Opinions and Legal Insights

Shifting Patient Engagement in the Era of COVID-19: HPM Facilitates the First Four Virtual Externally-Led PFDD Meetings, Moderating the Fifth Today

By Larry J. Bauer, Senior Regulatory Drug Expert & James E. Valentine

We all have had to adapt to a world with COVID-19 concerns.  One of the challenges has been how to continue having Externally-Led Patient-Focused Drug Development (PFDD) meetings in these times of social distancing, travel restrictions, mask requirements, and other efforts to “flatten the curve”.

On June 9, 2020, the first ever virtual Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting took place for the Hepatitis B community.  This virtual format, rather than the usual face to face meetings, was planned to avoid social contact and possible health consequences for attendees due to the COVID-19 pandemic.  HPM’s James Valentine and Larry Bauer helped the Hepatitis B Foundation in the planning and leading of this meeting.  On the day of the meeting, James was the meeting moderator, working from a local studio, and Larry gave a virtual meeting summary.

Since the success of that meeting, there have been three additional meetings for people living with Hypertrophic Cardiomyopathy (HCM); Facioscapulohumeral Muscular Dystrophy (FSHD); and, most recently, Pompe disease.  Today the National Kidney Foundation in partnership with NephCure hosts the fifth virtual meeting of its kind.  HPM helped in the planning and moderation of all these meetings.

Leveraging HPM’s Longstanding Experience with PFDD

PFDD meetings were a 2012 initiative originally conceived and sponsored by FDA to try to more systematically capture patients’ voices regarding their disease symptoms, the impacts of those symptoms on daily life, their current treatments, and their wishes for future treatments.  The goal of the meetings was to hear from patients and their caregivers to better incorporate their wishes and ideas throughout the drug development life cycle.  Information from these meetings is a valuable tool aiding FDA review staff when reviewing new drugs and treatments.  Part of each review is an evaluation of the potential benefits and risks from a new treatment.  Having direct patient input helps ensure that patients’ needs and wants are being considered.

James and Larry were both working at FDA at the time of the creation of this program. James served as a patient liaison, helping create and launch the PFDD program, and Larry served on several FDA panels at these meetings on behalf of the Rare Diseases Program.

Several years of success with these meetings followed; then, after conducting 24 such internal meetings, FDA decided to share the responsibility for PFDD meetings with patient organizations.  They encouraged interested groups to plan and conduct “externally-led” PFDD meetings with support from FDA.  These meetings typically follow a similar format to the original FDA meetings and result in a publicly available Voice of the Patient document that summarizes the meeting.

The first externally-led meeting took place in November of 2015 and was for people with Amyloidosis.  Since then, there have been meetings for a variety of conditions, both common and rare, including Spinal Muscle Atrophy, Friedreich’s Ataxia, Barth Syndrome, Cystic Fibrosis, and others.  HPM has been very active in planning many of these meetings, aided in 27 out of 37 meetings (73%), and moderated all but one of those meetings (see a complete listing in table at end of post).  In addition to James and Larry, other HPM colleagues bring their regulatory expertise to aid in organizing these meetings (e.g., Frank Sasinowski helped organize the EL-PFDD meetings on epidermolysis bullosa and pachyonychia congenita).  This extensive experience with PFDD meetings, both FDA- and externally-led, was leveraged to reimagine the virtual PFDD format when confronted with the COVID-19 pandemic.

Adapting the PFDD meeting format to a virtual environment

When we first considered shifting EL-PFDD meetings to a virtual format, there were many questions and doubts.  Would the meeting have the same impact? Would FDA attend virtually? How would we manage all the technical aspects of having a virtual meeting? How would we deal with any kind of technical failures on the day of the meeting? Would the virtual meetings be as interesting as in-person meetings? A virtual meeting format was not entirely new to the HPM team.  On October 29, 2018, the Cystic Fibrosis (CF) community held an EL-PFDD meeting for their community with help in planning and moderating from James Valentine.  Due to the nature of CF and the potential for cross-infection between patients, it was decided that the patients would call into the meeting.  A tele-townhall style approach allowed the moderator and meeting attendees to engage with remote patients from an in-person style meeting.  This experimental hybrid meeting format was very successful and critical for this community.

As the COVID-19 pandemic took ever greater hold on the country, it became clear that future EL-PFDD meetings would have to shift to virtual if the meetings were going to continue.  The Hepatitis B Foundation was the brave patient advocacy organization that decided to test the waters and planned a meeting for June 2020.  The Foundation enlisted the help of HPM as well as the media firm Dudley Digital Works to plan and produce the meeting.  When considering having a meeting, the Hepatitis B Foundation had these thoughts:

We have only a limited understanding of the broad impact of chronic hepatitis B on affected individuals,” states Dr. Chari Cohen, DrPH, MPH, Senior Vice President of the Hepatitis B Foundation.  “Many aspects of the disease, including the experiences of living with and being treated for chronic hepatitis B infection, have not been formally captured in clinical trials or public health studies.

The new virtual format gradually evolved.  It was decided that the patient panelists would pre-record their 5-minute statements to avoid any possibility of technical failure on the day of the meeting.  It also gave them the opportunity to practice their statements to ensure the best video quality.  For the first meeting, moderator James Valentine, was live in the Dudley Digital studio behind a desk to create the feeling of a news feature program.  For the subsequent meetings, James has had a co-host who was a representative from the patient advocacy community to provide additional commentary and moderate written comments.

Communication with attendees was key.  This was primarily done through an enhanced landing page for attendees to register in advance and get information about how to access the meeting on the day of the event.  This helped capture information about who was watching and participating in the meeting.  Live polling of participants was very similar to in-person meetings with patients and caregivers being asked polling questions by the moderator and then responding from their cell phones or other web-enabled device to give feedback in real time.

One of the biggest differences between in-person and virtual meetings was in how meeting attendees could participate in the live discussion part of the meeting.  During in-person meetings, the moderator asks questions and walks around the room with a microphone and people raise their hands to contribute to the meeting.

In a virtual meeting, there were now three possible ways of getting attendee feedback.  The first was through a panel of five pre-chosen people with the specific disease or condition that were all participating simultaneously in a Zoom call.  We called these people ‘discussion starters,’ since the moderator could ask them specific questions related to the discussion topic while waiting for callers to enter the queue.  The second way to participate was through emailed, written comments.  Participants could respond to the moderator’s questions by sending in an email with their thoughts and responses.  The third way of participating was through calling into a call center that took calls in real time.  James could call on people by name and ask them to respond to the discussion question.  These meetings really feel like a documentary style news show with a variety of inputs including photographs and videos to be shown during patient testimony.  Many FDA staff have attended these meeting virtually, and each meeting has had an FDA representative give opening remarks.  The FDA’s Patient-Focused Drug Development Program staff in CDER were very helpful in guiding the planning of these meetings.

This new virtual format has gone extremely well and put most of our doubts to rest.  Not unlike the in-person meetings, there have been few technical glitches, but the meetings have generally gone smoothly.  Some of the lessons learned have been to start planning early, make sure there are a lot of visuals to hold viewers’ attention, have the planning team communicate regularly, and to have two live meeting hosts instead of just one to balance the increased number of live inputs.

Parting Thoughts

COVID-19, unfortunately does not have any signs of going away soon.  It looks like virtual EL-PFDD meetings are the way for at least the near future and may remain a valuable option for patient communities that may have difficulty traveling or have other health risks.

We look forward to today’s meeting and continuing to develop the virtual format to make it even better. In fact, several organizations are already planning meetings for the coming months.

Table: 37 Externally-led PFDD meetings held to date (unofficial count)

Disease Patient Organization Date
Amyloidosis* Amyloidosis Research Consortium November 15, 2015

(prior to FDA guidelines)

Myotonic Dystrophy* Myotonic Dystrophy Foundation September 15, 2016
Acute Porphyrias American Porphyria Foundation March 1, 2017
Osteoarthritis Arthritis Foundation March 8, 2017
Spinal Muscular Atrophy* Cure SMA April 18, 2017
Friedreich’s Ataxia* FA Research Alliance June 2, 2017
Tuberous Sclerosis (& LAM)* Tuberous Sclerosis Alliance June 21, 2017
C3G, a rare kidney disease* National Kidney Foundation August 4, 2017
Lupus* LADA, LFA, & LRF September 25, 2017
Hyperhidrosis International Hyperhidrosis Society November 13, 2017
Duchenne Muscular Dystrophy Parent Project Muscular Dystrophy March 5, 2018
Hypereosinophilic Syndromes Am. Partnership for Eosinophilic Disorders March 23, 2018
Pachyonychia Congenita* PC Project April 6, 2018
Epidermolysis Bullosa* debra of America April 6, 2018
Sleep Apnea American Sleep Apnea Association June 8, 2018
Barth Syndrome* Barth Syndrome Foundation July 18, 2018
Juvenile Idiopathic Arthritis Arthritis Foundation; CARRA August 2, 2018
Alport Syndrome* National Kidney Foundation August 3, 2018
Chemotherapy-induced hearing loss in pediatric cancers* Children’s Cause for Cancer Advocacy September 13, 2018
CMT & inherited neuropathies* Hereditary Neuropathy Foundation September 28, 2018
Chronic hypophosphatemias* XLH Network October 5, 2018
Cystic Fibrosis* Cystic Fibrosis Research, Inc. October 29, 2018
Major Depressive Disorder Depression and Bipolar Support Alliance November 16, 2018
Niemann-Pick Type C Ara Parseghian Medical Research Fund March 18, 2019
Mitochondrial Diseases* United Mitochondrial Disease Foundation March 29, 2019
IgA Nephropathy* National Kidney Foundation August 19, 2019
Myeloproliferative Neoplasms (MPN)* MPN Research Foundation September 16, 2019
Pyruvate Kinase Deficiency (PKD)* NORD September 20, 2019
Atopic Dermatitis* National Eczema Association

Asthma & Allergy Foundation of America

September 23, 2019
CDKL5 Deficiency Disorder (CDD)* LouLou Foundation November 1, 2019
Lennox-Gastaut Syndrome LGS Foundation November 1, 2019
Pancreatitis* National Pancreas Foundation March 3, 2020
Chronic Hepatitis B* Hepatitis B Foundation June 9, 2020
Adult Hypertrophic Cardiomyopathy* Hypertrophic Cardiomyopathy Association June 26, 2020
Facioscapulohumeral Muscular Dystrophy* FSHD Society June 29, 2020
Pompe Disease* Muscular Dystrophy Association July 13, 2020
Focal segmental glomerulosclerosis (FSGS)* National Kidney Foundation August 28, 2020

*Meetings HPM helped plan.  Aided with VOP report only.