“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
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: firstname.lastname@example.org.