{"id":59079,"date":"2019-12-03T13:00:35","date_gmt":"2019-12-03T18:00:35","guid":{"rendered":"https:\/\/rarediseases.org\/nords-work-with-fda-gives-patients-a-chance-to-be-heard-in-drug-development\/"},"modified":"2019-12-03T13:00:35","modified_gmt":"2019-12-03T18:00:35","slug":"nords-work-with-fda-gives-patients-a-chance-to-be-heard-in-drug-development","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/nords-work-with-fda-gives-patients-a-chance-to-be-heard-in-drug-development\/","title":{"rendered":"NORD’s Work with FDA Gives Patients a Chance to Be Heard in Drug Development"},"content":{"rendered":"

By Debbie Drell, Director of Membership<\/span><\/i><\/p>\n

My sister was diagnosed with a rare disease, pulmonary hypertension, in September of 1998. At some point during her diagnosis, she was told she wouldn\u2019t live to see her 30<\/span>th<\/span> birthday. She was 28. There was one drug recently approved for her disease, and while it had serious and painful side effects, it was keeping her alive.<\/span><\/p>\n

Fast forward nearly two decades later and she\u2019s sitting on a panel speaking with other patients to the US Food and Drug Administration (FDA), talking about her hopes for better living with the disease and better therapy options. The panel was part of a larger \u201cPatient-Focused Drug Development\u201d meeting with the FDA, but I did not care what it was called. I wanted them to know that my sister would deal with the painful side effects if it meant more time to see her children grow older and have children of their own. My sister has always dreamed of having grandchildren to play with.<\/span><\/p>\n

As her caregiver, I did not really know the extensive role that FDA played in drug development, I just thought they approved drugs or rejected them based upon some set of criteria that scientists and researchers decided. Do scientists really know what risks patients are willing to take? How could they know what patients deem benefits worthy of the risks? Do patients have a say? I never thought they did.<\/span><\/p>\n

FDA is working with the National Organization for Rare Disorders (NORD) through a special partnership to bring voices like my sister\u2019s (and mine!) to their scientists to help understand what matters to patients for new drugs being reviewed by the FDA.\u00a0<\/span><\/p>\n

The people who are impacted the most by disease, patients and their caregivers, have been involved in the past, but this is a new way for scientists and FDA regulators to really listen to them share their very honest dreams, hopes, fears and wishes for their loved ones and themselves.<\/span><\/p>\n

The FDA created the Rare Disease Patient Listening Sessions in partnership with NORD. After a one-year pilot phase, these Listening Sessions offer patients and caregivers an opportunity to speak directly to FDA staff about what it is like to live with a specific rare disease. They help FDA staff understand the patients\u2019 perspective about their disease, needed treatment options and meaningful outcomes.\u00a0<\/span><\/p>\n

Our colleagues at the Patient Affairs Staff <\/span>recently wrote<\/span><\/a> about some of the specific ways they\u2019ve learned from these Listening Sessions including:<\/span><\/p>\n