRIALS I DON’T KNOW WHAT WILL. Value of guinea pig’s life is clearly zero. She has staff, she could direct one of number of people working for her to get this information from Galmed, but she did not. If you don’t like the bank protecting their investment analogy how about this? She signed Informed Consent, she is responsible for everything in it including insurance. Now she is just pointing fingers. And further she is simply lying by directing me to get this information from Galmed. She knows I asked and got nowhere and this is the reason  I filed complaint with AG. But why is it so hard to obtain insurance information from Galmed? Because it does not exists in my opinion. You will read further why.  So why approve informed consent to give prospective trial participant sense of security? I am aware FDA sometimes asks for marketing materials to determine if clinical trial participants  were not tricked in to sign up for trial.  I am guessing this is common practice and having this insurance was one of the reasons I signed up for her trail with potentially dangerous investigational drug. If this is not proof of lack of care, professionalism on behalf of Dina Halegoua-Demarzio, read no further. You clearly someone she is looking for her trials. She actually told me her trial participants cant even count pills possibly implying they are so dumb

My Investigator Dr. Dina Halegoua-Demarzio works in big not for profit research hospital. While it is unlikely to find for profit research hospital it is important to consider investigator’s employer. I will write about Jefferson in separate post.

Investigator is medical doctor hired by Sponsor (Galmed) to conduct clinical trial.

what investigator is supposed to do

The most important thing to understand from my blog, clinical trial is about research and not about treatment. The biggest mistake you will make if you think investigator is your doctor. In my case I was referred to investigator by my doctor and  when Dr. Halegoua had no further use for me she sent me back to my doctor.

Dr. Halegoua has 10-15 years of experience as a medical doctor, it is highly unusual that she is in charge of a hepatology department at a major hospital. Doctors heading such department will have decades of experience.

I blame Dr.  Halegoua-Demarzio for this painful experience I incurred at her hands. Anyone who considers her as a doctor should read this.

Dr. Halegoua has only 5 star reviews posted about her in all review sites, according to reviewers she saves their lives, she is best doctor ever. They are not saying what exactly she did for them. Liver transplant?  There is no treatment for NASH. Did she tell them to lose weight? Take Vitamin E? She does have a pleasant disposition, does that qualify her as a good doctor? Is  this reason for 5 star reviews?

I suggest to ignore all these 5 star reviews. Everything I say has proof. It really takes “special” person to do what she did to me.

But she has no remorse and it is worth noting that she is still recruiting participants for Galmed trial. These are the current trials she is conducting:

Regenerate study NCT02548351, Navigate study NCT04365868, Voyage study NCT04173065, NCT04399538, ARMOR Study NCT04104321. Possibly she will get new study from Galmed for new drug called Amilo-5mer.

Clearly she thinks what Galmed did to me is perfectly acceptable and she wants to continue to do business with Galmed. Clinical trials are business. I don’t know how Jefferson operates but independent clinical research companies have business development departments.

My informed consent starts with wording to ask as many questions as I want. I took it at face value and asked lots of questions.

  1. I asked Dr. Halegoua when Galmed Phase 2 Aramchol results will be published on clinicaltrials.gov.  She assured me it will be published.  Unless FDA approves Aramchol as marketable drug it is highly unlikely Galmed will publish results. As of today Galmed is over 2 years late.
  2. I asked Dr. Halegoua how FDA knows that results submitted are real. She said she does not know. She should know.
  3. I asked Dr. Halegoua what will happen with trial if another covid lock down order issued. She assured me Galmed will come up with plan.  They did in fact come up with such a plan, just not for me
  4. Dr. Halegoua’s last email to me before my appointment to start on investigational drug was a promise to take care of me and leave worrying to her.  She only took care of herself in this trial.

When I showed up for my first trial appointment, person who runs gastroenterology and hepatology research department at Jefferson, Cindi Miller, RN was yelling her lungs out at me for sending so many emails, asking so many questions. She has 35 year career at Jefferson. She must think she owns the place and can abuse patients because she is the one bringing clinical trial money to Jefferson. She was screaming at me that her department people including Investigator lives do not revolve around me. (I never marked any emails as urgent).  She then proceeded  with crazy accusations that I should not be listening to Galmed earning calls because somehow me registering to listen to a call will unblind me to Galmed.  I am assigned patient number in clinical trial, my name is concealed.  Several of my questions to investigator were from listening to Galmed calls. I urge anyone who is participating in clinical trials listen to calls and talk to companies.  I talked to other NASH sponsors after I was removed from trial, no one saw it as a problem. Why would Cindi Miller wanted to keep me in the dark? Ms. Miller also told me that she will remove me from trial if I don’t stop my behavior.   That patient abuse appointment was in front of Dr. Halegoua-Demarzio. She never said single word to Ms. Miller to stop this abuse.  There is a record of my very high blood pressure at Jefferson after that incident. 

I started with the trial on that day and continued with all my trial appointments while COVID positivity rate was about 3%. As 6-month trial visit approached positivity rate was at 15%. I asked Dr.  Halegoua-Demarzio if I can have COVID accommodations as outlined in FDA document. Or what some sponsors did to allow flexibility during covid. She said she will check with Galmed and subsequently wrote me letter on 1/19/2021 stating that Galmed would not allow it. And day before I was supposed to tell her if I was coming in for trial visit, she sent me email informing me she removed me from the trial using “various” wording of Informed Consent. Basically Informed Consent I signed allowed removal from trial without any reason.

While I was negotiating with her in good faith to have my 6 months visit according to FDA guidelines this is what she wrote to me on 1/8/21
“ I am so sorry that this is causing you so much stress. It is upsetting to us as well. I am checking with our IRB on best approach. I wish I could get you the vaccine but do not have that power”.
So she knew about my stress and my hypertension and Jefferson was giving vaccines left and right. While I will never know why she threw me out on 1/20/2021 is it possible she was just coordinating with Jefferson legal department, IRB and Galmed to throw me out?

She refused to discuss dismissal with me. She refused to have end of trial visit with me as outlined in Informed Consent.  Basically she had no need for me anymore so she threw me out without any explanation.

I am presuming she signed FDA Form 1572 that clearly states in comments portion that she has the power to make changes in protocol to protect safety of participants. She refused to make changes according to FDA recommendations.

Is Dr. Halegoua-Demarzio what her Google reviews portray her?

  • “She is the most kind, caring, thorough, thoughtful, amazingly skilled Doctor. She takes the time to address all of your concerns.”
  • “Dr. Halegoua is the best. She saved my life. Very understanding, a professional. And listens to her patient first hand.”

Or is she someone who tricks patients to take potentially life threatening investigational drug and then throws them out when she no longer needs them?  I have proof of what happened, so I think latter.

Letter that she sent to me on 1/19/2021 also had unproven allegations that I repeatedly asked research department staff if I was on placebo.  This allegation simply shows what she thinks of trial participants intelligence.  To accuse me that I don’t understand what randomized double blind trial is simply crazy. The only trial that is acceptable for NDA application to FDA is randomized double blind trial I was on. I certainly did keep track of my tests, but to accuse me that she could possibly know if I was on placebo is simply insane. It defeats entire purpose of trial. Only a handful of people at Galmed knew if I was on real drug. I asked her to remove such baseless accusation from her letter or at least provide basis for such accusation.  She refused but she also acknowledged that  letter was sent to others. I have no idea to whom she sent this letter. But I find it interesting that other investigators running NASH clinical trials would not respond to my emails.  That is highly unusual. Getting email from person willing to take investigational drug and stating I have qualification for trial you are running is a gift to investigator. Why would they refuse such a gift?

There is another important aspect to consider when deciding to participate in one of Dr. Halegoua  clinical trials. Galmed prepared informed consent and provided it to Dr. Halegoua , who probably approved it because her signature is there.  The only signature is there is hers and mine.  Informed consent, page 21 provides wording for clinical trial insurance to cover sponsor legal liability in respect of claims for injuries resulted from study drug or procedures required by study. I relied on this representation when deciding to enroll in clinical trial conducted by Dr. Halegoua.  I felt with Galmed being foreign corporation with no record in of any approved drugs having insurance was one of the reasons I signed up with Dr. Halegoua. When I asked Galmed to provide name and contact for such insurance company, Galmed did not respond.  I filed complaint with PA Attorney General who contacted Jefferson Hospital on my behalf. Jefferson replied that such trial insurance exists, but they don’t know name and I should contact Galmed for such information. When I told AG rep that I am unable to get such info from Galmed, I was told that Galmed is a foreign corporation and out of AG control. I asked my Congressman office to find out if clinical trial insurance is required in US.  FDA response:

“Following up on your constituent’s question, “Can you confirm with FDA that there is no federal law requiring any insurance for trial participants,” please see our response below. Adherence to the principles of good clinical practice (GCP), including human subject protection (HSP), is universally recognized as a critical requirement to the ethical conduct of research involving human subjects.  The Food and Drug Administration (FDA) regulations for the conduct of clinical trials, which have been in effect since the 1970s, address both GCP and HSP.

The FDA regulations do not require that a sponsor company carry insurance for clinical trials.  However, we are aware that some countries may require a sponsor to carry clinical trial insurance.

FDA’s informed consent regulations (21 CFR 50.25(a)(6)) do require that, for research involving more than minimal risk, the informed consent form provide an explanation as to whether any compensation and an explanation as to whether any medical treatments are available if injury occurs and, if so, what they consist of, or where further information may be obtained.  FDA expects investigators to ensure that subjects have access to reasonable medical care during their participation in the clinical investigation (see FDA’s Guidance for Industry Investigator Responsibilities –Protecting the Rights, Safety, and Welfare of Study Subjects).”

I think it is safe to assume that Galmed did not obtain such clinical trial insurance for patients recruited in US trial. I never saw it mentioned in any of informed consents I read from other sponsors or even in open label trial Galmed was conducting in different location. What would be implication of having non existing insurance for legal liability of sponsor in foreign country in informed consent Dr. Halegoua approved?  Did she want to give prospective participants comfort of insurance protection so more people would sign up? Or just plain ignorance on her part? Or maybe both? I have no idea, but what ever reason is for such unacceptable and incompetent behavior, do you want to trust your life to Dr. Halegoua  with investigation drug that may kill you or seriously damage you?  I would be very afraid of  Dr. Halegoua. Indication of how she approved informed consent may be indication how she conducts her clinical trials. 

And why she is agreeing to be investigator in trials that sends patient straight to dangerous biopsy without MRI assessments? Fibroscan I had before she decided to send me to biopsy is not reliable. From every trial I read on clinicaltrias.gov standard is Echosense fibroscan, mri, biopsy. Echosense is FDA cleared and not approved device. If you want to read about FDA clearance loophole read my FDA post. It may save your life. Did she want trial so badly that sponsor saving $5000 for MRI was more important than endangering prospective participant lives with invasive procedure? I do not know. Just asking questions.

There is another instance of how well Dr. Halegoua pays attention to study she is getting paid for. She released all study results to me after she reviewed them. She released biopsy results to me that had typo which stated incorrect fibrosis number. I pointed it to her in email and she had radiologist to correct typo. But this resulted in unnecessary stress to me as fibrosis number was dangerously high.  If you want to be in her trial be ready to do her job. Make sure you have expertise.

If you read her 1/19/21 letter to me (page 2)  she states about future promising drugs to treat NASH. As of 1/19/21 phase 3 trials for NASH failed to meet endpoints, stopped or NDA was rejected by FDA. Does she know that fibrosis is progressing disease leading cirrhosis and cancer? Did she read any studies about how long it takes to progress each stage of fibrosis? Or cirrhosis or cancer? Does she have any inside information that FDA will eventually approve NASH drug? I am guessing answer is no to all of the above. Or she just want to feed me lies knowing well enough I know that she is lying?

You are not going to read this in 5 star reviews about her. Everything I say about her is based on facts.   I have no idea why this sloppiness, possible negligence on part of Dr. Halegoua. Is it possible she just overworked? Dr. Halegoua shows pictures of herself with 2 small children.  May be her family obligations take priority over her clinical trial obligations to patients? May be writing research papers to have her name known take priority? There are could be many things I have no way of knowing.  But all of it should be consideration in enrolling in one of her trials including fact that research department run by Cindi Miller who exhibited herself in very unprofessional matter in my opinion. If after you read all this and ask Dr. Halegoua to tell you her side of the story, she may tell you nobody ever complained, my situation is unique, etc. My answer to this it took COVID 19 to reveal who Dr. Halegoua really is. A cruel person who only cares about getting rich and advancing her career at the expense of people like me.

What would be explanation for such life endangering investigator behavior? While I have no idea of her career goals, here are my thoughts.

In NASH field there are maybe 5 investigators that could be identified as Principal Investigators. Sometimes Principal Investigator can be investigator at a specific site. What I am instead referring to here is a Principal Investigator who has authority to make presentations for Sponsor. Dr. Halegoua is not one of these. These people have decades of experience in hepatology and extremely well compensated for this experience. They consult, lecture at all major pharma. They present at conferences, write articles in professional magazines. They sit on advisory boards of drug companies. They are key opinion leaders (KOL). They get equity ownership in drug company start ups. They have front row seat to see if drug works. While trials are double blind meaning neither investigator nor participant know if investigational drug participant takes is placebo or real drug, Principal Investigator has access to all data while regular investigator like mine will have access to data for the handful of participants that they have enrolled. My trial was supposed to enroll 2000 worldwide. As such a Principal Investigator is in a much better position to anticipate the end result of the trial. In no way am I suggesting that Principal Investigator knows who is on placebo.

A Principal Investigator can use this inside information to their advantage, taking ownership in the most promising stocks. When Allergan took over Tobira, Tobira stock increased 10 fold.   It is all legal and some investigators report stock ownership. I think it should be law to require all doctors disclose stock ownership. If your investigator sold stock in company you are in clinical trial, you may want to rethink your participation.  While I am not 100% sure I remember reading research hospitals are exempt from requirement for doctors to report stock ownership.  If this is correct you want to go with research hospital that overrides that exemption and requires doctors to report such stock ownership.  But this may explain why most investigators work in hospitals and not in for profit research companies where clinical trials business can bring 10 million annually.   Researchers employed by major hospitals can still get 250K salary and have all free support provided by hospital.  They bring lots of business to hospitals. This profession offers potentially great rewards and fame. I am guessing most investigators you encounter will strive to become someone who can participate in KOL events and reap all the benefits of this association.

Some doctors find themselves employed by biotech pharma and then start biotech companies, raise hundreds of millions in IPO (Initial Public Offering). Drug development takes decades while the company milks Wall Street for more money. Executives have pretty nice lifestyle at shareholder expense. Millions in compensation. And if drug actually works and approved, doctors can make billions.  Not bad career path for someone who is not even 40. And who is helping her along? You.  And what big biotech she already consults or sits on board? Gilead.  And which medical doctor led division at Gilead and now CEO of biotech company with 2  billion market cap? It is CEO of NGM pharma

Again I have no idea what her plans are but everything she done so far pointing to one or all I described.

It is never about you. You, the participant, are just a tool to provide data. And as long as you understand this, you should never believe anything related to general trial info investigator tells you. You should always consider the source.  I was told by someone on Dr. Halegoua’s staff how lucky I am to receive such good care from investigator and Galmed. What a lie it turned out to be. Read my Galmed post so you will see how well Galmed took care of me. I wish I never met Galmed or Halegoua or left Jefferson at the first sign of how they treat patients like garbage. And how can any Investigator become so well-known and so well compensated? By being loyal to sponsor because sponsors pays them and there are only handful of sponsors. NASH is epidemic and silent disease that can lead to liver failure and death. Any big hospital will have non ending supply of potential trial participants. All investigator staff needs is to look for elevated liver enzymes and few other things hospital already has to start qualifying someone for trial.

Does Dr. Halegoua-Demarzio want to become Principal Investigator? I have no idea. But she presented at liver conferences and publishes extensively in research  publications.  And where does she get data for her publications? From you. You are nothing more than a provider of data to investigators, sponsors, IRBs and FDA.

Dr. Dina Halegoua-Demarzio  linkedin profile https://www.linkedin.com/in/dina-halegoua-b780739b states she is member Biopharma Physicians group network possibly this https://www.bpng.org/  Just another example that she appears to be about advancing her career as top priority and may be if she was spending less time on networking and more time reading informed consents for her Guinea pigs, I would not be writing this blog. Check your doctor linkedin profile, will tell you a lot about him/her

Dr. Halegoua, There is a good chance you are reading this. I noticed when I post a negative review about you on doctor review sites, immediately another 5 star review shows up from a person who never reviews anything. You may want to refresh your memory, you did not take The Hippocratic Oath such long time ago. You show pictures of yourself  with 2 young sons. I know college is expensive but there is still way to make money and treat trial participants like human beings.  Nothing wrong with making as much money as possible as long as it is not done at expense of old sick person like myself. On 1/26/2021 Galmed CEO Baharaff said publicly that he is unblinding all Phase 3 double blind participants. While you threw me out from Phase 3 a few days prior, if you have any decency please ask Baharaff to tell me if I was on the real drug because whatever I was on made me sick.

Galmed Informed Consent

Dr. Halegoua-Demarzio Letter 1/19/2021

Dr. Halegoua-Demarzio Response to my complaint to PA Attorney General

Galmed

The most important part of my Galmed story is what transpired after they removed me from the ARMOR double blind Phase 3 Aramchol trial. Shortly thereafter the Phase 3 trial was suspended and Galmed CEO Baharaff stated that participants would be unblinded (notified whether they were receiving Aramchol or placebo). But having been already removed they would not unblind me in spite of my repeated pleas. This alone captures the essence of Galmed. Galmed is rotten to the core. If you are considering participation in such a trial understand that you are nothing more than a guinea pig and collateral damage. That said here is my Galmed story.

Trial starts with participant signing Informed Consent. FDA guidelines establish important but non-binding guidelines. Section 1.28 states “informing of all aspects relevant to the participant decision to participate in trial”. Informed Consent created by Sponsor (Galmed) approved by Institutional Review Board (IRB) omits several key points of information while misrepresenting others

  • Aramchol drug that was approved for Phase 3 study had patent expiring in 2023 and so would never be marketed. Instead Galmed’s intent was always to petition FDA to substitute with another similar drug called Meglumine with patent protection to 2035 without reverting back to Phase 2.
  • Informed Consent states “Clinical trial policy insurance to cover the Sponsor’s legal liability … for injuries that directly result from the study drug or procedures required by the study protocol will be maintained by the Sponsor”. In fact such insurance is not required in U.S. as it is in other countries. I asked for the name of the insurance provider and no name was provided. Informed Consent further states “If you have a research related injury reasonable and necessary medical care will be available to you. The cost of care … will be paid by the Sponsor of the study”. Since Sponsor is apparently not maintaining liability insurance in U.S. and Sponsor is located in Israel the chances of recovering such cost of care probably slim.

Had I been made aware of these things I would not have signed up for the trial.

In July 2020 I started on the trial and made all required trial protocol appointments. March 2020 FDA published document describing how to conduct clinical trials during COVID. Sponsors and investigators were given option to conduct video visits, to collect blood using local labs, and mailing study drugs. November 2020 earnings call Galmed gave investigators discretion in conducting the trial. January 25, 2021 scheduled visit I asked investigator to allow me such discretion. I expected that I would be receiving COVID vaccine shortly due to my age and co-morbidities. Investigator contacted Galmed who refused such. Interesting to note that investigator who is sitting on Galmed advisory board wrote this article. On January 20, 2021, one day before deadline to inform investigator whether or not I would show up, I was informed that my participation was being terminated. I was the only participant at this particular location (Jefferson Hospital in Philadelphia PA). Other locations throughout the U.S. were unable to recruit any participants due to strict Phase 3 requirements. Or possibly prospective participants were not so naive and wanted to have nothing to do with foreign corporation with no track record of having marketable drug or knew FDA had nothing to do with approving Phase 3 of double blind trial. Galmed expected 2000 participants worldwide and was only able to recruit about 50 (i was told about such small enrollment by investigator in another location). In fact the most difficult part of NASH trials is participant recruitment.

Having had to endure 6 months of pre-trial qualifications, painful and dangerous biopsy and other tests, another 6 months in trial taking investigational drug, multiple blood draws, only getting paid to cover parking and gas expenses. While I have no way of knowing if I was receiving the drug or just placebo, my results showed no improvement. While Galmed shared my test results with Halegoua who in turn shared them with me, I was told Sponsors test for significantly more things than they share with investigators. Such tests may include biomarkers such as proc-3, elf, and other sophisticated tests only research community knows about. Is it possible Galmed had tests that showed significant deterioration in my condition?

In December 2020 Galmed announced an open-label trial (NCT04104321) to run concurrently with the double-blind trial that I was participating in. All participants in the open-label trial would receive the actual drug. Galmed stated that all participants in the double-blind trial would be given the option of transferring to the open-label trial. I informed Investigator that I would like to exercise that option. Investigator informed Galmed and then I was removed from the trial. While I will never know reasons Galmed wanted me out of Phase 3 trial (expected to last 5 years), my suspicion is that they knew I am on the real drug and not placebo, and that the drug is ineffective in treating neither my NASH or fibrosis conditions. I pleaded with Galmed CEO Baharaff not to throw me out from trial I gave year of my life and did not even get a response. On 1/26/2021 Galmed presentation to investors, CEO Baharaff stated all Phase 3 participants will be unblinded. While I was kicked out of the trial on 1/20/2021 it seems like a decent thing to do for Galmed to inform me if I was taking the real drug. But they did not.

While I can only speculate that I was on real drug and Galmed wanted me out, at the same time I was kicked out from Phase 3 trial, Galmed prepared informed consent for open label trial participants. All accommodations I asked for now offered to new participants

“In case of circumstances directly related to the COVID-19 pandemic preventing holding the visits at the site, the study staff will make every effort for you to remain in the study. They may communicate with you to coordinate an alternative location for holding the visit. Dr. xxxxxx may also choose, for safety reasons related to the pandemic, to replace the face-to-face conversation during the visit, with a phone or video call. In such case you will be notified in advance and the call will be coordinated with you. Do not hesitate to contact the site for any related questions you may have”

This is part of email from Dr. xxxxxx (I am not releasing name because this doctor did not do anything bad to me unlike Dr.Halegoua-Demarzio who in my opinion is a monster for what she did to me). Answer to my question why conduct open label study “Regarding Galmed, I understand that the decision to lend open label access to drug is the incentive that if folks opt to enroll into clinical research during this time of COVID, then we need to minimize risk for receiving a placebo.  However, the study will open back up to randomization to placebo in the future, but render access to open label drug to those who elect to participate now.  This was strictly an effort to optimize recruitment during a very difficult and challenging time”  I am not buying this answer because I don’t think it is possible to get NDA (new drug application) approved with trial that is not randomized and double blind.  I am using this quote to show that Galmed was accommodating patients in Covid just not me.

I also noticed in open label informed consent something quite interesting. When I signed mine side effects were itching, now Galmed stating they had one person with lung blood clots and one with gallbladder inflammation.  And this is all from handful of people they signed up for double blind Phase 3 last year?

As far as my results? My blood sugar went a lot higher, my liver enzymes did not change, my bilirubin became dangerously high. And I developed shortness of breath and chest pains. Plus I am worried now about liver damage cause by investigational drug toxicity. And I am still unable to see doctors in person or have any tests. And when I finally get that COVID vaccine which Jefferson had ability to provide to me but did not, I expect my medical expenses from tests will cost me thousands. And who is supposed to pay for all this? Obviously me. So much for Galmed responsibility outlined in Informed Consent. Pretty good way to deal with such “responsibility” to throw participant out of the trial and pretend we never heard about you.  Here is safe investigational drug such as Aramchol that  Dr.Halegoua-Demarzio repeatedly told me as main reason she “selected” Galmed for her trials. And I told her about what happened to me,  her response was “I am not your doctor”, go where you came from to get treated. Not even decency to ask Galmed what exactly they fed me.  This is Dr.Halegoua-Demarzio with her angelic looking face and 5 star reviews (that cant be possibly be real if you read content) in action. And when I complained to WIRB about my symptoms I was called a liar. Here is organization that is supposed to protect trial participants and who pays for this “protection”? Galmed. And where do some WIRB executives work before they joined IRB? FDA. And this is full circle how sponsors, investigators, IRB and FDA all in it together to allow abuse of trial participants. (Circle actually completes with FDA executives move to become drug companies executives. Probably unlikely considering Galmed is foreign company. But there is a very good example how statins were approved in US https://www.fda.gov/media/110452/download. It is interesting that FDA sees this as success to approve such widely used medication that can do may horrible things to person with liver disease)

And as far as advising open label participants that the drug they will sign up taking may be substituted if FDA gives Galmed approval, not in new informed consent. I am guessing if Galmed wanted to keep them on original drug they would have said so.  But why do anything beyond FDA requirements? Interesting wording in new consent: “Clinically relevant information pirating to NASH or of this research will be communicated with you when we Dr. xxxxxxx get it” Is it a joke? Insult to trial participants? Any wording of mysterious liability insurance is gone from new Open Label Informed Consent.

If you are considering getting on one of their trials think about what they can do to you. I urge every prospective participant to consider my experience. I hope others will write about their experience in clinical trials.

More About Galmed

why it is important for investors to know about patent but not for trial participant risking his life for drug that will never be marketable drug? Because Galmed is public company listed on NASDAQ stock exchange in USA. Securities Exchange Commission (SEC) requires Galmed to disclose all important information to investors. Food & Drug Administration (FDA) does not require any such disclosure to trial participants.  Is it because trial participants second class citizens? I hope anyone reading this will post comments below. I think reason is rich people invest in stocks and participants of clinical trials according to statistics I read are mostly poor and uneducated. So why would anyone care about Guinea pigs? Is it even possible for Galmed CEO with annual compensation of $1,269,700, company that employs 19 people treat trial participants as human beings? In my opinion Allen Baharaff does not care.

I thought long and hard why Baharaff failed to show any humanity towards me, unpaid volunteer who trusted him for 66% chance I will get aramchol which was promised to me as safest NASH experimental drug. What would be Baharaff getting out of it if aramchol was approved by FDA? As CEO who owns 20% of Galmed, Baharaff may get few hundred million dollars.  But Baharaff has no respect for people who endanger their lives to make him that obscene amount of money. He treated me like garbage and here are my thoughts why it is not possible for Baharaff to act like human being expected to act. As I read about him, Baharaff and his wife Roni Gilat-Baharaff part of Tel Aviv super rich elite. Articles written how he is fighting liver disease but in in my opinion the only thing he is fighting is his bottom line.  I doubt Baharaff can understand that ordinary people like myself actually have feelings and care that I was possibly hurt by his investigational drug. She is managing director of famous Christie auction house, he is on board of art museum and member of Israel bar association. He is elite snob and you just Guinea pig and he does  not give a damn about you. 

Galmed filed annual report around the same time Informed Consent for Phase 3 trial was prepared.

Page 29

“The composition of matter patents directed to Aramchol expired on March 25, 2019 worldwide. We will not be able to submit an NDA seeking approval of Aramchol (free acid) prior to the composition of matter patents’ expiration date. However, because Aramchol is regarded as a new chemical entity, or NCE, following approval of an NDA, if we are the first applicant to obtain NDA approval, we may be entitled to up to five years of patent term extension in the United States with respect to such NCE, and provided that the use patent with respect to Aramchol in the treatment of fatty liver will still be in force when the approval of the NDA is received from the FDA. The non-extended patent term for such use patent, is due to expire on April 15, 2022 worldwide and on April 17, 2021 in Israel. The U.S. patent was extended by a patent term adjustment of 567 days, resulting in an effective expiration date in the U.S. of November 3, 2023. In addition, a term of data exclusivity of up to 5 years will be available for the first approved clinical use of this NCE in the U.S., if Aramchol receives regulatory approval. Although we believe that we may be able to protect our exclusivity in our field of activity through such use patent portfolio and such period of exclusivity, the lack of composition of matter patent protection may diminish our ability to maintain a proprietary position for its intended uses of Aramchol. Moreover, we cannot be certain that we will be the first applicant to obtain an FDA approval for any indication of Aramchol and we cannot be certain that we will be entitled to NCE exclusivity.”

Galmed talked about uncertainty of continuing with new compound Aramchol salts with investors but not with trial participants. Even if FDA grants galmed right to substitute aramchol acid with aramchol salt in phase 3 trial, would you as trial participant want to know if aramchol salt went thru the same phases as aramchol acid?  Will it be in informed consent? I doubt it.

“Our potential development of Aramchol salts may not result in improved bioavailability compared to the existing form of Aramchol. Furthermore, although we have submitted patent applications for our Aramchol salts in development, there is no assurance that we will receive any patents for them, and even if we receive one or more patents for our Aramchol salts in development, they may be of little or no commercial value.

As part of our research and development studies, we have confirmed that several Aramchol salts have improved solubility as compared to the existing form of Aramchol acid. In 2014, we submitted new patent applications to protect such salts and we will need to conduct an appropriate bioequivalence study, or studies, of the biological equivalence of two proprietary preparations of a drug.

If we commence animal PK studies and formulation development in order to test the bioavailability of the Aramchol salt compounds, the results might not support the claims sought by us. Success in our earlier pre-formulation studies does not ensure that later studies will be successful, and the results of later studies may not replicate the results of our prior pre-formation studies. Furthermore, either or both of the animal PK and formulation development studies may fail to demonstrate that the Aramchol salts result in an improvement in solubility and bioavailability. Any such failure may cause us to abandon the Aramchol salt compounds and may delay development of other product candidates. If the animal PK studies do not support our claims, the completion of development of such potential product candidates may be significantly delayed or abandoned, which will significantly impair our ability to generate revenues and will materially adversely affect our results of operations.

There can be no assurance that the U.S. Patent and Trademark Office, or the USPTO, will issue any patents based on the patent applications that we submitted to protect our Aramchol salts, nor, should the USPTO issue any patents to us with respect to the Aramchol salts, that we will be provided with adequate protection against potentially competitive products. Furthermore, if the USPTO issues us one or more patents for the Aramchol salts, there can be no assurance that the issued patents will be of any commercial value, or that private parties or competitors will not successfully challenge these patents or circumvent these patents in the United States or their counterparts abroad. In the absence of adequate patent protection, our business may be adversely affected by competitors who develop “comparable technology or products”.

When a company in S.E.C. filing talks about materially adverse effect, what they say to investors we are unlikely to remain as a business entity and our stock will become worthless. What Informed Consent should be saying is that the trial will be stopped if such materially adverse effect takes place.

New patent was given to Galmed on  12/1/2020 long after I signed informed consent. Galmed initially filed for patent in 2014 and said above there is no guarantee US Patent office will grant such application. Why trial participants not entitled to know this? Because Galmed will do the absolute minimum that is required by law. I saw other biotech companies clearly showing patent expiration on their website. This kind of disclosure shows respect for patients and investors.

Even if patent granted it is still unknown if FDA will allow Glamed to substitute Aramchol acid with Aramchol salt. Aramchol Meglumine is salt version of Aramchol and considered to be NCE (New Chemical Entity) and was granted patent.

http://patft.uspto.gov/netacgi/nph-Parser?Sect1=PTO2&Sect2=HITOFF&p=1&u=%2Fnetahtml%2FPTO%2Fsearch-bool.html&r=1&f=G&l=50&co1=AND&d=PTXT&s1=10849911&OS=10849911&RS=10849911

And if you don’t want to read company reports provided to investors here is clinicaltrials.gov, primary source to find trials

https://www.clinicaltrials.gov/ct2/show/NCT04104321?term=galmed&cond=nash&draw=2&rank=1#contacts. Double blind study expected to complete in 2024 after Galmed lost or will lose patent protection. While Galmed does not refer here if participants will receive patent losing Aramchol acid or unapproved Armachol salt with patent to 2035, how you person considering trial will know what you are going to be on for duration of the trial expected to complete in 2027. If you go on assumption FDA will do right thing and do not allow substitution unless Aramchol salt is safe to substitute, I wish you luck.  By definition there is no such thing as safe investigational drug. Right thing for patients  for FDA to do is to require Galmed to go back to Phase 2 with Aramchol salts with the same number of people as initial Phase 2 people (247) and don’t allow Galmed jump line because it will cost Galmed lots of money. FDA has responsibility to give 2000 future Phase 3 double blind trial  priority over Galmed money and schedule to get to NDA.

And lastly note Galmed added open label trial to Phase 3 double blind trial I was kicked out from with no explanation. If you considering it ask some independent party what is the purpose of doing this? How would FDA actually know at the end of the trial if participant got better by itself or on Aramchol? This is why double blind placebo controlled trials are done. The only sense I got out of one researcher about open label trials, they done for very sick group of patients.  Expanding trial to participants with F1 fibrosis (less sick than Galmed double blind trial) makes no sense.

I had many conversations with investigators at different locations in US.

  1. When you hear person breathing heavily and says ” to be honest it is been very difficult trial ….”. it tells you there are some people where honesty comes before Galmed money
  • Investigator with many trials including Galmed told me in email “I am not fan of Galmed trial”

You may want to call investigators who participated in Phase 2 and not doing open label or Phase 3 double blind or open label and ask them why not. May be they tell you more than standard “difficult to recruit”.

FDA

In this entire ordeal, FDA surprised me the most. It is understandable Galmed will do what is best for Galmed and Galmed stock price. Investigator employed by Jefferson hospital has contract with Galmed and probably was told by Galmed not to provide any COVID accommodations for me. Investigator needs stream of sponsors such as Galmed to generate profit for her employer.  FDA is expected to protect clinical trial participants but it turns out that FDA allows all this abuse. Besides usual reason such as pharma industry lobby, I think answer is quite simple. FDA employs over 15k and has budget of more than 3 billion.  For that kind of taxpayer provided money FDA expected to produce results, meaning approve as many drugs as possible to benefit population. Why would FDA protect clinical trial participants if there so few of them and yet millions will benefit from their participation? Considering that clinical trial participants are volunteers that get few pennies thrown at them for expenses and considering that most who participate are poor and uneducated, FDA thinking probably they deserve everything they get for being so stupid.

So below is collection of this madness I discovered.

It was a huge surprise to me to learn that sponsors pretty much run entire show from time IND is approved until application for NDA is made. There is no such thing as permission from FDA to proceed from Phase 1 to Phase2 or Phase2 to Phase3 of clinical trial. There are thousands of patients involved in all phases of clinical trial and FDA is nowhere to be found in this process except negligible amount of audits.

So far everything I read that FDA recommends is non binding. This is why sponsors can get away with just about anything.

  • My main concern was always FDA audits. How does FDA know that results submitted for approval are real results? Results are submitted directly by sponsor.  While  I still waiting answer to this  question for over 6 months, this what I was told by FDA patient affair office
    • Q: what percent of clinical trials get audited? is this percent higher or lower when sponsor is overseas?
    • A: The FDA’s Office of Regulatory Affairs (ORA) conducts inspections of clinical trials through the Bioresearch Monitoring (BIMO Program) and may have information to answer these specific questions. The ORA’s web page is found at Office of Regulatory Affairs | FDA and includes information about how to contact the ORA. The FDA posts BIMO clinical trial inspection metrics by fiscal year on our web site at BIMO Inspection Metrics | FDA. The individual inspection metrics include a breakdown of both domestic and foreign inspections conducted that may be helpful to help address these particular questions.
  • It is unlikely I will ever get percentages, but if you enter recurring on clinicaltrials.gov you get over 50,000 trials, numbers in these links above go into few hundreds so I say chances to get audited are very slim.
  • As far as providing information for trial participant to make informed decision, this is what I got from patient affairs
    • Q: is providing patent expiration that is very near expiration in trial sponsor conducting should be part of informed consent?
    • A: The FDA informed consent regulations do not have a requirement to include sponsor patent information.
  • Every clinical trial listed on clinicaltrials.gov requires to answer if Data monitoring committee (DMC) is established.
  • But YOU AS TRIAL PARTICIPANT NOT ALLOWED TO KNOW IF THERE IS SUCH COMITTEE.
    From patient affairs:
    • Q: should providing DMC name be part of informed consent?
    • A: The FDA informed consent regulations do not have a requirement for including the name of a Data Monitoring Committee (DMC) if the sponsor has established a DMC for a study.

So let’s say you decided to enroll in study based on the assertion on clinicaltrials.gov that a Data Monitoring Committee is employed by Sponsor. DMC is authorized to stop the trial if some investigator reports serious adverse effect. But you have no way of knowing if DMC is actually employed by sponsor other than the unverified assertion. IRB, Investigator, Sponsor at best will respond that such information is confidential. Participant is at the mercy of the DMC (if it exists) or Investigator and Sponsor that SAE (Serious Adverse Effect that could be death), will give the participant welfare priority over the financial loss to Sponsor / Investigator.

And lastly another reply from patient affairs regarding liability insurance

  •  Q: is liability insurance sponsor claiming to hold in informed consent mandatory in US?
  • A: The FDA informed consent regulations do not have a requirement for including information on liability insurance, if held by the sponsor.
  • Q. I understand that sponsor can conduct clinical trial without IND (Investigational New Drug) approval. But how can participant of clinical trial obtain such IND?
  • A. Sponsors of IND applications may obtain advice and guidance from FDA at any stage of IND development. A sponsor may consult with the Agency before formal submission of an IND application and send a pre-IND meeting request (PDF – 145KB) to an appropriate Review Division responsible for overseeing products in the therapeutic area relevant to the IND application.

Please note that due to confidentiality laws, the FDA cannot divulge information related to applications that may or may not have been submitted to the Agency. All information on an unapproved drug product is confidential and belongs to the sponsor. Individual researchers may be able to provide you additional information on their specific study or studies, and information may also be available through clinicaltrials.gov

My interpretation: So good luck getting copy of that IND

So you may wonder FDA does not require anything, how it is possible?

  • My most painful discovery came from fact that FDA does not enforce it is own laws.  So how does a Sponsor induce Investigators to recommend their trial to a prospective participant? Sponsors publish results in professional magazines,  fly investigators to conferences where results presented.  But most sponsors will never publish results on clinicaltrials.gov. and when investigator promises you results will be published, it is most likely a lie to entice you in a trial. Here is http://fdaaa.trialstracker.net/ there is existing law passed in 2007 and not single fine imposed by FDA. If you interested in trial put sponsor name in search box and you will see how many years they are late in reporting results. Why this is dangerous for patients considering enrolling in clinical trials with non complying sponsors? I suggest you google everything that site founder Dr. Ben Goldacre wrote including FAQ on his site. Sponsors have no excuse for not reporting results, Dr. Goldacre explains it so well.  But if you are a sponsor and you know you never going to pay single dollar in fines, why spend few dollars preparing and reporting these results? It is a lot easier to prepare some marketing materials to distribute to investigators.   This is probably the only time you as patient have any leverage. If you decline to participate in a trial unless results are reported on clinicaltrials.gov, investigator will stop returning your emails, but if enough people will say it may be things will change. Or you can do homework and find ethical sponsor such as Pfizer.

Writing to your elected officials and asking for change as Dr. Goldacre recommends is waste of time.  How exactly your congressman or senator can force FDA to enforce existing laws?  It is all by design and your elected official has plenty of contributions from pharma lobby. In fact I was told by my senator office that they don’t get involve with FDA.

  • It is question you need to answer for yourself. Do I want to be in a trial with zero transparency that will possibly endanger my life and nobody is going to be there to treat me if I get sick from investigational drug or provide for my family if I die from it? The way things stand right now my answer is no.  You can try to ask for changes in informed consent to make it not totally one sided in favor of Sponsor or Investigator. You can  try to negotiate for more money for trial visits. Ask Investigator to show contract provided by Sponsor. How much money Investigator gets for pushing paperwork  around and telling you ½ truths while you endangering your life?
  • While I don’t have access to Dr. Dina Halegoua-Demarzio contract with Galmed I found contracts Galmed possibly signed 7 years ago with another research institution. Look towards bottom of link below. Stunning amount of money goes to investigators. At least 600k just to one institution for one trial https://www.lawinsider.com/contracts/6blm94lWrmJY2jxAhPBIXy/galmed-pharmaceuticals-ltd/clinical-trial-agreement/2015-03-31

If you ever want to get information out of FDA, you have to do FOIA requests and you have to pay for that. And any requests asking for information for unapproved drugs will get automatically denied.

If you ever file complaint with FDA don’t expect any response as far as resolution. Maybe you can do Freedom Of Information Act (FOIA) requests to see what they decided if anything. Most likely waste of time. FDA will invariably put Sponsor’s interest first and zero consideration for participant.

There are number of contact emails for FDA and someone needs manual to understand who does what.

Below few email addresses if you inclined to verify if it is really that bad.

What do I think about FDA role in clinical trials after I learned everything is to learn? It looks like most of things FDA does is honor system.  Ask the hundreds of patients who participated in Phase 3 Fibrogen trial how honor system turned out for them. As Phase 3 was completed right before approval of NDA, FDA asked for advisory committee. On 4/6/2021 the company admitted not reporting correct safety results to FDA. Where things can end up when someone know they can get away with it?

http://www.hcpro.com/HOM-36473-1303/Avoid-fraud-and-abuse-issues-in-connection-with-clinical-trials.html

Everything is based on very small percent of audits. And what exactly happens is audit fails? Especially in foreign country? Especially in country that does not extradite it citizens to US? Does FDA even reports offenders to foreign authorities?

So when I read story like this I am not even surprised.

There is no better example than 510 (K) loophole for medical devices that was enacted by congress to allow FDA to do what outlined in this video.  https://www.youtube.com/watch?v=-tIdzNlExrw or in this article https://www.greenlight.guru/blog/fda-clearance-approval-granted

FDA lists approved devices for each year https://www.fda.gov/medical-devices/recently-approved-devices/2021-device-approvals

Here is list for FDA cleared devices so you may want to know to how dangerous they could be.  https://www.fda.gov/medical-devices/510k-clearances/510k-devices-cleared-2021

FDA is nothing more than huge bureaucracy that owned by lobbyists. After everything I learn about clinical trials it feels like FDA is fully owned subsidiary of Citizens United, llc. Corporation are people according to former presidential candidate, looks like FDA protects these kind of people before real people. Sometimes FDA does right thing and asks for advisory panel such as panel that was created with covid vaccines. Or rejects Intercept drug that did very little in fibrosis reduction and had serious side effects. But overall it feels totally hopeless as far as FDA protecting trial participants. But no matter who you vote for that will never change. You the volunteer, and drug industry needs you for clinical trials a lot more than you need them.

Don’t even think complaining to IRB. They approve useless informed consent and sponsor pays them and FDA rules what suppose to be in informed consent are simply laughable. https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=50 and non binding

And how do you know purity of investigational drug? Ask your investigator, sponsor suppose to disclose purity. And if you happened to be on placebo (obviously you would not know what you on), FDA does not regulate contents of placebo. Don’t assume you could be on harmless sugar pill. https://www.motherjones.com/politics/2010/10/placebo-ingredients-drug-trials/ demand to know purity contents, and where it is made. My bottles showed manufacturing company with headquarters in Germany but didn’t say “made in Germany”. Company has facilities worldwide. I don’t want a drug manufactured in China. Their U.S. facility has glassdoor complaints about quality control. I am entitled to know where the drug is made.

I am victim with millions of Americans who took generic blood pressure medication manufactured in China with Chinese government requirement only to inspect factories by advanced appointment.  The result of this compliancy on behalf of FDA cancer causing compound https://www.webmd.com/hypertension-high-blood-pressure/valsartan-losatran-bp-med-recalls-2018-19

If you think my experience is unique because Galmed is small company following is list of email addresses I asked trial specific questions:

I got no help from them, whole system designed to send you to investigator who will peddle you stuff according to their interests not what is best trial for you. Patient advocacy? Does not exist. Any Google searches will point to websites created by drug companies with videos of happy trial participants (aka guinea pigs) promoting this multi billion dollar industry. And main partner in this is FDA which on paper looks great with all this documentation what Informed Consent should be but in reality Informed Consent Galmed created for me and approved by Investigator and IRB was designed to keep me in the dark.

I am not saying stay away from clinical trials. All I am advocating is for disclosure what I am risking my life for by taking investigational drug.  And for FDA to protect trial participants and protect integrity of data submitted to FDA and to enforce existing laws and to stop this bs of notifying foreign drug company/lab/drug manufacturing plant by email,  giving them advance warning we are showing up on such date and time.  If some foreign country by law requires FDA to do that, FDA has power to tell said country to keep drugs developed or manufactured in this country for their own citizens.

And if you read in informed consent that you have legal rights, that is fantasy. You are volunteer, you signed informed consent that 100% on side of sponsor, investigator and IRB. Consent is legal, you have no case if you decide to sue any of these 3 entities. Unless they breached the informed consent, you have nothing. Withholding material information is legal. One sided informed consent is legal. But even if you are able to prove breach of contract, good luck finding lawyer to represent you. Lawyers represent investigators, IRBs and sponsors. Representing you will be conflict of interest. I am not giving you legal advice, but time to look for one is before you sign that consent. I contacted numerous lawyers in order to attempt legal action. Most did not respond and those that did said they could not represent me due to conflict of interest.

In conclusion I am wondering what exactly taxpayers getting for this 3 billion annual budget.  Not much in my opinion except providing employment to huge bureaucracy. And fact that it is impossible to get straight answer from that bureaucracy must be by design. My senator office told me from outset they don’t get involved with FDA. With tiny amount of audits it looks like drug approval process is simply honor system. FDA does not enforce laws to fine sponsors for not disclosing trial results. Is not this gift to drug companies and grave danger to trial participants?  Fact that liability insurance not required for participants is gift to tort lawyers.  FDA clearance with 510k loophole is sad joke. Even if company wanted to get approval presumably by paying more for process FDA would not allow it.

Food? Dangerous metals in baby food tells you all you need to know.

Not inspecting Chinese plants that manufacture drugs? Gift to generic companies.

 And if you say is not this the way government agency supposed to work? Why this is different from 1.5 trillion wasted on failed f-35 jet? If FDA did not hurt you in any way I guess it is ok, but when human lives involved we US taxpayers deserve better FDA.

Jefferson Hospital

Don’t think that not for profit is some kind of charity. It is just a scheme not to pay taxes and get all kind of benefits as outlined in this article.

It is stunning  how much money Jefferson makes and how executives getting millions in compensation. Billions in revenue, at least 30 millions in executive compensation and yet treat patients like garbage. And speaking of garbage, what percent of that executive compensation Jefferson executives need to give up and pay city of Philadelphia for garbage pickup?  Or lessen tax burden on Philadelphia taxpayers?  This is not for profit for you. It is all about profit.

Clinical trials are source of income for Jefferson. And this is just from private companies. N.I.H (U.S. Taxpayer funding) is where money is. $151M according to Jefferson.

You probably will be in clinical trial in institution you have some experience with. And it is very important to consider your prior experience when deciding to participate.

My overall experience with Jefferson over 10 years with one exception is horrible.  CEO Klasko could not care less about patients subsidizing his 5 million plus salary (double average non-profit hospital CEO salary). This culture comes from the top, so if your experience prior to trial is bad it will likely continue during trial.

I had one doctor telling me that his residents (medical doctors right out of med school) practically walk on water and if I ever complain again about them no matter what they do, I will be asked to leave practice.

 I had experience of having complicated procedure and Jefferson did not disclose that resident will be doing it. I had very serious complications and had I known in advance would never have agreed to have it done.

I had one doctor in charge of entire specialty practice who admitted that he never reads research publications and he is only one who decides what is best for patient.  That incompetence on his part ended up with me getting totally wrong prescription.

And lies with billing are non ending. They tell you test is covered in overall procedure, just to find out they lied when you get huge bill. Test that would cost $60 in Quest, you get bill for $500. And I was told that test is part of surgical fee. Ultrasound that takes few minutes at doctors office is $1200. Why? Because Jefferson owns that ultrasound machine and not doctor who just runs practice out of Jefferson building. And if you walk few feet to Jefferson outpatient imaging the same sonogram is $100. And who owns the same Sonogram machine there? Jefferson.

Pain pill after surgery I told them I don’t want, was $37, they still charged insurance and insurance did not want to pay.

Jefferson practice performed test they did not have contract with insurance that allowed them to do that test, Insurance denied. I can always receive bill for thousands because when practice is not in network like that test, Jefferson is allowed to charge just about anything. It is not that uncommon when insurance prints on their explanation of benefits that you are not responsible for non-network charges. Do not ignore it. Bills will come and collections will follow. So should I expect Dr. Halegoua-Demarzio not to be part of that culture?

But not all hospitals are the same. There are hospitals and doctors who actually lots better than Jefferson hospital and Jefferson’s doctors. It is all about culture from the top.  Jefferson treated me like garbage over 10 years while getting thousands from my insurance why I should be surprised that Dr.  Halegoua-Demarzio treated me like garbage as well? Email me, I will tell you how to find better ones. Don’t look at review sites, unless people describe something specific.  But in general good doctors are very few and most I met retired. In general younger the doctor the more likely he/she going to think he/she is hot stuff. I guess when they spent ½ million dollars on medical school they feel they entitled to treat you like garbage.

Conclusion

You may want to ask how is it possible Dr. Halegoua-Demarzio has 5 star Google  reviews  and not a single person ever complained about her?  Most reviews say she listens to patients. She is not a psychiatrist as far as I know.  Most reviews are from people who never review anyone and some don’t even have real name. All reviews at Jefferson are controlled by Jefferson so I would completely ignore them. Answer lies to sample population she gets for her trials.  In Pennsylvania 69% of adult population are not college educated. People have a tendency to trust doctors. Prospective participants should understand that she is not their doctor. People tend to believe government, specifically FDA, is there to protect them.  People generally don’t have inclination to research and ask for second opinion. Most doctors you ask if you should be on clinical trial will say it more likely to hurt than benefit you. That what I was told.  Most prospective participants will have no idea that only very small percent (maybe less than 58%) of clinical trials succeed. Failure rate in NASH trials is much higher. Few prospective participants will research best trial for them, read business analysts reports, research publications. It is almost impossible for most.  And even then it may turn out that the best trial for you is thousands of miles away. Will you move? Obviously not. And then you have your friendly investigator peddling you her trial because company that is a leader in NASH trials with great results did not give her business. Is there a reason for getting mediocre trials?

And then investigator will try to scare you how bad your disease will get if you don’t get on a trial. Lets say you have stage 1 fibrosis. It takes 6-7 years progress to next stage.

By the time it gets really bad FDA may approve NASH drug. This is what I was told “The natural history of progression from F3 to F4 disease (based on results from prior Gilead simtuzimab studies) is approximately 20% by 30 months.   The risk of F4 disease progressing to a clinically significant clinical event is also approximately 19% over 30 months.” This is why I decided to stay away from trials and concentrate on losing weight because likelihood of benefitting from study is very small and chance to get hurt by it a lot greater.

And lets say you did all homework and decided to participate:

  1. Then you should consider finances of the company. It may be less of concern with big pharma. But huge concern with small biotech company especially with limited pipeline (drugs in development). Take Galmed, they did IPO may be 5-6 years ago and raised 70 million on Nasdaq. They continue to burn money and continue to raise more money on Nasdaq by issuing more stock. Last offering 2/21 they were able to raise 9 million.  This is nothing for company that needs to conduct phase 3 trial with expected enrollment of 2000.  I read average company expected to spend 20k on each participant in NASH trial.  More stock they issue more difficult is to raise money. It is real possibility company can run out of money and stop trial.  If you Google “biotech cash burn reporting websites”, you can find website to get free trial and estimate if company you are considering has enough money to complete trial.  That website just takes information from financial reports files with SEC. Bottom line if company can’t get money, trial will be stopped.  Do not even think to rely on investigator opinion. They have no clue.
  2. There is always wording in informed consent that allows investigator to remove you from trial for any reason. Or as they call it your best interest, which really means investigator or sponsor best interests.  It is unlikely investigator or sponsor will agree to put specific reasons you can be removed from trial. But remember they can throw you out at any time without any reason. Just make sure you are ok with it. From my experience it hurts like hell to be treated like piece of garbage.
  3. Look for wording promising you 5-7 years of free drug. This is just marketing ploy to sign you up.
  4. Make sure you have guarantee in writing that all tests investigator receives will be shared with you.
  5. Make sure you have health insurance. It is unlikely investigator will agree to cover your medical expenses if you get hurt from investigational drug. But process of going after sponsor should be outlined. Make sure you have everything including contact information. And verify it directly  with sponsor. Obviously with small company like Galmed you should absolutely have  health insurance.
  6. You need to make decision if you want to be in trial where sponsor is breaking law and not reporting  results on clinicaltrials.gov. If you decide to take risk with such company make sure you get all marketing materials from investigator and read business analysts reports to see what brokers told investors about success of specific drug. You should ignore all reports done by brokers that sell (underwrite) sponsor stock.
  7. You need to have clear understanding how FDA approves drugs. https://www.fda.gov/drugs/information-consumers-and-patients-drugs/fdas-drug-review-process-continued To summarize there is very little FDA involvement after IND is granted (which is unlikely you will ever see) to multiyear process that leads to NDA application. Where is FDA role in this process that takes years with thousands of people taking investigational drugs for any given trial? To the best of my understanding is in tiny amounts of audits I already documented in my FDA post. Ask your investigator who approved whatever clinical trial phase you considering to be on. And see how she/he will answer. You may be told you have nothing to worry, DMC is in charge. While you will unlikely to know if DMC actually exists for your trial, this article shows how many things can go wrong https://www.nejm.org/doi/full/10.1056/NEJMra1510066 In 3/18/21 earnings conference call Galmed CEO announced “I’m happy to inform you today that the addition of the open-label part to our ongoing Phase 3 was approved in the US, Canada, Australia, UK, France, Belgium, and Spain and is expected to be approved in the coming weeks in Israel, Korea, Turkey, Mexico and Chile”. If you know which entity approved open label please post in comments below
  8. You need to understand every word of informed consent which will be totality one sided in favor of sponsor and investigator. Fact that it was approved by IRB and ethics committee is useless. They are all in it together to take advantage of you
  9. You should ask Investigator about purity of investigational drug. Sponsor suppose to disclose it in Investigator handbook.  And you should ask question such as “what is half life of real drug I may be taking?” Knowing half life is important for many reason including participation in any other trials as long half life will preclude you from some trials.  If answer is “proprietary”  as pretty much what Cindi Miller was answer to almost every question I asked, tell investigator to take that drug and shove it in whatever part of his/her body you think is appropriate.  And investigators will know thru epic system or social networking about your prior trial participation
  10. You should look into using existing approved drugs off label to treat NASH. I started to look in Ozempic but side effects scared me.
  11. You should pay close attention how you are qualified for trial. Currently standard set by FDA is liver biopsy. Most likely you will have fibroscan, MRI and then liver biopsy. I did not have MRI and I think it is mistake and probably was trial design by Galmed who wanted to save money on testing. I was told that liver MRI in clinical trial can cost $5000, so if sponsor of your trial wants you to go straight for biopsy and bypass MRI, knowing what I know now I would look for another trail. You want to have as much non evasive testing as possible before you agree to have liver biopsy. This may include PRO-c3, ELF tests, new devices that cleared by FDA based on handful of participants. You may hear that you don’t need biopsy and your disease diagnosis is clear enough for you to start on trial. But at the end you will be taking investigational drug for disease you may not have. Biopsy considered gold standard and don’t let anyone talk you of it. In my opinion possible danger of biopsy is magnitude of factors less than “safe” investigational drug you will be recruited to take. There are white papers circulating in Hepatology journal written by investigators probably with push from sponsors trying to convince FDA that liver biopsy is not reliable way to qualify for trial. I suspect this is done because sponsors can not recruit and investigators can not get paid due to such strict requirements as biopsy. I doubt it is so hard to run study presented in hepatology journal to create results someone want. I did not read any specific article on this subject as they are not free. I urge you to not fall for this tactic if FDA caves in and allows less strict testing to qualify for study. You are qualifying for study to take potentially dangerous investigational drug. You need to make sure you really have disease you are told you have. Biopsy is gold standard and if you told you don’t need it you may want to look for other study. Liver regenerates. You want to have best diagnosis possible. And you want to make sure your slides examined by more than one pathologist to make sure they arrive to the same conclusion. Insurance usually pays for second opinion, it is not that expensive to send slides some place else to make sure you have diagnosis you told you have.
  12.  If you still need answers before or when in a trial I would advise not to look for message boards that suppose to help patients. I found one called smartpatients. Run by person stating to be former Google executive. It has moderator, totally unqualified person that most experience is in virtual handholding and no substance.  You would expect someone with resources previously working for Google will spend money to get someone qualified to help people to navigate thru clinical trials. When I brought it up I was promptly removed from the message board. Most people on smartpatients board with advanced cancer. But what struck me the most is one person asking if she should participate in some Phase 1 study because her potential investigator told her that investigational drug will kill her cancer. Not single person including myself told her that investigator has no way of knowing and Phase 1 is extremely dangerous, this is why it is phase 1.  But this shows how no one should believe investigators on efficacy and safety of investigation drugs unless they can show something written to support that statement. And then you should still consider who wrote it and why.

About ½ century ago clinical trials were conducted on prisoners.  Maybe best leave to that crowd.

They can always advertise here for professional guinea pigs here

You need to think long and hard why you want to get involve in trial.

Global pandemic, life threatening disease such as cancer is one thing. NASH, I am not sure risks are justified. And fact that everyone is against you is very important consideration.

Please look at results only and take them to a doctor that doesn’t do trials. And assess your risk. You life is not to make investigator and sponsor rich. Sponsor will make hundreds of millions with successful NASH drug.  Assuming you are not F4 which probably will disqualify you from most trials anyway you should try to lose considerable amount of weight and see if your liver enzymes got better. I know easier said that done, but I was able to do it once.  You should never ever forget you can die from investigational drug. Phase 2 is done on few hundred people. What kind of indication of safety is that? And that is assuming best case scenario. No biases in reporting results, etc.

If you still decide to participate in trial you have to be ok  with idea you are nothing more than Guinea pig, don’t expect anything out of investigator and sponsor. You can be thrown out at any time. You have nobody to complain to. You will hear “you were told everything in consent, you signed it“. You are unpaid volunteer.  You are to be used and thrown out when we no longer need you for whatever reason. 

I don’t think there is entity left that I did not complain, Jefferson CEO  Klasko, Jefferson President Webster, WCGIRB, Jefferson ethics committee, FDA, PA AG, senators, congressman, state representatives.  Not even a response. The most I got out of Jefferson. We are still going to treat you as a patient (meaning we want your insurance money), don’t think because we thrown you out from clinical trial it has any bearing on your continuing treatment (abuse in my experience) at Jefferson. Needless to say I left Jefferson entirely.

The only place I have left to complain about Dr.Halegoua-Demarzio is PA medical board. I am sure result will be no different from all my other complaints.

I decided to stay away from trials and let someone else to endanger their lives from taking investigational drug. There are 40 trials going on now, most phase 2. Which I would never do anyway. Most phase 3 failed so far.

NASH drug from Intercept probably will be approved this year. I don’t want this drug, NASH / fibrosis improvement is small, risks with increased LDL is huge. Madrigal drug looks promising but reducing fibrosis is not primary endpoint. Akero, NGM, Hanmi reporting impressive results but all are Phase 2. All of them injectable. Number of big pharma doing trial as well. So I will take my chances and wait for approved drug.  And I will read everything about that drug to decide if I should take it.

I understand new drugs need to be developed and they only way to develop them is to conduct clinical trial. But unless clinical trials conducted in transparent matter, there is liability insurance that sponsor must pay for,  Investigator employers are responsible for medical treatment of trial participants if there is even small connection to investigational drug, FDA audits all sponsors, all FDA guidance has  legal enforcement and trial participants actually get paid money that have some resemblance of what investigators, IRBs, DMC get and possibly some loyalty payments if drug is approved and becomes huge financial success, I no longer want to be taken advantage of. But how this dream above can become reality?  By Guinea pigs demanding to be treated like human beings. This is how change is made. You dont need them, you are a tool to make billions from approved drug.  They cant do it without you. Clinical trials participants union anyone? There are 100,000 of us at any given time. Lets change US from green to red.

There is big debate going on about paying clinical trial participants to participate in trials. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5728432/

I think it is just pretense to save money for sponsors.  Take Galmed, I got $50 for each blood draw visit and only if Jefferson actually able to get blood from me.  I actually had to come back at my own expense because Jefferson does not train nurses to take blood from people with difficult veins. So lets say Galmed would pay me 100 for visit or 200 or more for each visit. Paid for my parking. Would that be enough to make me risk my life for investigational drug?  There is no money that would make me to do that.  I decided participate for 67% chance of getting real drug.  Would it be incitement to some people? Probably. Did NIH/FDA/IRB ever do study what % of sick people do it for money? No and they never will because it is a lot easier to offer some idiotic logic in interests to save money to cheap skates like Galmed (that has no problem paying over million to ceo and ½ million to other executives). I saw informed consent of other nash sponsor and they paid 3x of money Galmed paying plus parking and hotel. And even with that 99% of intelligent people would not consider as enticement.