Yesterday the President’s Council of Advisers on Science and Technology (PCAST) released another report (H/T ScienceInsider). This time the focus is innovation in drug discovery and development. Given the work being done at the Food and Drug Administration and the National Institutes of Health to make it easier to address drug shortages and other challenges in pharmaceuticals, this is a timely report.
The report focuses on all aspects of what it calls the drug development ecosystem – biomedical research, clinical research in hospitals, and drug discovery and development. The report identifies at least two so-called valleys of death (language more familiar to technological development, but comparable):
The translational research valley of death (which is the focus of the National Center for Advancing Translational Sciences) – the gap between mature biomedical advances and the validated targets for therapeutic drugs. The old silos of ‘pure’ and ‘applied’ research are a significant hurdle here.
The efficiencies of scale valley of death – between the complex requirements of clinical trials and the low private return on investment for certain diseases, some drugs aren’t going to be developed in the traditional commercial research system.
Besides addressing these gaps, the report seeks to address the challenge of post-approval study. Due to the complexity of drugs and their interactions with the human body, our understanding of a newly approved drug will be incomplete. There needs to be a better job of monitoring drugs post approval, and there needs to be more communication to the public about that monitoring and how our understanding of drugs can change over time. Think of the resistance to changes in recommendations on screenings for certain types of cancer. Changes in medicine recommendations post FDA approval could meet with similar kinds of resistance – and not just because the public often considers scientific questions to be more settled than they are.
While the challenges ahead are by no means easy, the PCAST report suggests, if not outright declares, that the current state of the pharmaceutical development process is not sustainable. Many of the advantages enjoyed by private companies are going to be challenged over the next few years for both economic and research reasons. New mechanisms and means of support for drug development and evaluation are needed. The complete list of recommendations from PCAST (see the report for more details):
A. Improving Drug Discovery and Development
Recommendation 1: Support Federal Initiatives to Accelerate Therapeutics
Recommendation 2: Catalyze the Creation of a Broad-Based Partnership to Accelerate Therapeutics
B. Improving Drug Evaluation
Recommendation 3: Expand the Use in Practice of FDA’s Existing Authorities for Accelerated Approval and Confirmatory Evidence
Recommendation 4: Create a New Pathway for Initial Approval of Drugs Shown to be Safe and Effective in a Specific Subgroup of Patients
Recommendation 5: Explore Approaches for Adaptive Approval Via Pilot Projects Under Existing Pathways, but Do Not Create New Adaptive Approval Pathways Through Legislation
Recommendation 6: Improve FDA’s Tools for Monitoring and Communication of Clinical Benefits and Risks
C. Improving FDA Management
Recommendation 7: Reform Management Practices at FDA
D. Economic Incentives
Recommendation 8: Study Current and Potential Economic Incentives to Promote Innovation in Drug Development