Regulators, industry recognize Rare Disease Day 2018

As part of Rare Disease Day 2018​, the US Food and Drug Administration (FDA) has announced several new actions​ to support the development of rare disease products, including a new pilot that aims to make orphan designation requests more efficient.

The administration is also entering into a new Memorandum of Understanding with the National Organization for Rare Disorders​ (NORD).

Per the announcement, “FDA and NORD will collaborate to promote scientific progress in innovation, patient education, drug safety communications, training, research, including risk evaluation and mitigation strategies (REMS), and adverse event reporting​.”

In 2017, FDA had its most approvals yet for rare disease indications.

“In 2017, there were over 700 requests for designation​,” said Scott Gottlieb, MD, Commissioner of Food and Drugs in a blog post. 

“This was more than double the number of requests received in 2012. Last year we also saw 80 treatments approved by FDA for rare indications, the highest number ever​.”

Looking back at the past year, catch up with some of our coverage of rare disease research and news:​

Patients as partners, communication key to designing rare disease clinical trials​​

Researchers are creating a “roadmap” for nephrology clinical trial design, which stresses the role patients and their families can play in rare disease study design.

How CROs and foundations are partnering to advance drug discovery and development​​

Disease-focused foundations and contract research organizations (CROs) are working together to bridge the gap between drug discovery and development.

PPD combines rare disease and pediatrics in new 'Center of Excellence'​​

The new center of excellence provides PPD clients with a team dedicated to clinical studies in rare disease and pediatric patient populations.

Are we moving fast enough? ARG talks rare disease clinical trials​​

Researchers and patients have more efficient tools for sharing information on rare diseases, and as knowledge continues to spread, rare disease studies will become less rare.

FDA, EMA propose more efficient approach to drug development​​

The US Food and Drug Administration (FDA) has released a new draft guidance as part of its stated commitment to assist sponsors and expedite drug development for rare pediatric diseases.

Brexit bad for orphans: Concerns about cost to rare disease-focused UK pharma​​

Eurodis says Brexit-related costs and admin burden placed on UK pharma may affect availability of drugs for rare diseases and discourage development of such products

Shire: Getting smart with delivery tech key in developing orphan drugs​​

Rare disease therapies need to be administered in a way that suit the patient according to Shire CEO Flemming Ørnskov, who says smart, flexible delivery devices are core to the firm's product development strategy.