The sands are shifting in the landscape of drug development. Large pharmas are merging and consolidating pipelines, quizzing more closely than ever the cost and rationale of clinical trials; some are moving research operations overseas where the work can be done faster and cheaper. Meanwhile small biotechs face a tough investor landscape and many are folding. And the future of healthcare and insurance is under hot debate. In the face of this change, two federal stalwarts, the Food and Drug Administration (which regulates all US drug approvals) and the National Institutes of Health (the premier funder of medical research in the US) were under the microscope at last week’s Partnering for Cures meeting with discussion panels asking ‘can they do it better?’
Concepts like ‘personalized medicine’ (predicting from your genetic information whether a drug is likely to be effective) are potentially revolutionary but are challenging for the FDA, who have to incorporate evaluation of such new technologies in their review of drug protocols, all the while keeping a weather eye on drug safety. To paraphrase FasterCures founder Mike Milken, the FDA needs to be strengthened the US is to remain competitive in drug development. To this end the FDA is streamlining its processes (e.g. moving from paper based to electronic document submission), regrouping and increasing staff. Meanwhile the NIH too faces change. Traditionally the 30+ NIH institutes have operated in ‘disease silos’ with institutes focused on one disease area (e.g. mental health) or organ system (e.g. eye). The emergence of genomics has turned this type of thinking on its head and looking forward we expect to see more cross-collaboration between institutes and promote innovative research that can be translated to healthcare such as early stage dug development, and the generation of biomarkers that will help accelerate clinical trials. On this theme NIH is working in partnership with FDA on a special biomarkers program, building a bridge between the two agencies. This needs to go further, to develop better diagnostics, and rethink clinical trials to be faster and cheaper, all the while keeping a strong communication with the FDA.
On a related note, some interesting comments emerged at Partnering for Cures about the healthcare system and the fact that (like the NIH) many hospitals operate on an organ based model. Recent medical knowledge advances in suggest the need to move toward a mechanism based paradigm. Doctors should seek annual CME accreditation in genetics/genomics to be more comfortable in understanding and enrolling patients in clinical trials.
Meanwhile once again patients and foundations step to the fore. Many groups are already ‘driving the bus’ for their disease area (see MMRF in yesterday’s blog) but we can do more: by engaging with the NIH and FDA, educating them on our disease area and how we do clinical trials; and lobbying Congress for more funding and synchrony for the FDA, NIH and healthcare system.
