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  1. FDA's Rare Disease Day

Helping Expand Access to Rare Disease Treatments

RADM Denise Hinton
Acting Chief Scientist

photo of Denise HintonIn her role as FDA’s acting chief scientist, Rear Admiral Denise Hinton has many responsibilities.  But few subjects are more critical to her than the work she and her colleagues do to help FDA expand access to safe and effective treatment options for patients with rare, debilitating, and sometimes fatal diseases.

As a scientist, she’s seen the struggles that patients face in the search for treatments and cures.  But as the niece of someone who faced recurring Gastrointestinal Stromal Tumors (GIST), Hinton learned first-hand that at least as important as bringing resources to research treatments for these rare diseases is making sure that patients can navigate the treatment process, that they are educated about the disease, and that they are connected with patient advocates and the companies who are doing important research and conducting clinical trials on the disease.

This understanding is why Hinton is especially excited about FDA’s recent expansion of its Expanded Access Navigator tool disclaimer icon. The Navigator is a comprehensive online information resource maintained by the nonprofit Reagan-Udall Foundation, and is designed to facilitate pre-approval access to drugs to those not eligible for any clinical trials.   This important tool was initially used for drugs that treat cancer, but the new expanded format means it can now be applied to any drugs that treat orphan diseases.

Widening the scope of the Navigator program increases the opportunity for patients to get access to promising treatments though clinical trials when these treatments are otherwise unavailable, and it helps advance the development of new drugs in a more accessible manner and collaborative manner.

This approach is familiar territory for Hinton, who previously served as Deputy Director of the Office of Medical Policy in FDA’s Center for Drug Evaluation and Research (CDER), where she focused on developing, coordinating, and implementing medical policy programs and strategic initiatives, including the integration of rapidly-evolving science and new technologies into the drug development and regulatory review processes.

As she explained, "Navigating the terrain of treatment and options in the world of rare diseases can be very challenging for the patient or lay person.  It’s important that we build a community to support them and make sure that the necessary tools and resources are brought together in the search for answers.  The new improved Navigator is a great achievement that helps us form the basis for even greater collaboration – which means greater promise for treatments and cures."

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