Maprium Ltd blog

Managed Access Programs: Building the Evidence Infrastructure for Rare Disease

romina.oxborough@maprium.com (Dr Romina Oxborough) — Fri, 10 Jul 2026 08:04:37 GMT

For many years, Managed Access Programs (MAPs) have served a single, vital purpose: providing patients with serious or life-threatening diseases access to promising therapies before they are commercially available or reimbursed. That compassionate mission remains unchanged.

What has changed is what the pharmaceutical industry now expects these programs to deliver. Increasingly, MAPs are being viewed not simply as a route to treatment, but as a strategic component of long-term planning. MAPs create an opportunity to generate the real-world evidence (RWE) needed to support reimbursement, demonstrate long-term value and reduce uncertainty surrounding rare disease therapies.

In many organisations, this represents a fundamental shift in thinking.

The evidence challenge in rare disease

The challenge is not a lack of innovation. The pipeline for orphan medicines continues to expand rapidly, driven by advances in gene therapies, cell therapies, precision medicines and RNA-based treatments.

The problem is that these innovations are often approved on relatively limited clinical evidence. Unlike therapies for more prevalent conditions, orphan medicines frequently reach the market based on:

Small patient populations
Single-arm clinical trials
Surrogate endpoints
Short follow-up periods
Limited long-term safety data
Uncertainty around treatment durability

These limitations reflect the realities of studying very small patient populations where conducting large, randomised clinical trials is often impossible. However, the uncertainty does not disappear once regulatory approval is achieved.

The questions regulators ask are no longer enough

Regulatory approval demonstrates that a medicine's benefits outweigh its risks. It does not necessarily answer the questions being asked by health technology assessment (HTA) agencies, reimbursement bodies and healthcare payers.

They increasingly want to know:

Does the treatment continue to work five years later?
Does it improve quality of life?
Does it reduce hospital admissions?
Does it delay disease progression in routine clinical practice?
Does it justify the expected value/price over time?

For many orphan medicines, those answers simply do not exist at launch. As a result, post-approval evidence generation has become an essential part of the commercialisation strategy.

MAPs are uniquely positioned to support RWE generation

MAPs occupy a unique position within the product lifecycle - providing treatment during the period between regulatory approval and reimbursement, or before commercial availability in individual countries.

Historically, the focus during this period has been operational:

Identifying eligible patients
Managing approvals
Coordinating supply
Maintaining compliance
Recording adverse events

Today, there is an opportunity to achieve considerably more. Every patient enrolled in a well-designed MAP represents an opportunity to generate structured, high-quality real-world evidence.

That evidence can include:

Long-term safety
Durability of response
Patient-reported outcomes
Quality of life
Healthcare resource utilisation
Treatment persistence
Natural history comparisons
Country-specific clinical experience

For therapies developed in very small patient populations, these data can become some of the most valuable evidence generated throughout the product lifecycle.

Momentum around RWE is building

Across Europe and internationally, regulators, HTA organisations and policymakers are placing increasing emphasis on real-world evidence to address the uncertainty that inevitably surrounds medicines for rare diseases:

The European Medicines Agency's DARWIN EU initiative has expanded the routine use of real-world data (RWD) in regulatory decision-making.
The European Parliament has highlighted the importance of patient registries, interoperable datasets and coordinated evidence generation as essential components of future rare disease policy.
Health Technology Assessment International (HTAi) has recognised that evidence uncertainty is inherent to orphan medicines and that decision-making increasingly depends on integrating real-world evidence alongside clinical trial data.

Industry studies have similarly shown that reimbursement submissions for orphan medicines increasingly incorporate real-world evidence to support pricing and access decisions.

The question is no longer whether real-world evidence should be collected, rather it is how to collect it effectively and without introducing additional administrative burden on both pharma and Healthcare Professionals (HCPs).

The operational opportunity

Unfortunately, many Managed Access Programs still rely on fragmented operational models.

Evidence collection is frequently managed through:

Spreadsheets
Separate electronic data capture systems
Individual patient registries
Multiple logistics providers
Standalone safety databases
Independent research platforms

This fragmentation creates familiar problems with inconsistent data, variable processes between countries and difficulty with full program oversight. HCPs in particular, find the process onerous with multiple sign-ons to multiple inconsistent systems. Evidence collection is treated as an additional project rather than an integrated component of program delivery. The aim should be to make evidence capture easier for healthcare professionals by embedding it into routine program workflows

Perhaps most importantly, valuable opportunities to generate meaningful evidence are often missed entirely.

Evidence-ready from the outset

Collecting high-quality evidence requires more than asking HCPs to complete additional forms. It requires deliberate program design with evidence objectives established before the first patient is enrolled.

Data collection should be proportionate, clinically meaningful and operationally sustainable with governance that ensures consistency across countries, vendors and participating centres.

Rather than evidence generation creating additional administrative burden, the operating system for the Managed Access Program should support this. Managed Access Programs should be evidence-ready by design.

A new role for Managed Access technology

This changing environment also changes what pharmaceutical companies should expect from technology. Historically, Managed Access platforms have focused on operational administration but the new generation of Managed Access Operating Systems need to support something broader.

They must provide the infrastructure to:

Govern programs consistently across countries
Coordinate multiple delivery partners
Maintain regulatory compliance
Capture structured operational and clinical data
Enable longitudinal follow-up
Support future evidence generation
Provide portfolio-level visibility across global programs

Managed Access needs to become an enterprise capability that connects patient access, operational governance and evidence generation.

Looking ahead

Therapies for rare diseases will continue to push the boundaries of science - and uncertainty will remain an inevitable part of drug development. The organisations that succeed will not be those that get their medicines to patients quickest but those that are able to reduce uncertainty. That reality should not be seen only as a challenge. It is also an opportunity to design better evidence strategies from the outset

From Operational Access to Strategic Advantage: How MFN Pricing is Reshaping Managed Access Programs

romina.oxborough@maprium.com (Dr Romina Oxborough) — Thu, 04 Jun 2026 16:52:48 GMT

One of the most significant consequences of the Trump administration’s Most-Favored Nation policy is the growing recognition that patient access programs play a strategic role within the broader pharmaceutical access ecosystem. Every decision regarding access now has greater potential implications for pricing strategy, launch sequencing, reimbursement negotiations and international reference pricing exposure.

A delayed commercial launch may increase demand for managed access. A managed access program may generate real-world evidence that supports reimbursement. A reimbursement decision in one market may influence pricing negotiations elsewhere. These activities are becoming increasingly interconnected, creating opportunities for more integrated and effective access strategies.

As a result, pharmaceutical companies have an opportunity to rethink the role of Managed Access Programs. Rather than serving purely as a mechanism for supplying treatment prior to commercial availability, MAPs are becoming an integral component of broader global access strategies.

This evolution creates an opportunity for organisations to align multiple priorities more effectively:

ensuring patients receive timely access to treatment;
protecting pricing and reimbursement strategies;
maintaining regulatory and compliance standards;
generating meaningful real-world evidence; and
supporting long-term commercial objectives.

Successfully leveraging these competing demands requires more than operational execution. It requires a strategic approach to access planning that considers the entire product lifecycle and the interdependencies between patient access, pricing and market access. This is where specialist managed access expertise has become vital.

At Maprium, we are seeing growing recognition that access programs should not be designed in isolation from broader commercial and market access considerations. As global pricing pressures intensify and launch strategies become more complex, organisations benefit from partners who understand not only how to deliver access programs efficiently, but also how to align them with evolving global access objectives.

Looking Ahead

One year after the introduction of MFN pricing, its most significant impact may not be on drug prices themselves but on how patients access medicines.

If international reference pricing starts to influence launch sequencing and commercial decision-making, demand for Managed Access Programs is likely to increase. At the same time, manufacturers will face growing pressure to ensure those programs align with broader commercial and pricing objectives.

For pharmaceutical companies, this creates both a challenge and an opportunity.

The challenge is maintaining patient access while navigating increasing pricing scrutiny and commercial complexity.

The opportunity is to use Managed Access Programs more strategically—to support patients, generate evidence, inform market access planning and bridge access gaps created by evolving pricing environments. The organisations best positioned for this future will be those that recognise patient access, market access and pricing strategy as complementary components of a unified global access cross-functional discipline.

At Maprium, we believe Managed Access Programs are becoming a critical strategic asset within the pharmaceutical access ecosystem. As healthcare systems seek greater affordability and manufacturers adapt to new pricing realities, the ability to design and execute effective global access programs will become increasingly important, as will the ability to use technology to do this.

The future of managed access is unlikely to be defined by whether these programs exist. Instead, it will be defined by how effectively they are integrated into broader global access strategies and how this cross-functional delivery is managed. Organisations will need solutions like Maprium to ensure transparency, visibility and control and to connect access activities with evidence generation within a single framework.

In a world increasingly shaped by international reference pricing, the companies that succeed will be those that treat patient access not as an operational necessity, but as a strategic capability that supports patients, informs market access, strengthens evidence generation and contributes to long-term commercial success.

Sources and further reading

RAND Corporation. Prescription Drug Prices in the U.S. Are 2.78 Times Those in Other Countries. RAND Health Care Research Report, February 2024. Available at: https://www.rand.org/news/press/2024/02/01.html
Assistant Secretary for Planning and Evaluation (ASPE), U.S. Department of Health and Human Services. Comparing Prescription Drugs in the U.S. and Other Countries. January 2024. Available at: https://aspe.hhs.gov/reports/comparing-prescription-drugs
U.S. Department of Health and Human Services (HHS). CMS and HHS Set Most-Favored-Nation Pricing Targets to End Global Freeloading on American Pharmaceutical Innovation. May 2025. Available at: https://www.hhs.gov/press-room/cms-mfn-lower-us-drug-prices.html
Matcha G. The Global Risks of America's "Most-Favored-Nation" Drug Pricing Policy. Petrie-Flom Center, Harvard Law School. May 2025. Available at: https://petrieflom.law.harvard.edu/2025/05/22/the-global-risks-of-americas-most-favored-nation-drug-pricing-policy/
Yadav P, Singh P. Executive Order to Lower U.S. Drug Prices Could Hurt the Poorest Countries. Think Global Health, Council on Foreign Relations. May 2025. Available at: https://www.thinkglobalhealth.org/article/executive-order-lower-us-drug-prices-could-hurt-poorest-countries
Certara. International Reference Pricing: Global Implications and Pharma Strategy. October 2025. Available at: https://www.certara.com/blog/u-s-international-reference-pricing-global-implications-and-pharma-strategy/
Rand LZ, Conti RM. International Reference Pricing for Prescription Drugs in the United States. Journal of Managed Care & Specialty Pharmacy. 2021;27(6):839-842. Available via PubMed: https://pubmed.ncbi.nlm.nih.gov/33840424/
Brookings Institution. International Reference Pricing for Prescription Drugs. July 2025. Available at: https://www.brookings.edu/articles/international-reference-pricing-for-prescription-drugs/
Office of Health Economics (OHE). The Trump Administration's US Drug Pricing Proposal: What Will Happen Next? June 2025. Available at: https://www.ohe.org/insights/the-trump-administrations-us-drug-pricing-proposal-what-will-happen-next/
ISPOR. International Reference Pricing Comes to America: The MFN Policies Explained. Available at: https://www.ispor.org/publications/journals/value-outcomes-spotlight/vos-archives/issue/view/preventive-medicine/international-reference-pricing-comes-to-america--the-mfn-policies-explained