Q&A: How RWE Is Reshaping Specialty Pharma Market Access

The role of real-world evidence (RWE) in pharmaceutical development and market access has undergone a fundamental transformation. Tommy Bramley, senior vice president of global consulting services at Cencora, has spent more than two decades working at this intersection of evidence generation, market access, and patient outcomes — and he has watched RWE evolve from a post-approval surveillance tool into an expected component of the evidence package that payers, providers, and regulators rely on throughout a therapy's life cycle.

This shift is especially pronounced in specialty pharma, where high costs, smaller patient populations, and complex comorbidity profiles make traditional phase 3 trial designs difficult to execute, he says. For these indications, synthetic and external control arms built from real-world data have emerged as a practical alternative, given that recruiting patients to non-treatment arms in conventional trials can be prohibitively expensive and time-consuming.¹ The managed care community has responded with increasing formality: the AMCP Research Institute, in partnership with IQVIA, payers, and pharmaceutical companies, published payer-focused RWE standards in the Journal of Managed Care & Specialty Pharmacy in September 2025, providing a structured framework of study types and endpoints to support coverage, formulary, and reimbursement decisions.²

Speaking at the Asembia AXS26 Summit in Las Vegas, Bramley sat down with Pharmaceutical Commerce to discuss how evolving evidence expectations are reshaping reimbursement strategy, why specialty drugs require a distinct RWE approach, and how early evidence planning can accelerate patient access and strengthen collaboration across manufacturers, payers, and specialty pharmacy.

PC: How Is Growing Demand for Real-World Outcomes Changing Evidence Expectations for Market Access and Reimbursement Decisions?

Bramley: There's a growing reliance on RWE and an expectation by payers and providers that there'll be label expansion or additional data that will come to better inform the decision-making process. On the payer side, that would be the formulary positioning, that would be the tiering, that would be any type of utilization management. On the provider side, they truly want that data to better target the therapies to their patients, so what is going to work so they don't have to go through a series of trial and error. And yes, the clinical trials are very important in saying what could work, but once you release it into the wild, it's a much different story in terms of comorbidities of the patients and medication-taking behavior, and do you truly get that return on investment either in health or lower utilization of precious healthcare resources. so, it's, an expected part, and it's that translation bridge between what's happening in, say, the laboratory, and what's happening in the real world.

How Should Companies Approach RWE Differently for Specialty Drugs Compared with Traditional Therapies?

I would say it's very similar, except you have very high-cost products for typically smaller populations, and that's where your traditional methods in phase three may not apply, because it's difficult to have a placebo control arm or find enough patients, and that's where you start to construct your external control arms or your synthetic control arms. So, being open to that, and the FDA and EMA has signaled that they're very open to that type of data package, either a supplement or in place of a true control arm. That's the way that pharma should be thinking. And then as you move into the post-approval period, it's thinking about that truth. It's thinking about outcomes-based contracts. It's showing the difference that these therapies can make in patients' lives and how they can lead longer, more fulfilling lives in ways that you can capture that through patient-reported outcomes or utilization measures to show this product really does lower the total cost of care.

How Can Companies Better Use RWE Earlier in Development to Speed Approvals and Improve Patient Access Later On?

It's very useful when you're starting to think about your place in therapy. An RWE can help you identify unmet needs that this therapy can fulfill. It can help you understand how a particular therapy may stand up against the standard of care and comparative effectiveness, and you can start to think through what are those endpoints that are important from a regulatory perspective, but also from an HTA or a payer perspective. And you're ready to have those good discussions, the good debates about where this fits into the place in therapy. As you move into the launch, you begin to collect data across more patients, more variety, and that becomes an important feedback loop for physicians in terms of what is the right drug for the right patient. It's a very, very powerful tool in terms of optimizing the access and maximizing the patient benefit.

How Do You See RWE Shaping Collaboration Between Manufacturers, Payers, and Specialty Pharmacies to Improve Patient Outcomes?

This truly is where the rubber meets the road, and so, once a product is launched, you want to make sure you are capturing those patient benefits, and indeed, what you saw in the clinical trial is happening in the real world. By having in-place either prospective studies or an ability to retrospectively capture the data, you're fulfilling that promise. And so, what we encourage pharma is to have those evidence generation plans in place early so you can support your launch and have a faster launch in terms of maximizing that benefit across the globe and for patients who are going to do very well on that particular therapy.

References

1. Jadhav S. 2025 trends: Leveraging real-world data for synthetic control arms. Applied Clinical Trials Online. Published September 2025. Accessed May 2026. https://www.appliedclinicaltrialsonline.com/view/2025-trends-leveraging-real-world-data-synthetic-control-arms
2. Lockhart CM, Gleason PP, Happe LE, et al. AMCP real-world evidence standards: Overcoming barriers to using real-world evidence in US payer decision-making. J Manag Care Spec Pharm. 2025;31(12):1230–1236. doi:10.18553/jmcp.2025.25108. https://www.jmcp.org/doi/10.18553/jmcp.2025.25108