By Mirella Zulueta
There are two bills in Congress at the moment related to the process of drug approval, specifically aiming to speed up the approval of new drugs. These two bills – FAST (Faster Access to Specialized Treatments) and TREAT (Transforming the Regulatory Environment to Accelerate Access to Treatments) – would essentially mediate that drugs are approved faster for serious disease conditions with no better treatment options. Such path would very much follow the precedent of the HIV/AIDS drug approval model. Language in FAST and TREAT states “that FDA should apply the accelerated approval and fast track provisions to the greatest extent possible to help expedite the development and availability to patients of treatments for serious or life-threatening diseases or conditions while maintaining appropriate safety and effectiveness standards for such treatments”. Key stakeholders may have different views on these proposals.
The FDA is less concerned with the pathway itself than it is with safety, and in its view, the rate-limiting factor is getting safe and effective drugs developed. Both FAST and TREAT specify the need to include early clear surrogate endpoints to predict benefit. According to Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the FDA, “The real problem is the paucity of worked-up endpoints, either as surrogates or as clinical endpoints that could be used for accelerated approval. It is not that we don’t accept them – it is that they don’t exist. People need to develop more interim endpoints and more surrogates.”
The need to identify valid new surrogate markers as well as novel clinical studies designs that would support accelerated approvals is apparent. It will take time and disruptive thinking to reach and implement consensus around new clinical and surrogate endpoints. Language in FAST encouraging the application of “modern scientific tools earlier in the drug development cycle” would likely expand the kind of data FDA reviewers consider acceptable for supporting accelerated approval.
Biotech companies support FAST. In the past, an accelerated approval process stimulated an explosion of investment in drugs to treat HIV/AIDS and cancer. The expectation now is that increased regulatory certainty and faster development times would stimulate investment, which in turn will ultimately increase the number of breakthrough drugs available to patients.
While biotech companies have endorsed FAST enthusiastically, pharmaceutical companies have not and remain mostly opposed to FAST. What are the possible explanations for the divide? The shorter time drug approvals require, the less that smaller biotechs would need funding for clinical trials and research from big pharma. While pharmaceutical companies have been more willing to undertake those partnerships rather than spend more for in-house R&D, it may have come at a price to the biotechs, i.e., a cut of their potential sales. It is certainly a possibility that it would be to big pharma’s advantage to continue resourcing to biotechs as generators of novel drugs and innovation, rather than give the smaller biotech companies the ability to launch drugs on their own if given the option to have their drugs tentatively approved after comparatively inexpensive phase II studies.
Independently of who takes a novel drug to the end through an accelerated approval, companies would have to do very careful post-marketing surveillance, and drugs could be pulled from the market if they didn’t live up to their pre-approval expectations.
Lastly, from the patient’s perspective faster approval of new drugs for severe conditions and rare diseases without better treatment would seem quite desirable.
Different stakeholders, different opinions … key questions remain as open-ended at this point: Is FAST likely to dramatically increase the number of new drugs developed or approved? Who will benefit from this process, biotechs, pharma, or the patient?
- Biocentury, March 12, 2012