Dr. Janet Woodcock (CDER, FDA) stated that for every 10 drugs that enter Phase I clinical trials, only 1 drug is approved. The cost of bringing an innovative drug to market often requires a decade and a billion dollars of investment. The paradigm where pharmaceutical companies invest heavily in research and development yet garner few drug approvals is unsustainable.
Woodcock suggests that academic researchers can contribute better methods and technologies to enable faster/better preclinical and clinical decisions to be made during drug development. Recommendations given include:
- Development of biomarkers that help identify not only safety risks but also identify patients most likely to benefit from a new, targeted therapy
- Greater emphasis on applied science (e.g., drug manufacturing and scale-up enhancements)
- Identification of biochemical pathways causal to disease states
- Identification of proof-of-concept/surrogate endpoints
- Enhanced understanding of how the body handles a drug
- Take a lead on developing orphan drugs, which have historically not been a priority for pharmaceutical companies
- Develop and implement new ways to conduct clinical trials (e.g., use of early biomarker identification to guide patient selection) with the goal of developing faster, better, smaller clinical studies to gain critical information more quickly ( e.g., work being done at Stanford University)
- To extend clinical trials into the community and region surrounding academic medical centers to facilitate patient access, recruitment, and to enhance compliance
The public has a decreased tolerance for risk, as evidenced by increased regulatory requirements for premarket evaluation of drug safety and efficacy. The hope is that academic researchers can drive changes in the required testing paradigms (nonclinical and clinical) to enable faster, better, and cheaper drug approvals.