Since the mapping of the genome, there has been a growing expectation that a flood of transformative therapies would re-define medical practice and patient care. But this flood is still a small trickle, and the Federal government is attempting to craft legislation to speed development and eliminate some logjams. Where are the logjams? Not at the FDA or NIH.
The much-discussed 21st Century Cures Act (introduced in the House of Representatives last May, passed on July 10, and now in the Senate), is intended to encourage innovation, give the FDA greater leeway to consider non-traditional sources of data and clinical studies, and shorten the time between application to the FDA and approval. The consensus among some industry experts is that the Act is a “mixed blessing.” This list juxtaposes some elements of the Act (in no particular order) with some recent commentary.
- The Act encourages the FDA to use biomarkers and other surrogate measures instead of clinical end points for evaluating efficacy. However...the FDA already uses surrogate end points in many new drug approvals.
- The Act instructs the FDA to consider non-traditional study designs and methods of data analysis to further speed approvals, and to develop criteria for relying on “clinical experience... observational studies, registries, and therapeutic use.” However... there is a reason why the randomized controlled clinical trial is the gold standard for assessing efficacy, although non-traditional study designs and data sources are valuable for monitoring drug safety post-approval. Most new drugs are already approved on the basis of data generated in studies of less than six month’s duration. In addition, the FDA already has a “breakthrough therapy designation” (BTD) intended to save both time and money in the development process. FDA’s experts provide guidance, beginning with phase 1 clinical trials, to expedite the process.
- The Act addresses the time needed for regulatory approval. However... the FDA approval process is remarkably efficient – the majority of new drug applications are evaluated within 10 months, and the FDA compares well with its counterparts in other countries.
A commentator in Forbes(2) opined that the 21st Century Cures Act is an attempt by Congress to placate patient advocacy groups. It is understandable that patients who have exhausted all other possibilities would want access to therapies in the developmental pipeline. However, approval of new therapies without appropriate risk-benefit analysis can only hinder the development of transformative therapies in the long run.
Ultimately, the FDA cannot generate innovation – it can only respond to what industry presents for review and approval. So where are the logjams? Why have we not seen the promised flood of new therapies? Why does development of a new candidate therapy and preparation of an Investigational New Drug (IND) application take 10 or more years?
Fisher BioServices is proud to support the researchers who are working on new therapies, and with every shipment — whether they are samples for use in assays, collection kits to support clinical trials, or a dose of a cell based therapy or new vaccine — we are aware that a patient is waiting. We are all working on the advancement of medicine and development of new therapies, even if indirectly.
We will soon post our latest eBook, which offers an insider's perspective on maximizing the value of biospecimen collections for the development of new therapies. Don’t miss it! In the meantime, check out our eBook Protecting Your Protocol: Operating a Flawless Clinical Agent Repository. This eBook discusses the process of how clinical agents are received, filling orders for clinical pharmacies, and ultimately how to minimize risk, all while adhering to FDA guidelines.
(1)Tufts Center for the Study of Drug Development
(2) LaMattina, John. “Is the 21st Century Cures Act Good or Bad for the Biopharmaceutical Industry?” Forbes Magazine. June 8, 2015. http://www.forbes.com/sites/johnlamattina/2015/06/08/is-the-21st-century-cures-act-good-or-bad-for-the-biopharmaceutical-industry/