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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

  • Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

    Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

  • Oliver Juenke, Germany

    Oliver Juenke, Germany

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Dr Shelly Hoover

    Dr Shelly Hoover

  • Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

    Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

  • Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

    Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

  • Liz Ogg, Diagnosed 2013 , MND Scotland, UK

    Liz Ogg, Diagnosed 2013 , MND Scotland, UK

  • Brigitte Wernli,  Association ALS Switzerland,  Diagnosed 2014

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014

  • Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

  • Jose Espinosa, Argentina

    Jose Espinosa, Argentina

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Dawn Morton, Diagnosed 2014 , MND Scotland, UK

    Dawn Morton, Diagnosed 2014 , MND Scotland, UK

  • Timmy, ALS Liga

    Timmy, ALS Liga

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Mike Small, Motor Neurone Disease (MND) Association, UK

    Mike Small, Motor Neurone Disease (MND) Association, UK

  • Liam Dwyer, England

    Liam Dwyer, England

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

    Danny Reviers, Diagnosed 1979 , ALS Liga België, Belgium

  • Jan Zuring, Diagnosed 2010 , The Netherlands

    Jan Zuring, Diagnosed 2010 , The Netherlands

  • Hollister

    Hollister
    hollister

  • Wilfried Leusing

    Wilfried Leusing

  • Marco Antonio Alvarez Mercado, Mexico

    Marco Antonio Alvarez Mercado, Mexico

  • Brian Parsons

    Brian Parsons

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

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

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

    David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

  • Norm MacIsaac,  ALS Society of Canada,  ALS Society of Quebec,  Diagnosed 2014

    Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Fabio Correia

    Fabio Correia

  • Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

    Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

  • Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

    Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

  • David Watson,  MND Scotland,  Diagnosed 2018

    David Watson, MND Scotland, Diagnosed 2018

  • Kirsten Harley,  Diagnosed 2013,  Australia

    Kirsten Harley, Diagnosed 2013, Australia

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Duncan Bayly , MND Australia

    Duncan Bayly , MND Australia

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

    Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

  • Ian Roberts

    Ian Roberts

  • 727747090571358167

    727747090571358167

  • Antonio Ventriglia,  ALS Liga Belgium,  Diagnosed 2013

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013

  • Den Haag, Diagnosed 2016 , The Netherlands

    Den Haag, Diagnosed 2016 , The Netherlands

  • Claudia Gotti, Brazil

    Claudia Gotti, Brazil

  • Nicholas (Nic) Bowman, MND Association of South Africa,  Diagnosed 2016,  Australia

    Nicholas (Nic) Bowman, MND Association of South Africa, Diagnosed 2016, Australia

  • Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

    Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

  • Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

    Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

  • Kirsty Gerlach, MND New Zealand, Diagnosed 2017

    Kirsty Gerlach, MND New Zealand, Diagnosed 2017

  • Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

    Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

  • Animesh Kumar, Diagnosed 2013 , Asha Ek Hope Foundation, India

    Animesh Kumar, Diagnosed 2013 , Asha Ek Hope Foundation, India

  • MNDaSG Group PALS & CALS, Motor Neurone Disease Association, Singapore (MNDaSG)

    MNDaSG Group PALS & CALS, Motor Neurone Disease Association, Singapore (MNDaSG)

  • Greg Heydet, ALS Hope Foundation, USA

    Greg Heydet, ALS Hope Foundation, USA

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