Achieving Innovation and Access Together

In today’s new era of medicine, breakthrough treatments are revolutionizing how we fight disease, providing hope to patient populations that 20 years ago could never have dreamed of using the word “cure” when talking about their conditions. From cancer treatments using genetically modified genes called CAR-T, to advanced, personalized gene therapies, biopharmaceutical researchers are advancing the definition of what is possible at a record-setting rate.

But for patients who can’t access these treatments, this progress is irrelevant. Dealing with chronic and acute illness is hard enough – no patient should have to also fight to secure the medicines they need. This topic was the subject of a recent discussion at Aspen Spotlight Health, moderated by Mary Woolley, president and CEO of Research!America, featuring Jeff Marrazzo, co-founder and CEO of Spark Therapeutics; Gary Reedy, CEO of the American Cancer Society; and Stephen J. Ubl, president and CEO of the Pharmaceutical Research and Manufacturers of America (PhRMA). The panel collectively delved into potential solutions to the challenge of balancing innovation and affordability in today’s health care environment.

“I want to be able to talk not just about the dollars and cents costs of areas like cancer care and gene therapy,” said Ms. Woolley. “But I also want to be able to talk about the sense of costs. What is sensible as we pursue new treatments, preventions and cures?”

PhRMA’s Ubl pointed out the need to examine treatment costs at the system-wide level, noting that both drug prices and drug spending are growing slower than the overall rate of inflation. However, due to system-wide forces, it doesn’t feel this way for patients, who have been facing rising out-of-pocket costs for years. This – coupled with the rise in high-priced, innovative gene-based treatments – requires a need to rethink the way we pay for medicines. One of these ways is called value-based contracting, or results-based contracting, which allows biopharmaceutical companies and insurers to share financial risk through innovative and flexible ways to pay for medicines that focus on results, lower out-of-pocket costs and enable patients to access the right treatments the first time.

Spark Therapeutics, which last year received U.S. Food and Drug Administration approval for and earlier this year launched the first gene therapy for a genetic disease in the United States, provides an example of what this looks like in practice. To help ensure access to patients, the company outlined several plans for payment and reimbursement models that both recognize the value of the therapy and remove extra cost and risk for insurers and hospitals.

“In our system today, we pay for volume, not the value or health that a patient gets,” said Spark’s Marrazzo. “We think we should be helping this system move from a 20th century design to a 21st century one, which would match up with the types of scientific explosion that’s going on.”

One area of agreement was that the future holds great promise for patients in all areas – including cancer, neurological and rare diseases, Alzheimer’s and infectious diseases – but only if patients are allowed access.

“The answer is not to push more costs down to the patient,” said American Cancer Society’s Reedy. “All of us are going to have to come to the table to get this system to where it should be so that we can have both affordability and innovation for the patient.”

With the caliber of minds represented by Aspen Spotlight Health attendees, we are certain we can find system-wide solutions that ultimately benefits patients.