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International Alliance of ALS/MND Associations

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US FDA Orphan Drug Designation

US FDA Orphan Drug Designation

Background

As our understanding of the underlying biology of ALS has improved, multiple experimental treatment approaches have been identified. Currently, there are numerous interventional drug trials testing a diverse set of targets using multiple drug approaches (e.g. small molecules, antibodies, stem cells, gene interface/silencing technologies). Drug regulators have also set up programs to provide special incentives for companies to develop drugs and biologicals for rare diseases that have a small market (fewer than 200,000 people such as the ALS community). One such program is the orphan drug designation (or sometimes “orphan status”) administered at the Food and Drug Administration (FDA) of the United States. Created in 1983, this program provides financial incentives and research subsidies such as partial tax credit for clinical trial expenditures, waived user fees, and eligibility for market exclusivity for that drug. The European Medicines Agency (EMA) of the European Union also has a similar program and defines a drug as “orphan” for a rare disease affecting fewer than 5 in 10,000 people. As of 2020, over 80 drugs have been designated by the FDA with the orphan drug status for ALS including recent example,s Cytokinetics’ drug reldesemtiv and Neuropore’s drug NPT520-34.

Benefit for patients
The National Organization for Rare Disorders along with many other organizations led the lobbying efforts for the passage and formation of this program. Companies will typically shy away from developing drugs for a rare disease area due to financial considerations, limited profit margins, as well as the scientific, ethical and operational complexities of conducting clinical research in small niche patient populations. This program motivates, supports and incentivizes drug companies to invest their research and development capital (both scientific and financial) with the hope that more medical breakthroughs will be made available for patients with rare diseases than otherwise would have been achieved.

Benefit for companies/sponsors
Companies benefit from this program as it allows them exclusive marketing and development rights and allows them to partially recover the costs of research and developing the drug. In addition to cost reductions, the FDA provides streamlining of regulatory processes and guidance for those drugs with such a designation. Additionally, investors in pharmaceutical companies often view orphan designation as a signal of higher company value, thus bringing in more capital needed for the drug discovery program.

What orphan drug designation means and does not mean
While the orphan drug designation can be an important milestone for a drug company, it is important to note that orphan drugs, like non-orphan drugs, are still required to show safety and efficacy prior to approval for use as a therapeutic. Orphan drug designations are typically given early on in development and orphan drugs must still follow the proper clinical development and regulatory process. It is also important to state that orphan drug designation should not be confused with other regulatory designations such as fast-track designation. Fast-track designation does not provide drug companies with any explicit financial benefits for developing its drug, however, it does provide more frequent meetings with the FDA, as well as an expedited review when submitting an application to bring a new drug to market.

Recommendation

The SAC hopes that drug companies take advantage of incentives and expedited pathways set up by regulatory agencies such as the orphan drug designation to discover and rigorously test new treatments for people with ALS.

 

International Alliance of ALS/MND Associations
July 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

  • Mark Miller

    Mark Miller

  • Claire Garry, USA

    Claire Garry, USA
    20200117_214643

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Mauril Bélanger, Diagnosed 2015 , ALS Canada

    Mauril Bélanger, Diagnosed 2015 , ALS Canada

  • Mike Rannie,  ALS Canada,  Diagnosed 2017

    Mike Rannie, ALS Canada, Diagnosed 2017

  • Dick Dayton, USA

    Dick Dayton, USA

  • Orlando Ruiz, Diagnosed 2001,  ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001, ACELA, Colombia

  • Inta Grubb, Diagnosed 2014,  MND Australia

    Inta Grubb, Diagnosed 2014, MND Australia

  • Daniel Hare

    Daniel Hare

  • Andrietta

    Andrietta

  • Zelina Brito, Diagnosed 2018, Brazil

    Zelina Brito, Diagnosed 2018, Brazil

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Den Haag, Diagnosed 2016 , The Netherlands

    Den Haag, Diagnosed 2016 , The Netherlands

  • Fabio Correia

    Fabio Correia

  • Richard Clark, MND New Zealand,  Diagnosed 2011

    Richard Clark, MND New Zealand, Diagnosed 2011

  • H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

    H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

  • Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

  • Chun Ju Xiao, China

    Chun Ju Xiao, China

  • Frank "Papa" Taylor, USA

    Frank “Papa” Taylor, USA

  • Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

    Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

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

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

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Conny van der Meijden, Diagnosed 2001,  ALS Netherlands

    Conny van der Meijden, Diagnosed 2001, ALS Netherlands

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Paul Launer, USA

    Paul Launer, USA

  • Kirsty Gerlach, MND New Zealand, Diagnosed 2017

    Kirsty Gerlach, MND New Zealand, Diagnosed 2017

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Mahmood Anwar, UK

    Mahmood Anwar, UK

  • Andrea Zicchieri, Associazione conSLAncio Onlus, Italy

    Andrea Zicchieri, Associazione conSLAncio Onlus, Italy
    AndreaZicchieri_conSLAncioItaly

  • Marco Antonio Alvarez Mercado, Mexico

    Marco Antonio Alvarez Mercado, Mexico

  • Armando González Gómez, ACELA, Colombia

    Armando González Gómez, ACELA, Colombia

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

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

  • Wilfried Leusing

    Wilfried Leusing

  • Philip Brindle,  MND Association,  Diagnosed 2015,  England

    Philip Brindle, MND Association, Diagnosed 2015, England

  • David Bishop

    David Bishop

  • Imelda Arenas, ACELA, Colombia

    Imelda Arenas, ACELA, Colombia

  • Hollister

    Hollister
    hollister

  • Debbie Craghill, USA

    Debbie Craghill, USA

  • Mauril Belanger

    Mauril Belanger

  • Lachlan Terry,  MND Australia,  Diagnosed 2015

    Lachlan Terry, MND Australia, Diagnosed 2015

  • March of Faces Photo Submission_ALEX_ELA ARGENTINA

    March of Faces Photo Submission_ALEX_ELA ARGENTINA

  • Lombana, Spain

    Lombana, Spain

  • Ian Gale, MND Australia

    Ian Gale, MND Australia

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

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

    Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

  • Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

    Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

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