Clinical Trial Results in the Public Domain

Following each clinical trial, the sponsor will compile a detailed clinical study report (CSR) which follows a format laid down by the Regulatory Authorities. It usually contains several hundred pages. Access to this report is usually limited to the sponsor themselves and the Regulatory Authorities assessing an application for marketing authorisation.

Summarised information from the CSR is, however, likely to come into the public domain via a number of routes.

European Public Assessment Reports (EPARs)

A European public assessment report (EPAR) is published for every human medicine application that has been granted or refused a marketing authorisation in the framework of the central authorisation of medicines. An EPAR provides public information on a medicine, including how it was assessed by EMA, together with a public-friendly overview.

This does not happen when the medicine is approved locally by one of the member states. In case of approval via the Mutual Recognition Procedure (MRP) or Decentralised Procedure (DCP) a similar public assessment report is published on the Heads of Medicines Agencies (HMA) website. The EPAR in the efficacy part contains a summarised version of the information in the corresponding CSR.

The EPAR is intended for a professional audience and is therefore technical in language. It is, however, accompanied by a summary for the public, which presents the key facts in non-technical language.

EPARs are displayed on the EMA website using four different sections containing different components of the EPAR. The below table provides an overview:

Section

Type of information

Overview

Public-friendly overview in question-and-answer format.

Authorisation details

Key details about the product and the marketing authorisation holder.

Product information

Package leaflet and summary of product characteristics; labelling; list of all authorised presentations; pharmacotherapeutic group; therapeutic indications.

Assessment history

Public assessment report for the initial authorisation; public assessment report(s) for any variation concerning major changes to the marketing authorisation; orphan maintenance assessment report or withdrawal assessment report (as of 17 January 2018); tabulated overview of procedural steps taken before and after authorisation.


Adapted from EMA (link)

Clinical Trial Registries

In Europe, the The EU Clinical Trials Register contains information on all interventional clinical trials of medicines performed in Europe (after May 1, 2004) in the EudraCT, (European Union Drug Regulating Authorities Clinical Trials Database). This is the database used by national medicines regulators for data related to clinical trial protocols. The data on the results of these trials are entered into the database by the sponsors themselves and are published in this Register once the sponsors have validated the data. As of July 2014, this database also makes trial summary results available to the public. For trials taking place in the EU starting after January 1st 2015, the results must be published regardless if negative or positive.

The World Health Organisation (WHO), through its International Clinical Trials Registry Platform (ICTRP), is setting international standards for registering and reporting on all clinical trials.

In the US, the registry clinicaltrials.gov is taking a similar approach.

Marketing Authorisation Product Information (see EPAR)

Although specific results of individual clinical trials are rarely presented in this way, an overall summary of the information available on a particular medicine is available from its summary of product characteristics (SmPC). This is a document aimed at healthcare professionals, however it forms the basis of the package leaflet (PL) (previously known as the patient information leaflet, (PIL)). This is aimed at the patient or user. These documents are normally available on the Internet depending on national regulations both from individual manufacturer’s sites or from regulatory authorities or sites run by independent organisations.

Journal Papers

The classic route for publication of clinical trial results is a research paper in a specialist medical journal. Virtually all modern journals have a peer-review process under which independent experts in the field review the manuscript and challenge any weak aspects of it before publication.

Conferences

A large number of international medical conferences are held every year, some with a quite general theme. However, many focus on a narrow specialist area. Clinical trial results are often presented at these conferences either as oral presentations or as posters displayed in public areas of the conference venue. Unfortunately, access to this information is often restricted to those who attend the conference and is not easily available to those who do not, unless the conference presentations are made available online. In many cases, however, the same trial will also be the subject of a journal paper.

Patient Organisation Websites

Many patient organisations provide help to patients with specific illnesses and many have websites that publish reports of relevant clinical trials. Interpretation by experts working with the organisation, together with the use of patient-friendly language, tend to make these reports particularly useful to patients.

Popular News Media

The accuracy and understanding with which television, radio and newspaper accounts present the results of clinical trials varies a great deal. However, as a general rule it is wise to approach such reports with the understanding that a sensational story is more likely than a sober account to sell newspapers.