The punch line of the briefing came toward the end when Steve Galson from Amgen opined that aEURoeThere are rooms of reports on innovation and the challenges aEUR" what will differentiate this one?aEUR? It was helpful to have it said out loud, because I felt a sense of dA(C)jA vu listening to the presentations and perspectives. Things may look tough now, but theyaEUR(TM)ll be much tougher if we donaEUR(TM)t wake up and smell the coffee.
• We heard about how innovation has played a huge role in advancing the health of our nation, but that the system is under stress.
• We heard about the key role the FDA plays in influencing the ecosystemaEUR(TM)s behavior, but even with changes at the agency, more needs to be done.
• We heard about how collaboration is central to future discovery, but the system doesnaEUR(TM)t consistently incent it.
• We heard about unmet need in the form of AlzheimeraEUR(TM)s, treatment for psychiatric diseases, cancer, and heart disease and stroke, but that we have been making some inroads with treatments for diseases like HIV/AIDS.
• We heard about how the entire system is struggling for many reasons aEUR" funding, patent cliffs, low-hanging fruit being picked, and overall costs of drug development rising, but that there are byways being built.
• We heard how patients and venture philanthropy are central to solving the problem, but there arenaEUR(TM)t groups in existence capable of taking on all the challenges.
• We heard there has been progress aEUR" at times breathtaking progress aEUR" but that there is still a long way to go to reap the benefit of the scientific promise that lies ahead.
• We heard how we need money to do all of this, but we donaEUR(TM)t know exactly where itaEUR(TM)s going to come from, especially in these tight times.
• And we heard weaEUR(TM)d better get moving.
There is a shift underway; during the briefing we heard people like Todd Sherer, who is the CEO of the Michael J. Fox Foundation for ParkinsonaEUR(TM)s Research, saying heaEUR(TM)s optimistic based on things heaEUR(TM)s seeing. And Bob Beall, president and CEO of the Cystic Fibrosis Foundation, talking about the game-changing approval of Kalydeco and the need to look at the PCAST report with the express purpose of figuring out what recommendations would speed things up. There was some discussion of consumers and patients needing a seat at the table. Well, we learned doing our report aEURoeBack to Basics: HIV/AIDS Advocacy as a Model for Catalyzing ChangeaEUR? that you donaEUR(TM)t get handed a seat at the table, you earn it. And you earn it by getting smart about the system and what solutions look like. IaEUR(TM)d like to see us moving beyond giving patients lip service to ensuring weaEUR(TM)re having a real dialogue with them about what they are already doing, and what is possible if and when they are engaged. As Sherer reminded us, "patients want to be part of a solution."
We know current resources are threatened by the possibility of sequestration, which would automatically cut the federal budget in January 2013. Future resources to address these pressing needs would also be slashed.
The PCAST report cites two primary needs: better methodologies and tools to translate basic biological insights to validated therapeutic targets and leads, and the need to have a more efficient clinical trial system, as it currently constitutes 40 percent of the biopharmaceutical R&D budget.
It posits recommendations in areas of improving drug discovery and development; improving drug evaluation; monitoring and communication about benefits and risks; improving FDA management; and economic incentives. There's a timeframe for action and a list of steps to be taken. We need to assess how this gets done, which entities can and should do it, in what timeframe, and also look at what agencies like the FDA are already beginning to do under the recently enacted PDUFA V agreement, and perhaps most importantly how to get the resources to do it. We were probably all raised with the phrase "money doesnaEUR(TM)t grow on trees," so we need to figure out creative ideas that will focus attention on the imperative for funding.
By focusing on specific solutions and not just the challenges, this report is different than many before it aEUR" but it canaEUR(TM)t be put on a shelf, action must be taken. As my husband often reminds me, "How do you eat an elephant? One bite at a time."