Advocates Seek to Elevate FDA Patient Access Program

FDA needs to expand and empower its Office of Health & Constituent Affairs (OHCA) and its Patient Liaison Program to better coordinate interactions with individuals and physicians seeking expedited access to experimental therapies. Elevating OHCA within FDA’s Office of the Commissioner would enhance its ability to coordinate access programs among the agency’s diverse Centers regulating drugs, biologics and medical devices and improve the agency’s processes for vetting compassionate use requests.

OHCA currently is “buried” in FDA’s Office of External Affairs, which handles a range of communications and information operations, observed consultant Diana Dorman, formerly vice president of the National Organization for Rare Disorders (NORD). She proposed at the July conference on expanded access sponsored by CBI that OHCA be placed on an equal footing with FDA’s Offices of Women’s Health and Minority Health, which report directly to the Commissioner. A more visible position would enhance its ability to manage patient requests for expanded access to medicines and better position it to provide more education for physicians and institutional review boards (IRBs) on FDA early access procedures. OHCA also needs more resources to perform these functions, and to effectively identify and recruit knowledgeable patient advocates to serve on FDA advisory committees and other review panels. Patient organizations are looking to allot a portion of industry user fees to provide added support for OHCA operations as part of negotiations for reauthorizing the fee programs in 2017.

Patient advocacy organizations acknowledge that OHCA approves almost all expanded access requests filed by physicians, but continue to press for initiatives to make the application process more manageable for doctors, patients and IRBs. OHCA has been working to simplify its form 3926 for physicians to submit access requests to FDA, but Richard Klein, director of OHCA’s patient liaison program, explained how complex it is for the agency to implement such a change.

Early access requests need to be balanced with clinical research programs, Klein noted, and an individual’s needs have to be weighed against the benefits to society of bringing a new drug to market. Advocates understand that sponsors often have difficulty accommodating early access requests, especially small firms with limited clinical supplies or problems enrolling subjects in cynical trials. More information on these issues would help patients understand the process, speaking to the need for better systems at FDA and biopharma companies for explaining access policies and programs to individuals.

Linking access to development

Steven Walker, co-founder of the Abigail Alliance, proposed that expanded access should be built into drug development and delivery systems in ways to help sponsors “get a faster ride through FDA.” He views functional, responsible access programs as useful in supporting drug development and believes that patients who obtain treatment outside of a clinical trials should be helpful in providing important quality-of-life information to support shorter, faster, smarter, cheaper clinical trials.

Similarly, a main goal of industry is to improve drug development and the clinical research process so that all patients can gain access to safe and effective drugs as expeditiously as possible, noted Kay Holcombe of the Biotechnology Industry Organization (BIO). But sponsors are concerned that expanded access may reduce incentives to enroll in clinical trials. Companies also fear that serious adverse events could stop or slow a development program, making it important for FDA to consider such reports in the context of the severity of an individual’s illness.

Early clinical data often appears more promising than it turns out to be, resulting in patient disappointment, Holcombe observed. And issues of fairness are inherent in situations where the sponsor cannot accommodate all expanded access requests. Emerging companies with no approved products, moreover, face very different issues than large firms, Holcombe explained, particularly where manufacturing capability is limited and can compromise equitable product distribution.

Most large biopharma companies have well-established policies for making unapproved products available through expanded access, Holcombe added, but still decide each request on a case-by-case basis, considering the individual’s medical situation and available data. Sponsors often have to say no to some, yes to others, and “it’s not fair to just give a drug to the first ten guys in the door,” she notes.  She also sees an “acute problem” in using social media to put pressure on companies to make experimental medicines available. But this use of the Internet will only increase, she predicts, and may become “really overwhelming.”

“Right-to-try” state legislation is “not the answer” to helping ill individuals gain access to experimental therapies, said Dorman. Richard Plotkin, vice chairman of the MaxCure Foundation, agreed that such laws give “false hope” to patients. Yet he and others noted that these local efforts reflect widespread patient dissatisfaction with the current clinical research system, which puts very sick individuals at risk of receiving a placebo in a randomized trial. To deflect right-to-try mandates, the advocates advise pharmaceutical companies to make expanded access policies and programs more visible and to provide a central point of contact for all access requests, actions that would be required by provisions in the “21^st Century Cures” legislation moving through Congress.