Communication of anticancer drug benefits and related uncertainties to patients and clinicians: document analysis of regulated information on prescription drugs in Europe

Abstract

ObjectiveTo evaluate the frequency with which relevant and accurate information about the benefits and related uncertainties of anticancer drugs are communicated to patients and clinicians in regulated information sources in Europe.DesignDocument content analysis.SettingEuropean Medicines Agency.ParticipantsAnticancer drugs granted a first marketing authorisation by the European Medicines Agency, 2017-19.Main outcome measuresWhether written information on a product addressed patients’ commonly asked questions about: who and what the drug is used for; how the drug was studied; types of drug benefit expected; and the extent of weak, uncertain, or missing evidence for drug benefits. Information on drug benefits in written sources for clinicians (summaries of product characteristics), patients (patient information leaflets), and the public (public summaries) was compared with information reported in regulatory assessment documents (European public assessment reports).Results29 anticancer drugs that received a first marketing authorisation for 32 separate cancer indications in 2017-19 were included. General information about the drug (including information on approved indications and how the drug works) was frequently reported across regulated information sources aimed at both clinicians and patients. Nearly all summaries of product characteristics communicated full information to clinicians about the number and design of the main studies, the control arm (if any), study sample size, and primary measures of drug benefit. None of the patient information leaflets communicated information to patients about how drugs were studied. 31 (97%) summaries of product characteristics and 25 (78%) public summaries contained information about drug benefits that was accurate and consistent with information in regulatory assessment documents. The presence or absence of evidence that a drug extended survival was reported in 23 (72%) summaries of product characteristics and four (13%) public summaries. None of the patient information leaflets communicated information about the drug benefits that patients might expect based on study findings. Scientific concerns about the reliability of evidence on drug benefits, which were raised by European regulatory assessors for almost all drugs in the study sample, were rarely communicated to clinicians, patients, or the public.ConclusionsThe findings of this study highlight the need to improve the communication of the benefits and related uncertainties of anticancer drugs in regulated information sources in Europe to support evidence informed decision making by patients and their clinicians.

Balancing ethical norms and duties for the introduction of new medicines through conditional marketing authorization: a research agenda

“…Research shows, however, that physicians often misinterpret the robustness of evidence used for regulatory decision-making by the FDA, leading to overestimated benefits and underestimated risks (23,(25)(26)(27). Moreover, uncertainties about the benefit-risk balance are seldom communicated by regulators to physicians, patients, or the public (28).…”

Patient centred care: filling in the gaps

“…Research by Courtney Davis and colleagues on new cancer drugs approved by the European Medicines Agency is concerning on this front (doi:10.1136/bmj-2022-073711). 1 The study found that important information about the benefits of cancer drugs was not included in patient leaflets and that concerns about the reliability of evidence for these benefits were omitted in communication to clinicians and patients.…”