Comments on EMA Policy 0070 on publication and access to clinical-trial data

2013-09-30

Comments from:

Institute for Quality and Efficiency in Health Care (IQWiG) Im Mediapark 8
D-50670 Köln
Germany

Please note that these comments and the identity of the sender (not contact details) will be published unless a specific justified objection is received.When completed, this form should be sent in Word format (not PDF) to: ctdatapolicy@ema.europa.eu

Comments on text

General remarks

IQWiG strongly supports the improved publication and access to clinical trial information described in EMA’s draft policy. Full trial information and results are needed for HTA agencies like IQWiG to be able to provide appropriate and meaningful assessments of drugs within their remit. As drug assessments conducted by HTA agencies support evidence-based decision making in health care systems, improved access to clinical trial data is in the interest of public health.

There is overwhelming evidence, that so far publicly available trial data are insufficient to provide a complete and unbiased picture of a given health care intervention. HTA needs additional independent and high quality data sources. Data submitted to regulatory agencies are therefore required by IQWiG and other HTA agencies. However, the data held by EMA are not only important for HTA agencies but also for other researchers supporting evidence-based decision making in health care and should thus in general be made publicly available.

HTA performed by IQWiG and other agencies specifically is aiming to describe comparative effectiveness. The methodology used by HTA requires

• information on all trials conducted with the intervention under assessment
• full information about clinicaltrial methods, e.g. forrisk of bias assessment
• full information about clinical trial results, e.g. for meta-Analysis
• extended information about patient populations included in clinical trials, e.g. to understand to what extent the study results are relevant for real life populations

In addition, comparative effectiveness research increasingly uses indirect comparisons. For this type of analysis full information on study methods including e.g. operationalization of study endpoints and on patient populations is required to allow for assessing assumptions of similarity of studies in a network for indirect comparisons.

IQWiG’s own work has shown that clinical trial documentation held by regulatory agencies provides substantial additional information compared to publicly available trial reports. A comparison of clinical study reports (CSR) with publicly available journal publications and reports from study registries has shown, that CSRs provided complete information on 88 % of relevant methods items, while journal publications included complete information only on 40 % of methods items and registry reports only on 31 % of methods items (1). Concerning clinical trial results, CSRs provided complete information on 86 % of patient-relevant trial outcomes while journal publications and registry reports presented complete information on only 23 % and 22 % of patient-relevant trial outcomes,respectively (39 % in the combined publicly available sources) (2). This additional information from CSRs can challenge published evidence on a given health care intervention or even reverse conclusions drawn based on publicly available Information (3).

These data clearly describe the information gainfrom one part EMA’s draft policy, i.e.making CSRs publicly available. Access to patient-level data willallow further research questions to be addressed. Our studies underline the relevance of improved public access to full clinical trial data according to EMA’s draft policy for evidence-based decision making and thus public health. Our studies also show, that alternative proposals like the EFPIA’s and PhRMA’s recently adopted “Principles for Responsible Clinical Trial Data Sharing” are insufficient to solve the problems associated with an incomplete public record of information on health care interventions, e.g. because they suggest publication of only limited information (synopses of CSRs orjournalpublications) on alimited range of clinical Trials.