Trinity, a leading global life sciences consulting firm, today announced, at the NORD Rare Disease and Orphan Products Breakthrough Summit in Washington, D.C., a new whitepaper titled ‘Assessing Value in Ultra-Orphan Markets’.
In the last five to 10 years, the concentration on orphan development has exploded. From acquisitions to creation of separate rare disease units, pharma companies of all sizes have an increased focus on the orphan market. Yet, while rare diseases have the attention of the industry and the incentives continue to be strong, finding success in the orphan market is not easy. This new white paper outlines the crucial clinical and commercial attributes that drive success for ultra-orphan, non-oncology products.
Key insights include:
- Potential products (to acquire or for portfolio planning) must first be assessed on several different commercial and clinical criteria specific to ultra-orphan markets.
- Success in the ultra-orphan market does not follow one formula. Soliris (Alexion) and KUVAN (BioMarin) are two assets that score high with regard to clinical and commercial opportunity, yet followed very different paths to success.
- The complexities surrounding success in the ultra-orphan market will continue to grow as competition rises alongside increased payer and pricing sensitivity.
“There are more than 7,000 rare diseases with only 500 approved treatments, which is a clear indicator of a large unmet need and opportunity for success in the orphan market space,” said Herman Sanchez, Partner, Trinity, and lead author of the report. “However, being aware that there is a large market opportunity and truly realizing that opportunity are very different things. Our whitepaper outlines key insights and findings based on our deep commercialization experience in the market, providing companies with a proven roadmap to recognize the critical metrics that determine success and the efforts needed to achieve them.”
Trinity, headquartered in Waltham, MA, with additional offices in San Francisco, New York City and Princeton, New Jersey, has extensive experience in orphan and ultra-orphan markets globally including NA, EUMEA, LATAM and APAC. The firm has more than 150 employees and has worked with six of the top 12 global pharmaceutical companies and 12 of the top 20 specialty and biopharmaceutical companies.
“The industry is driving to more targeted therapies, including new and exciting gene therapies, and many of them could become success stories with the right considerations. We’ve worked with many companies in the U.S. and around the globe on their commercialization strategies and are excited to share key learnings in our new whitepaper,” added Neal Dunn, Partner, Trinity and co-author of the whitepaper.
NORD’s Rare Diseases and Orphan Products Breakthrough Summit is the largest and most meaningful multi-stakeholder event of its kind – featuring more than 20 speakers from the FDA, participation from over 80 patient organizations and the Pharma/Biotech industry’s foremost experts in orphan product innovation, investment and commercialization.