Evolving a Market Access Strategy to Improve Patient Access

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.

Payer negotiation as a gateway to access

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).

Payer lens on clinical development

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

1. Is the trial population reflective of clinical practice?
2. Is the comparator relevant for my market?
3. What is the long-term efficacy?
4. What is the budget impact?

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.

Market access role throughout clinical development

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.

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1. In the early stage (pre-clinical, Phase 1, 2a), market access teams can strategically pursue early payer feedback through health technology assessment (HTA) scientific advice to help the company understand how to position a new therapy for the best possible access outcomes. For example, the European Network for Health Technology Assessment (EUNETHTA) offers the option to pursue parallel payer and regulator early clinical design consultation to “help generate optimal and robust evidence that satisfies the needs of both regulators and HTA bodies.”² It should be noted, however, that formal early HTA scientific advice is a resource intensive process that requires approximately six months of preparation and should thus be pursued selectively. Assets that benefit from early HTA advice typically have the following characteristics:
   
   
   1. Large commercial opportunity that would justify early investment
   2. Complex disease, e.g., with complex treatment pathway, undefined patient populations and/or endpoints
   3. No HTA precedence, e.g., first-in-class or disruptive assets entering a well-developed market
   4. Willingness and ability to implement advice, pre-pivotal assets early enough in clinical development such that HTA recommendations can be acted upon.
      
      
2. In the late stage (Phase 2b, 3), market access teams typically can guide clinical development by testing pivotal trial protocol with payers through primary payer research and advisory boards comprised of former payers. Pivotal trial components commonly tested with payers include trial endpoints, duration, comparator, subgroups, and site selection. Understanding payer expectations often differ from those of regulatory bodies, it is essential to understand any discrepancies before locking in the pivotal trial protocol to enable optimal access. For example, if the choice of appropriate comparator differs by market, a solution can be to add a targeted subgroup analysis of patients with specific treatment history. The location of the trial sites and planned launch sequence are other key considerations because some geographies require testing to be conducted on the local population.³
   
   
3. In the launch and post-launch stages, market access teams can negotiate reimbursement conditions with payers as regulatory approvals are gained in various geographies. In this phase, global and local market access teams should collaborate to jointly prepare payer negotiation strategies based on global team materials (payer value messaging, market access and pricing guidance,) as well as local market and competitive insights.
   
   The negotiation strategy for key markets can be further tested in mock negotiations. The preparations are extensive because negotiations with payers determine patient access to therapy. In those negotiations, market access teams advocate for patient access in local markets and agree on access criteria, including starting rules, treatment history requirements, continuation criteria, stopping rules, and price. It is worth emphasizing that payer value messaging used in negotiations is largely based on evidence generated in clinical development, which is again why it is so important to consider payers in the early trial stages in order to generate data which may later help secure optimal patient access to treatment.
   
   
4. In the life cycle management (LCM) stage, market access teams can ensure payer perspective is accounted to ultimately gain access for indicated patients. Payers typically value LCM indications that provide treatments for patients most in need of more efficacious or safer therapies. They also value significant cost savings in cases where clinical outcomes are similar to the standard of care.

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).

Increasing pressure on market access needs

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

References

1. Lebmeier M., Chandler F., Godfrey J., et al. (2020) The impact of payer and reimbursement authorities evidence requirements on healthcare solution design for Muscular Dystrophies. DOI: 10.5772/intechopen.92553
2. https://eunethta.eu/services/early-dialogues-for-pharmaceuticals/, accessed on 2/20/2021
3. Bancroft C. (2020) Meeting clinical trial data requirements in Asian markets. Clinical Leader
4. Batta A., Kalra B.S., Khirasaria R. (2020) Trends in FDA drug approvals over last 2 decades: An observational study. J Family Med Prim Care 9:105-114
5. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/accelerated-approval, accessed on 2/20/2021
6. Bognar K., Romley J.A., Bae J.P. et al. (2017) The role of imperfect surrogate endpoint information in drug approval and reimbursement decisions. Journal of Health Economics 51:1-12

Acknowledgements

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.