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Obtain and Manage Grants and Funding for Orphan Drug Development

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Title: Obtain and Manage Grants and Funding for Orphan Drug Development


1
Obtain and Manage Grants and Funding for Orphan
Drug Development
Debra Lewis, OD, MBA Associate Director FDA
Office of Orphan Products Development FDA
Seminar 2009 - Copenhagen November 3-4, 2009
2
Overview
  • Orphan Background
  • Grants Programs (Orphan and Pediatric Device
    Consortia Grants)
  • FDA OOPD Grant Program Application and Review
    Additional Resources
  • Warning No Fear these very dense slides are for
    reference purposes only

3
Objectives
  • FDAs Office of Orphan Products Development
    (OOPD) focusing on Grants Processes
  • Understand regulatory requirements for grants
    before applying, including grants.gov electronic
    application system
  • Ensure a successful program and continued funding
    through study conduct and grants management

4
A Common Perspective of Grant Review
5
The Mission of The Office of Orphan Products
Development
To assist and encourage the identification,
development, and availability of safe and
effective products for people with rare
diseases/disorders.
6
How does OOPD serve its Mission?
  • Conducts review of orphan product and
    humanitarian use device designation requests
  • Serves as liaison for medical product companies,
    FDA review divisions, patient advocacy groups,
    and other government agencies.
  • Awards and administers grants to defray orphan
    product clinical study costs and to advance
    Pediatric Device Development

7
Orphan Diseases
  • Rare defined as prevalence of lt200,000 in the
    U.S.
  • Includes over 6,000 rare diseases
  • Collectively affects approximately 25 million
    Americans
  • Frequently serious/life threatening

8
Orphan Products Grants Programs
9
Grants Programs in OOPD
  • Pediatric Device Consortia Grant Program (New as
    of FY 2009)
  • OPD Clinical Research Grants (R01) for Orphan
    Diseases

10
The Pediatric Device Consortia Grant Program
  • .charting a course for the development of
    medical devices for children

11
Pediatric Device Consortia Grant Program
  • Established from FDAAA 2007
  • Does not have to be Orphan Administered by
    OOPD, but encompasses devices used in all
    pediatric diseases, not just rare diseases.
  • Receipt date this year was June 15, 2009 Paper
    submission
  • See RFA at www.fda.gov/orphan

12
The Challenges
  • The development of pediatric devices currently
    lags five to ten years behind those for adults.
  • Many pediatricians are forced to adapt adult
    devices to suit their pediatric patients
  • Children differ from adults in terms of their
    size, growth, development, and body chemistry.

13
The Law
  • The Pediatric Medical Device Safety and
    Improvement Act of 2007
  • Calls for Non-Profit Consortia Grants to
    Stimulate Device Development

14
2009 Omnibus Appropriations Bill
  • Provided 2 million for demonstration grants for
    improving pediatric device availability, as
    authorized in FDAAA of 2007.
  • The program will provide grants to nonprofit
    pediatric medical device consortia, which will
    assist scientists and innovators with technical
    and financial resources to improve the number of
    devices available to children.
  • The Office of Orphan Products Development is
    responsible for carrying out this program.

15
Pediatric Medical Device Safety and Improvement
Act of 2007
  • Establishes Non-Profit Consortia to Stimulate
    Pediatric Device Development
  • What is a consortium?

www
www.images.inmagine.com
16
A nonprofit consortium that receives a grant or
contract under this section shall facilitate the
development, production, and distribution of
medical devices by--
  • Encouraging innovation and connecting qualified
    individuals with pediatric device ideas with
    potential manufacturers
  • Mentoring and managing pediatric device projects
    through the development process, including
    product identification, prototype design, device
    development, and marketing.
  • Connecting innovators and physicians to existing
    Federal Sources and non-Federal Resources.

17
A nonprofit consortium that receives a grant or
contract under this section shall facilitate the
development, production, and distribution of
medical devices by--
  • Assessing the scientific and medical merit
  • Providing assistance as needed on business
    development, personnel training, prototype
    development, post-market needs and other
    activities consistent with the purpose of this
    section

18
H.R. 3580, Title 3, Section 305
  • Demonstration Grants for Improving Pediatric
    Device Availability
  • Typically when we at FDA think of grants, we
    think of grants to directly fund research.
  • This is a grant program that will fund consortia
    that in turn support pediatric device developers
  • A RESULTS Driven Provision, aimed at moving
    products forward along the development continuum.

19
H.R. 3580, Title 3, Section 305
  • Coordination,
  • NIH
  • Each consortium . shall coordinate with the
    NIHs pediatric device contact point or office
    (Dr. Steven Hirschfeld)
  • Provide to NIH
  • any identified pediatric device needs that the
    consortium lacks sufficient capacity to address
    or
  • those needs the in which the consortium has been
    unable to stimulate manufacturer interest.

20
H.R. 3580, Title 3, Section 305
  • Coordination, continued.
  • FDA
  • Each consortium .shall coordinate with the
    Commissioner of Food and Drugs and device
    companies to facilitate the application for
    approval or clearance of devices labeled for
    pediatric use.

21
H.R. 3580, Title 3, Section 305
  • Coordination, continued.
  • Effectiveness and Outcomes
  • Each consortium. shall annually report to the
    Secretary of Health and Human Services on the
    status of pediatric device development,
    production, and distribution that has been
    facilitated by the consortium

22
H.R. 3580, Title 3, Section 305
  • There are authorized to be appropriated to carry
    out this section 6M for each of fiscal years
    2008 through 2012.
  • 2008 - no appropriation no program
  • 2009 2 M appropriation March

23
PDCGP Timeline
  • March 2009--
  • Draft Request For Applications Federal Register
    Notice
  • Schedule initial meetings with key stakeholders
    both within and outside of FDA
  • April 2009--
  • Clear RFA and FR Notice through the agency and
    with NIH Guide get them circulated publicly by
    May 1st.
  • June 15, 2009
  • Receipt of Applications Deadline
  • July 2009
  • Hold Review Panel of Proposed Grants
  • September 2009
  • Council Review by National Cancer Advisory Board
  • Distribute Funds to Consortia

24
The Pediatric Device Consortia Grants Program
  • 2 Million Dollars were distributed between 3
    Consortia
  • Using the P50, Center Grant Mechanism
  • Grant proposals were critiqued on the Consortias
    Organization/ Capability AND Portfolio of
    Proposed Projects

25
WHO?
  • James Geiger, MD and the Michigan Pediatric
    Device Consortium, 1,000,000.
  • Michael Harrison, MD and the UCSF Pediatric
    Device Consortium, 500,000.
  • Pedro del Nido, MD and the Pediatric
    Cardiovascular Device Consortium, 500,000.

26
Points of Contact
  • Linda C. Ulrich, M.D., F.A.A.P
  • Pediatric Consortia Grants Program
  • (301) 827-7252 Linda.Ulrich_at_fda.hhs.gov
  • Debra Y. Lewis, O.D., M.B.A, Associate Director,
    OOPD and Director of the HUD Program 301 827-0059
  • OOPD Main line (301) 827-3666

27
Points of Contact
  • Grants Management
  • Camille Peake,
  • Grants Management Specialist
  • (301) 827-7175
  • Camille.Peake_at_fda.hhs.gov

28
QUESTIONS?
www.elderoptionsof texas.com
29
Rare Disease Grant Opportunities
  • FDA OPD Grant Program (R01)
  • Other Federally funded grants
  • Grants.gov- search under find grant
    opportunities
  • Small business funding opportunities
  • Small Business Innovation Research (SBIR) program
  • Small Business Technology Transfer (STTR) Program
  • NIH grants http//grants.nih.gov/grants/guide/in
    dex.html
  • NORDs Research Grant Program
  • Small grants to academic scientists studying new
    treatments or diagnostics for rare diseases.
    (www.rarediseases.org)

30
Orphan Products Grants Program
  • Goal to advance clinical development of drugs,
    biologics, medical devices, or medical foods, for
    use in rare diseases.
  • Academic and industry sponsored research.
  • Domestic or foreign, public or private,
    for-profit or nonprofit entities
  • The disease must affect lt200K persons in the
    U.S.), but orphan designation is not a grant
    requirement

31
Orphan Products Grants Program
  • A practical program advancing marketing
    approvals and relevant publications that impact
    care
  • lt 100 applications/year
  • Competitive grant program 30 success
  • Fund about 15-20 new grants per year
  • Request for Application (RFA) available at
    www.fda.gov/orphan
  • Application, review, and scoring much like NIH
    grant application
  • Electronic submissions Grants.gov

32
FDA OPD Grant Program Overview
  • Eligibility/Requirements
  • Any entity except DHHS Federal agencies
  • Clinical study of an orphan disease or condition
  • A study must advance info towards a market
    approval
  • Must have active IND (not on clinical hold)
  • Evidence that drug product is sufficiently
    available
  • Funding dependent on quality of application and
    availability of Federal funds
  • For more information visit the OPD Grant Program
    website http//www.fda.gov/ForIndustry/Developing
    ProductsforRareDiseasesConditions/WhomtoContactabo
    utOrphanProductDevelopment/ucm134580.htm

33
OPD Grants Program Budget
  • The current annual budget for grant funding is
    approximately 14 million.
  • Clinical trials cycle award limits
  • 200K/yr (Phase 1) for up to 3years
  • or
  • 400K/yr (Phase 2 and 3) up to 4 years.

34
Annual Number of OPD Grant Applications
35
Overview Timeline FY 2011 Grant Program
  • Next receipt date - February 3, 2010 (October
    15, 2010 will be a receipt date only for
    resubmissions)
  • IND/IDE needs to be in effect at time of the
    grant application submission and must include the
    protocol for which funding is requested)
  • Protocol should be submitted to IND by January 4,
    2010 (earlier if submitted with original IND)
  • Application summary statement - August 2010
  • Earliest start date for award - November 2010
  • All FY 2011 funding completed by September 2011

36
Types of Products Supported by Active OPD Grants
Excluding no-cost extension and supplemental
grants
37
Composition of Orphan Grants
  • 25 have a Phase 1 component
  • 55 have a Phase 2 component
  • 20 have a Phase 3 component
  • About 25 of awards are to companies
  • About 50 are company/academic institution
    collaboration

38
Grants Statistics
  • To date, since 1983, FDA has provided more than
    246 million for more than 500 grants for studies
    on rare diseases.
  • Current annual budget 14 million
  • 44 FDA approved products were at least partially
    funded through the OOPD Grants Program.

39
OPD Grants Program KEY Requirements
  • Clinical studies to be conducted under
  • ACTIVE IND or IDE
  • Good Clinical Practices (GCP)
  • Human Subjects Assurance from OHRP Federal-Wide
    Assurance or FWA (www.hhs.gov/ohrp)
  • IRB approval

40
Grant Application Process
  • Grants.gov
  • Submit electronically through www.grants.gov
  • Follow instructions under Apply for Grants
  • Search using RFA information (FD09-001)
  • Download copy of application package (SF-424RR)
    and instructions
  • Applicant Help section provides Users Guide,
    FAQs and other support
  • Complete offline
  • Upload and submit via grants.gov web site
  • Track status of application via grants.gov
  • FOA link in NIH Guide at http//grants.nih.gov/gr
    ants/guide/rfa-files/RFA-FD-09-001.html

41
Application Process
  • Grants.gov
  • Documents completed in Adobe are added as
    attachments
  • Text attachments must be PDF files
  • Format specifications for attachments
  • Registration
  • Request a DUNS number
  • Register with CCR (Central Contractor
    Registration)
  • Register with Credential Provider
  • Register with Grants.gov
  • Register eRA

42
Specifics on Application Process
  • See website for requirements such as page limits,
    appendices, character restrictions, and face page
    requirements
  • http//www.fda.gov/ForIndustry/DevelopingProducts
    forRareDiseasesConditions/ucm124552.htm

43
Sections of Application
  • Key Application Sections
  • SF 424 RR Form
  • Applicant info, Type of application, Title,
    Project Period, Contact
  • Senior / Key Person Profiles
  • Biographical sketches limited to 4 pages per
    person- use CV to complete
  • Other Project Information
  • Human Subjects, IRB, Project Summary/Abstract,
    Narrative
  • Project/Performance Site Locations
  • Budget
  • OPD requires non-modular budget

44
Sections of Application
  • PHS 398 Cover Page Supplement
  • PI info, New Investigator, Organization Contact
  • PHS 398 Checklist
  • Application Type, Program Income if applicable,
    Change Investigator if applicable
  • PHS 398 Research Plan
  • Use study protocol and IND documents to create
  • Proprietary Information
  • Only include what is necessary for application
    evaluation
  • Mark confidential information with ()

45
Sections of Application
  • PHS 398 Research Plan
  • Sections
  • Introduction
  • Specific Aims
  • Background and Significance
  • Rationale for study contributing to product
    approval
  • Orphan prevalence data
  • Preliminary Studies / Progress Report (if
    applicable)
  • Research Design and Methods
  • Inclusion Enrollment report
  • Progress Report Publication List

46
Sections of Application
  • PHS 398 Research Plan - Sections Contd
  • Human Subjects Sections
  • Protection of Human Subjects (e.g., DSMB)
  • IRB approval required following peer review
  • Inclusion of Women, Minorities, Children
  • Targeted/Planned Enrollment
  • Other Research Plan Sections
  • Consortium/Contractual Arrangements
  • Letters of Support
  • Contracts with CRO, consultants, service
    providers (e.g., labs)
  • Appendix
  • Protocol, informed consent, DSMB charter,
    Investigators Brochure, FDA meeting minutes,
    product availability

47
Grantee Hints on the Process
  • FDA grant submission process for grants.gov is
    similar to the NIH process. 
  • 1. Do your homework.
  • 2.  Develop a submission plan.
  • Who will be responsible for completing the
    different forms within the application?
  • When must the forms be completed in order to
    allow time for the actual submission?
  • 3.  Prepare to submit.
  • Register with Grants.gov and NIH eRA Commons
  • Registration can take 2-4 weeks start early!

48
Grantee Hints on Process
  • 4.  Verify successful submission.
  • Login to eRA Commons to see if any errors or
    warning were identified for your submission. An
    application must be error-free to complete the
    electronic submission process.
  • 5.  See Check Submission Status for details.
  • You are responsible for checking eRA Commons to
    ensure successful submission of your
    application. 
  • 6.  View your assembled application.
  • Once an error-free application is received, FDA
    holds that application for two days
    (Monday-Friday, excluding Federal holidays) to
    allow submitters and PIs a chance to view the
    final assembled application in eRA Commons.

49
OPD Grant Program Review
  • Primary Review Grants scored by independent ad
    hoc expert panels for technical merit
  • Criteria are in the RFA
  • Funding based on scores (100-500 lower scores
    are better)
  • The recent fundable range was 100-190
  • Summary Statements contain review specifics
  • Second Level review by a National Council
    (process approval)
  • OPD Project Officer checks prefunding
    certifications prior to funding (check IRB,
    foreign sites, etc.)

50
OPD Grant Program Review
  • Ad hoc expert panel reviews application based
    on the following scientific and technical merit
    criteria
  • Soundness of study rationale and design
  • Appropriateness of statistical powering and plans
    for results analysis
  • Evidence that the proposed number of subjects can
    be recruited in the requested timeframe
  • Qualifications of the investigator and support
    staff and availability of resources
  • Justification for financial support request
  • Adequacy of plans for protection of human
    subjects and study monitoring
  • Ability of applicant to complete study within its
    budget and within the time limits of the grant

51
OPD Grant Program Review
  • Numbers Overview
  • lt 100 applications per year
  • Ave. 30 of new applications awarded per year
  • 82 active grants about 15-20 new grants each
    year
  • Future years of noncompetitive continuation of
    support depends on
  • Performance during the preceding year
  • Compliance with regulatory requirements of IND
  • Availability of Federal funds

52
Approved Products Supported by Orphan Grants
  • 44 products funded by OPD grants approved for
    marketing
  • Examples
  • Elaprase (idursulfase) Enzyme replacement
    therapy for patients with MPS II (Hunter
    Syndrome).
  • BabyBIG (botulism immune globulin) infant
    botulism
  • ORFADIN (nitisinone) tyrosinemia type 1
  • VEPTR (vertical expandable prosthetic titanium
    rib) thoracic insufficiency syndrome

53
10 FDA Hints Useful Homework
  • Start early, plan carefully, write clearly, and
    objectively
  • Establish good relations w/FDA review divisions
    via IND/IDE process. OPD invites FDA review
    divisions to the review as a resource (FDA does
    not score the application).
  • Read the RFA and instructions carefully not just
    for deadlines
  • Use the Grant Writing Tips from NIH Extramural
    Programs http//grants.nih.gov/grants/grant_tips.
    htm
  • Call OPD for program clarifications
    301-827-3666

54
10 FDA Hints Useful Homework
  • Call Grants Management for budget help contact
    Vieda Hubbard (301-827-7177) and electronic
    submissions help contact Marc Pitts
    (301-827-7162)
  • If you do not have expertise for issues, provide
    letters of collaboration for the needed expertise
  • Panel Reviewers are busy, so say it in fewer
    words if possible
  • Use outside readers improve the quality of the
    proposal
  • Dont be discouraged read summary statements
    and address all critiques

55
Responsibilities after Review
  • Complete OPD Pre-Certification Form sent by OPD
    to those applications with scores in fundable
    range
  • Send response to Summary Statement Critiques
  • Submit IRB approval letter
  • Submit FWA information
  • IND must be active (not on hold) and protocol
    must match protocol going to be used for funded
    study
  • Verify an adequate supply of study product is
    available

56
Responsibilities after Funding
  • Set enrollment goals
  • Clinicaltrials.gov registration and maintenance
  • IND maintenance
  • Must comply with all regulations to keep IND in
    effect (not on clinical hold), Annual Report
    submissions
  • Keep OPD Project officer aware of key IND
    submissions (e.g., IND Annual Report, Protocol
    Amendments)
  • Project officer periodic monitoring including
    Phone calls, correspondences and Site visit from
    OPD
  • Comply with GCP

57
Responsibilities after Funding
  • Quarterly progress reports
  • Purpose is to ensure there is acceptable progress
    in enrollment and study conduct is kept within
    the scope of the grant
  • Summary of study progress, enrollment, problems
    or challenges, adverse events, changes in
    protocol, study monitoring activities that ensure
    integrity of the data collected in study, and how
    you are ensuring compliance with the protocol.
  • Publication of study results encouraged
  • Submit IRB approval renewal yearly to OPD
  • Submit Type V Applications for following years
    with Progress report

58
Summary
  • FDA OPD Grant Program
  • Practical program to facilitate marketing
    approval of products for rare diseases
  • Up to 400,000 x 4 years
  • Competitive program (30 success)
  • Key recommendations include
  • Plan resources and timing
  • Regulatory compliance and adequate study progress
    critical to continued grant funding

59
Useful Links
  • OPD Website
  • www.fda.gov/orphan or (301) 827-3666
  • FOA
  • http//edocket.access.gpo.gov/2008/E8-30061.h
    tm or at NIH guide
  • SF424 (RR) Application Guide
  • http//grants.nih.gov/grants/funding/424/SF424_RR
    _Guide_General_Ver2.doc

60
A Better Perspective of FDA Grants
61
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