FDA Introduces New Options for Patients in Observance of Rare Disease Day


Several new approaches may increase drug development efforts for rare diseases.

Approximately 1 in 10 Americans has a rare disease, including cancers or genetic metabolic conditions. These conditions largely affect pediatric patients.

Today, February 28, 2018, is Rare Disease Day, a date set aside to raise awareness about the more than 7000 diseases that affect millions of Americans. Growing awareness is crucial because many rare diseases do not have treatment options and if they do, the drugs are costly.

For many of the conditions, developing a treatment can be difficult and present significant challenges, according to a blog post from FDA Commissioner Scott Gottlieb, MD.

Successful orphan drug development requires collaboration between researchers, drug sponsors, regulators, lawmakers, and patient advocacy groups, Dr Gottlieb wrote.

Related Coverage: How Have Orphan Drugs Changed the Specialty Landscape Over the Past Few Years?

In observance of Rare Disease Day, the FDA reviews the progress it has made thus far and determines what more can be done in the future to facilitate accelerating drug approvals, according to the agency.

The Orphan Drug Act was enacted in 1983 as an effort to spark interest in rare disease drug development. Legislation passed in 1990 also created the Humanitarian Device Exemption, which created a path for medical devices for patients with rare diseases.

Currently, there are more than 650 drugs indicated for rare diseases and 72 medical devices that have gained orphan indication. Prior to this legislation, Dr Gottlieb said there was a lack of drugs and biologics for this population.

Due to the growing interest in personalized medicine, applications for rare disease treatments have increased, reaching 700 submissions in 2017, according to the post. Also, 80 treatments were approved under the orphan drug pathway in 2017.

Related Coverage: What Was the Most Notable Orphan Drug Approval in 2017?

Dr Gottlieb wrote that there has been significant progress for ultra-orphan diseases as well.

Although great strides have been made, more efforts are needed to address the unmet needs of thousands of patients without treatments. A recent FDA survey indicated that innovative medical devices are also needed for pediatric and adult patients with rare diseases, according to the post.

In June 2017, the FDA announced the Orphan Drug Designation Modernization Plan, which is an effort to develop a more efficient and modern approach to rare disease drug approvals. The agency’s Orphan Drug Grants Program recently provided $17 million in funding to support clinical trials, according to Dr Gottlieb.

For Rare Disease Day, the FDA also announced multiple new actions to demonstrate its commitment to developing rare disease drugs and devices.

The agency launched a novel pilot to streamline orphan designation requests to make it easier for applicants and reviewers. This program also includes a tutorial and standardizes FDA communication processes, according to the post.

Further, the FDA entered into a new Memorandum of Understanding with the National Organization for Rare Disorders to conduct outreach for strategies that incorporate patient experience into regulatory decisions. Dr Gottlieb noted that early engagement can improve clinical and regulatory understanding of rare diseases, while also providing a more in-depth understanding of urgent patient needs and informing drug development.

Lastly, the agency is planning a meeting that will help prepare for changes in orphan drug development caused by the growth in targeted therapies and molecularly-defined conditions, according to the post. The FDA is also seeking input on scientific and regulatory issues related to targeted cancer therapies.

“Over the course of 2018 we’ll continue our efforts to increase the consistency and efficiency of our reviews of rare disease products,” Dr Gottlieb wrote. “We remain committed to supporting rare disease research on diagnostics, therapies, and potential cures.”

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