Much discussion surrounds how our society will pay for curative and high cost therapies, including cell and gene therapy. And while COVID-19 challenges the execution of clinical trials and further raises affordability concerns, it has also created opportunities—including unprecedented collaboration and increased use of telehealth—that can support future innovative therapies.
A cross-sector panel recently sat down (virtually!) to discuss these topics at the Biotechnology Innovation Organization’s BIO Digital 2020. Representing the pharmaceutical manufacturer perspective were Cristina Masseria, M.Sc., Ph.D., Vice President of Patient Health and Impact, Vaccines at Pfizer, and Doug Danison, Senior Vice President and Head of Access at bluebird bio. They were joined on the commercial payer side by Steve Miller, M.D., Chief Clinical Officer at Cigna. Investor and former pharmaceutical executive Neal Masia, Ph.D., rounded out the panel, speaking to third party financed mechanisms for bringing innovative therapies to market.
The impact of COVID-19 on development and access to innovative therapies was inevitably top of mind. Dr. Masseria, a health economist, highlighted the uncertainty core to the situation: “We don’t know what the economic and health care system profile is going to look like six months from now, or one year from now.”
Panelists highlighted the challenges employer, commercial, and public payers currently face as their budgets are stretched, further challenging drug access and heightening the need for data and evidence to support drug value. Summarized by Dr. Miller: “Affordability is going to be the number one issue.”
The panel also expressed affordability concerns related to curative therapies for Medicaid, which is largely funded by a strained pool of state tax dollars. Dr. Masia noted the prevalence of pediatric indications among gene therapies, with affected children often covered by Medicaid. He raised real-world evidence (RWE) as one potential solution, which can help “spread out payments over time” and support payer coverage of a certain outcome.
Regarding innovative payment mechanisms for access, Mr. Danison commented that “the system is not set up” to cover one-time treatments with potentially long-term benefits. Therefore, new payment models are needed to accommodate them, and bluebird identified core principles to guide them through this process—including a focus on putting value at risk. Dr. Masia further noted that innovation will require the use of third parties, and that “RWE will need to track people over time, in aggregate, in a very credible way.”
Further discussion centered on the potential of secondary markets to develop, which can help bring liquidity and smooth the flow of payments over time. Example market participants may include reinsurance companies, or innovative players focused on ways to separate the types of risk associated with a drug, like performance or pricing risk. Nonetheless, Dr. Masia cautioned, “solutions are largely to come.”
One such emerging solution is Embarc Benefit Protection℠, developed by Dr. Miller and his colleagues at Cigna and its Express Scripts unit. The solution is based on the belief that, as Dr. Miller explained, “we have to be as innovative on the payment side as the biotech industry is on the science side.” The platform functions as a mechanism for employers and other payers to access high-cost gene therapies with no patient co-pay, at a flat per member per month (PMPM) rate for their populations. Today, Embarc includes Spark’s Luxturna® and Novartis’ Zolgensma®, and is looking to expand to other areas such as sickle cell disease and thalassemia as new gene therapies are approved. As Embarc grows, Dr. Miller said the underlying mechanisms must evolve to serve a larger population and broader set of diseases; “we’re working on that right now,” he noted.
Comments on silver linings from COVID-19 were plentiful, including unprecedented collaboration across the health care industry to develop treatments and vaccines. “We have never seen this level of cooperation across manufacturers, payers, providers, and others,” Dr. Miller said. “And, when you think about the scientific collaborations that are going on now, companies that previously would never have worked with others are now collaborating.”
Dr. Miller also raised the growth of telehealth and digital health innovations as a positive result of COVID-19, as people are looking to meet their health care needs without leaving home. For example, Cigna uses Bluetooth-enabled glucose monitors to follow over 100,000 patients with diabetes, and help them manage their care. They plan to use similar tools to support members with hypertension, heart failure, and other diseases, and already have a program running with remote monitoring through asthma inhalers.
The panel’s pharma participants echoed Dr. Miller’s optimism regarding collaboration, particularly with the public sector. “The level of trust between the government, the issuer, the payers, and pharma has increased enormously,” Dr. Masseria said. By working closely with payers and governments, Dr. Masseria hopes to see progress in policy changes that weren’t previously possible. Mr. Danison emphasized the need to capture this moment to achieve legislative progress: “We are in a unique period in time, and I hope this gives people time to focus on the need for change.”
Ultimately, the group highlighted that in order to bring innovative gene and cell therapies to market, it is critical to work across sectors and to think differently than we have in the past. And this panel discussion was a representation of just that.