Senate Committee Promotes Additional Expanded Access To Investigational Drugs

Michael J Werner is a Partner at Holland and Knight's Washington, D.C. office

Today, the Senate Health, Education, Labor and Pensions Committee (HELP) passed legislation designed to provide patients more information about drugs that could be available under company expanded access programs – programs that allow patients to get access to unapproved drugs outside of the clinical trial process.

The Committee endorsed S. 934, the Enhanced Clinical Trial Design Act, considered along with legislation governing FDA user fees, that would require FDA to analyze patient access to investigational drugs under expanded access programs and develop appropriate guidance or regulations as needed. The Act also specifies when companies and distributors must publicize their expanded access policies. The bill is seen as an alternative to state-based "Right to Try" laws that attempt to allow patients access to investigational drugs without FDA involvement.

The 21st Century Cures Act, enacted last year, requires all biopharmaceutical companies and distributors with investigational drugs to diagnose, monitor, or treat serious disease to develop an expanded access policy and make that policy publicly available on the internet. The Cures Act also specifies the content of the policy to include:

• contact information for the manufacturer or distributor;
• procedures for making requests to obtain investigational drugs outside a clinical trial;
• the general criteria the manufacturer or distributor will use to evaluate such requests;
• the length of time the manufacturer or distributor anticipates will be necessary to acknowledge receipt of such requests; and
• a hyperlink or other reference to the clinical trial record containing information about the expanded access for such drug.

The new bill specifies that companies must develop and publish a policy the earlier of the first initiation of a phase 2 or phase 3 study with respect to the drug or 15 days after receiving a designation as a breakthrough therapy, fast track product, or regenerative advanced therapy.

The Act also calls on FDA to issue guidance on eligibility criteria for clinical trials. These guidance shall address ways manufacturers can: broaden eligibility criteria for clinical trials, especially with respect to drugs for the treatment of serious and life-threatening conditions or diseases for which there is an unmet medical need; and develop eligibility criteria for, and increase trial recruitment to, clinical trials so that enrollment in such trials more accurately reflects the patients most likely to receive the drug. It requires the FDA and NIH to convene a public meeting to inform the guidance. Moreover, FDA is required to report to Congress on patients' success in accessing expanded access programs under existing laws.

FDA is also required to issue new guidance or regulations, or revise existing guidance or regulations, to streamline the institutional review board review for individual pediatric and adult patient expanded access protocols.

Prospects for these provisions in the House is unclear though expanded access to investigational drugs remains a high priority for many in Congress. 

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