FDA – IDSA - ISAP Workshop April 15, 2004

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					FDA – IDSA - ISAP Workshop
               April 15, 2004

     Continuing Discussion on Incentives
              George H. Talbot MD

             On Behalf of the AATF

 13 Apr 04                                 1
            Presentation Objectives

   Update workshop attendees on AATF’s
    efforts to clarify factors responsible for
    decreased antibacterial R&D

   Discuss the full range of possible solutions…
    not just “financial” incentives

13 Apr 04                                        2
       What has AATF Learned?
   There is a problem
     – Complex, multifactorial etiology
     – Susceptible to oversimplification: “If only….”
 No easy, single solution
 Potential approaches are apparent
 Progress requires
     – Long-term commitment
     – Active collaboration of essential partners

13 Apr 04                                               3
             The Problem
“Drug options for treatment of infections are
becoming increasingly limited, largely as a
result of growing antimicrobial resistance.
Many generic but essential antibiotics are in
short supply, and the development of new
antibiotics has been severely curtailed….
Only 4 large pharmaceutical companies with
antibiotic research programs remained in
existence in 2002….”
      (Institute of Medicine Report, 2003)
13 Apr 04                                       4
                            The Problem
               Total Approved Antibacterials: US
                                                 Total # New
       6                                         Antibacterial Agents
            1983- 1988- 1993- 1998- 2003
            1987 1992 1997 2002

            Spellberg et al, CID 2004 in press
13 Apr 04                                                           5
       What has AATF Learned?
            Interactions with Stakeholders

 Pharmaceutical companies
 VCs
 The scientific and lay press
 Congress

13 Apr 04                                    6
      What Has AATF Learned?
                      Issues for Pharma
   Many individuals and groups within pharma
    are deeply concerned about, and committed
    to, the future of anti-bacterial R&D

   “Big” pharma is becoming disengaged
     – There are notable exceptions
     – Greatest concern -- the dearth of resources
            being applied at the Discovery level
13 Apr 04                                            7
      What Has AATF Learned?
              Issues for “Big” Pharma
 “Big” Pharma sees better return from the
  treatment of chronic diseases.
 In contrast, antibacterial therapies are:
     – Costly to develop
     – Short course, used for acute illnesses
     – Not embraced by the marketplace (cost,
       resistance, “satisfied” market)
     – Rarely “blockbusters”

13 Apr 04                                       8
      What Has AATF Learned?
            Issues for “Big” Pharma
 Any further uncertainty is a disincentive
 Further IP protections - ? upside
 Tax credits – interesting
 Because of enormous hurdles for Discovery
  infrastructure, we must keep those
  companies that are “In” – “In”

13 Apr 04                                     9
      What Has AATF Learned?
               Issues for “Small” Pharma
   “Small” Pharma is more engaged
     – Financial return better matched to size
     – Market opportunity is more clear
     – Regulatory uncertainty: a lesser concern?
   Focus
     – For some, in-licensed compounds only
     – Others, robust Discovery efforts
            Will it be enough?
13 Apr 04                                          10
      What Has AATF Learned?
                   Issues for VCs
 Access to venture capital is – of course -
  essential for new companies
 In a totally unscientific sampling:
     – Some VCs see the dearth of discovery efforts as
       an opportunity
     – Others consider the risks high because of
       restrictions on use of marketed products
     – Late-stage anti-infective products are drivers of
       some financing decisions
13 Apr 04                                              11
       What has AATF Learned?
                       Issues for FDA
   FDA understands the problem
     – Wishes to partner in finding solutions
     – Regulatory uncertainty, when present, further
            clouds the development process
             (FDA’s 2004 “Critical Path” report)
 Maintaining scientific rigor
 Limited flexibility per statutory constraints
     – e.g., waiver of user fees not possible

13 Apr 04                                              12
       What has AATF Learned?
            Issues for CDC, NIAID, HHS
   Substantial, relevant efforts proposed
     – PHS Action Plan to Combat Resistance (2001)
     – NIH Roadmap for Medical Research (2003)

   More funding needed for critical efforts

   More could be done to foster inter-agency
    collaboration, training, and outreach
    regarding antibacterial drug development
13 Apr 04                                            13
       What has AATF Learned?
     Issues for the Scientific and Lay Press
   Pipeline concerns -- of interest to both the
    scientific community and the public

   Articles directed to a wide readership

   Our communications must highlight not
    only measures to decrease resistance, but
    also those to ensure the pipeline

13 Apr 04                                          14
            “Bad Bugs, No Drugs”
            in the Scientific and Lay Press
Lay Press
   Boston Globe, "Firms Abandoning Antibiotics Research;
    Drugs That Make More Money Sought"
        Christopher Rowland, Mar 13, 2004

   USA Today, "Scientists: Research Cuts are Troubling"
       Anita Manning, Sept 15, 2003

   Washington Post, "Tougher Bugs, Few New Drugs"
       Roxanne Nelson, Mar 30, 2004

13 Apr 04                                                   15
            “Bad Bugs, No Drugs”
            in the Scientific and Lay Press
General Scientific Readership
   Nature, "Drug Companies Snub Antibiotics"
        Tom Clarke, Sept 18, 2003

   Science, "Orphan Drugs of the Future?"
        Robert Service, March 19, 2004, pg 1798

   The Lancet, "Antibiotic Development Pipeline Runs Dry"
        Roxanne Nelson, Nov 22, 2003

13 Apr 04                                                    16
            “Bad Bugs, No Drugs”
            in the Scientific and Lay Press
ID Specialty Journals
   The Lancet Infectious Diseases, "Covering the Parts Other
    Bioshields Don't Reach"
                editorial, Jan 2004

   The Lancet Infectious Diseases, "Incentives to Lure Drug
    Companies to Antimicrobials"
               Marilynn Larkin, Jan 2004

   Clinical Infectious Diseases, “Trends in Antimicrobial
    Drug Development: Implications for the Future"
                 Spellberg et al, in press 2004
13 Apr 04                                                      17
       What has AATF Learned?
               Issues for Congress
 Some vocal & effective supporters for
  addressing the issues, but
 Focus of policy-makers is elsewhere
     – Bioterrorism
     – Everything else…
 BioShield I offers some hope of solutions,
  but has substantial constraints
 More attention & action are needed

13 Apr 04                                      18
            What is IDSA’s Role in
             Defining Solutions?
   Raise awareness of the the problem
     – Multiple venues
     – Always speak to the needs of patients
   Brainstorm on possible solutions
     – Many are not within IDSA’s area of expertise
     – Partners needed
     – IDSA can act as a catalyst

13 Apr 04                                             19
            Raising Awareness
 Visit with Dr. McClellan, senior FDA staff
 Visits with senior pharma executives
 Interactions with CDC, NIAID
 Legislative contacts
 Outreach to scientific and lay press
 “White Paper”, to be released in May

13 Apr 04                                      20
            Potential Solutions
            Partnering of Stakeholders
   IDSA, FDA, CDC, NIAID, Pharma, &
    Congress in the interest of public health

   IDSA hopes that it can continue to play an
    important, constructive role in partnering

13 Apr 04                                        21
             Potential Solutions
            Changes in the Marketplace
 Greater marketplace receptivity to new
  antibacterials could alter economic equation
 But, potential changes are constrained
     – Cost, concern re promotion of resistance
     – Desire to hold antibiotics of last resort

         Change unlikely, without scientific data
    to justify different usage patterns
13 Apr 04                                          22
            Potential Solutions
         Regulatory adjustments
 Publish updated guidelines (on-going)
      – Periodic, timely review and revision
 Encourage novel clinical trial designs to
  gather information on drug efficacy against
  resistant pathogens
 Define surrogate endpoints, PK/PD
  parameters, & preclinical data that could
  reduce # of clinical studies
13 Apr 04                                      23
            Potential Solutions
                Initiatives by NIAID
 Responsible for implementing “Roadmap”
  for translational research
 Foster antimicrobial R&D
     – Collaborative planning with industry, academia
     – Fellowship curriculum in AI clinical trials
     – NIAID-FDA programs to streamline
     – Fund research into rapid diagnostics
     – Fund placebo-controlled trials in AECB, AS
13 Apr 04                                           24
             Potential Solutions
   Ongoing
     – The Project Bioshield Act
     – Best Pharmaceuticals for Children Act
     – Senators Gregg and Reed: GAO study

   Future
     – S.666 (Lieberman and Hatch)
     – Unique problems require unique solutions

13 Apr 04                                         25
             Potential Solutions
For investments in priority antibacterials
 Incentives successful elsewhere to spur R&D
    – e.g., R& D tax credits
   Supplemental IP protections
    –   e.g., wild card patent exclusivity
   Mechanisms to attract smaller companies
    – e.g., waiver of user fees for supplemental NDAs

13 Apr 04                                           26
             Potential Solutions
     Commission on Antimicrobial Resistance
       – Broad representation from stakeholders
       – Charges
           Identify priority pathogens

           Decide which antibiotics should receive the
            benefits of legislated incentives

13 Apr 04                                                 27
            Potential Solutions
Increase Funding for Essential Programs

 CDC antimicrobial resistance program
 NIAID antimicrobial resistance research
 FDA programs re review/approval of

13 Apr 04                                   28
                Potential Solutions
   Industry can point with pride to many pro
    bono initiatives for human health
     –      GSK: Malaria effort with the MVI
     –      Merck: Ivermectin effort with WHO
     –      AZ: TB discovery effort, Bangalore
     –      Many others
   If this impending crisis explodes, the public
    will need your help

13 Apr 04                                        29
            Potential Solutions
     These considerations to be discussed in
      detail in IDSA’s White Paper, to be
      released in May

13 Apr 04                                      30
 There is a problem
 Etiology is multifactorial
 No single, “easy” solution
 Essential partners are engaged
 Potential solutions apparent
 IDSA stands ready to make a long-term,
  constructive commitment to help address
  this brewing public health crisis

13 Apr 04                                   31

            Bad Bugs, No Drugs….
            How Can IDSA Help?

             We Want Your Input

13 Apr 04                          32
                       Final Notes
   Potential Conflicts
     – No external financial support for AATF effort
     – Some AATF members provide consultative
            services to industry
   Acknowledgements
     –      AATF members
     –      Factual input from J. Powers & D. Ross
     –      IDSA staff, esp. R. Guidos & D. Olson
     –      The many people with whom we have spoken
13 Apr 04                                              33