ITP Conference 2019, which took place on July 26-28 in our Nations’ Capitol, gave patients and caregivers a rare opportunity to have their voices heard. On the first day of the conference, Friday, July 26, PDSA organized an externally-led patient-focused drug development meeting (EL-PFDD). As North America’s leading organization dedicated to empowering ITP patients, PDSA was approved by the U.S. Food and Drug Administration (FDA) to lead this workshop to enhance the understanding of ITP and the patient experience by regulators, researchers, clinicians and industry.
View the Voice of the Patient Report
The purpose of PFDD meetings is to hear directly from patients, their families, caregivers and patient advocates. The FDA has conducted over 25 disease-specific PFDD meetings to more systematically obtain the patient perspective on specific diseases and their treatments. The FDA recognizes that there are many more disease areas that can be addressed beyond the PFDD meetings planned and conducted by FDA, so they are giving patient advocacy organizations the opportunity to host their own PFDD meetings, with input and guidance from FDA. After submitting a letter of intent to FDA, PDSA was honored to be selected to organize and host an EL-PFDD as part of our annual ITP conference.
According to FDA “The patient perspective is critical in helping FDA understand the context in which regulatory decisions are made for new drugs. PFDD meetings give FDA and other key stakeholders, including medical product developers, health care providers, federal partners, an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during our review of a marketing application."
What can you expect from an externally-led PFDD meeting?
If you have attended any of PDSA’s past conferences, you know we always end the conference on Sunday with a patient panel, where patients share their personal journey with ITP. Always insightful and inspiring, if not often heartbreaking, patients learn a tremendous amount about their disease from other patients and caregivers. Over the years, we have had patients say to us “Why don’t you have these patient panels earlier in the conference so that the doctors can hear what it is like to live with ITP?” Well, think of the EL-PFDD program as a larger patient panel session. We had two patient panels of 4-6 patients/caregivers each who talked about the symptoms and burden of living with ITP and the impact of treatments and their side effects. PFDD meetings are also facilitated large group discussions where all patients and caregivers in the room have a chance to share what it is like to live with their condition; discuss the daily impact of ITP, tell stories to highlight their unmet needs, and describe the shortcomings and advantages of current therapies and treatments. As experts in what it is like to live with their condition, ITP patients and caregivers are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation.
The EL-PFDD was a half-day meeting and ran from 11am-4pm on Friday, July 26. Following the EL-PFDD meeting, PDSA hosted a patient mixer from 4-5pm with appetizers and non-alcoholic beverages. With a number of new ITP drugs in the pipeline, this was a rare opportunity to have your voices heard and truly make an impact on the understanding of the unmet medical needs and quality of life issues that matter most to ITP patients.