Access to Investigational Drugs: FDA Expanded Access Programs or “Right‐to‐Try” Legislation?

Abstract

PURPOSE The Food and Drug Administration Expanded Access (EA) program and “Right-to-Try” legislation aim to provide seriously ill patients who have no other comparable treatment options to gain access to investigational drugs and biological agents. Physicians and institutions need to understand these programs to respond to questions and requests for access. METHODS FDA EA programs and state and federal legislative efforts to provide investigational products to patients by circumventing FDA regulations were summarized and compared. RESULTS The FDA EA program includes Single Patient IND (SP-IND), Emergency SP-IND, Intermediate Sized Population IND, and Treatment IND. Approval rates for all categories exceed 99%. Approval requires FDA and Institutional Review Board (IRB) approval, and cooperation of the pharmaceutical partner is essential. “Right-to-Try” legislation bypasses some of these steps, but provides no regulatory or safety oversight. CONCLUSION The FDA EA program is a reasonable option for patients for whom all other therapeutic interventions have failed. The SP-IND not only provides patient access to new drugs, but also maintains a balance between immediacy and necessary patient protection. Rather than circumventing existing FDA regulations through proposed legislation, it seems more judicious to provide the knowledge and means to meet the EA requirements.

Chimpanzee adenoviral vectors as vaccines for outbreak pathogens

“…This would require fulfilment of certain conditions including that no comparable or satisfactory therapy is available, that the risk of harm from the vaccine is not greater than the risk of disease and that there is sufficient evidence of the safety and effectiveness of the product to support its use in the given circumstances. 46 In this context, a vaccine for an outbreak pathogen, based on a well-developed platform, such as ChAd vectors, with evidence of efficacy from a relevant animal model would be likely to gain approval for use in a limited setting. Based on research, manufacturing and clinical trial costs for the ChAd3 vectored vaccine developed for Ebola, vaccines might be stockpiled for just $50 million per disease, representing a fraction of the cost of bringing a vaccine through to licensure.…”

Going “social” to access experimental and potentially life-saving treatment: an assessment of the policy and online patient advocacy environment for expanded access

“…Based on data from the FDA, numerous applications are submitted each year, with the agency receiving an average of 1,206 requests annually (based on data from October 2009–September 2014) with an overall approval rate of 99 % (Fig. 2 ) [ 42 – 44 ]. The vast majority of these applications are for single-patient, non-emergency (48 %) or emergency use (44 %) INDs (versus intermediate-size patient population access programs with multiple patients with the same disease or condition seeking access to the same drug), and are assessed by the FDA on a case-by-case basis [ 8 , 42 ].…”

Right now, in the right way: U. S. Food and Drug Administration’s expanded access program and patient rights

“…Most established IRBs will have had experience with EA and can serve as an important resource for assuring a timely and authoritative review of the consent document without imposing a barrier to access. In our experience with IRBs at academic health centers, there is typically no charge for review and advice for single patient EA review [10]. Independent IRBs may charge for review of EA requests but, at this time, WIRB-Copernicus Group provides EA IRB review at no charge [20].…”