2015 Policy Landscape: A Biomedical R&D Planning Guide

Transcription

1 2015 Policy Landscape: A Biomedical R&D Planning Guide A FasterCures Webinar Jan. 29, 2015

2 Action FasterCures is an action tank driven by a singular goal to save lives by speeding up and improving the medical research system. A center of the Milken Institute, we are a nonprofit and nonpartisan organization that works with all the sectors of the medical research and development ecosystem.

3 Efficiency Our work is focused on bringing efficiency to the medical research R&D process by identifying and eliminating the roadblocks that slow medical research down.

4 Innovation train.fastercures.org

5 Webinars Purpose Who s logged on? 2015 topics Real-time sharing of ideas, best practices, trends, and lessons learned Amplifies meaningful solutions, productive tools, and encourages action needed to spur medical progress Government 5% Academia 8% Biotech/ Pharma 15% Nearly 200 registrants from: Other 15% Nonprofit 57% R&D policy Science of patient input Venture philanthropy

6 Speakers Dave Zook Chair, FaegreBD Consulting Andrew Emmett Managing Director, Science & Regulatory Affairs, BIO Margaret Anderson, Executive Director, FasterCures MODERATOR

7 Margaret Anderson Executive Director, FasterCures MODERATOR

8 Dave Zook Chair, FaegreBD Consulting

9 Congressional Outlook 2015 The Path Ahead...

10 Congressional Outlook 2015

11 Congressional Outlook

12 Congressional Outlook

13 Congressional Outlook 2015 SGR Reform

14 Congressional Outlook 2015 King v. Burwell

15 Congressional Outlook st Century Cures

16 Congressional Outlook 2015

17 Margaret Anderson Executive Director, FasterCures MODERATOR

18 Andrew Emmett Managing Director, Science & Regulatory Affairs, Biotechnology Industry Organization

19 Overview 21 st Century Cures PDUFA 6 FDA Funding Drug Approval Trends

20 21 st Century Cures

21 21 st Century Cures If we want to save more lives and keep this country the leader in medical innovation, we have to make sure there s not a major gap between the science of cures and the way we regulate these therapies We are looking at the full arc of this process from the discovery of clues in basic science, to streamlining the drug and device development process, to unleashing the power of digital medicine and social media at the treatment delivery phase.

22 21 st Century Cures Launched in May 2014 Unprecedented Public Engagement 5 white papers 12 Hearings and Roundtables 64 Press Releases Significant Public Comment Discussion Draft Released January 27 th, 2014 Nearly 400 pages of legislative text

23 21 st Century Cures Priority Topics

24 Title 1: Putting Patients First by Incorporating Patient Perspectives into the Regulatory Process and Addressing Unmet Medical Needs Patient Focused Drug Development Surrogate Endpoint Qualification and Biomarkers Approval of Breakthrough Therapies Antibiotic Drug Development (ADAPT Act & DISARM Act) Expanded Access to Investigational Therapies Scientific and Medical Communication / Social Media Incentives to Develop Dormant Therapies (MODDERN Cures Act) *Select provisions only

25 Title 2: Building the Foundation for 21 st Century Medicine, including Helping Young Scientists 21 st Century Cures Consortium Act Regenerative Medicine Genetically Targeted Platform Technologies for Rare Diseases Clinical Data Sharing Real-World Evidence Combination Products Longitudinal Study on Chronic Conditions Precision Medicine (TBD) *Select provisions only

26 Title 3: Modernizing Clinical Trials Central IRBs Adaptive Trials Designs and Baysean Statistics Post-approval Studies Pediatric Research Networks *Select provisions only

27 Title 4: Accelerating the Discovery, Development, and Delivery Cycle and Continuing 21 st Century Innovation at NIH, FDA, CDC, and CMS National Institute of Health Provisions Vaccines Access, Certainty, and Innovation Reagan-Udall Improvements FDA Hiring, Travel, and Training (TBD) FDA Succession Planning Local and National coverage Decision Reforms *Select provisions only

28 Title 5: Modernizing Medical Product Regulation 21 st Century Manufacturing Controlled Substances Advisory Committee Process *Select provisions only

29 Prescription Drug User Fee Act (PDUFA 6)

30 About the PDUFA Program Established in 1992, PDUFA has reduced overall review times by more than a year and has contributed to the approval of more than 1,200 new therapies User fee resources facilitate the hiring of additional FDA medical reviewers Clear and predictable performance goals 6 month Priority Review 10 month Standard Review

31 PDUFA Reauthorization Themes Reauthorized Every 5 Years PDUFA 1 (1992) Reducing the Drug Lag PDUFA 2 (1997) FDA Modernization PDUFA 3 (2002) Biodefense and Preparedness PDUFA 4 (2007) Lifecycle Approach to Safety Surveillance PDUFA 5 (2012) Promoting Biomedical Innovation PDUFA 6 (2017) -

32 PDUFA 6 Timeline: ERG Report & Public Meeting (March) Parallel Meetings with Stakeholders (Sept-March) FDA-Industry Agreement (March 2016) Publish in FR, Public Meeting (Sept 2016) PDUFA V Expires (Oct 2017) FR Notice, PDUFA 6 Public Meeting (June 23 rd ) Submit to HHS (April 2016) Submit to OMB (June 2016) Transmit to Congress (Jan. 2017) Strategic Planning January-August Technical Discussions Sept-March Clearance Process April-Sept Brief Hill Oct-Jan Legislative Process January-September Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q st Century Cures Election

33 33 New Molecular Entity Review Program

34 Source: FDA, Jenkins, John, Director, Office of New Drugs, CDER New Drug Review: 2014 Update, FDA/CMS Summit, December 11, 2014,

35 Patient Focused Drug Development Disease Areas 1. Alpha-1 antitrypsin deficiency; 2. Breast cancer; 3. Chronic Chagas disease; 4. Female sexual dysfunction; 5. Fibromyalgia; 6. Hemophilia A, hemophilia B, von Willebrand disease, and other inheritable bleeding disorders; 7. HIV; 8. Idiopathic pulmonary fibrosis; 9. Irritable bowel syndrome, gastroparesis, gastroesophageal reflux; 10. Lung cancer; 11. Myalgic encephalomyelitis/cfs; 12. Narcolepsy; 13. Neurological manifestations of inborn errors of metabolism; 14. Parkinson s and Huntington s disease; 15. Pulmonary arterial hypertension; 16. Sickle cell disease

36 Structured Benefit/Risk Assessment 5-year plan issued February 2013; public comments being reviewed Key factors in a structured approach What s known or unknown (necessitating assumptions) Knowledge of disease/condition and existing treatment What s clear and unclear about benefits and risks Can risk be managed adequately? What are the conclusions and rationales?

37 Structured Benefit/Risk Framework Decision Factor Analysis of Condition Evidence and Uncertainties Conclusions and Reasons Current Treatment Options Benefit Risk Risk Management Benefit-Risk Summary Assessment

38 FDA Funding

39 FDA Funding Consolidated and Further Continuing Appropriations Act for Fiscal Year 2015 FDA budget of $2.588 billion, an increase of $36 million compared to FY14 FY16 budget expected soon Impact of sequestration

40 Drug and Biologic Approval Trends

41 Approval Trends 41 Drugs Approved in an 18 year high 12 biologics approved 15 orphan drugs 7 Breakthrough Designated Products 9 Accelerated Approvals Breakthrough Therapy Designations 17 indications approved 74 designations granted (66 CDER, 8 CBER) 270 requests received 139 requests denied

42 Source: FDA, Jenkins, John, Director, Office of New Drugs, CDER New Drug Review: 2014 Update, FDA/CMS Summit, December 11, 2014,

43 Q&A Dave Zook Chair, FaegreBD Consulting Andrew Emmett Managing Director, Science & Regulatory Affairs, BIO Margaret Anderson, Executive Director, FasterCures MODERATOR

44 View an archive of this Webinar

45 Register for upcoming Webinars train.fastercures.org February topic: Venture Philanthropy 101: What You Need to Know About Investing in Companies more details coming soon

46 Keep up with fastercures.tumblr.com

47 The latest developments in medical research delivered to you every Tuesday and Thursday to keep you current on relevant news. Subscribe

48 Connect fastercures.org

49 TRANSFORMATION IS HAPPENING robust social networks adaptive clinical trials health IT connected, networked world new healthy delivery models EMPOWERED PATIENT precision medicine scientific advances

50 FasterCures' public comments on FDA activities for patient participation in medical product discussions Dec. 4, 2014 Recommendations have one aim: to facilitate an intentional evolution from FDA s traditional engagement with individuals who serve as spokespersons for a disease/condition to an evidence-based means of understanding the range of patients experiences FDA s patient engagement activities should be: purposeful reciprocal dynamic transparent

51 Is anyone here willing to take this risk? Photo courtesy of Tarek Hammad, Merck

52 Proactive interest in patient perspective is expanding Industry sponsors are building patient perspectives into the unmet need case for access to expedited regulatory pathways Patient preferences are also informing sponsors reimbursement strategy FDA is directly seeking patient input on what symptoms they would most like resolved

53 partneringforcures.org Partnering for Cures plenary: The future of U.S. research leadership Nov. 17, 2014

54 Understanding the power of patients: The HIV/AIDS Model

55 The HIV/AIDS Model