Facing such a furious rate of innovation, and so much medical data being collected outside formal clinical trials, is the regulatory system capable of keeping pace? Will the future of medicine be ungoverned?

Biomarker Drugs

3X

Better chance that trials using genetic selection criteria will clear the approval process

BIOMARKER DRUGS WILL END THE ERA OF BLOCKBUSTER DRUG ECONOMICS

Research into genetic biomarkers has dramatically helped figure out why drugs work in some patients and not in others. There are already 62 cancer treatments approved for specific genetic subpopulations, and 238 overall. When they work, they tend to work far better. This raises the odds of approval considerably. Clinical trials that use genetic selection criteria have a 3x better chance of negotiating the full approval pathway. Critically, given that Phase 3 trials are so expensive, those odds of approval double.

All of this reduces development risk, but the tradeoff is clear: the genetic screening means the market size for any of these drugs is much smaller.

There is a bright spot, however: Pharma companies’ libraries of previously failed compounds can now be reanalyzed by biomarker interactions. They might have worked for certain genetic profiles. Potentially, many of those compounds can be brought back to market with low R&D investment.

Read the Full Can the FDA Keep Up? Report