Drug development is one of the most rigorous processes in any industry. Years of preclinical work, clinical trials, and regulatory review must clear an evidentiary bar that grows higher every decade. When a medicine wins approval, it’s a genuine achievement.
And then, in many cases, patients still can’t get it.
This is the part of the story that doesn’t get told often enough. Regulatory approval and patient access are not the same thing. Between the two sits a landscape of reimbursement decisions, health technology assessments (HTAs), payer negotiations, and pricing agreements that determines whether an approved medicine actually reaches patients. Health economics sits at the center of all of it.
The Access Problem Is a Value Problem
Payers and HTA bodies aren’t asking whether a medicine works. Regulators already answered that. They’re asking whether it works well enough, for enough patients, at an acceptable cost, to justify inclusion in the system they’re responsible for managing.
Different question. Different evidence.
Outcomes data, real-world evidence, quality-adjusted life years, cost-effectiveness — these are the currencies of reimbursement decisions [1]. A development program with strong clinical trial data but no answer to what payers need has left a gap in its evidence package — and that gap is often discovered too late.
How big is the gap? The EFPIA Patients W.A.I.T. Indicator, which tracks access to innovative medicines across 36 European countries, found that of 173 medicines approved by the European Medicines Agency between 2020 and 2023, only 46% are available to patients — and only 29% are fully reimbursed [2]. The average wait from EMA approval to patient access has now reached 578 days, with disparities between countries exceeding sevenfold: patients in Germany wait 128 days on average, while patients in Portugal wait 840.
A comparative analysis of 1,405 HTA assessments across seven OECD countries identified low quality of evidence and unresolved uncertainties around clinical benefit, cost-effectiveness, and economic model inputs as significant predictors of rejection [3]. The science was usually sound. The health economics case was not.
Why This Has to Start Early
One of the biggest shifts in pharmaceutical development over the past decade: health economics can’t wait until the end.
The evidence needs of payers shape which endpoints get selected, which patient populations get studied, which comparators get chosen, and how the whole evidence package is structured. As the ISPOR Special Task Force on US value assessment frameworks emphasized, value-based thinking has to be built into development strategy, not bolted on after [4].
That changes the job description for everyone who touches the development process. Clinical teams now have to think about how trial designs will be received not just by regulators but by HTA committees. Medical affairs needs to articulate value in economic terms, not just clinical ones. Market access can’t be a function that gets pulled in at the end.
Medical Affairs Steps Up
No function has felt this shift more acutely than Medical Affairs.
Historically a post-approval function focused on scientific communication, Medical Affairs has evolved into something considerably more strategic — a critical interface between the clinical evidence generated during development and the external stakeholders who decide whether that evidence translates into access [5]. McKinsey’s analysis found that only 20% of leading pharmaceutical companies currently develop a truly cross-functional, integrated evidence generation plan across a product’s life cycle — a gap that Medical Affairs is increasingly being asked to close.
Payers. HTA bodies. Key opinion leaders. Patient advocacy organizations. Healthcare providers. These are the audiences Medical Affairs now engages — and engaging them effectively requires fluency in health economics [6]. Real-world evidence studies, health-related quality of life assessments, budget impact models, outcomes research — these activities are now frequently led or co-led by Medical Affairs.
In short: Medical Affairs has become the function that connects clinical science to commercial reality. Health economics is the language that connection runs on.
Value Demonstration Crosses Borders
The complexity compounds geographically. A medicine that achieves reimbursement in one market faces an entirely different set of requirements in the next. HTA processes and value frameworks vary significantly across countries, payer priorities differ, and the evidence that satisfies a committee in one jurisdiction may be insufficient in another [7].
For development organizations operating across multiple markets, the evidence generation strategy has to be built with this heterogeneity in mind. That means understanding what evidence is needed and where it needs to work and how different audiences will evaluate it. It means having professionals who can navigate the UK’s NICE, Germany’s G-BA, France’s HAS, and their counterparts in markets around the world — and who can build a coherent value story that holds across all of them.
This is sophisticated, demanding work. The industry needs more professionals equipped to do it.
The Bottom Line
For anyone working in medical affairs, market access, clinical development, or any function that intersects with how medicines reach patients, health economics is no longer a specialist domain. It’s a foundational competency — one that shapes the outcomes of development programs, the breadth of patient access, and the impact that medicines have on the people they’re designed to help.
Module 2 of the GMDP Academy Certification in Medicines Development program is built around this reality. It explores how Medical Affairs operates as an effective interface with external stakeholders to make sure the right medicines reach the right patients — covering evidence generation, health economics, commercialization, and the application of digital technologies to support market entry across countries.
It approaches these disciplines not as theoretical frameworks but as practical competencies — the tools professionals need to navigate the gap between regulatory approval and genuine patient access, and to close it.
Available as a standalone module or as part of the full ten-month CMD program in partnership with King’s College London, Module 2 is designed for professionals who understand that getting a medicine approved is only part of the work — and who want to be equipped for all of it.
References
[1] National Institute for Health and Care Excellence (NICE). Guide to the Methods of Technology Appraisal 2013. Process and Methods [PMG9]. London: NICE; 2013. https://www.nice.org.uk/process/pmg9
[2] EFPIA. Patients W.A.I.T. Indicator 2024 Survey. European Federation of Pharmaceutical Industries and Associations; May 2025. https://www.efpia.eu/media/oeganukm/efpia-patients-wait-indicator-2024-final-110425.pdf
[3] Papadopoulos F, Visintin E, Kyriopoulos I, Kanavos P. Why drugs fail health technology assessment: a comparative analysis of health technology assessment rejections across seven OECD countries. Health Economics, Policy and Law. 2025;20(3):264–283. doi:10.1017/S1744133125100212
[4] Garrison LP Jr, Neumann PJ, Willke RJ, et al. A health economics approach to US value assessment frameworks — summary and recommendations of the ISPOR Special Task Force Report [7]. Value in Health. 2018;21(2):161–165. doi:10.1016/j.jval.2017.12.009
[5] Algazy J, Garcia A, Ryan S, Westra A, Zemp A. A vision for medical affairs 2030: five priorities for patient impact. McKinsey & Company; 2023. https://www.mckinsey.com/industries/life-sciences/our-insights/a-vision-for-medical-affairs-2030-five-priorities-for-patient-impact
[6] Leal Beckouche M, Resler Plat G, Marinich JA, et al. The Evolution of Medical Affairs Central Teams: Shaping Strategic Action and Driving Healthcare Transformation Through Scientific Interaction. Pharmaceutical Medicine. 2026;40(1):9–18. doi:10.1007/s40290-025-00592-9[7] Oortwijn W, Sampietro-Colom L, Habens F. Developments in value frameworks to inform the allocation of healthcare resources. International Journal of Technology Assessment in Health Care. 2017;33(2):323–329. doi:10.1017/S0266462317000502
[7] Oortwijn W, Sampietro-Colom L, Habens F. Developments in value frameworks to inform the allocation of healthcare resources. International Journal of Technology Assessment in Health Care. 2017;33(2):323–329. doi:10.1017/S0266462317000502
Disclaimers
- The material in these reviews is from various public open access sources, meant for educational and informational purposes only
- Any personal opinions expressed are those of only the author(s) and are not intended to represent the position of any organization(s)
- No official support by any organization(s) has been provided or should be inferred
