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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

  • Dawn Morton, Diagnosed 2014 , MND Scotland, UK

    Dawn Morton, Diagnosed 2014 , MND Scotland, UK

  • Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

    Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

  • Hollister

    Hollister
    hollister

  • Liam Dwyer, England

    Liam Dwyer, England

  • Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Brian Lovell, Diagnosed 2011 . MND Australia

    Brian Lovell, Diagnosed 2011 . MND Australia

  • Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

    Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • March of Faces Photo Submission_ALEX_ELA ARGENTINA

    March of Faces Photo Submission_ALEX_ELA ARGENTINA

  • Wendy Hendrickson, ALS Hope Foundation, USA

    Wendy Hendrickson, ALS Hope Foundation, USA

  • Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

  • Kirsty Gerlach, MND New Zealand, Diagnosed 2017

    Kirsty Gerlach, MND New Zealand, Diagnosed 2017

  • Robbie Caliste, UK

    Robbie Caliste, UK

  • Enzo Maccarrone, AISLA ONLUS, Italy

    Enzo Maccarrone, AISLA ONLUS, Italy

  • Maurice Leclerc, Canada

    Maurice Leclerc, Canada

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

    Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

  • Claudia Cominetti, Associazione conSLAncio Onlus,  Italy

    Claudia Cominetti, Associazione conSLAncio Onlus, Italy

  • Roxana Canova, Diagnosed 2012 ,  Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 , Asociación ELA Argentina

  • Shera Mukherjee, Diagnosed 2013,  Asha Ek Hope Foundation, India

    Shera Mukherjee, Diagnosed 2013, Asha Ek Hope Foundation, India

  • Susan Keldani, Les Turner ALS Foundation, USA

    Susan Keldani, Les Turner ALS Foundation, USA

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Claire Garry, USA

    Claire Garry, USA
    20200117_214643

  • Imelda Arenas, ACELA, Colombia

    Imelda Arenas, ACELA, Colombia

  • Charlie “Hark” Dourney, Diagnosed 2007 , Hark ALS, USA

    Charlie “Hark” Dourney, Diagnosed 2007 , Hark ALS, USA

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • Paul Launer, USA

    Paul Launer, USA

  • Steven Spencer, Diagnosed 2014 , MND New Zealand

    Steven Spencer, Diagnosed 2014 , MND New Zealand

  • Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Ian and Teresa Roberts

    Ian and Teresa Roberts

  • IMG_1211

    IMG_1211

  • Armando González Gómez, ACELA, Colombia

    Armando González Gómez, ACELA, Colombia

  • Dr Shelly Hoover

    Dr Shelly Hoover

  • Eric Von Schaumburg, USA

    Eric Von Schaumburg, USA

  • Steve

    Steve

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

    Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Angela Jansen, Deutsche Gesellschaft für Muskelkranke e.V.-DGM, Diagnosed 1995, Germany

    Angela Jansen, Deutsche Gesellschaft für Muskelkranke e.V.-DGM, Diagnosed 1995, Germany

  • Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

    Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

  • Carlos Alberto Báez Murillo, ACELA, Colombia

    Carlos Alberto Báez Murillo, ACELA, Colombia

  • Chris McCauley, Diagnosed 2015 , ALS Canada

    Chris McCauley, Diagnosed 2015 , ALS Canada

  • Bayley, Australia

    Bayley, Australia

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

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

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

    Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

  • IMG_2658

    IMG_2658

  • Irene McCaughey, Diagnosed 2011,  MND Australia

    Irene McCaughey, Diagnosed 2011, MND Australia

  • Sébastien Batiot, Diagnosed 2012 , ARSLA, France

    Sébastien Batiot, Diagnosed 2012 , ARSLA, France

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