Accelerated Approval of Cancer Drugs: Improved Access to Therapeutic Breakthroughs or Early Release of Unsafe and Ineffective Drugs?

Abstract

AA oncology NMEs are safe and effective, although development times are not accelerated. A return to endorsing phase II trial designs for AA for oncology NMEs, particularly for orphan drug indications, may facilitate timely FDA approval of novel cancer drugs.

Reevaluating the Accelerated Approval Process for Oncology Drugs

“…Some have criticized the FDA as being lax in their oversight of postmarketing commitments; others have voiced concern that the FDA is making accelerated approval increasingly difficult to obtain (2)(3)(4)(5). Two events in particular intensified this concern.…”

Applying Oncology Formulary and Benefit Design Innovations to the Management of Multiple Myeloma in the Managed Care Setting

“…11 For some tumor types, response of the surrogate marker is predictive of an OS benefit-an outcome subsequently confirmed on further investigation-but this is not always the case. 12 In multiple myeloma trials, complete response is often the targeted endpoint; however, trial data may not be sufficiently robust to definitively indicate that the directional change in complete response is predictive of prolonged OS. 13 This lack of confidence in trial endpoints creates challenges when designing the pharmacy benefit and for therapeutic decision making.…”

Critical Stages in the Development of the First Targeted, Injectable Molecular-Genetic Medicine for Cancer

“…Despite the enlightened intentions of the U.S. FDA Accelerated Approval program to shorten the development times of promising new drugs for serious medical illness -that is, to grant accelerated approval for new molecular entities on the basis of compelling Phase II trial data, followed by confirmatory post-approval trials -there has been a discernable reversion, in recent years, for the U.S. FDA to restrict Phase II efficacy endpoints and to encourage sponsors to design accelerated approval applications on the basis of interim analyses of protracted Phase III trials (Richey et al, 2009). Moreover, a number of adverse i n s t a n c e s i n t h e d r u g a p p r o v a l p r o c e s s h a v e r a i s e d l e g i t i m a t e c o n c e r n s b y t h e F D A Oncology Drugs Advisory Committee (ODAC), which has essentially created new hurdles, for both clinically effective and ineffective agents alike, and has increased the focus on postmarketing studies (Goozner, 2010(Goozner, , 2011.…”