In the article below, we summarize key takeaways from this webinar. You can access the first and second parts of the series here:
Part 1: “The Foundation: The Basics of Market Access and Why They Matter Before Phase III”
Part 2: “Making It Real: What Does an Early-Stage Market Access Strategy Look Like?”
Building off the foundational lessons provided in parts one and two of this year’s Demy-Colton webinar series on U.S. health care market access, Alice Valder Curran, partner in the Hogan Lovells health practice, emphasized that pre-commercial companies should focus on their market access strategies as early as their Phase 1 trials to increase their probability of success. Market access is the most important factor of success in the U.S. market, Valder Curran noted, and must be a research and development imperative as well as a commercial imperative, not least because it impacts the design and execution of a company’s clinical trial strategies.
Panelist Michael Rothrock, President, Allegheny Strategic Partners LLC, provided a payer perspective from his experiences on behalf of national and regional health plans and pharmacy benefit managers (PBMs). Rothrock provided a timeline of best practices for how and when a manufacturer should engage with managed care payers, including key topics to cover at each stage of clinical trial development. Rothrock noted that the therapeutic category of the product could be relevant to determining when earlier engagement is most recommended. For example, orphan drug, rare disease, gene therapies and oncology therapeutics could benefit from Phase 1 or even pre-Phase 1 engagement with prospective payers on topics such as “outcomes” in assessing trial endpoints. Valder Curran posed the important question of how companies without a market access team could still engage with payers at this stage. According to Rothrock, even absent an internal market access team, manufacturers could still reach out to medical directors, pharmacy directors, trade directors at specific payers. Rothrock cautioned that while some payers or PBMs will not engage at this stage, others, depending on the disease state, will want to think ahead about drugs to include on their formulary. Continuing along the product development timeline, Rothrock noted that for therapeutic categories where payers tend to have a better understanding of existing treatments and alternatives (e.g., diabetes, cardiovascular, or respiratory conditions), engagement starting at the time of Phase 2/3 trials could be appropriate. This would allow a manufacturer to help align on what could help differentiate their drug to make it more valuable for pharmacy and therapeutics (P&T) or formulary approval. Post-Phase 3, re-engagement with the payer tends to focus on a review and discussion of trial endpoints, focusing on the “patient journey” and alignment of the new drug with other possible therapies. Also at this stage, payers would expect to discuss specific pricing and distribution options in anticipation of an FDA approval date. Rothrock noted that the FDA approval date starts the “P&T time zero” to revisit the label and package insert, as well as final distribution options.
Lowell M. Zeta, a partner in the Hogan Lovells pharmaceuticals and biotechnology practice and former Senior Counselor to the FDA Commissioner, provided a perspective on tools that manufacturers can use when engaging with FDA to support a value based strategy for their products. Zeta identified three key areas which management should integrate into their strategic plans. First, Zeta discussed the use of patient-generated data and digital tools for data acquisition. “As payers are becoming more focused on outcomes, FDA and [industry have] been more focused on patient-focused drug development”, Zeta noted, in making sure that therapies work as intended. Early, meaningful engagement with FDA is crucial in helping the Agency evaluate the value of innovative tools, such as digital technologies or novel biomarkers. Zeta also discussed how the Agency has been grappling with regulatory decision making around real-world data/real-world evidence (RWD/RWE) and has issued several helpful and on-point guidance documents. Second, Zeta discussed how companion diagnostics can impact strategic planning. Zeta noted that it is important to consider early on whether a companion diagnostic is essential for the safe and effective use of a corresponding therapy, and if so, whether it will be stipulated in the final labeling. Zeta shared that laboratory developed tests (LDTs) may be used during early development, and highlighted the need for manufacturers to think through the studies that may be needed to bridge the gap between LDTs and companion diagnostics. Rothrock added that payers like to see companion diagnostics link to a binary “yes or no” of a patient response to the drug; more challenging from the payer perspective are companion diagnostics which only identify a “better versus poorer” responder. Third, Zeta discussed confirmatory trials, which can support, for example, the accelerated approval program, which gives conditional approval based on a more limited set of data. Manufacturers should closely monitor these areas as the regulatory frameworks evolve including through key changes proposed in industry user fee legislation.
Panelist Peter Rubin, Executive Director, No Patient Left Behind (NPLB) discussed how manufacturers can convey the value of their product to a broader audience prior to launch. Rubin provided an overview of the “ISPOR value flower”, which provides an enhanced societal view of a therapy’s cost-effectiveness, as compared to the “simple math” budgetary view of innovation espoused by many health economists. Rubin suggested that a traditional cost-effectiveness analysis (CEA) often comes in less than market price because economists overlook many benefits that medications provide to society as a whole. Rubin encouraged manufacturers to highlight these “real world values,” such as genericization and relieving caregiver burden, to better capture the societal value of innovation and justify a likely market price. Rubin advised manufacturers to design studies to support a generalized cost-effectiveness analysis (GCEA) and be prepared to “frame the debate” in providing a broader rationale for innovative therapies in supporting patients and families. Adding in the regulatory approval perspective, Zeta noted that confirmatory studies have been focused on verifying safety and effectiveness in the clinical setting. However, Zeta noted, there is also recent thinking on how to link this with RWD to support patient voice and quality of life, and potentially, to expanded indications. Data generated to support the FDA regulatory process can also guide other decisions to support the overall value proposition.
Particularly for early stage companies having a finite amount of resources and personal time to devote to product development, Valder Curran reminded of the importance of “starting early” and on “making purposeful decisions about what you are focusing on, when you are engaging, how you are framing the product as opposed to being boxed in at the end because you haven’t thought about it”.
Our summaries of the first and second webinars in the series are available online here (part 1) and here (part 2). The first session, “The Foundations: The Basics of Market Access and Why They Matter Before Phase IIIs” discussed the key questions that new drug sponsors must consider early on in planning their clinical and market access strategies. The second session, “Making It Real: What Does an Early-Stage Market Access Strategy Look Like?” walked through real life hypotheticals that incorporate the key learnings from the first webinar.