Report: Trends and challenges in rare diseases

The Access Insights 2024 H1 Benchmarking Report polled audience members from January 2024 through July 2024 at Access events on various topics. Here are the results for rare diseases.

If considering pipeline development strategy adjustment, 36% of delegates surveyed emphasized eliminating therapeutic areas that are not a part of their strategic development roadmap as a tactic in their new approach, while 29% said they would increase their focus on pharma solutions for targeted therapeutic areas.

Other key tactics include:

• (17%) increased focus on acquiring assets in high-growth, low-competition landscape; and
• (14%) reduction in R&D in the number of therapeutic areas and drug classes being developed.

When asked about the most important area of interest for the industry to tackle, 50% of audience members voted reduce barriers to research participation and improve data landscape, while 30% chose increase equitable access to genomic sequencing. Only 12% said invest in accessible and culturally relevant resource, according to the survey.

The polled found the main challenges in ensuring patient access to rare disease medications are lack of medical and pharmacy staff to provide patient education, issues with cost support, signatures, high PAP utilization, and location proximity affecting access.

Source: H1 Access USA conference session data and audience polling from January 2024 through July 2024

DepositPhotos/photoraidz