”Perhaps the questions we are asking are not the questions that are important to patients.”
Matthew Galsky, MD, Associate Professor Medicine, Mt. Sinai, in ASH Clinical News
Why we all – patients, drug companies, physicians — need a patient advocate on MPN clinical trials.
Now that Dr. Margaret Warner has entered the MPN Patient Advocate program and progress has been made toward placing her in a pilot MPN clinical trial, the end of a long journey is in sight. We’re not there yet, but we’re closing in.
The old top down model of drug development is crumbling. For science and for profit, drug companies would develop targeted therapies, secure FDA approval for clinical trials and recruit trial sites, investigators and patients to participate. There was virtually no patient input into this system until the final step. Now with recruitment difficulties, with growing awareness among patients and physicians of pitfalls in clinical trial, and with recent highly public failed and faulty trials, the old model is evolving into a patient-centered system.
The FDA officially noted this direction as early as 2007 when it partnered with Duke University to form the Clinical Trials Transformation Initiative (CTTI). According to the FDA: “The initiative was generated from the realization that the clinical trials system in the United States has been suffering as a result of increasingly longer study start-up times, slowing enrollment of patients into trials, increasing clinical trial costs, and declining investigator interest in participating in clinical trials.”
Six years later an earthquake shook MPN clinical trials as the much anticipated Sanofi international JAKARTA clinical trial of SAR302503 (Fedratinib) was nearing its conclusion. Suddenly, only weeks before a much anticipated FDA filing for approval, the company aborted the trial. Inexplicable patient injuries and death, widespread ignorance of adverse events on the trial and the sudden expulsion of MF patients on the trial without recourse or compensation electrified the MPN community.
MPN patients, caregivers, physicians and friends united behind the Zebra Coalition in November, 2013 to petition the FDA Commissioner to permit a Patient Advocate to participate in future trials. A high ranking panel of FDA department heads gave MPNforum and the Zebra Coalition — appearing on behalf of those who signed the petition — the green light to proceed,.
The clinical trial climate has changed in the new age of patient advocacy.
Today, for example, Sanofi is leading a Patient Advocate effort through its PAIRS program and Celgene’s Patient Partners program has a highly aggressive recruitment effort to get patient advocates involved in clinical trials.
These and many other industry efforts are aimed at involving Patients both before and after clinical trial. The MPN Patient Advocate program is designed to place a Patient Advocate within the trial site itself, available through e-mail, phone, or Skype to patients and investigators alike during the full course of the trial.
Entering a clinical trial as patients we’re normally sick, out of established therapeutic options and both worried and hopeful. Around us is a sea of administrators and white coats taking notes, giving directions. We need an advocate, a fellow patient available to answer questions, communicate problem issues to the site medical team, assist in non-medical concerns like travel and economics, and provide a backup safety system.
Drug companies and medical institutions need an MPN Patient Advocate to encourage patient enrollment in a trial and to assist in compliance with study protocols as well as offload some of the non-medical work from the site team.
In the arrival of Dr. Margaret Warner, an MPN patient and physician, we have our first candidate. The process of developing a cadre of MPN Patient Advocates, training. certifying, and fully defining the role lies ahead. Together, we can do it.
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