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ALS Clinical Trial Recruitment and Retention Action Summit: Three Takeaways to Create Positive Change


I AM ALS aims to transform the ALS research system by addressing system-wide barriers to medical breakthroughs for ALS. This initiative is built on the foundational principles that (1) better communication, collaboration, and transparency throughout the ALS research system will accelerate scientific progress, and (2) that meaningful progress cannot be accomplished without a commitment to authentic partnership with those most impacted by ALS.

In 2019, I AM ALS conducted a national survey to assess ALS patient and caregiver knowledge of and barriers to enrollment in clinical trials. The results represent the lived experiences of hundreds of people, and illuminate real issues with the clinical trial system for ALS, which manifest in long enrollment periods, high dropout rates, a general lack of knowledge and understanding about clinical trials, and avoidance of trials by some people living with ALS. The results of this survey and our more recent conversations with a range of stakeholders have revealed an urgent need for (1) improved knowledge and understanding of clinical trials in ALS by clinicians and people impacted by ALS, (2) more patient-centric trial designs and practices, and (3) improved diversity and equity in clinical trial access and participation (race, age, type of ALS, geographical location, etc.). 

In response to these concerns, I AM ALS hosted a collaborative Action Summit on March 2-3, 2022 to engage stakeholders in the ALS community to improve the clinical trial experience for people living with ALS and their caregivers. Summit participants included people living with ALS, loved ones and caregivers, physicians, clinical research coordinators, advocacy organizations, biopharma allies, and key government stakeholders. The participant breakdown is presented in table 1 below. 

Table 1: Participants’ Stakeholder Categories

Stakeholder category Percentage of participants
ALS Community Members 25%
Clinical Researchers/Clinicians 18%
Advocacy Organizations 24%
Biopharma Allies 16%
Government Partners 9%
Bioethicists 4%
Other 4%

With conversations focused on clinical trial recruitment and access, diversity and equity in clinical trials, and standardizing patient-centric clinical trial design and operations, participants discussed the following questions during the two-day summit:

  1. How can we ensure all people living with ALS and their loved ones are able to easily find and understand comprehensive information about current ALS clinical trials? 
  2. How can we meaningfully and sustainably increase racial and ethnic diversity in ALS clinical trials? 
  3. How can we sustainably improve operations at clinical trial sites to be more patient-centric?
  4. How can we as an ALS community work to standardize equitable and patient-centric practices across all ALS clinical trials? 

In a review of the discussion notes, we identified the following key takeaways for action and change. These are not meant to be exhaustive, but to place us firmly on the path to ensuring equitable access to clinical trial information and participation, and patient-centric trial experiences.  

  1. We need to invest in education and training. Healthcare professionals (HCPs) need support to be well-equipped to understand ALS, the diagnostic process, the value and availability of clinical trials, and more. The lack of a standardized system for ongoing education and training for all HCPs caring for a person living with ALS can lead to them feeling overwhelmed and unprepared to appropriately support and care for a person living with ALS. Additionally, we need to focus on workforce development at clinical trial sites to ensure that sites are appropriately staffed to establish timely and effective screening and enrollment practices. Lastly, we need to raise awareness of existing protocols and guidelines related to clinical trial recruitment and operations such as the Airlie House guidelines, PaCTD rating system, ALS Signal and Morris ALS Principles to help ensure a patient-centric clinical trial experience.
  2. We need to create new and evolve existing clinical trial resources for stakeholders. This includes centralizing and improving online clinical trial databases to be more user-friendly and customized for ALS, such as ALS Signal, providing resources to develop informed consent and trial information in more languages and using medical translators to review the forms and information with individuals to ensure their understanding, investing in Patient Navigators at one or multiple sites and more. It is also essential to publish post-trial information and provide access to results for trial participants to build trust with the community.
  3. We need to champion collaboration. To create lasting change, all stakeholders must work together to establish processes to incorporate patient advisory boards for patient-centric clinical trial design, and encourage sponsors to implement these processes. There is also an urgent need for:
    1. Protocols to ensure equitable access to clinical trials by including a plan to address diversity, equity and inclusion in clinical trial recruitment, enrollment and participation, and
    2. Cross-site learning and collaboration to define, share and grow best practices at clinical trial sites. 

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