Evaluating the impact of REMS on burden and patient access

Transcription

1 Evaluating the impact of REMS on burden and patient access Doris Auth, Pharm.D. Team Leader, Division of Risk Management Office of Medication Error Prevention and Risk Management Center for Drug Evaluation and Research U.S. Food and Drug Administration

2 Overview REMS assessment background REMS assessment metrics Examples of assessment data Stakeholder feedback Conclusions 2

3 REMS ASSESSMENT BACKGROUND 3

4 Assessment Requirements* 1. Per the timetable for submission of assessments of a REMS in REMS (minimum 18 months, 3 years, 7 years) 2. With a supplement for a new indication for use (i.e., efficacy supplement) 3. If Agency determines that an assessment is needed to evaluate whether the approved strategy should be modified a. To ensure the benefits of the drug outweigh its risks b. Minimize the burden on the healthcare delivery system of complying with the REMS *FD&C Act, Sec 505-1(g)(2) 4

5 Assessment Requirements* With respect to each REMS goal: An assessment of the extent to which the REMS, including each element: Is meeting the goal; or Whether 1 or more such goals or such elements should be modified Modifications may be required when REMS programs not meeting goals, or to minimize the burden on the healthcare delivery system of complying with the REMS. *FD&C Act, Sec 505-1(g)(3) 5

6 Assessment Challenges Lack of comparators without REMS Small patient populations Low utilization of REMS drugs Representativeness of survey samples Ability to monitor outcome of interest Assessments conducted by applicant For these reasons, use of multiple metrics and surrogates necessary for evaluation 6

7 ASSESSMENT METRICS 7

8 Assessment Plan Development Proposed by applicant; further developed with FDA input Outlines metrics and methodologies used to evaluate program effectiveness FDA requests that applicants provide assessment methodologies (drug use, surveys) for FDA review and feedback 8

9 REMS Assessment Metrics Process and Compliance Knowledge Behavior Healthcare system burden and patient access Health Outcomes Generally easier to measure Generally harder to measure 9

10 REMS Assessment Metrics Launch date, website availability and utilization Geographic location of pharmacies and prescribers Number of patients treated and patient demographics Number of prescribers certified Number of prescriptions by certified vs non-certified prescriber Program compliance Surveys of prescribers, pharmacists, nurses, patients/caregivers Prescriber behavior Health outcomes Drug utilization Pharmacy switch data Shipment delays Call center information Stakeholder feedback 10

11 REMS ASSESSMENT EXAMPLES 11

12 Assessment Data that may Inform Burden and Access Shipment delays Pharmacy switch data Drug utilization Call center information 12

13 Example 1: Shipment Delays Source of information: Specialty pharmacies May inform: patient access issues including treatment delays or interruptions in therapy Information gaps: Doesn t distinguish whether delays related exclusively to REMS processes Doesn t provide information on outcome of shipment delay Unavailable for other dispense types (e.g., retail, hospital) 13

14 Example 2: Pharmacy Switch Data Source of information: Retail pharmacies that use switch for REMS adjudication May inform: Patient access issue if prescription delayed due to multiple REMS rejections Pharmacy burden if multiple REMS rejections, requirements to contact prescriber Information gaps: Doesn t provide outcome of REMS rejections Comparisons between delays for other adjudications and REMS edits 14

15 Example 2: Pharmacy Switch Data Time to authorization for a prescription that experienced at least one initial REMS-related rejection Total mean time for prescription to be authorized (days) Chain Independent outpatient Closed system Current reporting period Previous reporting period Cumulative Total median time for prescription to be authorized (days) Chain Independent outpatient Closed system pharmacies 15

16 Example 3: Drug Utilization Data Sources of data: Applicants REMS database, claims data May inform: Patient access issue Prescriber burden 16

17 Example 3: Drug Utilization Data Information gaps: Anticipated usage No comparator Differentiation of Appropriate vs Inappropriate use Impact of other actions on utilization (regulatory, other) Decreases in utilization prior to or concurrent with implementation of REMS due to media coverage, public meetings, safety communications 17

18 Example 4: Call Center Information Source of data: Applicant s REMS database May inform: Both program burden and barriers to patient access Information gaps: Relies on patients, pharmacies, prescribers to report issues Confusion between REMS-related issues with other issues Start up vs maintenance issues 18

19 Stakeholder Feedback 19

20 Stakeholder Feedback Source of data: Applicant or direct contact of stakeholder with FDA May inform: Prescriber, pharmacy, infusion center burden Patient access Information gaps: Representativeness of population providing feedback 20

21 Examples: Stakeholder Feedback Burdensome to have outside entity to collect labs; can t order ourselves Follow up phone calls to prescribers not burdensome and allow us to reinforce the requirements and safe use conditions Form could be revised to include another prepopulated field so not as much writing required REMS does introduce burdens, however, provides valuable safety checks and information on risk that allow prescribers to feel more comfortable in prescribing than other products without REMS 21

22 Other sources that may inform burden and access Survey data? May inform both access and burden Need to identify potential patients and prescribers Focus groups? Time-in-motion studies? Initial vs maintenance Root Cause Analysis? Failure modes effect analysis (FMEA)? Others? 22

23 Conclusions Burden and access issues must be considered on a case-bycase basis What data is available and what are the limitations of the data source? How should the data be analyzed and interpreted? Discussion and agreement between applicant and FDA as to potential sources of burden; patient access barriers during REMS development Effects of other approvals, regulatory actions, other messaging 23