FDA/CMS Summit Offers Opportunity to Focus on Value

From the 21st Century Cures Act to negotiations about reauthorizing the Prescription Drug User Fee Act (PDUFA), much of this year has been focused on a range of health care issues—including how to ensure patient access to appropriate therapies, and how best to share and communicate about data throughout the health care community. And it’s a safe bet that attention won’t be shifting any time soon. In fact, the two-day FDA/CMS Summit for Biopharma Executives beginning today signals the continuation of an important conversation about these issues and more.

From the 21st Century Cures Act to negotiations about reauthorizing the Prescription Drug User Fee Act (PDUFA), much of this year has been focused on a range of health care issues—including how to ensure patient access to appropriate therapies, and how to share and communicate about data throughout the health care community. And it’s a safe bet that attention won’t be shifting any time soon. In fact, the two-day FDA/CMS Summit for Biopharma Executives beginning today signals the continuation of an important conversation about these issues and more.

One key—and incredibly complex—topic slated on the Summit’s agenda? How best to determine the value of biopharmaceuticals for patients.

Determining the value of treatment has inherent challenges. First and foremost, unlike the way most of us think about value in other parts of our lives, the value of a therapy is much more than simply its cost. Rather, the value of a drug also includes for its benefits to patients (e.g., clinical benefit, improved quality of life), our health system (e.g., cost offsets for other medical expenses) and society (e.g., improved productivity, reduced caregiver burden). And, because all patients won’t respond to treatments the same way, it is also important to consider each individual patient’s access to the treatment that works best for them when we think about value. In short: a one-size-fits-all approach to assessing value just doesn’t work for biopharmaceuticals.

As our health care system continues to shift toward value-based care models and away from focusing on volume-based care, identifying the best way to measure value and create a system to account for it remains an ongoing debate. Today, there are a range of payment models—from value-based insurance designs, which links how beneficial a treatment is to how much a patient pays, to consumer-directed health plans, which raise concerns because of the financial burden placed on patients —that are working to address this issue. And as these and other models are tested and implemented, it will be important to identify payment systems aimed at rewarding high value, while also working to eliminate barriers to treatment.

Moreover, knowing how to determine what actually counts as value is critical to creating a system that works for all patients. To this end, several health organizations, such as the Institute for Clinical and Economic Review and the American Society of Clinical Oncology, have created “value assessment frameworks.” The National Pharmaceutical Council (NPC) has reviewed the existing frameworks and has several key concerns with the methodologies for each. NPC has continued to provide comments and feedback on these developing value frameworks to help improve upon current methodologies and ensure that such frameworks aren’t misused or don’t inadvertently cause barriers to treatment, particularly with new drugs and breakthrough therapies.

Another important issue included at this week’s Summit is a discussion about patient outcomes. Using research and real-world patient examples help payers know what works the best for patients, but there are limits on how this comparative effectiveness research can be shared and communicated. While payers are interested in that data, biopharmaceutical companies may be unable to share such data because of ambiguities in regulations that govern those communications. Companies must rely on legal counsel for clarification around the policies, even if changes are made via legal decisions, legislation or FDA guidance.  

While there are many policy areas that affect the biopharmaceutical industry, access to research and clarifying what value means are key topics that will influence future treatments. 

To follow along with the FDA/CMS Summit on Twitter, please follow the hashtag #FDACMS. More information about the Summit is available here