Will 21st Century Cures Modernize Drug Discovery And Development?

Here are a few facts that are familiar to many who work in the life sciences:

• There are 10,000 known diseases, and 7,000 of them are considered rare.
• We have treatments for around 500 diseases.
• It takes approximately 14 years and close to $2 billion to develop a new drug, and 95 percent of them fail.

With those grim statistics, Marilyn Weber Serafini, VP for policy at the Alliance for Health Reform, kicked off a dinner discussion on the topic of 21st Century Cures. I was honored to take part in this discussion, which also included notable representatives from across the life science industry. Taking part in the dinner conversation were representatives from AstraZeneca, Quintiles, Avalere, National Pharmaceutical Council, National Journal, Personalized Medicine Coalition, National Association for Rare Disorders, Aetna, Alliance for Health Reform, and more.

After a year of discussions, white papers, roundtables, and the vetting of issues by stakeholders across the industry, both democrats and republicans are ready to move forward with legislation that would attempt to bring the drug discovery and approval process into the 21st century. The congressional head of this effort, Congressman Fred Upton of Michigan, has indicated he would like to see action on this bill by Memorial Day (the discussion draft was released on April 29th).The main goals of the initiative are to incorporate the patient voice into the clinical trials process, build a foundation for 21st century medicine, streamline clinical trials, support continued innovation at federal public health agencies, and modernize medical product regulation.

One attendee noted one of the problems we face is the dichotomy in this industry between tremendous success and tremendous failure. The success is that over the last 40 years this nation has invested billions into biomedical research, which is now revolutionizing the world. But at the same time, we still have 10,000 diseases and only 500 cures. “There is frustration over why this situation exists,” he notes. “Why can we not accelerate the ability to go from an exponential development of new knowledge to a point where we are rapidly delivering medicines to patients that save lives and improve health? That’s the problem.”

The purpose of the bill, of course, is to look at the ecosystem of discovery, development, and delivery in order to solve that problem. Unfortunately, another participant built on those comments by noting the systems we currently have in place are simply not designed to make this process happen quickly. Of primary concern is the regulatory system, which does not seem to be designed for what scientists and pharma companies are doing today. The system needs to change to catch up to the advances being made in science.

Measuring Success Is Difficult

If one of the goals of 21st Century Cures is to improve innovation in the industry, what is the measure of success we should use, and how will we know when we achieve it? One attendee stated FDA approvals is likely not the best measure. The industry’s goal should never be the number of molecules on the market. The goal should always be how those molecules will make lives better. Unfortunately, measuring quality of care and whether or not a drug is working is difficult at best, and many quality of care measures were not designed for that purpose.

One measure would clearly be to look at metrics. If in five years we see the number of new cures rising, quality of life for patients improving, and fewer drug companies fleeing the U.S. for better opportunities elsewhere, then we could conclude the industry is making progress and moving in the right direction.

Still, congress is facing a slightly different problem. Politicians likely understand they will need to invest more money in drug discovery. But if we make the investment without changing the way we do business, it will simply result in money being wasted. This will not result in congress, pharma, or patients getting the solutions they desire.

Reasons For Hope And Dismay

While there are many challenges pharma must overcome, 21st Century Cures gives many in the industry reason to be hopeful. Contained in the legislation is the ability to talk about real world evidence, which the pharma industry has not been able to do. “The legislation states there should be some examination of what can be shared,” said one participant. “As the industry continues to produce more non-randomized evidence, we need to know if we can talk about it and if the information is valid.”

There is also recognition in the bill that science is moving fast, and systems we have in place need to move fast to keep up. If the systems we have in place are not keeping up with the science, the first step in fixing the problem is recognizing that a problem exists.

It’s pretty clear from draft guidance already released that the senate will be focused on regulatory reform and NIH. Some of the other initiatives may fall away or be pared down, only to resurface again a few years down the road. Even so, attendees at the dinner questioned whether a 400 page bill (currently a discussion draft) would be passable. Congress passes big bills all the time, but one that is half the size would likely stand a better chance of making it through both chambers. A politician spending this much time addressing the FDA is certainly a watershed moment for the industry. The bill may end up being both impressive and wide reaching, but the size of this one could also be the thing that seals its fate.

In Part 2 of this article, I will discuss comments from the dinner attendees related to roadblocks that may impede progress in making the hope of 21st Century Cures a reality, and possible solutions to overcome them.