Why Only 1 in 4 People with ALS Can Realistically Access Clinical trials

Introduction

As part of an ongoing collaboration between myTomorrows and The ALS Association to improve clinical trial access for people living with ALS, between August 2025 and June 2026, we analyzed 280 individuals seeking trial opportunities and assessed 35 actively recruiting ALS clinical trials based in the U.S.A.

While most people matched multiple potential trials, only about 24% could realistically access a recruiting study. The primary barriers were not a lack of available trials or limited patient interest. Instead, access was lost through a combination of diagnostic delays, missed symptom-onset eligibility windows, referral lag, and operational constraints that closed the enrollment window before many people could reach a study site.

These findings build on previous work from the myTomorrows and ALS Association partnership, examining ALS trial recruitment and providing new evidence that ALS trial access may be more constrained by timing and process than by trial availability itself.

In the United States, an estimated 33,000 people are living with ALS at any given time. Despite a growing number of recruiting studies, many never reach a realistic enrollment opportunity.

Key findings at a glance

• 96% of patients found trial matches with a median of ~5 potential trials; only 9 of 280 had no match at all
• 32 of 35 trials were gene-agnostic – genetics was rarely the barrier
• Just ~1 in 4 could realistically access a recruiting trial
• Missed symptom-onset windows drove the largest single loss (31.6%), followed by referral lag (18%) and diagnostic delay (14%)
• Active trial sites were available in 33 states + DC; only 5 of 231 patients were unwilling to travel

Most people with ALS had trial options – so why is enrollment so low?

One of the most notable findings was that trial inaccessibility was rarely caused by a complete absence of clinical trial opportunities.

• Patients matched to a median of approximately five potential trials
• Only 9 of 280 patients had no identified trial match
• 32 of 35 assessed trials were gene-agnostic

These findings suggest that the ALS clinical trial landscape may no longer be primarily limited by trial availability in the USA. Most patients had at least one potential trial option, and many had several. The challenge emerged when those opportunities were filtered through eligibility requirements, referral timelines, and operational constraints.

The ALS trial access funnel: where patients drop off

We followed each patient through five sequential access gates. Attrition happened at each stage.

The patient funnel reveals that attrition occurred at every stage of the clinical trial matching process. Of the 280 patients initially profiled, only 55 patients remained realistically able to access an actively recruiting ALS clinical trial. While many patients appeared to have potential trial opportunities on paper, successive barriers related to eligibility, timing, geography, patient preferences and, trial operations reduced the accessible patient population to just 24% of the consulted patients. The result is a substantial gap between theoretical trial eligibility and real-world trial accessibility.

These findings suggest that the challenge in ALS clinical trial participation is not solely the availability of ALS clinical trials. Rather, the pathway from potential eligibility to realistic access is constrained by a series of structural and operational barriers that together progressively prevent people living with ALS from reaching any realistic enrollment consideration.

Importantly, the barriers identified in this analysis are not unique to this patient cohort. Previous studies have identified many of the same barriers observed in our analysis, including diagnostic delays, restrictive eligibility criteria, referral challenges, and logistical barriers associated with ALS clinical trials. Our findings demonstrate how these barriers accumulate in real-world settings, substantially narrowing access even when multiple clinical trials are actively recruiting.

Among the barriers identified in this analysis, time emerged as a recurring theme. Diagnostic delays, missed symptom-onset eligibility requirements, referral timelines, and screening procedures all consume valuable time within a finite enrollment window. Understanding how quickly this window closes helps explain why many people living with ALS lose access despite appearing eligible at first review.

Why do people living with ALS lose trial eligibility so quickly?

Many ALS clinical trials require enrollment within a defined period from symptom onset, often 24 months or less. As a result, the pathway from symptom onset to enrollment becomes a race against time. Published research consistently reports diagnostic delays of approximately 10 to 16 months between symptom onset and confirmed ALS diagnosis². Even before patients are referred to clinical trial opportunities, a substantial portion of common eligibility windows has already elapsed before a trial is even discussed. The question, then, is where that time goes.

What are the biggest barriers to ALS clinical trial access?

The analysis revealed that three barriers in particular account for most lost access:

Beyond these, several structural barriers also limited access:

• Remaining geographic gaps
• Trials not yet recruiting
• Site-level constraints
• Functional status requirements
• Trial specific inclusion and exclusion criteria

Where is time lost between symptom onset and trial matching?

As a result of many ALS clinical trials requiring enrollment within a defined period from symptom onset, every delay between this point and trial matching reduces the likelihood that a person will remain eligible for enrollment.

Among the 73 individuals in our analysis who fell outside common symptom-onset eligibility windows, we identified two distinct causes of lost access.

1. For 32 individuals (44%), the time between symptom onset and confirmed ALS diagnosis alone exceeded common trial eligibility windows. In these cases, access was effectively lost before trial discussions could even begin. This reflects the well-documented diagnostic challenges associated with ALS, including misdiagnosis, multiple specialist referrals, and delays in neurological assessment.
2. For the remaining 41 individuals (56%), diagnosis occurred within the eligibility window, but access was lost later. These individuals were still potentially trial-eligible at diagnosis but were not connected to trial-matching services until much later. On average, more than two years elapsed between diagnosis and intake, suggesting that referral pathways, awareness, and navigation challenges can be just as important as diagnostic delays.

Together, these findings suggest that clinical trial access is lost at multiple points. About half of the loss occurred before diagnosis, while the other half occurred after diagnosis but before individuals were connected to clinical trial opportunities.

Every month of delay reduces the remaining enrollment window. By the time many people living with ALS reach clinical trial matching, they are already approaching or have exceeded common eligibility thresholds, leaving little opportunity to identify, evaluate, and enroll in a study.

Geography has improved, but access gaps remain

Geographic access is better than commonly assumed. Active recruiting sites were identified across 33 states and Washington, D.C., resolving several previous trial-access deserts. However, notable gaps remained in South Carolina, Mississippi, North Dakota, and Oklahoma.

Crucially, only 5 of 231 consulted patients were unwilling to travel for a trial, suggesting that patient willingness may not be the primary limitation. Recent research similarly found that geographic distance alone does not adequately explain low participation in ALS clinical research. Instead, trusted healthcare providers, trial awareness, navigation support, logistical assistance, and access to current trial information often play a larger role in participation decisions.

As a result, travel support, decentralized trial elements, and patient-navigation programs may generate greater improvements in participation than expanding site counts alone.

ALS trial access is primarily a process problem

Taken together, these findings suggest that ALS trial-access barriers are driven more by process and logistics than by genetics or a lack of available studies:

• Patients had a median of approximately five potential trial opportunities
• 32 of 35 trials were gene-agnostic
• Only a small minority of patients had no matching trial option
• The largest losses occurred through eligibility windows, referral delays, diagnostic timing, and operational constraints

The challenge therefore appears to be less about creating trial opportunities and more about helping patients reach them before they become inaccessible.

Five ways sponsors can widen ALS trial access

1. Accelerate diagnostic and referral pathways: Reduce the number of patients who lose eligibility before their diagnosis is confirmed or before they reach an actively recruiting trial site.

Our analysis suggests that delays before ALS clinical trial matching occur at two critical points. Among patients who fell outside common symptom-onset eligibility windows, nearly half exceeded common enrollment cutoffs before receiving a diagnosis, while the remainder were diagnosed within the eligibility window but reached trial-matching services too late. Earlier recognition of ALS symptoms, faster specialist referral, and earlier discussion of ALS clinical trials could preserve substantially more opportunities before enrollment windows close.

2. Strengthen referral networks: Improve coordination between community neurologists, multidisciplinary ALS clinics, advocacy organizations, and trial centers.

Trusted healthcare providers strongly influence participation decisions yet often report limited time and limited access to current trial information. Strengthening referral pathways and increasing clinician awareness of actively recruiting studies may thus help eligible individuals reach clinical trial opportunities sooner.

3. Identify and assess people living with ALS earlier: Reduce delays between initial interest, trial assessment, referral, and enrollment consideration by collecting and structuring relevant information earlier in the patient journey.

Earlier identification of potentially eligible individuals can help preserve access while reducing administrative burden on patients, caregivers, and clinical teams. As enrollment windows become increasingly compressed, even modest reductions in processing time may help prevent otherwise eligible individuals from losing access.

4. Expand travel support and decentralized participation: Reduce geographic and logistical barriers through comprehensive travel reimbursement, travel concierge, remote assessments, home-based services, and hybrid and platform trial models. Expanded Access programs may also provide an additional pathway for people who cannot participate in a clinical trial or who lose access because of eligibility or operational constraints.

Support should reflect the needs of both patients and caregivers, including accessible transportation, accommodation, lost working time, mobility requirements, and the increasing difficulty of travel as ALS progresses. Where scientifically appropriate, local laboratory testing, remote follow-up, home nursing, and flexible visit schedules can reduce burden and improve retention. Initiatives enabled through the ACT for ALS Act may further expand access to investigational treatment options when traditional trial participation is not feasible.

5. Reevaluate symptom-onset eligibility windows: Assess whether current symptom-onset limits appropriately balance scientific rigor with the realities of ALS diagnosis and referral.

Any change to eligibility windows involves trade-offs. Even where a wider window might admit more patients on paper, some would still be lost during pre-screening, medical-record collection, site review, and formal screening. Protocol feasibility assessments should therefore weigh both the eligibility window itself and the time required to move a patient from identification to enrollment, rather than treating the window in isolation.

Conclusion

Our analysis suggests that the ALS clinical trial challenge may not primarily be a trial supply problem. Most patients had multiple potential trial options, and only a small minority had no matching opportunity at all. Instead, access appears to erode through a series of timing and process barriers: diagnostic delay, referral lag, eligibility windows, and operational constraints.

This aligns with growing evidence from the broader ALS community that improving access will require more than opening additional trials. It will require helping patients reach those trials before the window of opportunity closes.

Findings also suggest that improving trial access will require more than additional studies and sites. It will also require earlier identification of people living with ALS, faster diagnostic and referral pathways, and patient navigation models that help connect eligible individuals to clinical trial opportunities before enrollment windows close.

For years, much of the field’s focus has been on creating more trials and more efficient trial infrastructure. Our analysis suggests that another challenge deserves equal attention: helping patients reach those opportunities before diagnostic delays, referral lag, and eligibility windows remove them from consideration.

Most people living with ALS do not lose access to research because no relevant trial exists. They lose access because diagnosis and referral delays consume the window of opportunity before ALS clinical trial enrollment can begin.