Innovation and competition in advanced therapy medicinal products.

Abstract

EMBO Mol Med (2019) 11: e9992[OpenUrl][1][FREE Full Text][2] Advanced therapy medicinal products (ATMPs), including gene therapy, cell therapy, and tissue engineering products, represent a paradigm shift in health care as they have great potential for preventing and treating many diseases (Food and Drug Administration (FDA), 2013). By way of example, only 367 (8.0%) of the 4,603 rare diseases and conditions listed by the NIH Genetic and Rare Diseases Information Center had at least one FDA‐approved drug therapy in early 2018. An estimated 3,038 (66.0%) of those rare diseases and conditions are congenital and genetic diseases that could potentially be treated by gene therapy. There are already ATMPs under development to address these and many other unmet medical needs (FDA, 2013; MIT NEWDIGS FoCUS Project, 2017) and for the treatment of prevalent conditions, such as cardiovascular, neurologic, and metabolic diseases. However, the high cost of ATMPs has given rise to concerns about the affordability of these breakthrough therapies for health care systems and patients. Furthermore, the projected increase of ATMP approvals in the upcoming decades will create a significant financial challenge for patients, insurance companies, and public health care schemes. During the past decades, the rise of generic markets for drugs and biologics has helped to drive down the costs for many drugs and other medicinal products; a similar market for generic versions of ATMP could therefore help to make these advanced treatments more affordable. Yet, there are several factors that may hinder a future competitive market for ATMPs and thereby affordability. In the USA and Europe, the regulation of ATMPs falls under the biologic licensing procedures of the FDA and the European Medicines Agency (EMA), respectively. Both agencies have implemented a regulatory framework to expedite the development and approval of ATMPs that address unmet medical needs or provide new therapies … [1]: {openurl}?query=rft.jtitle%253DEMBO%2BMol%2BMed%26rft_id%253Dinfo%253Adoi%252F10.15252%252Femmm.201809992%26rft_id%253Dinfo%253Apmid%252F30770338%26rft.genre%253Darticle%26rft_val_fmt%253Dinfo%253Aofi%252Ffmt%253Akev%253Amtx%253Ajournal%26ctx_ver%253DZ39.88-2004%26url_ver%253DZ39.88-2004%26url_ctx_fmt%253Dinfo%253Aofi%252Ffmt%253Akev%253Amtx%253Actx [2]: /lookup/ijlink?linkType=FULL&journalCode=embomm&resid=11/3/e9992&atom=%2Fembomm%2F11%2F3%2Fe9992.atom