Orphan Drugs

This area is one of the largest areas of research in the pharmaceutical industry and Cornerstone has had the good fortune to be involved in market access support for many of the world’s most prominent orphan drugs.

The costs associated with the development of all pharmaceutical products – orphan or otherwise – are immense. However, the per-patient prices of orphan therapies are commonly higher than those of non-orphan drugs due to several factors:

  • The small market for orphan drugs typically demands higher pricing to allow sustainable return on investment
  • The vast majority of orphan diseases are genetically inherited and the continual discovery of new target genes contributes to high research costs
  • Material and/or production costs may be higher for some orphan drugs, such as biologicals/enzyme replacement therapies
  • Investment into clinician awareness may be required, both to promote recognition of the orphan disease and the availability of novel and potentially life-saving treatments

It is well-recognized that the high costs associated with orphan drugs present challenges to their funding; yet for this same reason, patients are typically unable to afford these therapies unless they are reimbursed. While a number of government programs currently promote the development of orphan therapies, these efforts are inconsequential unless there is an additional commitment to make these drugs available and affordable to patients in need.

Cornerstone has experience with a number of orphan products. We have developed global value dossiers, global and local models, payer detailers, product monographs, and slide decks that have strategically supported their adoption and reimbursement.

  • Bronchietisis
  • Cryopyrin-Associated Periodic Syndromes
  • Cystic Fibrosis
  • Fabry disease
  • Dupuytren’s Contracture
  • Familial amyloid polyneuropathy
  • Homozygous familial hypercholesterolemia
  • Myelodysplasic syndrome
  • Myelofibrosis
  • Myeloma kidney
  • Multiple myeloma
  • Pompe disease
  • Systemic juvenile idiopathic arthritis

Contact us for more information regarding our experience or for support in demonstrating the value of your orphan product.

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