Increasing Enrollment in ALS Clinical Trials: Four Things Patients Need

Clinical trials are research studies performed in people in order to understand (i) more about a disease and/or (ii) if a potential treatment is safe and can slow, stop, modify or reverse a disease’s progression. They are a critical step in making real potential new treatments for ALS patients, and patient participation in trials is crucial to their success. In February 2019, Ipsos launched a national survey on behalf of I AM ALS to assess ALS patients’ knowledge of, barriers to enrollment in, and ways to eliminate barriers to clinical trials. The survey was designed in partnership with the ALS community, including patients, caregivers, researchers, physicians, pharmaceutical companies, non-profit organizations, and others.

The survey, which received a total of 551 responses from patients with ALS, was distributed on-line and over email by ALS TDI and I AM ALS. The full results of the poll are available here and we hope you will take the time to read through it and share. These results represent the lived experiences of hundreds of ALS patients—experiences that for years were ignored when it came time to design clinical trial protocols. They illuminate real issues with clinical trial designs—issues that produce longer enrollment periods, higher dropout rates due to unnecessarily burdensome and/or lengthy trials, and a general avoidance of trials by too many patients. By hindering patient participation in trials, these design issues slow scientific advancement and delay the development of potential new treatments. We can and must do better.

There are literally dozens of key takeaways from this poll. One that stood out to us was that 78% of respondents strongly agreed or agreed that they are more willing to participate in trials to help find a cure for ALS even though they themselves will not benefit from them because this disease is fatal. This altruism of the ALS patient community is, in a word, astounding.

This altruism alone, however, means little if we don’t have trials that are readily available to patients and which are designed to give every patient a chance to contribute to the fight. In this vein, we have identified some key takeaways from the poll below. These are not meant to be exhaustive, but to start the conversation about how we build more patient-centric trials and speed the development of the treatments we all seek.

1. We need effective education about clinical trials. Only 20% reported that they knew a great deal about clinical trials, and 24% reported that they understood the differences between phases very well. The majority of respondents (59%) mentioned that they have not participated in any clinical trial. Many (32%) respondents reported that they knew a little about clinical trials, and 30% reported that they understood little about the differences between clinical trial phases. Every patient diagnosed with ALS should be educated about their role in research and clinical trials.

2. We need to improve access to clinical trials. Of those who noted they had not participated in trials (n=324), 17% reported that they were not aware of any trials, and 13% reported they didn’t know how to find information about trials.

Most respondents reported that they found information about trials through other organizations/associations for their disease (54%), their doctor or medical teams (53%), and ClinicalTrials.gov or other government websites. Forty-two percent of patients surveyed also noted they find information about trials in web searches, and 26% noted through social media.

Additionally, we have to address financial barriers to accessing clinical trials. Many respondents (39%) reported that they knew people with ALS who can’t afford to participate in clinical trials, even though they wish they could. Many respondents also noted that they are more likely to participate in clinical trial phases 1 (74%), 2 (79%) and 3 (83%) if travel costs for them and a caregiver were covered.

3. We need to make clinical trials more patient-friendly. Clinical trials can sound daunting, and 25% of respondents noted they were afraid of them. This is one of the reasons why we need to design a clinical trial process that incorporates patients’ values and supports them throughout the process. Most respondents (72%) reported they would be more likely to consider participating in a clinical trial if they had a patient navigator or advocate at their primary hospital to help with participating in trials, including helping them with coordinating logistics of the trial.

Also, most respondents (87%) indicated being willing to travel by car anywhere from 30 mins to over 6 hours to participate in a trial, and many were willing to travel by air anywhere in the United States (24%) and the world (28%) to participate in a trial. More than half of respondents also stated that they were more likely to participate in a trial if an organization provided loaner equipment during travel for the trial such as hoyer lifts and wheelchairs.

Most respondents (82%) noted that they would be willing to participate in televisits to supplement in-person visits, which could greatly reduce travel burden, and related financial costs, on patients and their caregivers. They also stated they would be willing to have a study team visit them at home to make assessments (83%), go to a local hospital or clinic for blood draws (82%), and wear a ‘wearable’ device to remotely monitor movement and other clinically relevant data (75%) and more.

4. We need innovative trial designs. Many respondents reported they were more likely to participate in a trial that was testing multiple drugs at once with different patient groups, producing more data more quickly across phases 1 (49%), 2 (55%) and 3 (60%). The most recently revised FDA Guidance for Industry on Developing Drugs for Treatment of ALS supports the implementation of such innovations.

Many respondents also noted they were more likely to participate in trials that were six months or less, and did not require them to stop taking other medications or supplements they are on. Most respondents also reported that they were more likely to participate in a trial if they had guaranteed access to the drug after the trial was over through expanded or post-trial access.