Advocacy Groups Ask FDA to Allow Patients with Rare Diseases to Give More Drug, Treatment Input

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In an effort to empower people who have rare diseases, 30 advocacy groups are asking the Food and Drug Administration (FDA) for more opportunities to provide feedback on important decisions involving the risks and testing of new drugs or treatments.

The groups are aiming for more communication between patients and medical reviewers at the FDA as they are determining risk tolerance and product applications for rare diseases.

Unlike patients battling more common diseases, people with rare conditions are often limited in their treatment and clinical trial options because of regulations on the amount of risk allowed for experimental treatments and research. This may be true for those diagnosed with mesothelioma. Between 2,000 and 3,000 new cases of mesothelioma are diagnosed each year in the United States. It represents 0.3 percent of all cancers, and there is no known cure.

The lobbying initiative, which is starting with a group letter to high-level officials at the FDA, includes organizations like the National Organization for Rare Diseases (NORD). It is a follow-up to a series of discussions between patient advocates and FDA officials.

“Patients need to have opportunities to communicate with FDA medical reviewers on the risks they are willing to run in exchange for a potential though unproven benefit,” NORD CEO Peter L. Saltonstall said.

The patient community does have an opportunity to give feedback about policy decisions by the FDA. But sometimes that feedback comes after important risk-tolerance decisions are already made.

“For example, a patient with a serious disease and no approved therapy may have a perception of risk that is very different from that of someone who has treatment alternatives. We believe the FDA can make more informed decisions about investigational products and about which products to approve if they hear directly from patients.”

In its letter, the group called for more “systematic processes” to let patients to participate in critical risk-tolerance decisions with the FDA. The advocates said FDA risk-tolerance policies are made throughout the regulatory life cycle of a new drug or treatment and that patients ought to be able to weigh in at at assigned times and before policies are final.

The organizations outlined two specific milestone periods when the patient community should be involved: when the application for a new investigational drug or device is filed and any time the FDA decides to delay or disapprove a proposed or existing clinical trial.

The groups sent their joint letter to Theresa Mullin, the FDA’s director of planning and informatics, and Jeffrey Shuren, director of the FDA’s Center for Devices and Radiological Health (CDRH).

The groups sent copies to Health and Human Services Secretary Kathleen Sebelius, among others.

Previous meetings between members of the patient advocate groups and the FDA came in April and May, leading to the official proposal and follow up letter.

“We are pleased that the patient community has been able to band together to bring these proposals to the FDA, and that senior FDA officials have been so receptive,” said Diane Dorman, vice president of public policy for NORD.

Some of groups advocate for rare diseases and conditions like Parkinson’s disease, cystinosis, Eosinophilic Disorders, histiocytosis, lymphangiomatosis and Gorham’s Disease, immune deficiency diseases, sarcoma cancers and a host of other unique illnesses.

According to NORD, approximately 30 million Americans with rare diseases are covered by these advocacy groups.


Mark Hall joined the Mesothelioma Center as a writer in 2011. Prior to joining the content team, Mark graduated from the University of Florida and then spent several years writing about business, entrepreneurship and technology for various online publications.

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