Portal de Periódicos da CAPES

Orphan Drugs and Their Impact on Pharmaceutical Development

2018; Elsevier BV; Volume: 39; Issue: 6 Linguagem: Inglês

10.1016/j.tips.2018.03.003

1873-3735

Misty M. Attwood, Mathias Rask‐Andersen, Helgi B. Schiöth,

Nuclear Receptors and Signaling

Orphan drugs constitute more than 40% of all innovative pharmaceuticals that have expanded the human drug target landscape from 1983 to 2017. Our dataset encompasses 803 potentially new drug targets in clinical trials, of which 40% are under investigation as potential targets for treatment of rare diseases. Orphan drugs have reached as high as 55% of all FDA approved NMEs in 2014 and have maintained 43% to 44% of NMEs for 2015 to 2017. Biological approvals have come to comprise ∼20% of all newly approved NMEs, with 40% of biological NMEs having orphan designation. Cancer research is one of the primary drivers in pharmaceutical development, and 60% of the approved antineoplastic agents have orphan designations. Cancer development for orphan diseases is returning large sales, with four of the top five approved oncology products having not only orphan designation but also blockbuster status. High levels of productivity, with an increasing number of approvals for new molecular entities (NMEs) by the FDA during the past decade, have coincided with the emergence of innovative drugs for treatments of rare diseases that have utilized the FDA orphan drug program. Since 2000, NMEs with orphan designation encompass a significant portion of approved drugs and constitute about 80% of the approved drugs that have established novel human genome-encoded products in recent years. Biological approvals are also expanding, with 40% of the approved biological agents having orphan designation. This trend illustrates a pivot within the pharmaceutical industry: from research programs that focus on canonical blockbuster indications and targets, towards the establishment of new treatments for rare and difficult to treat diseases. High levels of productivity, with an increasing number of approvals for new molecular entities (NMEs) by the FDA during the past decade, have coincided with the emergence of innovative drugs for treatments of rare diseases that have utilized the FDA orphan drug program. Since 2000, NMEs with orphan designation encompass a significant portion of approved drugs and constitute about 80% of the approved drugs that have established novel human genome-encoded products in recent years. Biological approvals are also expanding, with 40% of the approved biological agents having orphan designation. This trend illustrates a pivot within the pharmaceutical industry: from research programs that focus on canonical blockbuster indications and targets, towards the establishment of new treatments for rare and difficult to treat diseases. Orphan Drugs and Their Impact on Pharmaceutical Development: (Trends in Pharmacological Sciences , 525–535, 2018)Attwood et al.Trends in Pharmacological SciencesOctober 13, 2018In BriefThe authors of this review wish to issue the following corrections: The first line of the main text 'Rare diseases are defined in the US as a disease or condition affecting less than one in 200 000 people' should read 'Rare diseases are defined in the United States as a disease or condition affecting fewer than 200 000 people'. In addition, on page 526, the statement 'For example, one group suggested a distinctly more positive innovative future related to the impact of technologies such as high-throughput screening and human genome sequencing' was incorrectly referenced to [1]. Full-Text PDF