2025 Market Access Check-In: Evaluating the Impact of Major P&MA Trends 

2025 was a pivotal year for market access, marked by the growing convergence between scientific innovation, regulatory reform, and pricing policies to ensure financial sustainability. European health systems are moving from fragmented to more coordinated approaches for assessing value and managing costs. At the same time, advances in technology, data, and therapeutic innovation are reshaping how evidence is generated and used. . The developments seen this year—including the implementation of the JCA, the growing recognition of real-world evidence (RWE) as a complementary source of value, and emerging approaches for managing price and access for high-cost therapies—suggest that the sector is undergoing a period of transition. Clear themes emerging this year include the balancing of innovation with affordability, and evidence with equity.  

Joint Clinical Assessments (JCA) is Here to Stay 

2025 marked the long-awaited launch of the Joint Clinical Assessment (JCA) process, a significant step in harmonising HTAs throughout the EU-27 since the EUnetHTA project was launched in 2006.¹ . A key objective of the EU HTAR is to improve efficiency and equity of access by offering a single, centralised approach to clinical evaluation that adheres to the evidence requirements of all EU member states. This harmonised approach has prompted a shift in how manufacturers and payers approach evidence generation and launch sequencing in Europe. 

Early Implementation and JCA Reality Check 

The JCA process launched on January 12^th, 2025, with new oncology and advanced therapy medicinal products (ATMPs) being the first to go though the new process. 17 JCAs for cancer medicines and 8 JCAs for ATMPs were predicted in 2025.² However, as the year closes, this number is slightly lower at 9 JCAs for cancer medicines (one being an ATMP) and just one additional ATMP assessment. The first wave included an ATMP for melanoma, AMTAGVI,³ and a cancer therapy called Tovorafenib; the most recent addition is Relacorilant, another cancer therapy.⁴ This lower-than-expected volume of submissions may be due to timing, with manufacturers potentially delaying applications until the first JCA reports are published, allowing them to learn from precedents before submitting their own dossiers. This is in line with the limited capacity⁵ of the Joint Scientific Consultation (JSC), a process that provides free consultation/advice to health technology developers for the evidence needed to have a successful JCA. There are also defined submission windows, which may be slowing down the overall pipeline.⁶ 

Key learnings from the JCA  

The first outcomes of the JCA have not yet been published. Despite this, there has been meaningful progress from the introduction of the JCA in 2025: 

• The introduction of the HTA-IT platform has streamlined dossier management, reducing errors and inefficiencies. It provides a single, standardised submission route for health technology developers, rather than multiple national applications.⁷   

• New dossiers now have a template and standardised guidance with file structure and naming conventions, ensuring consistency across submissions. Something that was not seen under the previous non-mandatory EUnetHTA 21.   

• Guidance on filling in the joint clinical assessment (JCA) dossier template – Medicinal products – Public Health 

• We now understand that manufacturers can take a more pragmatic approach to evidence generation for JCA dossiers. Guidance states that ‘the data presented can be limited to the evidence which provides the highest certainty on the relative effects of the new medicinal product versus the comparator’,⁸  

What’s Next 

While the JCA offers the potential for greater efficiency and improved patient access, its ultimate effect on national access pathways remains uncertain. Much will depend on how effectively it balances EU-wide harmonisation with the autonomy of individual member states in pricing and reimbursement. As the JCA matures, stakeholders should continue to monitor early JCA reports to anticipate how member state input impacts PICO predictions, and as a result, influence expectations for evidence generation and value dossier formatting. Also, engaging with JSCs early on to align evidence strategies will remain crucial. 

Navigating Rare Disease Uncertainties 

Emerging Gene-Editing Therapies

In 2025, NICE approved exa-cel for NHS use in England, a one-time £1.65 million therapy for sickle-cell disease and β-thalassaemia^9,10 prompting payer scrutiny around whether short-term efficacy could reasonably be extrapolated to lifelong remission and thus justify such a substantial upfront cost. In contrast, the US approval of k-abe, the first in vivo CRISPR therapy used under compassionate grounds for a single patient with CPS1 deficiency¹¹, raises a different set of questions: how to regulate, evaluate, and reimburse n=1 genetic interventions. Together, these cases highlight the widening spectrum of market-access challenges posed by advanced therapies and illustrate how innovation is evolving faster than current assessment and reimbursement models.  

Evolving UK Rare Disease Landscape 

Still on the topic of rare diseases, the UK Rare Disease Framework has continued its efforts to deliver on four priorities: faster diagnosis, improved clinician awareness, better coordination of care, and broader access to advanced therapies. In 2025, there were three critical actions added to this framework:¹² 

1. Integrated multi-system disorder clinics aim to reduce the need for multiple appointments. 

2. MHRA-led reforms under the Medicines for Human Use Regulations 2024 to streamline and speed up approvals for rare-disease trials. 

3. The development of an NHS framework for delivering personalised genetic therapies by building infrastructure for single, high-value interventions.   

These measures demonstrate policymakers’ continued commitment to strengthening the UK’s ability to deliver treatments for rare diseases. However, meaningful improvements in patient access will ultimately depend on whether therapies can meet the UK’s stringent cost-effectiveness requirements, which is still the key hurdle for rare disease medicines. The extent to which these initiatives will translate into real-world access therefore remains to be seen. 

Rare Diseases Reshaping Clinical Trials 

Decentralised clinical trials (DCTs) were once a pandemic necessity, now DCTs are increasingly being seen as a high value for research on ultra-rare diseases¹³. DCTs have been shown to: 

• Halve recruitment times and cut per patient costs from $15-30k to $ 10-20 k.¹⁴ 

• Improve patient retention by 35% and achieve a 92% patient satisfaction rating.¹⁴  

• Increasingly being accepted by regulators. For instance, the FDA issued new guidance on conducting trials using decentralised elements,¹⁵ and the EMA released papers recommending their use across Europe.¹⁶ 

Indeed, 68% of new trials now incorporate DCT elements, which can take the form of electronic patient-reported outcomes or telemedicine platforms.¹⁴ DCTs can broaden global reach and enable continuous real-world data collection, reducing both financial and evidentiary barriers.   

Real World Evidence (RWE) – Tangible as a Complementary Source of Evidence 

RWE has been transformed from a complementary data source into a vital tool for market access. By demonstrating real-life effectiveness, supporting value-based pricing, and showcasing the durability of outcomes, RWE is increasingly playing a pivotal role in securing reimbursement for promising innovative treatments with immature data packages.  

Evolving Role of RWE in Rare Diseases

Between 2014 and 2025, RWE played a key role in securing full reimbursement in 7 out of 16 European pricing and reimbursement cases for orphan medicines and provided supporting input in conditional agreements for the remaining cases.¹⁸ However, agencies acceptance and use of RWE are variable: 

• NICE and AEMPS tend to accept external comparators and natural history data. 

• AIFA tends to rely on RWE primarily for outcome-based agreements. 

• HAS uses RWE to inform re-evaluations. 

• G-BA, by contrast, has stricter with data collection systems,¹⁹ so it limits RWE use mainly to ultra-rare diseases.¹⁸ 

Despite these disparities, we could be seeing the role of RWE converging between European markets as JCA drives further harmonisation.¹⁸   

Globally, regulatory openness to RWE is also growing both the FDA and EMA now accept real-world studies in support of approvals and label extensions, under initiatives such as the FDA’s Real-World Evidence Framework and EMA’s Adaptive Pathways Pilot.²⁰  

NICE Redefining RWE Framework 

NICE’s 2025 update to its Real-World Evidence (RWE) Framework¹⁷ in particular marks a shift toward treating RWE as a strategic, rather than supplementary, evidence source. This reflects a broader trend toward greater rigour and transparency in the use of real-world data for HTA.  

The updated framework strengthens methodological standards by providing detailed guidance on validating and reporting free-text electronic health record data, and by introducing a reporting checklist for target trial emulations to improve transparency, peer review and interpretation. These measures enhance the credibility of RWE and increase confidence among regulators, clinicians, researchers and patients. However, uncertainties remain, particularly around the variability in data quality across systems, the ability of developers to meet these higher standards, and how quickly improved RWE practices will translate into smoother appraisals or greater acceptance in areas with limited clinical evidence. 

Increasing role of AI in P&MA 

In 2025, national pricing and reimbursement agencies began issuing targeted guidance to address the growing role of artificial intelligence (AI) in healthcare decision-making. For instance, in 2025 we saw AI tools starting to directly influence market access outcomes through their use in real-world data analysis, literature screening, and evidence generation, prompting several national P&R agencies to issue guidance on its use.²¹

GLP-1 Drugs 

Demand Spike Has Increased Pressure on Payer Budgets 

GLP-1 drugs were once exclusively used for diabetes; however, in 2025, they took centre stage in market access debates as payers grappled with whether to and how to reimburse them for obesity. The challenge has been accentuated due to the larger than expected population seeking treatment, limited long-term clinical data and the potential for high budget-impact. Although demand is growing through various channels, these clinical and economic uncertainties mean that payers remain cautious about allowing expanded reimbursed access.^25,29 

This pressure is further intensified by three interconnected trends:  

1. Oral GLP-1s – this potential shift from weekly injectables to convenient pills promises greater uptake. Products such as Orforglipron are currently in Phase 3 trials for both obesity and diabetes and will be submitted for regulatory approval in late 2025 and 2026.²⁶ This will alleviate supply constraints on injectables, but the net effect will be faster volume growth, not cost relief.  

2. Label Expansion – the ELAD trial tested liraglutide, a GLP-1 used for both diabetes and obesity. While the primary end point of improving brain sugar use was not met, secondary results showed a slower mental decline and less brain shrinkage, showing potential for these drugs to be used against early-stage Alzheimer’s. The EVOKE studies are building on these using semaglutide, which is currently in phase 3 clinical trials²⁷ with data to be analysed and released towards the end of 2025 or early 2026.²⁸  

Spain, Brazil, Denmark, and the UK have all noted concerns within their national HTA reports regarding the significant budget impact issues caused by using GLP-1s for obesity. Only the latter two countries produced formal mitigation measures for this issue.³¹  

NICE’s 12-year recommended rollout of tirzepatide enables phased eligibility, capacity building, and RWE generation to handle the initial demand and budget impact from the rollout of this drug.³²  

Denmark’s response to the budget impact concerns was to trial a risk-sharing mechanism with manufacturers, allowing for the limitation of runaway expenditure while ensuring some level of patient access.³¹  

These models signal a new era in obesity treatment reimbursement, where access is deliberately sequenced to protect health-system capacity and manage long-term affordability.  

Will Digital Coaching Ease the Load?  

Stakeholders are showing a growing interest in value-management tools, particularly digital coaching that incorporates flexible medication dosing. This approach has the potential to relieve some of the financial pressures on payers by enhancing patient adherence, maximising health outcomes, and minimising waste.  One notable study demonstrated that combining behavioural support with personalised dosing led to a remarkable 16.7% reduction in weight. Notably, this result aligned with findings from clinical trials, even when using only half of the standard dosage.³⁰ These insights suggest that digital support may not only optimise medication use but also holds promise for reducing overall costs and mitigating budget-impact concerns. 

US Pricing in 2025: Rising Accountability and Implications for Market Access 

The US pharmaceutical landscape in 2025 has been characterised by a clear shift towards greater price transparency and accountability. This shift is driven by the continued implementation of the Inflation Reduction Act (IRA), the introduction of a broad Most-Favoured Nation (MFN) Executive Order (EO), with major policy adjustments intended to balance affordability with innovation incentives. 

IRA Bill Upheld Despite Pushbacks 

Legal challenges have failed to derail the IRA, with courts repeatedly affirming Medicare’s authority to negotiate drug prices. The first set of negotiated prices, covering ten high-expenditure medicines, will take effect in 2026 and reflect an average 38% reduction compared with 2023 list prices. A further fifteen medicines, including high-profile treatments such as Ozempic, entered negotiation in 2025 for 2027 implementation.³³ 

While this signals enduring policy momentum, the 2025 Budget Reconciliation Bill (HR1) narrows the IRA’s scope by limiting which medicines can be selected for negotiation. This pushes the favour slightly back into the hands of manufacturers and protects innovation by ensuring expected returns are not dropping for all drugs. However, this is predicted to increase Medicare spending by at least $5 billion in the long run. 

MFN Executive Order Deepens Price Pressure Across the Globe 

Concurrently, the May 12 2025 executive order on the Most Favoured Nation (MFN) pricing model sought to tie US drug prices to the second lowest country-specific manufacturer-reported net price in peer developed countries, adjusted by gross domestic product per capita using a purchasing power parity method, thereby flattening global price differentials.³⁴ 

These shifts have prompted manufacturers to align drug prices more tightly across markets, accelerate launch sequencing in high-price markets, and rethink how they capture value globally. For example, European list prices are reportedly being increased in some cases to narrow the gap with the US, pre-emptively mitigating MFN exposure.³⁵ 

Strategic Implications for Market Access 

2025 marked a big year for market access. These combined trends illustrate a heightening need for robust value demonstration, supported by emerging approaches in clinical trial design and AI applications. In particular, robust RWE capturing patient-centred outcomes has become essential to justify pricing, support negotiations, and reinforce value arguments for both rare disease therapies and high budget-impact therapies, such as GLP-1s. Lastly, the shift toward heightened US price scrutiny, driven by the MFN policy, signals a renewed need for strategic, price-protective global launch sequencing. These trends have culminated in manufacturers ending the year by reassessing where and when they launch to protect long-term global revenue. 

Looking ahead to 2026

Many of the pressures that defined market access in 2025, from pricing reform and EU HTA implementation to the growing role of RWE and patient voice are set to intensify next year.

We’ve mapped out what this means for evidence, pricing and access in our Market Access & Pricing Trends for 2026. Read HERE

References

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