Meg Oeller, DVM FDA Center for Veterinary Medicine 1

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. 2

Need-to-know acronyms MUMS – Minor Use & Minor Species (all fish are minor species) OMUMS – Office of Minor Use & Minor Species Animal Drug Development ONADE - Office of New Animal Drug Evaluation INAD – Investigational New Animal Drug PMF – Public Master File NADA – New Animal Drug Application 3

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

A little background 5

The Goal Safe and Effective New Animal Drugs approved for, and/or available for, legal use in U.S. Aquaculture 6

The Main Problem Drug approval is expensive and the aquaculture market is small 7

What do we need? More drug approvals thru: MUMS incentives Research partnerships 8

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 9

Incentives 10

Incentives for sponsors User fee waivers Designation Grants Exclusive Marketing Rights Conditional Approval Guidance and assistance Research partners & Public Master Files 11

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 12

OMUMS Programs Designation 13

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 14

Designation… The responsibilities of the sponsor: Apply Submit annual reports to show due diligence Keep current with modifications, if needed Terminate, if project abandoned NOTE: If grants are desired, apply for designation early in the process. Just after the “product development” presubmission conference. 15

So, How’s that MUMS Grant Program working out? 118 designations granted 84 are for aquaculture Started awarding grants in grants awarded to date 22 for aquaculture uses Remember – grants are competitive 16

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) 17

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 18

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 19

Changes to improve the program – see announcements Limiting indirect costs to 15% No longer using NIH 2 nd 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) 20

Who you gonna call? Dr. Stuart Jeffrey is in charge of Designation and the MUMS Grant Program You can contact her at: (240)

OMUMS Programs Indexing 22

Indexing The Index of Legally-marketed Unapproved New Animal Drugs for Minor Species (The Index) Faster and easier than approval For non food minor species only Ideal for ornamental fish Also 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 23

Who you gonna call? Dr. Dorothy Bailey is in charge of Indexing You can contact her at: (240)

OMUMS Programs 25 FDA Liaison to NRSP-7

National Research Support Program #7 (NRSP-7) of USDA The regulatory affairs role for the program Work with the NRSP-7 technical committee Establish INADs Set up meetings Submit protocols & studies Dr. Amy Omer is taking over this role, contact her at: (240)

Stakeholder outreach 27

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) 28

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 29

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… 30

Who are the partners? Pharmaceutical sponsors Researchers private academic Part FDA/CVM 31

Partnerships for drug approval Multiple entities complete the NADA technical sections – Communication is key Multiple INADs are possible for a single project The pharmaceutical sponsor’s files are proprietary The public entities usually use the Public Master File (PMF) model Project managers in ONADE can help 32

New Public Master File Process 33 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 34

Federal & State Programs Many entities are involved in this work All are under funding pressure Stakeholders need to support these programs 35

Examples USFWS AADAP has a long list of INADs – see Upper Midwest Environmental Science Center is involved with several INADs – see NRSP-7 currently has active projects for Erythromycin & Strontium chloride – see Others - USDA Agricultural Research Service, etc. 36

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 37

Addresses MUMS website: rovalProcess/MinorUseMinorSpecies/default.htm rovalProcess/MinorUseMinorSpecies/default.htm Designation & Grants: rovalProcess/MinorUseMinorSpecies/ucm htm rovalProcess/MinorUseMinorSpecies/ucm htm Indexing: rovalProcess/MinorUseMinorSpecies/ucm htm rovalProcess/MinorUseMinorSpecies/ucm htm PMFs: rovalProcess/MinorUseMinorSpecies/ucm htm rovalProcess/MinorUseMinorSpecies/ucm htm 38

Thank you! 39

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. 40