Our friends at FasterCures prepared this list of the top 10 medical research issues and trends to watch in 2015, first appearing in The Huffington Post. Each one of these issues impacts children with cancer.
(Huffington Post) – At FasterCures, we are always thinking about ways to innovate the medical research system so that better treatments and cures get to patients faster. Read below to find out the top 10 issues and trends that we are watching this year:
- Crossing the aisle for cures. Kicking off 2015 will likely be the release of a draft of the 21st Century Cures legislation the U.S. House Energy & Commerce Committee has been developing under the leadership of Chairman Fred Upton and Representative Diana DeGette. The process itself has been a rare bright spot of comity and bipartisanship in the Congress, and a positive undertaking by members and staff to address a number of the entrenched systemic challenges in medical research that have become status quo. Will it make it through the wringer that it takes for an idea to become a bill and then a law? Perhaps, if patient groups and advocates like what they see and encourage their members to move it along.
- PDUFA VI: Regulations for patients? Also kicking off in 2015 will be the process of discussions leading to a new Prescription Drug User Fee Act (PDUFA), due to be passed in 2017. PDUFA V introduced some important initiatives to improve the regulatory process; what add-ons will the new round of negotiations bring? We’ll be looking for continued improvement in the patient-focused drug development process that began with PDUFA V (specifically the inclusion of patient perspective in the assessment of desired benefits and tolerable risks, not just the understanding of disease and adequacy of existing treatments), a commitment to the sustainability of existing accelerated approval mechanisms like “breakthrough therapy designation,” and more focus on the Food and Drug Administration’s (FDA) regulatory science needs.
- Making the value case for new therapies. There was plenty of good news in FDA’s unofficial year-end report, beginning with the fact that FDA approved a record 41 new drugs and biologics in 2014, the most since 1996, giving new options and hope to millions of patients. This included the highest number of orphan drugs and new biologics ever approved in one year, and 43 percent of new approvals were first-in-class drugs. While these numbers seem to bode well for the state of innovation – FasterCures’ Senior Fellow Bernard Munos suggested earlier this month that we are on the cusp of a hyper-innovation age – how will the payer marketplace react? There may be a lot of very costly new drugs coming at us. Forbes’s Matt Herper noted that the most important immunotherapy drugs just approved will carry price tags of $150,000 per patient per year. How will the value these new therapies deliver to patients be defined and evaluated? There is wide agreement that the current paradigm of uncertainty about how innovation will be paid for cannot be sustained, but will stakeholders be willing to align their interests with those of patients?