Living with ALS: What we heard from patients

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First listening session with ALS patients

Health Canada wanted to hear from people living with ALS (amyotrophic lateral sclerosis). We held our first listening session with representatives from Canada's ALS community on December 7, 2021. Joining the session were members of the ALS Society of Canada and ALS Action Canada.

The members:

ALS (also known as Lou Gehrig's disease) is a progressive nervous system disease that affects nerve cells in the brain and spinal cord, causing loss of muscle control. Over time, as the muscles of the body break down, people with ALS lose the ability to walk, talk, eat, swallow and eventually breathe.

Currently, there is no cure for ALS and treatment options for those living with the disease are limited. About 80% of people with ALS will die within 2 to 5 years after diagnosis.

The main theme of the session was access to ALS therapies in Canada. Health Canada gave the participants several questions in advance. The questions focused on what we wanted to learn more about and what participants wanted us to know.

Participants gave a number of points for Health Canada (and others involved in the access to medicine process) to consider.

Patients have high tolerance for risk

Many ALS patients have a high tolerance for risk in potential treatments, due to both the debilitating and lethal nature of the disease, as well as the lack of available treatment options. Many attending the session said that no risk is too great. One participant said, "If patients were offered a treatment that had a 50/50 chance of completely curing their ALS or of being fatal, 100% of patients would take the treatment".

Participants said patients, not regulators, should have the final say on whether a potential treatment poses too high a risk. Currently, they can assess the risk of a product when they choose to participate in a clinical trial. Yet, Health Canada makes the final decisions regarding whether a product that has undergone clinical trials will be authorized (approved) for sale in Canada.

List a product's risks on the label and let the patient decide whether to take the product.

Incremental progress is key

ALS is a diverse disease. How it progresses in someone varies greatly from person to person. This means that a single drug will likely not work for all ALS patients. Many different drugs may be required.

Participants emphasized the need for regulators to not wait for a home-run drug that is very safe and effective for all patients. Progress is made in stages and requires taking a calculated risk.

Give priority review to ALS therapies

Health Canada should review all ALS therapies on a priority basis. Participants cited the rapid and degenerative nature of the disease and the few treatment options that are available in Canada. To ensure that future ALS submissions are reviewed quickly, participants urged Health Canada to update the criteria for priority review and to develop new accelerated pathways.

Faster access through innovation

Participants said the most meaningful thing that Health Canada can do for the ALS community is to help improve access. Authorizing new treatments is a crucial first step, but is only one piece.

After we authorize a drug, the Canadian Agency for Drugs and Technology in Health (CADTH) or l'Institut national d'excellence en santé et services sociaux (INESSS) will review the drug. These organizations assess a drug's "value for money" and make recommendations to the payers of the drug, which are the provinces and territories. The provinces and territories then decide whether they will cover the drug.

This process, from new drug submission to reimbursement, can take a long time, between 2 and 3 years on average.

Participants recommended that all groups involved in the drug review and approval process look at innovative ways to speed up the access time. Some of their recommendations include the following:

Health Canada thanks everyone who participated in this listening session. We are grateful for your thoughts and feedback, which we will use to strengthen our capacity as a regulator. We will consider your input in future regulatory initiatives to support access to new drugs for patients with life-threatening or severely debilitating conditions.

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