ALS clinical research learning institutes (ALS-CRLI): empowering people with ALS to be research ambassadors

Abstract

Objective: Patient engagement in research is increasingly recognized as important across many countries and fields. In 2008, we conducted surveys that suggested a need for improved patient engagement in ALS research. We decided to create an ALS Clinical Research Learning Institute (ALS-CRLI) to facilitate direct interactions between researchers and people with ALS and their caregivers, toward ultimately improving engagement. Methods: Initially modeled after a similar program in Parkinson’s disease, our ALS-CRLI is a multi-day collection of formal courses for people with ALS and their caregivers, moderated by clinicians, scientists and patient advocates. Previous graduates (called ALS Research Ambassadors) engage with the current class participants before, during and after the courses. Prior to the courses, Research Ambassadors serve as “mentors” to the participants, offering guidance and setting expectations. Feedback during the courses is used to change the way researchers design and advertise studies, and feedback after the courses is used to improve the agenda for subsequent ALS-CRLIs. Funding is provided by patient advocacy groups including the ALS Association and ALS Hope Foundation. Research Ambassadors are provided with ongoing mentoring and notifications about opportunities for engagement via regular teleconferences with the Northeast ALS Consortium’s Patient Education and Advocacy Committee and their own Facebook page. Engagement and advocacy efforts are tracked using a tool on the Northeast ALS Consortium’s website. Results and Conclusions: We have now held 15 ALS-CRLIs at various locations within the United States, resulting in over 320 graduated ALS Research Ambassadors. From these engagements, researchers have been prompted to formally include patients in the design process, to design more patient-centric trials and to create new ways to help patients find trials. Research ambassadors are improving awareness and clearing up misconceptions about participation in research, improving research availability, and helping to create more patient-centric trial designs. In addition, we are now creating an ALS-CRLI Toolkit that will facilitate ALS-CRLIs throughout the world. This will be housed on the Northeast ALS Consortium website.

Keywords:

• Engagement
• involvement
• advocacy

Introduction

Patient engagement, defined as “meaningful and active collaboration in governance, priority setting, conducting research and knowledge translation (1),” is now internationally recognized as important across many research fields (1–8). Patient engagement can facilitate the design of research questions (9) and outcomes (10) that are of greater interest to patients, trial designs that are more patient-centric (11), consent forms and study summaries that are more understandable to the target population (12), improved enrollment (12,13) and retention rates (12), and reduced trial costs (14).

In 2008, we identified an opportunity for improved patient engagement in ALS research (15). Surveys we conducted of researchers (15) and people with ALS (PALS, 16) suggested a lack of awareness of research options, misconceptions about research and dissatisfaction with trial design features. All of these appeared to contribute to surprisingly low enrollment rates in ALS trials (15,16). Other fields noticing the same problems already had programs underway to address these (17–23). We were particularly impressed with a program called the Parkinson’s Learning Institute (23). Over the course of several days, formal courses helped patients and caregivers to better understand research and showed them specific ways and places they could impact the process. Patients and caregivers helped faculty better understand their own frustrations and engagement barriers. Ongoing facilitated connections between graduates and researchers afterward optimized engagement at many levels. Given this success, we decided to start an ALS Clinical Research Learning Institute (ALS-CRLI) in 2011.

Methods

Agenda

The agenda for the initial ALS-CRLI mirrored that of the Parkinson’s Learning Institute. Following each subsequent ALS-CRLI, graduates (referred to as ALS Research Ambassadors) are surveyed to help modify the agenda for the next class. The agenda for the upcoming 2019 Clearwater FLORIDA ALS-CRLI is shown in Figure 1. Each topic is led by a clinician, scientist or Research Ambassador, and discussion is encouraged throughout each learning session. There are several small group exercises that provide the opportunity for faculty to learn from the patients and families in attendance

Figure 1 Agenda for the 2019 Clearwater FLORIDA ALS-CRLI.

Faculty

Faculty are mostly physician and scientist members of the Northeast ALS Consortium who volunteer their time. Representatives from patient advocacy groups such as the ALS Association and ALS Hope Foundation have presented. Research Ambassadors have become faculty as well.

Attendees

A “call for nominations” is announced prior to each ALS-CRLI. Anyone can nominate a person with ALS and/or a caregiver via a simple application form that asks for an explanation of why they would be a good Research Ambassador. We typically seek to enroll 24–26 attendees in pairs of PALS and caregivers. If more patients and caregivers apply than the class can accommodate, faculty vote on the applicants to select the final 24–26. Application forms are posted on a secure online survey system and each faculty member is asked to select the 24–26 applicants they think would make the best Research Ambassadors. Those 24–26 with the most votes are then invited to attend.

Funding

Funding for 12 of the 15 ALS-CRLIs conducted to date has been provided by the ALS Association (ALSA). For all 9 Clearwater FLORIDA ALS-CRLI’s funding was received from the National ALSA Office (24). Three CRLIs were funded by local ALSA chapters (Sacramento CA, Phenix AZ, St. Louis MO). Three Philadelphia ALS-CRLIs were funded by the ALS Hope Foundation (25). Budgets varied depending on the ALS-CRLI location and the geographic range over which faculty and attendees were solicited. Travel and lodging expenses for faculty and research ambassadors are usually covered. Clearwater FLORIDA ALS-CRLI’s have been the most expensive and typically cost around $30,000 (Table 1).

Table 1 Budget for the 2019 Clearwater FLORIDA ALS-CRLI.

Item	Amount	Number	Totals
Travel	$700	26	$18,200
One Night Hotel	$234	18	$4,212
Meeting Rooms & Food	$100	34	$3,400
Welcome Dinner	$100	34	$3,400
Material for Meeting	$50	26	$1,300
GRAND TOTAL			$30,512

Mentoring

To ensure that Research Ambassadors continued to receive mentoring and specific opportunities for engagement after they graduate, a NEALS subcommittee called PEACe (Patient Education and Advocacy Committee) was started. The PEACe Committee is comprised of all the Research Ambassadors and any interested NEALS clinicians and scientists. Monthly teleconferences are held in which various stakeholders within the ALS community present opportunities for Research Ambassador input/engagement. Research Ambassadors, in turn, provide feedback and present issues and projects for clinician or scientist input or assistance.

Social media

A Facebook page was created for ALS Research Ambassadors to facilitate networking activities between ambassadors and faculty. Between PEACe meetings, Research Ambassadors utilize this page to share relevant information regarding research, and physicians and scientists can use this page to announce specific opportunities for Research Ambassadors. This page is open to all in the ALS community, and Research Ambassadors and faculty encourage others to view the shared information and submit information as applicable.

Tracking

Research Ambassadors are asked to update a “tracker” on the NEALS website, identifying their research and advocacy efforts (Table 2). Categories tracked include speaking engagements, organization or participation in ALS events, interviews, interactions with congressmen, creation and posting of videos, publishing articles, serving on ALS-related committees, and mentoring other patients and caregivers.

Table 2 ALS-CRLI research ambassador tracker as of September 23, 2019.

Outreach efforts by research ambassadors	Number of each
ALS speaking engagement (fundraiser, support group, political event, etc.)	864
Organization of or participation in an ALS event	167
Interview (TV, radio, web, or print)	92
Speaking or writing to a congressman	322
ALS video (YouTube, social media, webinar, etc.)	27
Creation of or contribution to a website, blog or social medial page	344
Published article/column (web or pint)	70
Serving on an ALS committee	40
Mentoring (PALS or CALS)	121
Other	11

Results and discussion

ALS-CRLIs have trained over 320 Research Ambassadors to date . They are actively helping to improve ALS research in the following specific ways:

1. They are broadening patient-awareness and clearing up patient-misconceptions about participation in research through more than 800 talks at fundraisers and support groups (Table 2), 400 contributions to media, social media, chat rooms or websites (Table 2), leading educational webinars (26), 120 specific mentoring sessions with other patients or caregivers (Table 2) and even showing up in clinics on days when they don’t have an appointment to answer questions for other patients. In the words of one Research Ambassador “I am starting to see evidence that our efforts work. For the first time our clinic is getting requests about clinical trials from patients. I have been speaking to support groups around our state and it seems to have piqued their interest! I am working with our clinic to make myself available on clinic days to provide information and guidance with the clinical trial process (27).”

2. They are improving research availability through fundraising and interactions with policymakers. Two early Research Ambassadors later helped start the ALS Ice Bucket Challenge which raised over $200 million in the Summer of 2014 (28) and led to several new studies (28,29). In the words of Research Ambassador and Ice Bucket Challenge co-founder Pat Quinn “With the Research Ambassadors program, I was able to develop the qualities necessary to become a leader in the ALS world. Through extremely persistent advocacy, I became a major catalyst and co-founder of the Ice Bucket Challenge” (30). Through more than 300 interactions with members of Congress (31), Research Ambassadors have helped obtain and maintain funding for the National ALS Registry (32) and the Department of Defense ALS Research Program (33). Said Research Ambassador Allison Pogemiller, “I had the honor of participating in the National ALS Advocacy Days on Capitol Hill in Washington DC, where I was able to personally meet with my congress men and women to share my journey with ALS, and help them to understand how their support could drastically assist families like mine. It was incredibly emotional to re-live the tragic loss of my husband, yet I felt empowered to make a difference for others, as I could put a real, relatable face to a tragic disease, and instill the urgent need for things such as maintaining funding for the National ALS Registry and the DOD ALS Research Program” (34). In the words of Research Ambassador Shelly Hoover, “I've participated in ALS Advocacy Day in Washington DC for the past six years. I've met with US senators, representatives, and staff members about appropriations and legislation pertinent to the ALS community. Each year we request ten million dollars for the ALS National Registry, the largest ALS research project ever conducted. This year, 2019, we requested and received 20 million dollars for the DoD ALSRP. This appropriation is twice the amount of previous years. As a US veteran, this program is especially important to me because veterans are twice as likely to be diagnosed with than non-veterans” (35).

3. They are educating trialists, sponsors and regulators about the challenges and opinions they have related to research. For example, feedback from several early Research Ambassadors regarding the difficulty they had finding open trials helped prompt NEALS to create a simpler trial search engine and identifying a “clinical trial liaison” that patients can call to get help finding a study (36). Ambassadors participated in the new Airlie House ALS Research Guidelines and helped generate specific suggestions that future studies incorporate patients into the design process, utilize patient-reported outcomes, expand access to the therapeutic being studied and publish results in open access journals (37). In the words of Cris Simon, one of these Ambassadors, “Airlie House ALS Research Guidelines was my introduction to the importance of PALS advocacy. The invitation, as one of four pALS, to participate in updating the guidelines with our voice taken into consideration was exciting and encouraging with hope for expediency for the numerous ALS trials both present and future. Consulting with esteemed doctors, researchers and statisticians who work tirelessly to find a cure was humbling and greatly appreciated” (38). Several Ambassadors participated in the FDA Guidance for ALS Drug Development and helped make it clear that patients with ALS want interim analyses of effectiveness as well as safety and are more risk-tolerant than those with other diseases (39–41). Brian Wallach, a Research Ambassador who recently started a group called I Am ALS, recently tweeted his appreciation for being “able to have open and honest discussions with them (the FDA) to push the Guidance to be patient-centric, forward leaning and focused on both now and the future” (42).

4. They are helping to create more patient-centric trial designs by interacting with investigators and companies that are planning studies. For example, Research Ambassadors’ suggestions for largely virtual visits, historical (rather than placebo) controls, and results available in real time were all heard by Dr. Bedlack and incorporated into the design of his clinical trial of Lunasin (43). Twelve Research Ambassadors’ suggestions are being incorporated into an upcoming Mitsubishi Tanabe Pharma America biomarker study (44).

In the words of Research Ambassador Madeline Kennedy, “the CRLI was the best two day learning experience of my ALS journey. I acquired a much more profound and realistic understanding of the research/trials process. I have participated on committees formulating patient positions on the design of several trials, and FDA guidelines, I was invited to and participated in the National ALS Registry Annual meeting. These opportunities were a direct result of the voice I found, the confidence I acquired and the people I met at The CRLI” (45). We are pleased with the results of the ALS-CRLI thus far and hope to see it expand to involve more PALS and CALS in more locations. To facilitate this, we are creating an ALS-CRLI “toolkit” which will be housed on the NEALS website (46). This will include suggested steps and timelines, sample agendas, budgets, and surveys, a list of faculty who are willing to participate, and slide decks from recent ALS-CRLIs that are free to utilize.

We are proud to add CRLIs to a growing international list of programs that facilitate patient engagement, including a newer, ALS-focused, United Kingdom-based program just reported (47). This is an exciting time in ALS research, with more ideas and products to test than ever before. Patient engagement in this will improve the chances of successful outcomes and, ultimately, better ALS treatments.

Declaration of interest

Richard Bedlack has research support from ALSA, MNDA, Orion, and Ultragenyx, and consulting support from ALSA, Cytokinetics, ITF Pharma, Biohaven, Mitsubishi Tanabe Pharma America, Mallinkrodt, Brainstorm Cell, Biogen and New Biotic Inc. Ms. Pogemiller is the president of “ALS Connect, LLC.” Dr. Cudkowicz is a consultant for Biogen, Sunovian, Avexis, Takeda.

Additional information

Funding

Funding for previous ALS Clinical Research Learning Institutes has come from the ALS Association and the ALS Hope Foundation.