Dataset Information

Evaluation of the Effectiveness of the Risk Minimization Measures of Sodium Oxybate in the European Union.

ABSTRACT:

Background

Sodium oxybate (Xyrem^®), approved by the European Medicines Agency (EMA) for narcolepsy with cataplexy, is only available through risk mitigation programs due to potential adverse effects including respiratory and central nervous system depression, neuropsychiatric events, and misuse.

Objective

We report findings from a survey evaluating effectiveness of the European Union Xyrem^® Risk Management Plan (RMP).

Patients and methods

A cross-sectional, online, multiple-choice survey was distributed to randomly selected healthcare professionals (HCPs) from six European countries (April 2016-May 2018). Eligibility criteria: current/potential Xyrem^® prescriber and/or sleep disorder specialist; contact information available; on the Xyrem^® RMP educational materials mailing list.

Primary outcome

proportion of respondents answering each question correctly (< 50% responses correct = unsatisfactory comprehension, 50% to < 70% = satisfactory, ≥ 70% = excellent), with precision assessed using 95% confidence intervals (CIs).

Results

Of the 709 HCPs contacted, 601 did not agree to take part, 108 were screened with 35/108 eligible for inclusion; 31 HCPs completed the survey. Of the 31 respondents, 29 (93.5%; 95% CI 84.4-100.0) reported receiving Xyrem^® safety information, commonly from a sales representative, EMA Summary of Product Characteristics (SmPC), or educational meeting; only 9/31 (31.0%; 14.3-50.0) recalled receiving mailed educational materials. The number of HCPs answering dosing-related questions correctly ranged from 24/31 to 31/31. All Xyrem^® contraindications were correctly identified by 26/31 (83.9%; 70.0-96.7) respondents. All respondents 'always' or 'sometimes' completed SmPC recommended activities upon treatment initiation. The majority indicated signs of abuses/misuse/diversion (23/31; 74.2%; 58.6-88.0) and criminal use (23/31; 74.2%; 59.4-89.3) should be monitored at follow-up.

Conclusions

These data demonstrate the importance of providing a range of educational materials. However, the low sample size limits interpretation; increased HCP engagement would improve understanding of how best to develop educational materials.

European post-authorization study (pas) register number

EUPAS15024.

SUBMITTER: Iranzo A

PROVIDER: S-EPMC7581670 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Evaluation of the Effectiveness of the Risk Minimization Measures of Sodium Oxybate in the European Union.

Iranzo Alex A Serralheiro Pedro P Schuller Jan-Christof JC Schlit Anne-Françoise AF Bentz Jürgen W G JWG

Drugs - real world outcomes 20201201 4

<h4>Background</h4>Sodium oxybate (Xyrem<sup>®</sup>), approved by the European Medicines Agency (EMA) for narcolepsy with cataplexy, is only available through risk mitigation programs due to potential adverse effects including respiratory and central nervous system depression, neuropsychiatric events, and misuse.<h4>Objective</h4>We report findings from a survey evaluating effectiveness of the European Union Xyrem<sup>®</sup> Risk Management Plan (RMP).<h4>Patients and methods</h4>A cross-secti ...[more]

PMID: 32989679

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Project description:IntroductionStudies measuring the effectiveness of risk minimization measures (RMMs) submitted by pharmaceutical companies to the European Medicines Agency are part of the post-authorization regulatory requirements and represent an important source of data covering a range of medicinal products and safety-related issues. Their objectives, design, and the associated regulatory outcomes were reviewed, and conclusions were drawn that may support future progress in risk minimization evaluation.MethodsInformation was obtained from risk management plans, study protocols, clinical study reports, and assessment reports of 157 medicinal products authorized for cardiovascular, endocrinology, and metabolic indications. We selected observational studies measuring, as outcomes of interest, the relationship between the RMMs in place and (1) implementation measures, such as clinical knowledge or physicians` compliance to recommendations contained in the RMMs; and (2) occurrence or reduced severity of the adverse drug reactions for which the RMMs were required.ResultsOf 59 eligible studies (24 completed, 35 ongoing), 44 assessed implementation measures, whereas only 15 assessed safety outcomes (1 study as a single endpoint and 14 studies with other endpoints). Fifty-one studies used non-experimental designs and 25 studies employed electronic healthcare databases for analysis. Of the 24 completed studies, 17 were considered satisfactory and supported immediate regulatory decision making, 6 were considered inconclusive and required new evaluations, and 1 was terminated early because new safety restrictions were required, thereby necessitating a new evaluation. Compliance with agreed deadlines was considered acceptable in 21 of 24 completed studies; the average time for a submission was 37 months (standard deviation ± 17), with differences observed by type of data source employed.ConclusionsThree important gaps in the evaluation plans of RMMs were identified: lack of early feedback on implementation, limited evaluation of safety outcomes, and inability to provide information on the effectiveness from an integrated measurement of different elements of a set of risk minimization tools. More robust evidence is needed to advance regulatory science and support more rapid adjustment of risk minimization strategies as needed.

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Dataset Information

Evaluation of the Effectiveness of the Risk Minimization Measures of Sodium Oxybate in the European Union.

Background

Objective

Patients and methods

Primary outcome

Results

Conclusions

European post-authorization study (pas) register number

Publications

Evaluation of the Effectiveness of the Risk Minimization Measures of Sodium Oxybate in the European Union.

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