How Public-Private Partnerships Benefit All Stakeholders Douglas C. Throckmorton, MD Deputy Director for Regulatory Programs, CDER, FDA

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1 How Public-Private Partnerships Benefit All Stakeholders Douglas C. Throckmorton, MD Deputy Director for Regulatory Programs, CDER, FDA September 10, 2013

2 Outline Need for improved science in support of medical product development Tools FDA has to support medical product development Role of public private partnerships Overall message: Why partner? BECAUSE WE HAVE TO

3 R & D Process DRUG DISCOVERY PRE CLINICAL CLINICAL TRIALS FDA REVIEW LARGE SCALE MFG PRE-DISCOVERY 5,000-10,000 COMPOUNDS ONE FDA- APPROVED DRUG IND SUBMITTED TO FDA PHASE 1 PHASE 2 PHASE 3 Number of Volunteers YEARS 6-7 YEARS NDA SUBMITTED TO FDA YEARS PHASE 4: POST MARKETING SURVEILLANCE 3 SOURCE: PhRMA 2008, Stages of Drug Development Process and attrition rate of compounds as they travel through the drug development process over time.

4 R & D Spending $80 $70 Total NIH Budget PhRMA Member Companies' R&D Expenditures $63.2 $63.7 $65.3 $67.4 Total R&D Investment (in $Billions) $60 $50 $40 $30 $20 $10 $15.2 $11.3 Entire Pharma Sector $22.7 $21.0 $19.0 $16.9 $15.6 $13.7 $11.9 $12.7 $26.0 $17.8 $29.8 $20.5 $31.0 $23.3 $34.5 $27.1 $47.6 $37.0 $27.9 $51.8 $39.9 $28.5 $56.1 $43.4 $28.5 $47.9 $29.0 $47.4 $29.3 $45.8 $30.6 $49.4 $31.0 $ Year 4 Source: Burrill & Company, PhRMA, NIH Office of Budget

5 5 CDER New Molecular Entity and New Biologic Entity Filings and Approvals * Calendar Year NME/NBE Approvals NME/NBE Filings *CDER data as of 11/30/2012. *Since applications are received and filed throughout a calendar year, the filed applications in a given calendar year do not necessarily correspond to an approval in the same calendar year. *Certain filed submissions are within their 60-day filing review period and may not be filed upon completion of the review.

6 Drug Development in Renal Disease in Past 5 Years Renal Disease 2 Phosphate Binders (new formulations) Renal Transplantation: Zortress 2010 (everolimus) Nulojix 2011 (belatacept) Astagraf 2013 (extended release formulation of tacrolimus)

7 FDA s Role In The Science of Drug Development Develop infrastructure and tools for product development (not focus on development of specific products but rather areas of need) Encourage collaborative efforts among government, academia, industry, and patient groups Develop relevant data standards and regulations Build support for relevant academic science Create opportunities to share existing knowledge and databases 7

8 Critical Targets: Drug Development Tools (DDTs) Clinical Outcome Assessments DDT Qualification Biomarkers Animal Models

9 Goals for DDTs: Efficient Medical Product Development, Greater Certainty about Benefits and Risks of Products Earlier High potential for return to invigorate drug development and improve efficiency of development Reduce or eliminate the need for clinical data Important for improving drug development Challenging endeavor in today s environment of safety consciousness

10 Challenges to Re-Energizing Development Time, money, people. Progress needs Focus on science that will make a difference Process that works CDER DDT Qualification Process Champion Collaboration. Balanced collection and review of data

11 Why is Focused Work on DDTs Needed? Current Development Case by case For drug development, within a specific IND/NDA/BLA/Labeling Update For a specific drug Driven by a specific drug developer s needs Without focus, progress is slow, reliant on general use accepted over extended period Scientific experience accumulates through varied uses Usually very extended time-frame Evidence collection not cohesively directed Inefficient, silo d Results are non-public

12 Power of Collaboration It s the most efficient game in town Multiple stakeholders with multiple needs No single company, university, or governmental agency will have sufficient resources, expertise, or information bas to undertake the work. Builds consensus, expanding use Power worked examples of success of collaboration PCAST report calls for it, IOM is applying it, work on clinical trials certification FDA is applying it in a variety of situations

13 Power of Collaboration: FDA Role (cont) FDA sees the opportunities and pitfalls FDA has experience in appropriate ways for government to partner Transparent, open, inclusive, rigorous Results broadly applicable for maximum value Caution: FDA consortia have an important role to support full and open scientific discourse and needed data collection and discuss potential approaches to challenges, not to set federal policy

14 Examples of Collaborative Activities CONSORTIA, PUBLIC-PRIVATE PARTNERSHIPS

15 Example: CV Consortia/ CSRC HESI-CSRC Collaboration Pre-clinical cardiac safety database Replacing the Through QT Human Study Collaboration to link nonclinical data to early phase human data Building on ILSI-HESI work Improve quality of nonclinical cardiac assessment Replace Through QT Study

16 Example: Renal Biomarker Qualification Consortium Predictive Safety Testing Consortium Coordinated by CPath Institute 17 pharmaceutical companies represented, sharing data First result: validation of non-clinical nephrotoxicity biomarkers Next step: bridge these same biomarkers to clinical use Other working groups-nephrotoxicity, hepatoxicity, vasculitis, carcinogenicity, muscle toxicity

17 Public-Private Partnerships CAMD Coalition Against Major Diseases Collaboration: C-Path and Brookings Institutes, Academic and Industry Meeting November 5, 2012 Focus on Parkinson s and Alzheimer s Aims to clarify natural history using shared data from placebo use (aim: natural history of the disease) Support disease modeling and improved trial efficiency

18 Public-Private Partnerships Kidney Health Initiative Collaboration: FDA, ASN, Industry, Academia Focus: Create transparent infrastructure and processes that facilitate collaboration and communication among the greater Nephrology community and the FDA Develop novel medical products to advance kidney care. Ensure all products on the market are safe for patients and their kidneys. Eliminate racial and ethnic disparities in kidney disease

19 KHI Member Projects Many interesting ideas to discuss in areas commonly addressed through consortial activities Drug Development Tools: Biomarkers, COAs both critical in developing new renal therapeutics Endpoints in renal disease I look forward to hearing the progress you make!

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21 Summary/Conclusions This is a transformational time in the healthcare system. Expectations, resources, and challenges all changing Current development paradigm is not sustainable Clear role for regulators in finding solutions Targeted work to improve the tools of drug development promises to help re-energize development Collaboration is keys to this work Public-Private Partnerships like the KHI promise to support the needed changes in medical products development Why collaborate? BECAUSE WE HAVE TO!

22 The art of progress is to preserve order amid change and to preserve change amid order ---Alfred North Whitehead