Background
The desire of someone living with ALS/MND to try experimental therapeutics that lack complete knowledge of their safety or efficacy is understandable. To many, the nature of the disease creates a situation where the potential benefit often outweighs the risk of getting worse or death.
However, the existence of Right to Try legislation in any form often does not result in extensive use by a country’s citizens, because it also requires the company that owns the treatment to comply.
For Right to Try to result in someone with ALS/MND receiving an unapproved, often experimental, treatment, the following three things must all be in place.
- Some form of Right to Try legislation must exist, where government regulatory barriers that would normally safeguard citizens against unproven and unapproved treatments are reduced or removed.
- Some countries have been willing to pass this type of legislation.
- An owner of a treatment, often a small or large pharmaceutical company, is willing to provide the treatment, usually while it is still in the clinical trial process.
- Many companies with experimental treatments will hesitate to provide them through Right to Try while they remain in clinical development, because any adverse events or misuse of the treatment under non-controlled conditions could jeopardize their program and their potential to bring the treatment to market. Such a situation may be detrimental not only to the company or owner but to everyone living with ALS/MND, if an effective treatment is called into question because of a Right to Try issue.
- Cost coverage for the treatment must be identified, either through the owner providing it free of charge or the recipient paying out-of-pocket.
- Without government oversight, an owner can set their own price, which may be very high and out of reach for some or many people.
- An owner may set prices at very high levels to compensate for the risk of providing the treatment during an ongoing clinical development program.
- Owners who do not wish to make individuals pay out-of-pocket for what are often very expensive treatments to manufacture and deliver will often be unable to afford to provide the treatment to everyone through Right to Try.
- Owners who decide to provide a limited amount of free treatment to a select group of people would likely be subject to scrutiny in a potential future market for them.
An additional potential consequence of Right to Try legislation is a reduced level of protection against illegitimate companies or owners with products that may be unsafe or knowingly ineffective. Government-legislated access to dubious treatments may give them a level of legitimacy on par with companies or owners who are taking the appropriate clinical trial steps to prove the safety and efficacy of their products.
Recommendation
The Scientific Advisory Council (SAC) recommends that members of the Alliance refrain from making any opinion-based statements about Right to Try and refer those with questions to this document. Members should be aware of the realities of Right to Try (listed above) and compassionately provide information that balances an understanding of the position people living with ALS/MND are in with the reasons why Right to Try legislation is not the only step in accessing experimental or unapproved treatments. Medical advice regarding any specific treatment should be deferred to an individual’s own clinician. Further objective information about what is known regarding a specific treatment in development may be sought through the SAC.
Further information
Companies often want to help people living with ALS/MND and truly care about developing and providing an effective therapy as quickly as possible. It is common for prominent clinical trials to offer extension studies, where all participants, regardless of whether they were on treatment or placebo during the trial, can receive the treatment at the company’s expense. These mechanisms allow the treatment to continue in a controlled situation where risk is minimized and safety data can be collected. Learn more on the Open Label Extension page.
International Alliance of ALS/MND Associations
June 2020
The original language of communication is English, and any translation cannot be guaranteed for accuracy of messaging.