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Prioritizing Patient & Caregiver Experience in ALS Clinical Development

By: Rich Bennett, Therapeutic Strategy Lead, Neuroscience, Worldwide Clinical Trials & Evy Reviers, Chairwoman, EUpALS

Given the rapid and relentless progression of this neurodegenerative disorder, an ALS patient, on average, has only one opportunity to participate in a clinical trial. This harsh reality forces them to make it count for themselves and for their community. Therefore, every patient has the potential to make an impact on their patient community and future treatment options for ALS. It’s critical that the study they select provides a reasonable likelihood of therapeutic benefit, meets study objectives, and creates learning opportunities for the wider research community. The clinical research industry has a responsibility to make this happen.

This ALS Awareness Month, Worldwide Clinical Trials is teaming up with the European Organization for Professionals and Patients with ALS (EUpALS) to highlight the critical and still underutilized role that the patient and caregiver experience needs to play in driving clinical development of ALS therapies. The levels of comprehensive engagement in the study and the quality of the participant experience must be a central part of study design and operational planning in every program.

Our Partnership

EUpALS prioritizes awareness of ALS in Europe, provides resources for people and families facing an ALS diagnosis, and works to create better access and better treatments for ALS patients. At Worldwide, we are focused on delivering patient-centric ALS studies through optimized protocols and tailored clinical operations solutions. As we believe in the power of aligned missions across stakeholder groups, our partnership facilitates more patient-centric trials, better pathways for enrollment and adherence, and improved treatments for ALS trials.

Our experience has shown that industry sponsors of ALS studies may accept advice on patient engagement from medical key opinion leaders or their CRO. However, they often fall short of actively engaging their ultimate study partners — the patients (pALS) and carers of people with ALS (cALS).

To truly be ‘patient-centric’ in ALS, you must go beyond an empathetic approach and ensure the patient experience is well-considered. Rather, it must be balanced with the scientific and methodological rigor of every ALS clinical trial. It also must prioritize the full carer or family journey, as pALS must rely on their family or carer to facilitate study visits and support in day-to-day tasks.

Hear Our Voice

Despite good intentions, the ALS patient voice is largely underprioritized by the pharmaceutical industry regarding study design. Instead, industry-sponsor engagement with the patient community tends to be reserved for meeting with patient advocacy organizations with direct engagement of pALS. These engagements are often restricted to requests for testimony during regulatory agencies’ advisory committee meetings in support of marketing authorization application reviews.

While there is certainly a significant benefit to industry-sponsor engagement with patient advocacy organizations, it’s critical that we listen to the patient voice through study-specific feedback more frequently. Hearing their voice in the early stages of the clinical development process adds significant value to study design and conduct, and it is appreciated not only by the pALS and cALS themselves but also increasingly by regulatory agencies. 

Engaging the EUpALS Patient and Carer Expert Board

EUpALS has built a Patient and Carers Expert Board (PCEB) to help facilitate better alignment between patients and research. The PCEB is a group of patients and carers based in the EU who provide the industry with input on specific study information based on their real-world experiences with ALS. This group is nine strong with members from Belgium, Iceland, the Netherlands, Spain, Sweden, and Turkey.

Univocally, their slogan is “nothing about us without us!”

Evy Reviers, chairwoman from EUpALS, adds:

“Our Patient and Carer Expert Board has the potential to be transformative for industry sponsors of clinical trials in ALS. By listening to patients and carers, practical solutions can be adopted that will enhance the experience of study participants and are likely to have a positive effect on the attractiveness of a study to potential participants.

By creating a study design that works for pALS and cALS, you lower the burden for their enrollment in your trial and increase their ability to remain in your study, which means that you will have less missing data, and the participants will more likely continue taking your experimental medicine throughout the disease course.”

The PCEB reviews study documents and plans, providing actionable input to Worldwide on:

  • Clinical trial protocols
  • Study synopsis/Schedule of events
  • Information sheets
  • Consent forms
  • Pharmacy instructions
  • Recruitment and retention plans
  • Travel and accommodation policy

In addition to protocol and study insights, Worldwide and EUpALS consider it relevant that the study’s operational specifications are shared so that the PCEB can provide feedback on the practical study logistics that will be used. While seldom included in a study protocol, these items are often important to the PCEB evaluation.

Engagement with the PCEB can be customized to your company’s needs and approach. A typical process is:

  1. Sign a confidentiality agreement with EUpALS, who in turn has confidentiality agreements in place with the individual members of their PCEB.
  2. Provide EUpALS an informative presentation and list of specific questions to be addressed by the PCEB. A clarifying video call can be scheduled to set the stage.
  3. Allow a two-week review by the PCEB. Feedback will be compiled and provided anonymously.
  4. If desired, a follow-up video call can be scheduled to further discuss the feedback by the PCEB.

Though not an official part of the process, the PCEB greatly appreciates hearing whether feedback was adopted and the rationale for any feedback not adopted.

Best Practices When Engaging Patient-Experts for Feedback:

When you engage with organizations like EUpALs, be mindful to:

  • Ensure engagement is early in the study design process so there is ample time to reflect and implement feedback.
  • Respectfully give patients and carers adequate time for review and their means of communicating feedback. Due to the manifestation of bulbar symptoms, verbal communication can be difficult for a given patient.
  • Understand that feedback from our pALS and cALS is personal and based upon their real-world experiences, despite their efforts to represent the attitudes and opinions from across the broad ALS patient community.
  • Avoid offering honorariums to the members of the PCEB, as all members commit their time and experiences for the greater good. Instead, consider your EUpALS Industry Partnership as a market-based remuneration to support the daily activities of the organization.

In addition to working with groups like EUpALS, there are readily available tools to guide your trial. Worldwide utilizes I AM ALS’s Patient-Centric Trial Design (PaCTD) rating scale to objectively rate humane and efficient trial designs. The PaCTD rating system is a 5-star rating system created by patients and caregivers that assesses clinical trial designs across nine elements based on the FDA ALS Clinical Trial Guidance Document. We highly recommend checking out this scale to learn more.

Successful Research Starts with the Patient

Balancing the various stakeholder needs and priorities in clinical research is not an easy task, but the more alignment there is, the better the outcomes for all stakeholders. When it comes to ALS research, we are all making strides to ensure better treatments and wider access as we move forward on the quest to ultimately provide longer lives for these patients. The path forward is clearest when we are united, intentional, and together.

Worldwide is committed to working with sponsors and patient organizations to advance treatments in ALS clinical development. We have amassed more than four decades in the field of neuroscience, developing key partnerships, KOLs relationships, and in-house expertise that allows us to deliver excellent solutions for our sponsors designed with the patient experience in mind. Contact us to learn more.


The EUpALS PCEB provides advice and recommendations to the EUpALS Board of Directors, the European ALS/MND Associations, and Industry partners. In particular, they provide input on how to incorporate the needs of pALS in ALS/MND research and clinical trials.

EUpALS PCEB members are people living with ALS/MND or primary (informal) carers to someone living/lived with ALS/MND. Professional (para)medical carers are excluded.

Candidates are nominated by a European ALS/MND Association that is a member of EUpALS. Two nominations of pALS or cALS from each country that are members of EUpALS are accepted.

Worldwide Clinical Trials

Worldwide is a global clinical research organization (CRO) founded by neuroscientists. Today, it holds several strategic focus areas, including neurodegeneration and neuromuscular disorders. Worldwide has conducted 15 full-service ALS studies in the last six years and has provided consultancy services to biotech clients regarding ALS drug development planning and clinical trial design in collaboration with ALS community leaders.


Worldwide Clinical Trials was the first CRO to become an industry member of EUpALS in 2022 and has continued its support into 2024.