Commercial Challenges: Perspectives from the Biotechnology Industry

Transcription

1 Commercial Challenges: Perspectives from the Biotechnology Industry Eric A. Rose CEO and Chairman, SIGA Technologies; Guggenheim Professor and Chair Dept. of Health Evidence and Policy Mount Sinai School of Medicine

2 Objectives o Define biotech industry role in MCM o Offer biotech industry perspective on BARDA and PHEMCE o Suggest potential policy remedies

3 Biotechnology Industry Role in MCMs Developers and manufacturers of all new CBRN biological countermeasures procured into the SNS BARDA partners for most advanced development contracts Mostly small, non-profitable companies sustained by private capital and government grants and contracts An essential effector limb of the PHEMCE implementation plan

4 BARDA Perspective o Advanced development o Acquisition o Post-acquisition

5 BARDA Advanced Development An excellent, responsive development partner on a steep learning curve Funding for direct project costs reasonable, realistic, and flexible Funding for indirect costs is a fraction of actual costs FDA coordination unclear Development activities deserve substantially higher funding

6 BARDA Acquisition Process o Transition trigger from advanced development to acquisition RFP unclear o Contract completion target dates frequently unmet (H1N1 an exception) o Negotiation process is sequential with technical and security issues resolved prior to pricing o Option rationale and triggers unclear o Overall industry impression: lengthy, opaque, unpredictable

7 BARDA Post-Acquisition o Perceived improved communication compared to acquisition process o FDA coordination unclear o Option exercise behavior very encouraging but strategy it reflects remains unclear

8 PHEMCE Perspective o Function of the enterprise o Industry impact o Implementation plan implications

9 PHEMCE Function While Material Threat Determinations are public, MT and Population Threat Assessments remain secret DHS/HHS requirement setting process and determinations remain opaque until RFP issuance provides limited visibility Industry understandably not included, but enterprise itself lacks business and capital markets expertise

10 PHEMCE Industry Impact o Some highly desirable predictability o Possible unintended consequences Category B,C pathogens ignored Acquisitions for <$100,000,000 either ignored or severely impaired in ability to raise capital o Missed opportunities in engaging private sector, particularly re capital markets

11 PHEMCE Implementation Plan Implications o Shopping list a guide for industry focus

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14 Shopping List Limitations o Checkbox approach obscures product shortcomings and regulatory gaps o Tables don t conform to concept of operations for countermeasures

15 Proposed Policy Solutions o Principles o Goals o Specifics

16 Principles o Biology eats technology for lunch. o Countermeasure development is difficult and failure is far more frequent than success. o Policies don t make drugs and vaccines. o Companies and people do. o Companies are primarily incented by the opportunity to succeed (i.e. profit) in commercial markets.

17 Policy Goals o A safe and effective broad array of MCMs o Alignment of stakeholders, products, and product uses o Appropriate transparency o Speed

18 Use-based MCM Acquisition Matrix (Healthy Adults 18-64) Threat Pre-Event Prophylaxis Treatmen t Post-exp. Prophylaxis General Prophylaxis Anthrax Vaccine Antibiotics, MoAb Antibiotics +/- Vaccine Vaccine Smallpox Vaccine None Vaccine Vaccine Plague None Antibiotics Antibiotics None Tularemia None Antibiotics Antibiotics None Viral Hem. Fevers None None None None

19 Policy specifics o Grow BARDA development o Simplify product acquisition process and use commercial pricing for new biodefense products o Integrate regulatory and development processes by Creation of and FDA Center for Emergency Preparedness and Response (CEPR) Formalize the pre-eua process

20 FDA CEPR o Approval authority for small molecule or biologic products for the indications of therapy, post-exposure prophylaxis, or general population prophylaxis in the event of a public health emergency related to CBRN, pan flu, and emerging disease threats. o Adequately funded and staffed with budgets prepared through a collaborative mechanism with BARDA.

21 Formal Pre-EUA Approval Process o Existing EUA mechanism works well as an FDA emergency response process, not a preparedness mechanism o Use existing criteria for EUA with the exception of the actual declaration of an emergency. o Process would differ from current encouragement of submission of pre- EUA data but requires no response

22 Extras o Generation of animal data in two or more models for a product demonstrating a substantial reduction in mortality and/or a major morbidity for pathogens for which there are no approved therapeutic or prophylactic agents should trigger an expedited BARDA development plan, an estimate of size and scope of a potential procurement, and a priority FDA process to generate a Special Protocol Assessment to create a clear pathway to pre-eua approval and subsequent licensure.

23 Distribution: An Orphan MCM o If rapid distribution were a drug, it would receive >$100,000,000 advanced development award o Budget for distribution far less o Resilient, resourceful public is not going to wait for postal delivery of MCMs. o FedEx, UPS, Wal-Mart, drugstore chains, etc. all potential distribution partners