Many companies with investigational drugs have posted an expanded access policy on their websites as required, but more than a few still have this task left over from the 2018 to-do list. As the time for New Year’s resolutions and new to-do lists is upon us, those companies, as well as those that already have policies available on-line, may want to start thinking about how they will handle the newest requirements that Commissioner Gottlieb has described as part of a new program coming in 2019.
In November 2018, FDA released the results of an independent assessment that evaluated the expanded access program at FDA and made recommendations for improvement. In reporting on the results of the assessment, Commissioner Gottlieb announced a number of program modifications that either had already begun or would begin soon at the Agency to streamline the process and to recognize companies’ concerns about how adverse events related to expanded access might complicate the approval process.
Recent statements about the coming program signal that FDA is now planning to become more involved in expanded access with the goals of facilitating and expediting access. As reported by BioCentury, under the new initiative, FDA staff will provide information by telephone to physicians and patients seeking expanded access. FDA staff also will complete forms for single-patient IND requests and send the completed forms to the physician for signature and then forward the request to the sponsor.
Particularly noteworthy is that, as reported by BioCentury, although companies will still have the discretion to deny requests for expanded access, under the new initiative, they will have to provide a reason for the denial. It remains to be seen how comfortable it will be for companies to maintain a policy against granting expanded access when faced with FDA’s queries even in the absence of a regulation requiring a response. But perhaps that’s the point. While FDA cannot require that investigational drugs be provided on a compassionate basis, the Agency is well aware of sponsors’ predisposition to maintain positive relationships with their FDA review team.
It was also reported that FDA will follow-up with the physician or patient who receives an investigational drug to obtain information about the outcome. The potential for poor outcomes in the expanded access setting and the potential for those outcomes to adversely affect the approval process has been cited historically by sponsors who have shied away from providing expanded access. FDA has been working to convey the message that providing expanded access is unlikely to lead to such a negative result. The Agency, however, has not and, under the Food, Drug, and Cosmetic Act, cannot assure sponsors that such a result is impossible.
Robert Temple, M.D., who was recently appointed to the new position of senior advisor within the Center of Drug Evaluation and Research, Office of New Drugs’ Immediate Office, and Peter Marks, M.D., Director of the Center for Biologics Evaluation and Research, addressed sponsors’ hesitation to make investigational products available through expanded access at the Reagan-Udall Foundation for the Food and Drug Administration program on using real-world evidence from expanded access protocols in November. Both officials emphasized their belief that sponsors should not fear that adverse events occurring in the context of expanded access use will negatively affect the prospects for approval of an investigational product. Dr. Marks referred to reports of negative effects as “urban lore,” and Dr. Temple noted that FDA understands that patients receiving treatment under expanded access are likely to be sicker and have multiple comorbidities, among other factors. Dr. Marks acknowledged that the expense and complications of manufacturing cell and gene therapies currently make providing these investigational products available through expanded access more difficult, but predicted that costs may decrease in the future making these therapies more accessible.
Although it has not yet been formally announced, as reported by BioCentury, FDA will be having one or more public meetings about the new initiative and plans to launch a pilot program for cancer drugs in 2019.