This article shares insights on how addressing payer needs in the product development process can help companies increase patient access to their novel treatments. It also explores the differences between payer and regulator considerations in clinical development, the role market access plays, and why market access involvement in drug development may become more important over time.
Securing drug reimbursement through negotiations with payers is among the surest ways to mitigate financial barriers to access. For example, in the European Union, after a drug company gains regulatory approval from the European Medicines Agency (EMA), the company must negotiate pricing and reimbursement criteria with each country’s payer separately. Likewise, after FDA approves a therapy, there are no guarantees private, commercial or government-funded insurers will cover it.
While regulatory bodies focus on and find confidence in clinical outcomes, safety, and efficacy, payers — whether privately held or government-funded — must prudently add economics to the equation. As such, successful negotiations with payers rely on a product 1) offering evidence-based, compelling efficacy and safety, 2) targeting a specific population with an unmet need and a clinically appropriate position in the treatment paradigm, and 3) coming in at a price that reflects product value. If successful, payer negotiations result in drug reimbursement in national-payer markets (e.g., the countries within the EU) or drug inclusion in health insurance plan coverage in fragmented-payer markets (e.g., the US).
Clinical trials historically are optimized to satisfy regulatory decision-making bodies, not payers, which generally leads to market access hurdles, including delays, misaligned value recognition, and/or access restrictions. Keeping in mind, regulators seek to determine whether the new treatment is efficacious and safe to administer, and if the data was collected in a rigorous and statistically powered way. Whereas, in addition to assessing whether the treatment works for the given indication, payers try to determine if the drug profile is aligned with the proposed cost.1 To that end, payers seek evidence to know how the drug will perform in their market, and ask questions such as
Determining “real-world” drug efficacy compared to available treatments is at the center of payer benefit assessment and most of the evidence presented to payers during this assessment comes from the clinical trials. Therefore, incorporating the payer perspective into clinical trial design is crucial to generate compelling evidence that will be accepted by payers, and result in optimal patient access.
Because payers will consider clinical evidence to determine whether to include a treatment option, it is critical to account for payer needs in each of the four stages of clinical development for optimal access.
In this stage, it also is important to consider whether a new treatment option can be expanded to address additional indications. If so, sharing indication expansion plans with payers during negotiations can enhance the value proposition for payers through the promise of future improved patient experience and cost savings (in case of overlapping patient populations).
The emerging trend toward faster regulatory approvals4 emphasizes the need for market access integration throughout clinical development. To enable accelerated approval, regulators more frequently allow the use of surrogate endpoints in clinical trials.5 Payers often consider these abbreviated endpoints suboptimal and react by requiring more evidence generation post-launch, restricting access, or denying access in some cases.6 The payer perspective is especially important in abbreviated trials, because of the increased risk that payers will consider the evidence base suboptimal. As such, including and acting on payer perspective during clinical trials ultimately will help improve patient access.
Looking ahead, each of the players involved in the process of drug development, approval, and reimbursement share the common goal of bringing life-improving therapies to the people who need them. Improving collaboration among pharmaceutical companies, regulators, and payers earlier in the drug development and clinical trial process is the best way to help ensure patients can access innovative therapies affordably.
David Bower is a Global Value & Access Lead for DUPIXENT in Dermatology indications at Sanofi Genzyme. Agata Wiśniowska is a senior consultant in Life Sciences at Guidehouse.
The views expressed in this article are solely those of the authors and do not necessarily represent the views of the authors’ employers
The authors thank Dr. Jean-Francois Ricci of Alira Health for advice on the payer lens on clinical development section. Additionally, the authors also thank John Etchberger of Guidehouse for advice on the role of market access in future indication planning.