On December 13, 2019, Nature Reviews Drug Discovery published an article that reviews the landscape of the development of drugs for rare diseases, both the progress and challenges. The article was authored by HP&M Attorney James E. Valentine along with co-authors from the BioPontis Alliance for Rare Diseases and representatives from NIH, FDA, and EMA, among others.
The article discusses the technological basis and rare disease applicability of today’s main therapeutic modalities, including small molecules, monoclonal antibodies, protein replacement therapies, oligonucleotides and gene and cell therapies, as well as drug repurposing. The article also discusses selected overarching topics in the development of therapies for rare diseases, such as approval statistics, engagement of patients in the process, regulatory pathways and digital tools (topics commonly covered on this blog, e.g. on gene therapies clinical trials here). This article serves as an important resource for anyone interested in the research and development of therapies for rare diseases, inspiring interest in funding and nurturing a culture that will lead to innovations that will alleviate the suffering of the very large number of patients affected by rare diseases in the U.S. and globally.
HP&M has a long legacy of thought leadership in rare disease product development, particularly in the application of the FDA regulatory framework to the review and approval of these products. For example, HP&M’s Frank J. Sasinowski authored the 2012 seminal report on FDA’s flexibility in orphan drug approvals, and he and James co-authored the 2015 update (see coverage here).
Frank, James, and colleagues are currently drafting a second update, so our blog readers should stay tuned!