A Third Seat at the Table: An Insider's Perspective on Patient Representatives

As the nation takes a fresh look at all aspects of medical care, advances in the health sciences—from genetic sequencing to stem cell technology—may give us opportunities to make health care better, faster, and more cost-effective. But first we must transform our approach to health care innovation, particularly in the regulation of new products that may signal clinical breakthroughs.

These issues intersect at the table where federal agencies, particularly the Food and Drug Administration, and innovative product development companies negotiate the regulatory approval process. At present, both parties are overly constrained. The agencies face too many "but what if?" disincentives: potential and unknown safety risks and the specter of adverse publicity. The sponsors are wary of investing time and resources in the absence of a defined regulatory pathway. Too often, inaction seems the safest course.

But for the people most directly impacted by disease, inaction is irresponsible, even unethical. Patients and their families have the greatest stake in the approval process. Their lives, and their quality of life, hang in the balance. They are in the best position to weigh actual risks and benefits. It is time to give patients, through carefully selected representatives, a seat at the regulatory approval table. If they serve as mediators, not just advisers, they can help regulators and companies identify new pathways for fostering innovation, building public trust, cutting costs, and addressing quality-of-life issues.

Three decades of experience demonstrate that informed and dedicated patient representatives can break through development impasses. And since 2005, designated "patient advocates" on the governing board of the California Institute for Regenerative Medicine (CIRM), which I have served on for the last four years, have shown that patients are uniquely qualified and motivated to help decide how medical products should be delivered responsibly and expeditiously.

AIDS and the FDA Sea Change

The concept of the patient representative came to prominence in the 1980s AIDS epidemic, which galvanized patient communities to unprecedented levels of scientific and political involvement. In the San Francisco Bay Area, patient advocates organized their own studies of HIV/AIDS drugs with local doctors and volunteer subjects. They conferred with pharmaceutical manufacturers and exerted pressure on the FDA and the National Institutes of Health. Their efforts helped bring about a sea change in AIDS clinical trials and drug regulation. The FDA revamped its approval criteria, and community advisory boards began working with institutions that received NIH AIDS-related grants.

Starting in the 1990s, the FDA invited patient representatives to serve on advisory committees that review products for life-threatening diseases. But patient members typically occupy less than 10 percent of the slots on those committees, and they continually struggle to exert real influence on product decisions.

After the FDA approved the multiple sclerosis drug Tysabri in 2004, manufacturers withdrew it when some patients in clinical trials developed a rare brain disease. Prolonged efforts by MS patient groups helped bring about the 2006 FDA reintroduction of the drug and a new government-industry program to educate patients about risks. As John Richert of the National Multiple Sclerosis Society said, "We just have to learn … how to balance those risks and benefits for each individual person who needs to be treated for their MS."¹

In 2007, an FDA advisory committee studying the prostate cancer drug Provenge endorsed its safety by a vote of seventeen to zero and its efficacy by a vote of thirteen to four. But the agency felt that it needed more data on safety and efficacy. Additional clinical studies resulted in several years of delay before approval was granted this past April. While the FDA may have been exercising due diligence in asking for further study, the delay caused an outcry from the national cancer patient community and was a key factor in the bipartisan introduction of the Access, Compassion, Care, and Ethics for Seriously Ill Patients (ACCESS) Act, legislation that is still pending in Congress.

The CIRM Experience

There is a longstanding ethical argument that patients afflicted with illnesses should be able to participate in the search for treatments and cures. I have gained...