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International Alliance of ALS/MND Associations

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Right to Try

Right to Try

Background

The desire for someone living with ALS to try experimental therapeutics that lack complete knowledge of safety and/or efficacy is understandable. To many, the nature of the disease creates a situation where the risk of benefit often outweighs the risk of getting worse or death.

However,  the  existence of a Right to Try  legislation in any form often does not result in extensive utilization 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 receiving an unapproved, often experimental, treatment, the following three things must ALL be in place.

    1. 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.
    2. An owner of a treatment, often a small or large pharmaceutical company, is willing to provide the treatment, usually while still in the clinical trial process.
      • Many companies with experimental treatments will hesitate to provide it through Right to Try while it remains in clinical development as any adverse events or misuse of the treatment under non-controlled conditions could jeopardize their program and potential to bring the treatment to market. Such a situation may not only be detrimental for the company/owner, but everyone living with ALS if an effective treatment is called into question due to a Right to Try issue.
    3. Cost coverage for the treatment must be identified, either through the owner providing it free of charge or the recipient paying out-of-pocket.
      • No oversight from government can allow for an owner to set their own price, which may be very high and exclusionary to some or many.
      • An owner may set prices at very high levels to compensate for risk of providing the treatment during an ongoing clinical development program.
      • Owners who don’t wish to make individuals pay out-of-pocket for what are often very expensive treatments to manufacture and deliver, often won’t be able to afford provision of the treatment to all 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 the reduced level of protection against illegitimate companies/owners with products that may be unsafe or knowingly ineffective. Providing government legislated access to dubious treatments may assign a level of legitimacy to them on par with companies/owners who are taking the appropriate clinical trial steps to prove safety and efficacy of their product.

Recommendation

The SAC recommends that members of the Alliance should refrain from making any opinion-based statements about Right to Try and should 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 with ALS 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

Often companies want to help people with ALS 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 expense. These mechanisms allow for the treatment to continue in a controlled situation where risk is minimized and safety data can be collected.

 

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.

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Advocacy

  • Advocacy Toolkit

  • Conny van der Meijden, Diagnosed 2001 - ALS Netherlands

    Conny van der Meijden, Diagnosed 2001 – ALS Netherlands

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Francisco Perez Palop, Diagnosed 2013 - FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 – FUNDELA, Spain

  • Roy

    Roy
    roy

  • Ali Var, Turkey

    Ali Var, Turkey

  • Denis Blais, Diagnosed 2015 - ALS Canada

    Denis Blais, Diagnosed 2015 – ALS Canada

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

  • Susan Anderson, Diagnosed 2014 - Hope Loves Company, USA

    Susan Anderson, Diagnosed 2014 – Hope Loves Company, USA

  • Angie Bordaen, Diagnosed 2014 - ALS Liga België, Belgium

    Angie Bordaen, Diagnosed 2014 – ALS Liga België, Belgium

  • Wilfried Leusing, Diagnosed 2010 - DGM, Germany

    Wilfried Leusing, Diagnosed 2010 – DGM, Germany

  • Danny Reviers, Diagnosed 1979 - ALS Liga België, Belgium

    Danny Reviers, Diagnosed 1979 – ALS Liga België, Belgium

  • Ian Roberts

    Ian Roberts

  • Willi Klein

    Willi Klein

  • Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Orlando Ruiz, Diagnosed 2001 - ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001 – ACELA, Colombia

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Chen Chun-Chin

    Chen Chun-Chin

  • Timothy Holman, Switzerland

    Timothy Holman, Switzerland

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

  • Len Johnrose, MND Association, Diagnosed 2017, England

    Len Johnrose, MND Association, Diagnosed 2017, England

  • Mike Rannie, ALS Canada, Diagnosed 2017, Canada

    Mike Rannie, ALS Canada, Diagnosed 2017, Canada

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Fabio Correia

    Fabio Correia

  • Daniel Hare

    Daniel Hare

  • Joy Blakeley, Diagnosed 2017 - MND Australia

    Joy Blakeley, Diagnosed 2017 – MND Australia

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Lachlan Terry, MND Australia, Diagnosed 2015, Australia

    Lachlan Terry, MND Australia, Diagnosed 2015, Australia

  • Inta Grubb, Diagnosed 2014 - MND Australia

    Inta Grubb, Diagnosed 2014 – MND Australia

  • Mary Thomas, Diagnosed 2013 - MND Australia

    Mary Thomas, Diagnosed 2013 – MND Australia

  • Mark Miller

    Mark Miller

  • Brian Lovell, Diagnosed 2011 - MND Australia

    Brian Lovell, Diagnosed 2011 – MND Australia

  • Robbie Caliste, UK

    Robbie Caliste, UK

  • Steven Spencer, Diagnosed 2014 - MND New Zealand

    Steven Spencer, Diagnosed 2014 – MND New Zealand

  • Roxana Canova, Diagnosed 2012 - Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 – Asociación ELA Argentina

  • Carlos Gomez Matallanas, Diagnosed 2014 - FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 – FUNDELA, Spain

  • Zelina-Brito-Diagnosed-2018-Brazil

    Zelina-Brito-Diagnosed-2018-Brazil

  • Claire Garry, USA

    Claire Garry, USA
    20200117_214643

  • Fernando Ocampo Cardona, Colombia

    Fernando Ocampo Cardona, Colombia

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

  • Ann Nicol

    Ann Nicol

  • Dorette Lüdi, Diagnosed 2014 - ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 – ALS Schweiz, Switzerland

  • Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

    Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

  • Erwin Coppejans, Diagnosed 2007 - ALS Liga België, Belgium

    Erwin Coppejans, Diagnosed 2007 – ALS Liga België, Belgium

  • Monica Soriano, Diagnosed 2011 - Asociación ELA Argentina

    Monica Soriano, Diagnosed 2011 – Asociación ELA Argentina

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Josée Kolijn-de Man, Diagnosed 2015 - ALS Patients Connected, The Netherlands

    Josée Kolijn-de Man, Diagnosed 2015 – ALS Patients Connected, The Netherlands

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