FDA’s Proposed Regulations to Expand Access to Investigational Drugs for Treatment Use:

March 2009

64 Food and Drug Law Journal 183-224 (2009).

On December 14, 2006, the Food and Drug Administration (FDA) proposed two new regulations in the Federal Register amending current regulations governing expanded access to investigational drugs for treatment use and charging for investigational drugs.

The proposals come at a time when FDA has found itself under new pressure to provide seriously ill patients with early access to investigational drugs outside the framework of clinical trials. In recent years, patient advocacy groups have filed citizen petitions with FDA asking the agency to provide specific criteria to patients and sponsors seeking expanded access or to create an early approval mechanism to permit easier access to investigational therapies. Further, FDA has seen proposed federal legislation intended to ensure early patient access to investigational treatments and nearly lost a lawsuit in federal court in which terminally ill patients sought a fundamental right of access to investigational therapies under the Due Process Clause of the Constitution.

The proposed rules seek to assuage patient activists, physicians, drug sponsors and other critics who contend that FDA must strike an appropriate balance between allowing patient access to promising treatments while protecting against undue risk and safeguarding the clinical trials process. Although FDA heralded the announcement of the rules as a key step forward to improving patient access, the proposal does not expand access beyond measures currently available under longstanding agency practice and, in fact, creates new regulatory barriers and disincentives to industry participation in expanded access programs.
This article examines the proposal in light of historical agency regulation and recent pressures to expand access. Section II describes the historical development of FDA’s

statutory authority to regulate drugs and the traditional new drug approval process. Section III describes the various methods through which FDA has allowed expanded access to investigational treatments since 1962. Section IV recounts various recent pressures on FDA to reform its expanded access procedures and describes the context in which FDA’s recent proposal arises. Section V examines the changes proposed in both the proposed rules to expand access to investigational treatment and charging for investigational drugs. Section VI evaluates the rules and argues that the proposal will fail to expand access for patients because new restrictions on charging provide no incentive for industry participation and the proposed regulations create increased regulatory barriers to access inconsistent with FDA’s statutory mandate.


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