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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

  • Jeff Sutherland

    Jeff Sutherland
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  • Lachlan Terry, MND Australia, Diagnosed 2015, Australia

    Lachlan Terry, MND Australia, Diagnosed 2015, Australia

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Claire Garry, USA

    Claire Garry, USA
    20200117_214643

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Mary Thomas, Diagnosed 2013 - MND Australia

    Mary Thomas, Diagnosed 2013 – MND Australia

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • England-Lee-Millard, UK

    England-Lee-Millard, UK

  • Guido De Mets, Belgium

    Guido De Mets, Belgium

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Roxana Canova, Diagnosed 2012 - Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 – Asociación ELA Argentina

  • Steve Lufkin, USA

    Steve Lufkin, USA
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  • Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

  • UK-Mahmood-Anwar

    UK-Mahmood-Anwar

  • Brian Lovell, Diagnosed 2011 - MND Australia

    Brian Lovell, Diagnosed 2011 – MND Australia

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • David Bishop

    David Bishop

  • Ann Nicol

    Ann Nicol

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

    Erwin Coppejans, Diagnosed 2007 – ALS Liga België, Belgium

  • Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

    Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

  • Chris McCauley, Diagnosed 2015 - ALS Canada

    Chris McCauley, Diagnosed 2015 – ALS Canada

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

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

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

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

  • Fernando Ocampo Cardona, Colombia

    Fernando Ocampo Cardona, Colombia

  • Christian Bär, Germany

    Christian Bär, Germany

  • Zelina-Brito-Diagnosed-2018-Brazil

    Zelina-Brito-Diagnosed-2018-Brazil

  • Osiel Mendoza, Diagnosed 2016 - ALS Therapy Development Institute, USA

    Osiel Mendoza, Diagnosed 2016 – ALS Therapy Development Institute, USA

  • Monica Soriano, Diagnosed 2011 - Asociación ELA Argentina

    Monica Soriano, Diagnosed 2011 – Asociación ELA Argentina

  • Richard Clark, MND New Zealand, Diagnosed 2011, New Zealand

    Richard Clark, MND New Zealand, Diagnosed 2011, New Zealand

  • Conny van der Meijden, Diagnosed 2001 - ALS Netherlands

    Conny van der Meijden, Diagnosed 2001 – ALS Netherlands

  • Karl Hughes, Diagnosed 2010 - IMNDA, Ireland

    Karl Hughes, Diagnosed 2010 – IMNDA, Ireland

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

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

    Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Mark Miller

    Mark Miller

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

    Susan Anderson, Diagnosed 2014 – Hope Loves Company, USA

  • Ali Var, Turkey

    Ali Var, Turkey

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Eddy LeFrançois, Diagnosed 1992 - ALS Canada

    Eddy LeFrançois, Diagnosed 1992 – ALS Canada

  • Mike Rannie, ALS Canada, Diagnosed 2017, Canada

    Mike Rannie, ALS Canada, Diagnosed 2017, Canada

  • Elkin Ramiro Gaviria Muñoz, Diagnosed  December 2018

    Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Fabio Correia

    Fabio Correia

  • Kirsten Harley, Diagnosed 2013 - Australia

    Kirsten Harley, Diagnosed 2013 – Australia

  • Joanne Pratt, Diagnosed 2011 - MND Australia

    Joanne Pratt, Diagnosed 2011 – MND Australia

  • David Watson, MND Scotland, Diagnosed 2018, Scotland

    David Watson, MND Scotland, Diagnosed 2018, Scotland

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