Update From the Office of Surveillance and Epidemiology

Transcription

1 Update From the Office of Surveillance and Epidemiology Gerald J. Dal Pan, MD, MHS Director Office of Surveillance and Epidemiology Center for Drug Evaluation and Research Presentation to FDA-CMS Summit Washington, DC 11 December

2 2 Office of Surveillance and Epidemiology Office of Surveillance and Epidemiology Gerald Dal Pan, Director Robert Ball, Deputy Director Administration Project Management Regulatory Affairs Regulatory Science Outreach and Communications Organizational Development Office of Pharmacovigilance and Epidemiology Office of Medication Error Prevention and Risk Management Division of Pharmacovigilance I Division of Pharmacovigilance II Division of Epidemiology I Division of Epidemiology II Division of Medication Error Prevention and Analysis Division of Risk Management

3 OSE Engagement & Collaborative Reviews Standard NME NDA & Original BLA Review Timeline Pre- Filing/Panning meeting (Day 45) Submission meeting Application Receipt Mid-Cycle Meeting Mid-Cycle Communication with Applicant Late Cycle meeting AC meeting Wrap-Up Mtg. Action Date Total Time Clock Time Months Conduct Filing Review Conduct Review Labeling/PMC/PMR/REMS OSE Pre-submission topics Preliminary discussion of REMS/ need for REMS Plans for proprietary name submission if not submitted in IND. Plans for post market safety monitoring Discussion on Human Factor studies if applicable OSE Review Activities Proprietary Name Medication Error Prevention Labeling including directions for use Carton Container Review Human Factors/Usability Review REMS/Risk Management Plans Specific Safety Reviews (i.e. hepatotoxicity) Review of epidemiology studies submitted to the NDA OSE Late Review Cycle Engagement Finalization of REMS with OND Post market safety monitoring Safety PMR protocol as needed Advisory Committee as needed Denotes milestone meetings with OSE in attendance 3

4 4 OSE s Roles in Postmarketing Drug Safety NDA/BLA/ANDA approval REMS Assessments Modifications Shared System REMS PMRs Finalized in the post approval window Review protocols and study reports of epidemiological studies Postmarket surveillance FAERs screening Biweekly screening 915 reviews BPCA/PREA reviews Epi and drug use data Sentinel Other Evaluation of signals Safety issues/safety label changes Product quality-related safety reviews 915 reviews BPCA/PREA reviews

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7 Mini-Sentinel Transition to Sentinel Awarded to Harvard Pilgrim Healthcare Institute 50+ healthcare and academic organizations Will build on existing infrastructure and capabilities 1) Summary tables precalculated tables 2) Modular SAS programs reusable, similar to SAS Procs 3) Protocol based assessments - custom SAS programs for in-depth assessments 4) PROMPT 7 7

8 Sentinel: PROMPT Characteristics Semi-automated analyses Partial adjustment for confounding Use of various epidemiologic design approaches (cohort, self-controlled designs) Use of complex statistical approaches Option of one time or sequential analyses allowing for looks at risk while uptake increases Pre-programmed modules for Design options Adjustment for confounding based on indications and outcomes Statistical approaches Allow rapid assessments 8

9 9 Sentinel: PROMPT Surveillance at a Glance Newly marketed product Define exposures, outcomes, etc Choose analysis approach Estimate the risk Repeated sequential analyses Aggregate results over time Apply alerting rules Report to FDA Consider results along with other data streams FDA reports to public when appropriate

10 Innovation in Medical Evidence Development and Surveillance (IMEDS) The IMEDS program is a public private partnership offered by the Reagan-Udall Foundation for the FDA through the FDA Amendments Act of 2007 ( IMEDS s primary objective advance the science and tools necessary to support post-market evidence generation on regulated products, including safety surveillance and evaluations, and facilitate utilization of a robust electronic healthcare data platform for generating better evidence on regulated products in the post-market settings 10

11 Innovation in Medical Evidence Development and Surveillance (IMEDS) 11

12 12 Innovation in Medical Evidence Development and Surveillance (IMEDS) IMEDS Methods The IMEDS Research Laboratory is available for use by investigators interested in participating in IMED s mission IMEDS Evaluation IMEDS Education 2014 Research Agenda has been implemented A core goal of the 2014 IMEDS Methods Research Agenda is to support the FDA s Sentinel system, including an assessment of differences in results between the Mini-Sentinel system and the IMEDS system (based on the Observational Medical Outcomes Partnership) 2015 Research Agenda is under development Secure computing platform housing research tools and 5 de-identified healthcare datasets Currently 20 projects underway by academic, industry and government researchers Apply lessons learned from IMEDS-Methods; leverage Mini-Sentinel (MS) tools and capabilities; and collaborate with MS data partners to enable non-fda entities (such as regulated industry and international regulatory agencies) to sponsor safety queries of marketed medical products Pilot project underway When developed, will offer educational opportunities in areas related to medical product safety surveillance, and methods research and application for scientific professionals

13 Number of Communication Plan and ETASU REMS 1 1 Data reflects the: Initial approval date of the Communication Plan or Element-To-Assure-Safe-USE (ETASU) REMS Number of REMS approved included shared system REMS, not the number of products approved with a REMS If a product has a REMS with both a Communication Plan and an ETASU, only the ETASU is counted 13

14 Standardizing and Evaluating Risk Evaluation and Mitigation Strategies (REMS) Report issued September 2014 Prescription Drug User Fee Act (PDUFA) V commitment: to measure the effectiveness of REMS, to continue to develop techniques to standardize REMS, and with stakeholder input seek to integrate REMS into the existing and evolving health care system. Pilot projects in four areas Source: US FDA. REPORT: Standardizing and Evaluating Risk Evaluation and Mitigation Strategies (REMS) September 2014; 14

15 REMS Priority Project Summary Areas under REMS Patient Benefit/Risk Information Prescriber Education Pharmacy Systems Practice Settings Identified project 1. Providing Patient Benefit/Risk Information by Improving Tools for Prescriber-to-Patient Counseling 2. Prescriber Education REMS and Continuing Education (CE) for Health Care Providers 3. Standardizing REMS Information for Inclusion into Pharmacy Systems Using Structured Product Labeling (SPL) 4. Providing a Central Source of REMS Information for Practice Settings 15

16 Project #1 Providing Patient Benefit/Risk Information by Improving Tools for Prescriber-to-Patient Counseling How the project addresses stakeholder feedback Stakeholders have communicated to FDA their concern that REMS programs are seen as potentially deterring providers from prescribing, and patients from taking, drugs that may, on balance, be appropriate for them simply because the product has a REMS. Patients value having information to improve their understanding about the product s serious risks, thereby facilitating more informed decisions (made together with a healthcare provider) to initiate or maintain therapy. Project overview Project deliverable(s) Conduct research into existing REMS patient counseling tools, other patient counseling initiatives, and counseling literature to identify the current state of patient counseling regarding medication benefits and risks. Seek public feedback to improve upon content, format, processes, techniques, tools, and delivery of effective counseling of REMS programs. Develop a report of findings, counseling processes, and tools that could serve as basis for designing new tools and validating them in demonstration projects. 16

17 Project #2 Prescriber Education REMS and Continuing Education (CE) for Health Care Providers How the project addresses stakeholder feedback Project overview Stakeholders have asked the FDA to facilitate the provision of CE for the education and training of health care providers who prescribe/dispense or anticipate to prescribe/dispense a drug product with a REMS program. Stakeholders confirmed that there are low participation rates for non-ce training programs and limited reach for Dear Health Care Provider letters. Assess if it is feasible to accredit CE with certain REMS so health care professionals will receive accredited CE when they complete prescriber education activities (such as study and self-assessment tests, modular web-based activities, webinars, etc.). Accrediting bodies will ensure that CE activities developed as part of this project will be in compliance with the CE standards for their organizations. Project deliverable(s) For new drugs, determine at what stage the development of CE would best fit in the drug approval process (e.g., pre- or post-approval). Provide an analysis of the time and resources related to developing and using CE to conduct REMS-related training and/or communication. 17

18 Project #3 Standardizing REMS Information for Inclusion into Pharmacy Systems Using Structured Product Labeling (SPL) How the project addresses stakeholder feedback Project overview Stakeholders have expressed concern that REMS materials, tools, strategies, and requirements are inconsistent, even across drugs with similar risks used in similar settings, and are not communicated to stakeholders in a clear and consistent manner. REMS materials and requirements may be difficult to locate, and specific activities and requirements of various stakeholders (e.g., prescriber, pharmacist, etc.) are not clearly outlined. Some stakeholders have difficulty integrating REMS materials and procedures into their existing health information systems and health care delivery processes. Stakeholders reported spending excessive time trying to understand and comply with different sets of REMS requirements and experiencing difficulty ensuring appropriate use of drugs with REMS. Work with standards development organizations (SDOs), if appropriate, FDA will identify approach to incorporating REMS information into SPL Project deliverable(s) Make structured REMS information available to health care providers, patients, and FDA 18

19 Project #4 Provide a Central Source of REMS Information for Practice Settings How the project addresses stakeholder feedback Project overview Project deliverable(s) Stakeholders shared that FDA REMS website should provide more information about the content of REMS programs, including what is required of specific stakeholders (e.g., whether certain parties need to enroll or become certified as part of the REMS). Stakeholders also asked that currently available information be provided in a more user-friendly format, and that key information found in the FDA-approved REMS document is summarized to provide a concise overview. FDA proposes a series of enhancements to its existing REMS website that will make the site more user-friendly and to add additional information about REMS programs. Where possible, FDA will leverage information captured through SPL. An enhanced FDA REMS website 19

20 Draft Guidance for Industry: Best Practices in Developing Proprietary Names for Drugs Issued May 28, 2014 Intended to promote development of proprietary names that do not cause or contribute to medication errors or the misbranding of the drug A systematic, standardized, and transparent approach to proprietary name evaluation 20

21 Contents of Best Practices for Developing Proprietary Names for Drugs I. Prescreening considerations for proprietary name candidates II. Consider attributes that may be misleading or error-prone III. Misbranding review Avoid names that overstate efficacy, minimize risks, or make unsupported suggestions of unique effectiveness or composition IV. Methods for Evaluating Safety of Proposed Proprietary Names Avoid names with orthographic, spelling, and phonetic similarity to other names that could result in errors 21

22 CDER/CBER Proprietary Name Submissions (type, by FY) *FY 2014 data from 1 Oct 2013 through 31 Aug

23 CDER PDUFA Performance for Name Review: % of Goals Met *FY 2014 CDER data from 1 Oct 2014 through 30 June

24 CDER Granted rates (% of names evaluated)** *FY14 data from 1 Oct 2013 through 31 Aug 2014 **Percentages calculated based on # of completed evaluations 24

25 CDER: FY14* Name Denial Reasons CDER FY 2014 Denials (n=33) Reasons Identified for Rejection (Misbranding or Safety) # Reasons** Misbranding 13 Safety (similar in spelling, speech and/or writing) 17 Other Attributes Within the Name That Posed Risk for Error or Found to be Misleading 5 *FY14 data covers from 1 Oct 2013 through 31 May 2014 **A name may have more than 1 reason for denial, so the total number of reasons exceeds the number of names denied 25

26 Thank you 26