FDA Announces 2 New Efforts Aimed at Drug Competition, Access

In the first week of October, FDA Commissioner Scott Gottlieb, MD, announced 2 new initiatives intended to make drugs more widely available: the first is designed to help developers of complex generics better navigate the approvals process, and the second aims to streamline access to investigational treatments for compassionate use.

On Monday, Gottlieb announced that the FDA has adopted a new set of policies to speed the approval of complex generic drugs, including injectable drugs, that are harder to produce as generics. Because development of complex generics poses additional challenges, innovator drugs face little competition even after losing patent protection. According to Gottlieb, “because brand-name versions of complex drug products are often higher-priced than many other brand name drugs, any steps we can take to encourage the development of generic competitors to complex drugs will have an outsized impact on access, and prices.”

In order to help reduce the number of review cycles necessary to approve these complex generics, the FDA has released new draft guidance, “Formal Meetings Between FDA and ANDA [abbreviated new drug application] Applicants of Complex Products Under GDUFA [Generic Drug User Fee Amendments],” that seeks to improve development timelines through earlier, better meetings between developers and the agency. The document describes the pathway for communication between the FDA and an applicant submitting an ANDA for a complex product, which the FDA defines as one with complex:

• Active ingredients (such as peptides)
• Formulations
• Routes of delivery
• Dosage forms
• Drug-device combinations
• Concerns with respect to approval that would benefit from early scientific engagement

The guidance also describes in detail the purpose, scope, and criteria for holding product development, presubmission, and mid-cycle reviews between the applicant and the agency.

The second initiative, announced on Tuesday, aims to expand patient access to investigational treatments for compassionate use. Gottlieb wrote in his blog that the “FDA recognizes that time is critical for these seriously ill patients who do not have alternative therapies, and who cannot take part in a clinical trial of an investigational therapy,” and announced that the agency is revising a policy that had the potential to slow patient access to investigational drugs. Previously, a physician seeking to prescribe such a treatment to a patient under expanded access would require approval from the full Institutional Review Board (IRB) at his or her facility, and infrequent convening of such boards could potentially cause delays. Moving forward, approval from just 1 IRB member, such as a chairperson, will be required.

Gottlieb pointed out, however, that drug makers cannot be compelled to provide investigational drugs, and that some companies are concerned about how adverse events (AEs) that occur in patients who receive investigational drugs could be viewed by the FDA. In attempting to address some of these concerns, the agency updated its guidance document “Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers.” In the guidance, the agency states that it is “not aware of instances in which [AE] information from expanded access has prevented FDA from approving a drug,” and said that its reviewers were aware that expanded access occurs outside a controlled trial setting, and that patients receiving expanded access may be experiencing more advanced stages of disease, may have comorbidities, or may be receiving additional therapies. It also clarifies the fact that AEs must be reported only if there is evidence that they were caused by the drug therapy.

Gottlieb also noted that the agency would continue to make simplifications and clarifications to its processes for expanded access in the days ahead.