-
The 2025 New England Rare Disease Statistics (NERDS) workshop, held on October 9-10 in Boston, brought together leading biostatisticians, data scientists, clinical researchers, and regulatory professionals from academia and industry. This workshop served as an important forum for exchanging innovative ideas aimed at addressing the unique statistical and regulatory challenges inherent to rare disease drug research and development. At PROMETRIKA, we continue to advance collective expertise and strengthen our contributions to successful clinical development.
-
Team members at PROMETRIKA are encouraged to stay abreast of scientific and regulatory advancements in biopharma by attending industry conferences. Earlier this year, I took this opportunity to attend the World Orphan Drug Congress 2025 in Boston. Some of the brightest minds in biotech, pharma, patient advocacy, and regulatory sciences met to discuss and brainstorm around one of healthcare’s most complex challenges: rare diseases. Urgency and innovation were highlighted through powerful keynote speeches, deeply personal fireside chats, and content rich panels focused on everything from patient identification to artificial intelligence in clinical care. I’d like to share some highlights from my experience and the sessions I joined.
-
PROMETRIKA was one of the proud sponsors of the MassBio Rare Disease Day Forum in Boston. Several of PROMETRIKA’s team members from Clinical Operations, Biostatistics, and Project Management came away from the event with new insights and increased dedication to contributing in the rare disease space.
-
Over the past 15 – 20 years, projected peak sales and return on investment (ROI) of new drugs has been shrinking (source: Statista and Deloitte Centre for Health Solutions). To offset this, pharmaceutical companies are pushing to get more drugs to market and multiple indications for each agent.
-
Home visits as part of clinical research have accelerated in recent years as we strive to find the right balance between facilitating study participation while accommodating participant’s busy everyday lives. Clinical trials in rare diseases are even more challenging than trials in other diseases due to a number of factors:
- Small number of eligible trial participants
- Complicated by heterogeneity among rare disease patients
- Most have no cure and manifest at a young age
- Less than 10% of rare diseases have a specific treatment
- Many have other debilitating conditions / physical limitations making it difficult to attend frequent study visits
The combination of home study visits and the right technology removes barriers to optimal patient recruitment, compliance and retention.
THOUGHT LEADERSHIP
