NORD and Other Patient Organizations Send Letter on Risk-Benefit to Senior FDA Officials
WASHINGTON, Oct. 5, 2011 /PRNewswire-USNewswire/ -- The National Organization for Rare Disorders (NORD) and about 30 of its advocacy partners have sent letters to key officials at the Food and Drug Administration (FDA) seeking more frequent and regular opportunities to provide input on decisions related to relative risks and potential benefit for new drugs and medical devices.
The letters are a follow-up to meetings the patient advocates had with senior FDA officials over the past few months. They include specific proposals for enhanced communication between the patient community and FDA to ensure that the voices of patients with chronic and rare diseases are heard in risk-benefit determinations and related policy decisions.
"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," said Peter L. Saltonstall, president and CEO of NORD. "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 that FDA can make more informed decisions about investigational products and about which products to approve if they hear directly from patients."
The letters were sent to Dr. Theresa Mullin, Director of FDA's Department of Planning & Informatics, and Dr. Jeffrey Shuren, Director of FDA's Center for Devices and Radiological Health (CDRH). Copies were sent to Health and Human Services Secretary Kathleen Sebelius, FDA Commissioner Dr. Margaret Hamburg, and Dr. Janet Woodcock, Director of FDA's Center for Drug Evaluation and Research (CDER).
The patient advocates note that, when they met with the FDA officials in April and May of this year, the officials asked them to follow up with a proposal outlining specifics for more frequent and regular communication between the patient community and FDA on benefit-risk determinations. While mechanisms currently exist for patient input on this topic, they add, the opportunity to provide such input doesn't necessarily occur at the time that critical issues are being deliberated and may not represent a broad spectrum of patient views.
"We have had discussions at length within the patient community as to what the most appropriate milestones should be for patient contribution," the letters note. They go on to say that the rare disease patient advocates have concluded that the two milestones should be:
1) When the IND (Investigational New Drug Application) or IDE (Investigational Device Exemption) is filed and,
2) Any point at which the agency (FDA) decides, based on issues related to risk, that it must disapprove or delay commencement of a clinical trial or impose an unscheduled suspension on an existing clinical trial.
In addition to laying out specific suggestions as to how and when patient input might be submitted, the letters propose that FDA create a portal on its website, www.fda.gov, where it can post specific requests for patient contributions to certain issues.
"The patient groups who have signed this letter represent a broad spectrum of diseases, some rare and some more common. But all patients with chronic or rare diseases know that the benefit-risk ratio for any new drug or device is an important consideration," said Diane Dorman, NORD's vice president of public policy. "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."
NORD is an umbrella organization providing advocacy on behalf of the 30 million Americans with rare diseases, working with its member organizations, which represent patients with specific rare diseases, and other advocacy partners.
The letters and the list of patient organizations signing them are posted on the NORD website.
SOURCE National Organization for Rare Disorder (NORD)