Fast track Approval process- Ethical considerations

Transcription

1 Fast track Approval process- Ethical considerations Sara Ingersoll, MS Program Assistant, Health Sciences Institutional Review Board Rutgers, The State University of New Jersey New Brunswick/Piscataway Campus

2 Learning Objectives Understand the fast track approval process of emergency drugs. Identify the role of different stakeholders and their ethical responsibilities in the fast track approval process. 2

3 Contents FDA Fast Track Designation History of the fast track approval process Laws, Regulations & Guidance Fast track approval process Vioxx timeline Zmapp timeline Ethical considerations reviewing the role of the different stakeholders 3

4 FDA Fast Track Designation Is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet need. 4

5 History of the fast track approval process Laws, Regulations & Guidance The Food and Drug Administration Modernization Act of 1997 (FDAMA) includes Section 112, Expediting study and approval of fast track drugs Amended by Section 901 of the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) 5

6 Regulations 21CFR312 Subpart E Purpose Scope Early Consultation Treatment Protocols Risk Benefit analysis in review of marketing applications for drugs to treat lifethreatening and severely debilitating illnesses Phase 4 studies Focused FDA Regulatory research Active monitoring of conduct and evaluation of clinical trials Safeguards of patient safety 6

7 Guidance Guidance for Industry Expedited Programs for Serious Conditions - Drugs and Biologics -5/ ances/ucm pdf. 7

8 Fast Track - Designation Request Performance Report ( All requests received March 1, 1998 through March 31,2013) Number Submitted Goal Within Goal Overdue Grante d Denied Pendin g Total % Grante d Denied Pendin g Total % days Source: 8

9 Approval Process Fast track Breakthrough Therapy Accelerated Approval Priority Review 9

10 Fast Track Determining whether a condition is serious or not Filing an unmet medical need providing a therapy where none exists or providing a therapy which may be potentially better than available therapy Fast track drug must show some advantage over available therapy Fast track designation what it is eligible for 10

11 Breakthrough therapy Preliminary clinical evidence that indicates the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint Break through therapy designation i) Fast track designation ii) Intensive guidance iii) Organizational commitment involving senior managers 11

12 Accelerated Approval To be approved based on a surrogate endpoint Priority Review Will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would bring significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. 12

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14 The Vioxx Story November 1998 Merck asks the FDA for approval of Vioxx January 1999 Merck launches the VIGOR May 1999 FDA approves Vioxx October 1999 First DSMB Positive results November 1999 Second meeting of the VIGOR safety panel December 1999 Last DSMB meeting 2000 Agree to analyze the data Numerous epidemiological studies point to Vioxx s increased risk of cardiovascular problems September 2004 Merck withdraws Vioxx 14

15 ZMAPP March 2014 Ebola outbreak August 2014 WHO gives the green signal for unproven interventions and potential treatments August 2014 Early Was given to infected aid workers in United States Feb Clinical trial 15

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17 Ethical Considerations With limited numbers of experimental drugs who should receive the interventions? Who has the power to decide and design the experimental trial? Conducting scientifically rigorous trials Is it realistic? Control group Should we have one or not? Post marketing surveillance - Who will provide this data? 17

18 Role of the Investigator Requirements for informed consent (part 50) Requirements for review and approval by institutional review boards (part 56) These safeguards further include the review of animal studies prior to initial human testing (312.23) The monitoring of adverse drug experiences through the requirements of IND safety reports (312.32) Safety update reports during agency review of a marketing application (314.50) Postmarketing adverse reaction reporting (314.80) 18

19 Role of the Institution and the IRB Requirements for informed consent (part 50) Requirements for review and approval by institutional review boards (part 56) These safeguards further include the review of animal studies prior to initial human testing (312.23) The monitoring of adverse drug experiences through the requirements of IND safety reports (312.32) Safety update reports during agency review of a marketing application (314.50) Postmarketing adverse reaction reporting (314.80) 19

20 Summary Objective of the fast track approval process Different pathways that can be used Real Scenarios Ethical Considerations 20

21 Questions?????? 21