The US Food and Drug Administration (FDA) is to support the research of treatment for rare diseases through a new $14m fund.

The money will support 15 research projects that are investigating new ways to tackle rare diseases, defined in the US as conditions that affect less than 200,000 people in the country.

The fund will be administered through the Orphan Products Grants Program, part of the Orphan Drug Act.

This Act was passed in the US to stimulate the development of ‘orphan drugs’ to treat rare conditions – traditionally seen as an unattractive market for pharma companies due to the limited market and complexity of many rare conditions.

Interest has increased substantially in recent years, however, due in part to more attractive regulatory environments in both the United States and the European Union with respective regulators providing incentives, such as tax benefits, fee reductions and an extended period of marketing exclusivity.

These incentives are encouraging companies to invest more in the area and tap the 30 million people in the US that are thought to be affected by at least one rare disease.

The FDA is also investing directly in the area, and its Orphan Products Grants Program has provided more than $300m to fund more than 530 new clinical studies that have supported the marketing approval of 50 products.

The latest round of grants includes funding for projects investigating treatments for cystic fibrosis, sickle cell anaemia, and the vision loss disorder Stargardt disease.

“The FDA is committed to fostering and encouraging the development of products for rare diseases, most of which have no available or adequate treatments,” said Dr Gayatri Rao, director of the FDA’s Office of Orphan Product Development.

“The grants awarded this year support studies in very vulnerable, difficult-to-treat populations who have no available options.”

Read more here.

2 thoughts on “FDA Sets Up New Rare Disease Research Fund”

  1. I’m willing to let you know about Bell’s Palsy that a lot of people don’t Know I’ve had it for 23 yrs. There’s a headache that i get about once or twice per month and my face swell’s and sometimes there is a server pain. My jaw starts to lock if i start to yarn,or laugh that’s very very painful. I which someone would update the system so they know what there up agents.

  2. What most people know it’s a Nerve blocked on either on the right side of the brain which operates the left side of the face. After seeing different nerve DR only one knew what i was going throw. Her son got it around the same time i did and has been suffering with it just like me but if you asked some DR what i just explained The DR would say i was not telling the truth.

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