The Prescription Drug User Fee Act (PDUFA) is passed every five years after the FDA and the drug and device industries re-negotiate user fees and regulatory priorities. This year is PDUFA V, the Food and Drug Administration Safety and Innovation Act. It will pass Congress this summer.
Today, let’s focus on an under-reported story: how the new “fast track” approval process in Title IX helps the industry negotiate the looming patent cliff. This new “fast track” process changes the ground rules on how the FDA approves drugs. The FDA is being told to approve drugs with less complete studies based on biomarkers, intermediate endpoints, and other time-saving short cuts. Essentially, companies with a product for a “serious or life-saving disease or condition” will be able to get conditional marketing approval with something akin to Phase IIB studies, rather than two or more Phase III studies. Here’s the multi-billion dollar payoff, saving the industry from the looming patent cliff:
- Shorten drug approval times by 2-3 years;
- Reduce pre-marketing clinical trial costs by 20-30%, saving hundreds of millions of dollars; and
- Focus innovation on “serious or life-saving” therapies.
The big risks are safety and efficacy. Bringing drugs to market with less information is akin to experimenting on the public during the first few years the drug is sold. Title IX does a couple things on safety:
- Companies must agree to undertake the full Phase III studies after marketing approval;
- The FDA reviews marketing materials in advance; and
- The FDA can pull the drug off the market if the safety or efficacy data is bad, if the company markets the drug with false or misleading information, or if the post-marketing studies aren’t completed on time.
Title IX is a huge boost to earlier sales of patented drugs, with significant but unknown safety and efficacy risks. Patient advocacy groups (often with drug company financial backing) support allowing patients to take those risks, so long as they are fully informed. If you think that in the real world, patients will make poor choices with these fast-track drugs, then you are likely to oppose Title IX.