Science & Medicine

The MPN Patient Rights Petition

“Nothing about us without us”

Patients need to have a voice in Clinical Trials.

By the time we get to clinical trial we are usually sick and our other options have run out. We’re weak and often desperate and in no shape to demand our rights. That’s why we need our one guaranteed right, informed consent, to be carefully monitored based on developments in the clinical trial.

We need our interests represented by one of our own.  Conflicting stakeholders, however high their motives and deep their commitment, are not patients. The hundreds of millions of dollars at stake in clinical trial, the need to move a drug to market, the professional reputations on the line… all that has nothing to do with us.

The need to petition for patient rights in clinical trial became obvious  November 18, 2013 when many of the safeguards we believed to be in place failed us. It’s not that risk management plans aren’t in place, they are… on paper and sometimes in fact.   The combination of conflicting agendas,  mandated Federal secrecy, corporate self-interest, minimal oversight, and the absence of a patient voice in the assessment and management of risk  creates a potentially deadly environment for us.

We seek minimal human rights.  Informed consent requires us to know when our clinical trial risks are increased.  We need to know when Severe Adverse Events may place our health and lives in jeopardy.

What follows is the draft of a petition to Dr. Margaret Hamburg, Commissioner of the FDA, to be distributed and publicized at the end of this month.

Your review and comments in the next few days can help strengthen this petition.

The Zebra Coalition — We’re not just dinner.

The Rights of MPN Patient-Participants in Clinical Trial

There already is a current FDA guidance that patients, the most vulnerable and core stakeholders in Clinical Trial, be made part of a safety oversight Data Mining Committee.  In our primary demand, we ask that this advice be elevated to a requirement and the voice of patients be an integral part of safety monitoring.

In this petition  we specifically ask that:

1. A Patient Ombudsman, drawn from the population from which the research cohort is formed,  be appointed prior to commencement of any clinical trial and be co-equally informed with site Investigators of every Severe Adverse Event (SAE) occurring in the course of the trial whether attributed to the investigational drug under study or not.

The Patient Ombudsman, selected by an independent committee of Investigators, Institutional Review Board representatives and Patients, shall be fully informed and empowered to communicate directly with patient-participants and with Investigators on behalf of patient-participants.   The costs of employing the Patient Ombudsman and covering expenses shall be borne by the Sponsor.

2. Any Unanticipated Severe Adverse Effects (USAE) from any trial site shall trigger a trial-wide safety review with patients advised of new risk levels, if any, not specifically described in existing consent forms. 

3. In the event of early trial termination, the Sponsor shall provide compensation to patients for resultant out of pocket costs and independent physical examination and testing


The need:

Clinical trial of investigational new drugs are an essential part of the process through which vitally needed new medicines reach the market.  While rules assuring patient safety are in place through the FDA, the EHA and participating institutions, compliance varies.  Despite an FDA guidance, there is no required Patient-participant involvement in monitoring the conduct of a clinical trial. Patients and caregivers are routinely excluded from monitoring their own clinical trials.

Nowhere was the need for changes in current practices made more evident than in the abrupt and unanticipated termination of Sanofi’s Phase III trial of SAR302503,  Fedratinib, a myelofibrosis JAK inhibitor drug on November 18, 2013,  just days away from filing of its New Drug Application with the FDA for approval and release to the general patient population.

Most investigators and all participating patients were taken unawares.  Due to corporate policies and legal restrictions on the FDA, two full months after the trial ended there is still no publicly available information on deaths, the extent of injury, or the chain of discovery resulting in termination of this trial.  Patients, including some who were benefiting from the drug  were thrown on their own resources without notice and or medical guidance beyond advice to see their doctors.

The ensuing backlash threatens the clinical trial system itself.

It’s slowly dawning on us as patient-participants that we must agree to remain in the dark about clinical trial events affecting our health and mortality.  For example:  Early cases of Wernicke’s encephalopathy (WE), both recognized and unrecognized,  cropped up in the course of the Fedratinib clinical trial.  WE is a brain disease easily treated at early stages and deeply debilitating or fatal as it progresses.   One case was confirmed with MRI imaging in the Spring of 2013 and results published in Neurology, a peer-reviewed Journal in August, 2013.  Despite these unequivocal findings the Fedratinib trial continued and unsuspecting patients, unaware of the potential damage, continued to get dosed with a potentially deadly drug.

It’s possible, even likely, that this report from a prominent neurology journal was not ignored but simply missed by the  FDA,  IRBs,  hematologists, Safety Committees and Sponsor. We cannot know what Investigators or the Sponsor knew since Sanofi won’t say and the FDA claims it cannot under Federal regulations. But the referral of a clinical trial patient taking an experimental drug for a neurological work-up is precisely the event that would have been followed closely by a patient  representative, the Ombudsman.

Whether SAEs are trial drug-related or not cannot always be easily determined.  A pattern of SAEs does not always emerge clearly.  We cannot rely on Investigator and Sponsor recognition and certification of an SAE as drug-related before we learn of new risks in our Clinical Trial.

As patients in clinical trial, we need to know that all SAEs will be reported to us promptly and the risk assessment conveyed to us as quickly as possible by an independent patient representative. Only when we are fully informed can we grant our informed consent.

* Please strengthen this petition by publicly sharing your comments  on this draft with other patients, caregivers and healthcare professionals in the COMMENTS box below.  If you need to express issues privately, contact:


Comments on: "The MPN Patient Rights Petition" (4)

  1. Mike mccallum said:

    There must be a voice and opinions like Zhen in order to secure clinical trial patients safety in the future after this Jakarta trial farce.
    The drug companies have to be transparent and honest when dealing with people’s lives or patients will not bother to enter these trials no matter what.
    They should also be brought to task by some appointed authority that can judge them when they try to hoodwink consultants with results and stop a trial abruptly without any aftercare whatsoever.
    Although they claim to spend millions on trials they also make a substantial profit when successful.
    Well done Zhen and the panel, keep up the good work.

  2. Woody Woodruff said:

    Remember, clinical trials are by definition explorations into the unknown. If they had given up on bone marrow transplants in the early days, when far too many patients died, they might not be an option today. So I’m not sure how a patent ombudsman, knowing about an adverse event, could form a reliable opinion on whether the trial was too risky or not. Some gene-based therapies provide benefits to only a small portion of the patient population, but if you’re one of those few, and the drug is your only option, adverse events among other patients are simply not relevant. I know all adverse events are reported to trial sponsors, and I do think they have an ethical obligation to stop the trial if they see a pattern of severe adverse events.

    • Woody, thank you. I agree with both your reservations about the effectiveness of a patient ombudsman to determine risk levels and the necessity of proceeding with clinical trials that are inherently risky. The described Patient Ombudsman function is that of information and communications, something sadly lacking in the JAKARTA trial. Shouldn’t we be concerned that two months after JAKARTA was terminated we have still haven’t learned the number of SAEs, the death and injuries incurred and the reason multiple Wernicke encephalopathy SAEs didn’t amount to a pattern until November 18?

      We do have reports from patients whose WE-like symptoms were ignored. A patient ombudsman would have helped. We had a confirmed Wernicke encephalopathy at the Mayo Clinic this Spring, an MF patient on clinical trial, with MRI results published in Neurology in August.

      At the least, throughout JAKARTA, there was a failure to recognize the danger, a failure to take prompt action after confirmation, and a failure of oversight. Patients had no idea of increased risk. A patient ombudsman who would act as a conduit between patients and trial investigators might have put all these incident reports together and, with direct patient contact, have helped identify this risk earlier

  3. A lot of thought has been put into this – thanks Zhen for watching our backs.

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