5 Key Trends in Global Market Access

The continued emphasis on rare and highly specialized diseases is perceived as important by U.S. and ex-U.S. payers

• Rare and highly-specialized disease continues to be a key area of focus, with many countries investing significant funding towards supporting rare disease research and manufacturing. Countries are also increasing efforts to optimize policies aimed at ensuring early access to new therapeutics in this space
• In 2018 nearly 28,526 clinical trials internationally studied 1,535 rare diseases, covering a fraction of the estimated total of 7,000+ types of rare diseases to exist
• In July 2024, the FDA announced the launch of a Rare Disease Innovation Hub, which will seek to publish new guidance for reviewing products indicated for rare disease populations
• In Saudi Arabia, although disease registries do not currently exist, rare disease-specific Health Technology Assessments are being considered

Both U.S. and ex-U.S. payers perceive oncology pricing pressures as important, with U.S. payers in particular underscoring movement towards sharing these pressures with institutions

• Entrants of high-cost oncology therapies (particularly cell and gene therapies) serve as major drivers of specialty drug spend, leading U.S. payers to indicate greater budget impact concerns
  • The FDA stated that it expects to approve 10-20 cell and gene therapies per year
• Within the U.S., novel reimbursement approaches and payer-manufacturer relationships drive oncology pricing pressures on institutions
  • Payers are increasingly putting pressures on institutions as a strategy to incentivize them to optimize administration processes through larger reimbursement agreements which are patient-agnostic
• To mitigate budget impact concerns, Germany is implementing a new hospital reimbursement system following the Ministry of Health’s end of the ORTG system​
• In Saudi Arabia’s healthcare transformation plan, special oncology centers are also in development in anticipation of novel oncology entrants

Payers continue to view weight loss management drugs as a trend; however, their perspectives are largely shaped by regulatory approvals and perceptions around chronic disease management

• There is broad uptake of weight loss management drugs on a global scale. Uptake has been directly impacted through broader governmental policies around the reimbursement of obesity-related drugs (e.g., depending on whether obesity is perceived as an indication/chronic disease or as a lifestyle)
• Payers’ perspectives of these medications varied based on their respective country’s regulatory statuses
• U.S. payers perceived weight loss management drugs as particularly impactful in 2024, indicating that uptake of these drugs could exponentially increase should Medicare grant approval
  • In late June, the Treat and Reduce Obesity Act of 2023 passed through the Way and Means committee into the full house, indicating the first step towards Medicare’s potential coverage of weight loss medications
• In contrast, Germany, along with other countries such as the Netherlands, has not approved these medications due to bans on the public reimbursement of “lifestyle” therapies, yielding a lack of perceived impact from German payers

Across all countries, payer opinions are mixed regarding expanding evidence and expectations. Some payers anticipate an increase in evidence and expectations with new entrants in established therapeutic areas while others foresee minimal change to current expectations

• Germany anticipates minimal changes to their evidence expectations given these are already high, whereas China expresses anticipation of moving towards stricter requirements for head-to-head data, robust data and high-quality data in the future​​
  • In China, a new rating system has been developed this year to assess new therapeutic advances, but it does not currently directly tie to reimbursement outcomes
• Some payers see evidence expectations continuing to expand whereas others are less optimistic due to a competing desire to ensure early access to innovative therapeutics, which may lack up-front data. As a result, it is expected that innovative agreements to share risk with manufacturers will become more common
  • These innovative risk-sharing agreements are still in early stages because payers find it difficult to determine, track and enforce the outcomes linked to these agreements
• In light of the IRA and increasing competition (especially in the biosimilar space), U.S. payers underscore indirect linkages to increase evidence expectations for entering products
• With the formal adoption of the joint clinical assessment (JCA), guidance on timelines and steps for conducting EU joint clinical assessments has been published. The implications of this guidance are still unfolding across applicable markets

Movement to in-house patient support programs has grown increasingly popular as manufacturers recognize the benefits of consolidating their patient support programs to improve patient relations and promote better patient adherence

• With increasing evidence of improved patient adherence and patient experiences, more manufacturers have begun moving their patient support programs in-house for increased control over their patient relations
  • Payers believe that low patient participation in support programs is largely due to a lack of awareness and familiarity with these programs. This issue is expected to be addressed by a consolidated manufacturer hub of patient support resources
• Despite over $5B spent on patient support programs annually, pharmaceutical companies have only been able to capture a ~3% patient participation rate
• U.S. payers acknowledge the associated benefits to moving patient support programs in-house but underscore that improved patient adherence is likely to have minimal impact on their management (and would instead translate to HCP utilization implications)​
  • Looking ahead to 2025, as we see further movement towards manufacturers directly running their own patient support programs. Positive impacts on patient adherence and access may translate to increased utilization among HCPs (which, in turn, could lead to preferential management in cases where all other clinical factors may be equal)