A Bird’s Eye View Of The Rare Disease R&D Landscape
The number of drugs in development for rare diseases have increased by 56%, with the number of rare diseases on the development map also increasing by 23% in just three years, demonstrating ongoing interest and seemingly increased enthusiasm for research within these areas of high unmet needs.
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Sweden’s Swedish Orphan Biovitrum and Italy’s Recordati are looking towards hemophilia and rare diseases to keep up the growth of their businesses, and Q3 results have highlighted the Swedish company in particular as a potential takeover target.
Slowly but surely, gene therapy projects are winning back confidence from the industry and as investment increases, so increase the reports on progress in this area; from sensational claims of restoring sight, to slightly more spurious claims of reversing the aging process.
Pharmaceutical companies, payers and other stakeholders need to work more collaboratively if the problems surrounding the pricing and reimbursement of orphan medicines are to be tackled successfully in Europe, says the rare disease organization EURORDIS.