The 2015 BIO International Convention was an all-out celebration of the innovative spirit of the biopharma industry. However, it was clear that not everyone defines innovation in the same way. In fact, during that four-day period, participants had dozens of interpretations of what it means to be innovative in the biopharma/biotech industries in 2015.
As speakers and participants weighed in on what drives innovation, the concept of “value” was prominent. During a super-session on “Accelerating Innovation in the 21st Century,” Paul Hudson, President, AstraZeneca US, explained, “We are shifting from a fee-for-service paradigm to a system where we are focused on measuring cost-quality outcomes and asking questions about the best ways to manage population health.”
Throughout the conference, Hudson and other biopharma executives continually referenced a shifting reality in which demonstrating value is a key determinant in patient access and market uptake.
In reality, innovation is about more than accelerating clinical trials with qualified biomarkers, or designing post-marketing programs to take into consideration long-term population-based outcomes. Innovation means being able to demonstrate value—in clinical and pharmacoeconomic terms.
But given the regulatory structure, communicating value in health economic terms has been challenging for many companies.
More guidance is needed
According to Sung Hee Choe, Director at Avalere Health, a Washington, D.C.-based healthcare advisory company, “As the healthcare system moves towards a value-based system, there is demand for real-world evidence related to health economics. Yet companies feel hampered in their ability to share that evidence, though payers and physicians are looking for that information.”
Recently, Avalere surveyed 14 small, mid-size, and large global biopharma companies about this issue. Overall, 86% of survey respondents said they would invest more in pharmacoeconomic and other studies to support the development of more economic information if more FDA guidance were available.
Biopharma companies are eager to share key value points about their products and to be able to frame their offerings in the context of health-economic decision-making, because they know that is what payers, physicians, and patients do every day.
In fact, payers are actively demanding more evidence-based information about the value of treatments, in terms of product value, effectiveness, costs and risks, as well as comparative health-economic information about how one treatment stacks up against an alternate treatment. Yet, there is still a great deal of uncertainty about what type of data drug makers can share.
Unfortunately, when it comes to sharing compelling, well-researched pharmacoeconomic data, there is a big question mark.
Enter 21st Century Cures
When putting together the key provisions of the 21st Century Cures Act, the House Energy and Commerce Committee, under the guidance of Fred Upton (R-MI) and Diana DeGette (D-CO), added a key provision to address this issue head on.
In Section 2101 “Facilitating Dissemination of Health Economic Information,” health care economic information is defined as any analysis (including clinical data, inputs, or other assumptions, methods, results, and other components underlying or comprising the analysis) that identifies, measures, or describes the economic consequences, which may be based on the separate or aggregated clinical consequences of the represented health outcomes, of the use of a drug. Such analyses may be comparative to the use of another drug, to another health intervention, or to no intervention.”
In short, the goal of this provision is for the FDA to provide better guidance for drug makers, so that these companies can communicate key health economic, comparative effectiveness and pharmacoeconomic data, including comparative data, in a commercial and clinical context.
The challenge of dealing with PBMs
In December 2014 when Express Scripts, the largest pharmacy benefits management (PBM) company in the United States, struck an exclusive deal for AbbVie's hep C combo Viekira Pak instead of Gilead’s Sovaldi and Harvoni in exchange for a significant discount, it signaled the dawn of a new pricing era.
Since then, payers have been mounting huge offensives against specialty drugs, including newly approved immunotherapy cancer drugs, such as Opdivo, as well as not-yet-approved PSCK9 inhibitors, a new class of anti-cholesterol drugs that lower LDL-C by as much as 60% and decrease cardiovascular events by half. Yet, payers, such as CVS and Prime Therapeutics, are already complaining about the costs of PCSK9 inhibitors and forecasting annual outlays in excess of $150 billion.
Even as innovative therapies gain approval, the ability for patients to gain access to those medications is not a foregone conclusion. And it’s not just the United States. Coverage denial is a regular occurrence in the U.K., as well as across Europe. So now the question is, “What good is innovation without access?”
Clarification bodes well for patients
The 21st Century Cures Act is a blueprint for innovative thinking, including a clarified, more expansive perspective on the role of health economic information in decision-making.
Assuming that this provision sticks and the bill is passed, the ultimate winners will be patients. But drug makers and payers stand to benefit as well.