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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

  • Rosie Riley, Les Turner ALS Foundation, USA

    Rosie Riley, Les Turner ALS Foundation, USA

  • Tison, USA

    Tison, USA

  • Ada Garrido Benavidez, Diagnosed 2016,  FYADENMAC, Mexico

    Ada Garrido Benavidez, Diagnosed 2016, FYADENMAC, Mexico

  • Bjarne Hytjanstorp, ALS Norge, Norway

    Bjarne Hytjanstorp, ALS Norge, Norway

  • Willi Klein

    Willi Klein

  • Richard Clark, MND New Zealand,  Diagnosed 2011

    Richard Clark, MND New Zealand, Diagnosed 2011

  • Mark Miller

    Mark Miller

  • Steve Lufkin, USA

    Steve Lufkin, USA
    IMG_3993

  • Hollister

    Hollister
    hollister

  • Ali Var, Turkey

    Ali Var, Turkey

  • Jean

    Jean
    jean

  • Chen Yin Xue, Taiwan MND Association, Diagnosed 1995, Taiwan

    Chen Yin Xue, Taiwan MND Association, Diagnosed 1995, Taiwan

  • IMG_1211

    IMG_1211

  • Mauril Belanger

    Mauril Belanger

  • Maurice Leclerc, Canada

    Maurice Leclerc, Canada

  • Dad

    Dad

  • Ana Lilia RodriguezApoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Ana Lilia RodriguezApoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Eric Von Schaumburg, USA

    Eric Von Schaumburg, USA

  • 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

  • Jack Buzby, USA

    Jack Buzby, USA

  • JP

    JP

  • Art Eggert, USA

    Art Eggert, USA

  • Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

    Camilla Heiberg Freiberg, Muskelsvindfonden, Denmark

  • Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

    Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

  • Steve Gallagher, ALS Society of Canada

    Steve Gallagher, ALS Society of Canada
    Picture1

  • Purningam Jacob, Diagnosed 2012 , Asha Ek Hope Foundation, India

    Purningam Jacob, Diagnosed 2012 , Asha Ek Hope Foundation, India

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

    Maria Santos Garcia Tellez, Diagnosed 2017 , FYADENMAC, Mexico

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

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • 83

    83

  • Ian and Teresa Roberts

    Ian and Teresa Roberts

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Len Johnrose,  MND Association,  Diagnosed 2017,  England

    Len Johnrose, MND Association, Diagnosed 2017, England

  • IMG_2658

    IMG_2658

  • Liz Ogg, Diagnosed 2013 , MND Scotland, UK

    Liz Ogg, Diagnosed 2013 , MND Scotland, UK

  • Claudia Gotti, Brazil

    Claudia Gotti, Brazil

  • Chen Chun-Chin

    Chen Chun-Chin

  • Paul Launer, USA

    Paul Launer, USA

  • Enzo Maccarrone, AISLA ONLUS, Italy

    Enzo Maccarrone, AISLA ONLUS, Italy

  • Graham Johnson, MND Australia

    Graham Johnson, MND Australia

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • Timmy, ALS Liga

    Timmy, ALS Liga

  • Inta Grubb, Diagnosed 2014,  MND Australia

    Inta Grubb, Diagnosed 2014, MND Australia

  • Frank "Papa" Taylor, USA

    Frank “Papa” Taylor, USA

  • John Dinon, MND Australia

    John Dinon, MND Australia

  • Mary Thomas, Diagnosed 2013 , MND Australia

    Mary Thomas, Diagnosed 2013 , MND Australia

  • Susan Keldani, Les Turner ALS Foundation, USA

    Susan Keldani, Les Turner ALS Foundation, USA

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