Rare Disease Day is always the last day of February, but this year the date falls on the quadrennial-only Feb. 29, making the day truly rare. And pharma companies and advocacy groups are making the most of it.
Founded by Eurodis 13 years ago, Rare Disease Day is meant to raise awareness for the more than 6,000 known rare diseases and help foster action. Only 5% of all rare diseases currently have FDA-approved treatments, although interest in the segment is higher than ever. According to EvaluatePharma, drugs designated as orphan drugs will grow by 12.3% per year and top $242 billion in sales by 2024.
Biopharma companies including Spark Therapeutics and Catalyst Pharmaceuticals plan to join the National Organization for Rare Disorders (NORD), which sponsors the day in the U.S., in its “Show Your Stripes” campaign—zebras are used in medicine to refer to rare disease—with activities and events including wearing stripes on the 29th.
Takeda, which bulked up its rare disease business by acquiring Shire in 2018, plans to celebrate the day by launching its “Constellation of Rare Stars,” highlighting the overall global impact. The effort shares the stories of 29 patients, families, caregivers, health care professionals and community members who work to reframe the thinking of rare disease and will be shared across social media.
In Ireland, BioMarin Pharmaceutical commissioned a new "Tree of Life" sculpture for its site in Cork to signify the company’s commitment to supporting rare disease patients. BioMarin already has a similar installation at its U.S. headquarters.
MassBio, the Massachusetts life science advocacy group, hosts a panel discussion that includes executives from Astellas, Blueprint Medicines, X4 Pharmaceuticals and Fulcrum Therapeutics. The day ends with an invitation to participate in a Sanofi Genzyme-sponsored Rare Disease Day run and walk on the Boston Common.
Also in Boston, Havas Health & You agency H4B Boston and joint data analytics venture HVH Precision Analytics gathered rare disease stakeholders, including Ipsen Pharma and advocacy groups Beyond the Diagnosis and Global Genes.
HB4 is working with Ipsen on its retinoic acid receptor gamma agonist treatment palovarotene, although the drug recently faced a pair of setbacks with a partial clinical hold and failed futility analysis.
“It’s a good opportunity to have a conversation about the things we need to think about when we’re marketing, servicing, supporting the rare disease community,” Leigh Hartnagel Hatcher, managing director H4B Boston, said. “Rare disease isn’t so rare anymore. More and more traditional Big Pharma companies are getting into the rare-disease market, but outside (the rare disease community) awareness, and even inside the healthcare professional community, is still low.”