Incentives for Drug Approval - How & Why to Use Them PowerPoint Presentation, PPT - DocSlides

Incentives for Drug Approval - How & Why to Use ThemAquaculture Drug WorkshopBozeman, MontanaJuly 31, 2013

Meg Oeller, DVM FDA Center for Veterinary Medicine

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Caveat

Please note that these slides have been altered slightly from those used in the presentation at the aquaculture workshop. This was done largely to facilitate making these slides 508 compliant for posting on our website. The pertinent content has not been changed.

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Need-to-know acronyms

MUMS – Minor Use & Minor Species (all fish are minor species)OMUMS – Office of Minor Use & Minor Species Animal Drug DevelopmentONADE - Office of New Animal Drug EvaluationINAD – Investigational New Animal DrugPMF – Public Master FileNADA – New Animal Drug Application

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Who am I?

Meg Oeller, DVM 20 years at FDA Center for Veterinary Medicine (CVM) Aquaculture drug reviewer in 1993 (with Tom Bell) FDA Liaison to USDA’s National Research Support Project #7 (NRSP-7) since 1995 Technical assistance to Congress for the Minor Use & Minor Species Animal Health Act of 2004 Director, OMUMS (the Queen MUM) since 2008

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A little background

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The Goal

Safe and Effective New Animal Drugs approved for, and/or available for, legal use in U.S. Aquaculture

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The Main Problem

Drug approval is expensive and the aquaculture market is small

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What do we need?

More drug approvals thru: MUMS incentives Research partnerships

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Why is approval best?

Studies to show a drug is safe & effective for its intended use Good information on dosing Good information on withdrawal times Extra-label use not permitted for medicated feeds Approved drugs are legal

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Incentives

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Incentives for sponsors

User fee waivers DesignationGrantsExclusive Marketing Rights Conditional Approval Guidance and assistance Research partners & Public Master Files

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Who has the lead in CVM?

OMUMS Designation Grants Indexing Liaison to NRSP-7 Stakeholder outreach

ONADE User fee waivers INAD review/PMFs Conditional Approval NADA Project managers Stakeholder outreach

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OMUMS Programs

Designation

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How does “Designation” help?

“Designation” is the MUMS equivalent of “Orphan Drug” status for human drugs Provides 7 years exclusive marketing rights starting when the intended use of the product is approved or conditionally approved Provides opportunity to apply for grants to support safety and effectiveness studies

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Designation…

The responsibilities of the sponsor:ApplySubmit annual reports to show due diligenceKeep current with modifications, if neededTerminate, if project abandonedNOTE: If grants are desired, apply for designation early in the process. Just after the “product development” presubmission conference.

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So, How’s that MUMS Grant Program working out?

118 designations granted 84 are for aquacultureStarted awarding grants in 2009 24 grants awarded to date 22 for aquaculture usesRemember – grants are competitive

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For a researcher to get a grant

The sponsor needs to get the designation The study needs to be intended to support approval for the designated use Must have protocol acceptance from ONADE Either the sponsor or a research partner can apply for a grant (whoever is doing the study) Research partnership needs to be official – copy of a letter to the INAD(s)

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Whoever is applying must

Register online with grants.gov, sam.gov, and eRA Commons Allow up to 4 weeks to process Check CVM website for open periods (Updates) – usually twice/year Apply for grant(s) at grants.gov to support safety or effectiveness studies

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How much?

Amounts vary based on the study Grants are available at 2 levels – up to $75K or up to $125K Usually $750K available per year

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Changes to improve the program – see announcements

Limiting indirect costs to 15% No longer using NIH 2nd level review Extended open periods for applications Stand-alone method validation studies now eligible (need protocol acceptance) Will expand eligibility to include some manufacturing studies (w/ protocol)

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Who you gonna call?

Dr. Stuart Jeffrey is in charge of Designation and the MUMS Grant Program You can contact her at: stuart.jeffrey@fda.hhs.gov (240) 276-8604

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OMUMS Programs

Indexing

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Indexing

The Index of Legally-marketed Unapproved New Animal Drugs for Minor Species (The Index)Faster and easier than approvalFor non food minor species onlyIdeal for ornamental fishAlso for some early non-food life stages of food producing species (oyster spat, not lambs)Not always clear – baitfish, fry, eggs …Out of 4 Indexed products, 2 are for ornamental fish

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Who you gonna call?

Dr. Dorothy Bailey is in charge of Indexing You can contact her at: dorothy.bailey@fda.hhs.gov (240) 276-9337

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OMUMS Programs

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FDA Liaison to NRSP-7

National Research SupportProgram #7 (NRSP-7) of USDA

The regulatory affairs role for the programWork with the NRSP-7 technical committeeEstablish INADsSet up meetingsSubmit protocols & studiesDr. Amy Omer is taking over this role, contact her at:amy.omer@fda.hhs.gov(240) 276-9331

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Stakeholder outreach

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Stakeholder outreach

OMUMS cooperates with other CVM Offices Work with researchers Assist new sponsors Manage our programs Act for NRSP-7 Give presentations Answer questions from the public Write Guidance for Industry (GFI)

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What about that GFI #61?

It is coming along  Identity crisis What it isn’t: Not a panacea – or even a very good cookbook What it is: One stop shopping for MUMS information along with pointers to specifics Especially for aquaculture, there is a lot of case-by-case information

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Take-home message

THINK OUTSIDE THE BOX ONADE is making a conscious attempt to accept alternate ways to meet approval requirements What has been done before is only one way to do it…

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Who are the partners?

Pharmaceutical sponsors Researchers private academic Part FDA/CVM

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Partnerships for drug approval

Multiple entities complete the NADA technical sections – Communication is keyMultiple INADs are possible for a single projectThe pharmaceutical sponsor’s files are proprietaryThe public entities usually use the Public Master File (PMF) modelProject managers in ONADE can help

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New Public Master File Process

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1

st

major public Technical Section Complete

CVM creates PMF – posted on the web

TSCs from

1 or more INADs added & credited

Sponsor can use data by reference for NADA

Pharmaceutical sponsors

Sponsors can work with universities and public labs to find products to develop (R&D) Need to establish partnerships with researchers to do studies to support approval of these products Designate the product for a MUMS intended use Complete sponsor Technical Sections of the NADA (manufacturing, labeling, All Other Info, Freedom of Information Summary…) File the NADA and then market the product after approval or conditional approval

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Federal & State Programs

Many entities are involved in this work All are under funding pressure Stakeholders need to support these programs

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Examples

USFWS AADAP has a long list of INADs – see http://www.fws.gov/fisheries/aadap/home.htm Upper Midwest Environmental Science Center is involved with several INADs – seehttp://www.umesc.usgs.gov/aquatic_toxicology.html NRSP-7 currently has active projects for Erythromycin & Strontium chloride – seehttp://www.nrsp7.org Others - USDA Agricultural Research Service, etc.

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What can/should we do?

Look at the investigational uses that have been around a long time What still needs to be done? Prioritize them and get them approved! Use MUMS incentives Look for new needs Maximize partnerships Support funding

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Addresses

MUMS website: http://www.fda.gov/AnimalVeterinary/DevelopmentApprovalProcess/MinorUseMinorSpecies/default.htmDesignation & Grants: http://www.fda.gov/AnimalVeterinary/DevelopmentApprovalProcess/MinorUseMinorSpecies/ucm064838.htm Indexing: http://www.fda.gov/AnimalVeterinary/DevelopmentApprovalProcess/MinorUseMinorSpecies/ucm070206.htm PMFs: http://www.fda.gov/AnimalVeterinary/DevelopmentApprovalProcess/MinorUseMinorSpecies/ucm279384.htm

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Thank you!

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Summary Slide

The FDA animal drug approval process is very important to ensure legal availability of safe and effective drugs. FDA/CVM is offering flexibility and incentives to encourage sponsors to complete projects for minor species like fish. Public/private partnerships are essential to complete these projects. Markets are too small and specialized for sponsors to go it alone. Public programs are under severe budget constraints and need to have their funding needs supported.

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