Regulatory Open Forum

 View Only

  • 1.  Establishing Prevalence of an Orphan Disease

    Posted 12-Nov-2014 17:54
    Does anyone know a good consultant for establishing the prevalence of an orphan disease?

    -------------------------------------------
    Clark Sullivan JD
    Troutman Sanders
    New York NY
    United States
    -------------------------------------------


  • 2.  RE: Establishing Prevalence of an Orphan Disease

    Posted 13-Nov-2014 02:54
    Clark,
    You may contact Voisin Consulting at voisin@voisinconsulting.com  
    or +33-6-60284702, Emmanuelle Voisin's direct number

    Best regards

    -------------------------------------------
    Manfred Ruthsatz PHD, RAC, RPH, FRAPS
    Global Head Regulatory Advocacy
    Nestlé Health Science
    VEVEY
    Switzerland
    -------------------------------------------


    Original Message


  • 3.  RE: Establishing Prevalence of an Orphan Disease

    Posted 13-Nov-2014 07:47
    Hi Clark -
    In a previous life at a biotech company that worked on Fabry disease, we conducted a literature search ourselves. If you look on Pub Med and search for papers on the given disease state, you should be able to find prevalance. You can then purchase the articles and provide them to the Office of Orphan Drug Product as supporting documentation.

    I hope this helps.
    Kind regards,
    Shannon

    -------------------------------------------
    Shannon Wehrendt MA
    West-Ward Pharmaceuticals
    Eatontown NJ
    United States
    -------------------------------------------


    Original Message


  • 4.  RE: Establishing Prevalence of an Orphan Disease

    Posted 13-Nov-2014 09:58
    I assume that you want this to support an orphan drug designation application.

    We can do this for you, as we have for a number of our clients.

    Shayne C Gad
    Gad Consulting Services
    scgad@ix.netcom.com

    -------------------------------------------
    Shayne Cox Gad ScD PhD
    Principal
    Gad Consulting Services
    Cary NC
    United States
    -------------------------------------------


    Original Message


  • 5.  RE: Establishing Prevalence of an Orphan Disease

    Posted 14-Nov-2014 10:29
    Clark,
    You may want to start off by visiting the NORD (National Organization of Rare Diseases) website:
    https://www.rarediseases.org/
    The Department of Health and Human Services (HHS) also maintains a database of this information>
    Good Luck,
    Marge
    -------------------------------------------
    Marge Walls-Walker
    Sr. Regulatory Specialist: Engineering
    Cook Endoscopy
    Winston-Salem NC
    United States
    -------------------------------------------


    Original Message


  • 6.  RE: Establishing Prevalence of an Orphan Disease

    Posted 17-Nov-2014 08:37
    We also do a lot of this work, and it is certainly tricky for many conditions.  Although websites like NORD's or Orphanet can lead you to other resources, they are not considered by regulatory agencies to be valid data sources.  The foundation of most prevalence sections in orphan drug designation requests is a thorough literature search.  Depending on what is found in the literature, there is usually a need for a certain amount of inference to build an argument.

    With best regards,
    Kathy

    -------------------------------------------
    Kathryn Wekselman
    Senior Director, Regulatory and Scientific Affairs
    CTI Clinical Trial and Consulting Services
    Cincinnati OH
    United States
    -------------------------------------------


    Original Message


  • 7.  RE: Establishing Prevalence of an Orphan Disease

    Posted 17-Nov-2014 19:48
    Clark,

    I would refer you to the recent guidance (e.g., rare pediatric disease review vouchers), which describes how to calculate prevalence estimates or http://regulatorydoctor.com/rare-pediatric-disease-priority-review-vouchers/


    Regulatory Doctor     LinkedIn Google Plus Twitter Facebook






    Original Message


  • 8.  RE: Establishing Prevalence of an Orphan Disease

    Posted 18-Nov-2014 10:19
    Hi, Clark -

    I worked for a mid-sized CRO (Cato Research, www.cato.com) for 7 years before moving to a large pharma company.  I would strongly recommend them for this type of work - they are great at regulatory/clinical research, and creative solutions for niche indications.  They are the polar opposite of the cookie-cutter approach you'll see from a lot of CROs.  Feel free to touch base with me on LinkedIn if you have questions...

    Best,

    Cathy 

    -------------------------------------------
    Catherine Anderson RAC
    Manager - Regulatory CMC
    Hillsborough NC
    United States
    -------------------------------------------


    Original Message