Orphan drug development: an economically viable strategy for biopharma R&D.

Orphan drug incentives have stimulated research into diseases with significant unmet medical need. Although the targeting of orphan diseases is seen by industry as an attractive strategy, there are limited economic data available to support its use. In this paper we show that the revenue-generating potential of orphan drugs is as great as for non-orphan drugs, even though patient populations for rare diseases are significantly smaller. Moreover, we suggest that orphan drugs have greater profitability when considered in the full context of developmental drivers including government financial incentives, smaller clinical trial sizes, shorter clinical trial times and higher rates of regulatory success. The data support the targeting of rare diseases as an important component of a successful biopharma R&D strategy.

Meekings KN ，Williams CS ，Arrowsmith JE 《-》

The evolving drug development landscape: from blockbusters to niche busters in the orphan drug space.

Strategy, Management and Health Policy Large pharmaceutical companies have traditionally focused on the development of blockbuster drugs that target disease states with large patient populations. However, with large-scale patent expirations and competition from generics and biosimilars, anemic pipelines, escalating clinical trial costs, and global health-care reform, the blockbuster model has become less viable. Orphan drug initiatives and the incentives accompanied by these have fostered renewed research efforts in the area of rare diseases and have led to the approval of more than 400 orphan products. Despite targeting much smaller patient populations, the revenue-generating potential of orphan drugs has been shown to be huge, with a greater return on investment than non-orphan drugs. The success of these "niche buster" therapeutics has led to a renewed interest from "Big Pharma" in the rare disease landscape. This article reviews the key drivers for orphan drug research and development, their profitability, and issues surrounding the emergence of large pharmaceutical firms into the orphan drug space.

Kumar Kakkar A ，Dahiya N 《-》

Medicines for rare (orphan) diseases: an enquiry and proposal for tax-incentives.

John JE 《-》

[Orphan drugs: drugs for rare diseases].

Schenk M 《-》

Raising orphans: how clinical development programs of drugs for rare and common diseases are different.

Orfali M ，Feldman L ，Bhattacharjee V ，Harkins P ，Kadam S ，Lo C ，Ravi M ，Shringarpure DT ，Mardekian J ，Cassino C ，Coté T ... -  《-》