Genetic Therapy and the Industrial Uncertainties

Last week, EMA approved Strimvelis for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). It is the second genetic therapy approved by EMA after Glybera was cleared in 2012. The usual discussion goes around rarity, investment and pricing, which have been set at very high levels with a few unknowns on efficacy and long term safety. But it has to raise a parallel question, from the industrial and economic perspective.

hitt col·labora amb el Col·legi de Metges de Barcelona pel desenvolupament d’un estudi sobre malalties rares

El Col·legi de Metges de Barcelona (COMB) i Health Innovation Technology Transfer (HITT) han signat un acord de col·laboració per a la realització de l’estudi “La valoració del finançament de les malalties rares”, amb l’objectiu de disposar d’una eina que ajudi al desenvolupament de millors i més adequades polítiques de salut en aquest àmbit.

Rare Diseases – Innovation in Funding & Reimbursement, and Insight into Societal Preferences.

Despite the continuing progress made in the R&D of orphan drugs to diagnose and treat rare diseases, the issue of rarity continues to ignite controversies and debates on the scientific, economic, and political grounds. While great efforts are being made both nationally and internationally to collaborate and advance in our knowledge and policies regarding rare diseases, little – albeit growing – attention is paid to societies’ values and preferences when it comes to the trade-offs to be made when allocating public resources.