Expansion of Expanded Access? Federal Right to Try Legislation is Introduced

By James C. Shehan –

Over the past year, the plight of seriously ill patients seeking access to unapproved drugs has led five states to pass laws that permit manufacturers to provide patients in those states with investigational drugs on an emergency basis without going through the FDA’s expanded access process (see here and here). Now the public debate over this issue has led to the introduction of a bill, H.R.5805, in the House of Representatives. Taking a different approach than the state laws, H.R. 8505 would not bypass FDA but instead supplement FDA’s expanded access process with provisions requiring companies to provide FDA and patients with more information about how they deal with requests for expanded access.

H.R. 8505, sponsored by Representative Michael McCaul (R-TX), would create the Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act. The bill is named after Andrea Sloan, an Austin resident who died on New Year's Day after unsuccessfully seeking access to a potentially lifesaving drug to treat her ovarian cancer.

The bill has five major provisions. First, all sponsors of “covered breakthough drugs,” must file with FDA their expanded use policies. The policies must designate a single point of contact for expanded access requests, contain procedures for making requests, provide minimum criteria for the sponsor’s consideration or approval of such requests and provide the amount of time the sponsor anticipates is needed to make a decision on an expanded access request.

The bill defines a “covered breakthrough drug’ as those designated as breakthrough therapies, fast track products or qualified infectious disease products under FDC Act § 505E, those given accelerated approval under 506, and those for which sponsors have been awarded priority review vouchers under FDC Act §§ 524 and 529.

The second major provision requires sponsors who deny requests for expanded access to give written notice of the denial within five days to the patient or doctor making the request and to provide a written explanation of the reason for the denial.

A third major provision of the bill requires the Government Accountability Office to analyze the current expanded use program and within 180 days of enactment submit a report to Congress. This report must qualitatively analyze the extent to which individual patients currently have access to investigational drugs and make recommendations for improving such access. The report must also identify trends and patterns in expanded access requests and denials, identify barriers to drug sponsors granting requests, examine how FDA evaluates safety and efficacy data submitted in expanded access requests, detail how long it takes physicians, sponsors and FDA to make and process requests, provide expanded use request approval and denial statistics, make recommendations for improving regulations and policies, and specify the effect of adverse events from expanded access has on rug development.

H.R. 8505 also establishes a nine member Expanded Access Task Force assigned to provide recommendations for improving the expanded access program. Eight members of this task force, including the chair would be appointed by Congress and one by the Secretary of HHS. The Congressionally appointed members would include representatives of industry (2), patients (2), payors (2) and a bioethicist. This task force would submit a report to Congress within 180 days of being convened, which would occur after the GAO report is submitted.

The fifth major provision of the bill requires FDA to finalize its May 2013 draft expanded access guidance for industry. The final guidance must take into account the GAO and Expanded Access Task Force Reports and clarify how FDA interprets and uses expanded access adverse drug event data.

The bill does not preempt state right to try laws.

Prospects for passage of the Andrea Sloan CURE Act are unclear but quick action is not expected. In the press release accompanying the bill, Congressman McCaul stated that he plans to re-introduce the bill in the next Congress.