News
Rare disease organizations ask FDA to allow more patients' views on drug risks and benefits
Washington DC — 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 Alpha-1 Foundation was among the patient advocacy organizations signing the letters.
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 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.
