The marketing application submitted by the manufacturer for a medicine includes data on the pharmaceutical quality of the medicine, and on efficacy and safety.

The firm requesting a marketing authorisation carefully selects these data. Obviously, the firm wants to show this product in its best light. Very often, the clinical trials have been designed merely to show that the medicine is just as effective as the existing treatments, as the current regulation does not demand that a new medicine be superior to available options.

The pressure on experts from regulators is very important considering the financial stakes. The experts who examine the data should therefore have critical appraisal skills in order to detect possible biases the firm could have introduced, and to come to an independent decision. Theoretically, they should be able to examine data not provided by the company, or at least data available in the scientific literature, in the agencies of other countries, in independent evaluation organisations and pharmacovigilance bodies.

The recent celecoxib (CELEBREX°) affair is a case in point. In order to present this product as having fewer serious effects on the gastrointestinal system than its competitors and to obtain a corresponding modification of the marketing authorisation, the company managed to publish in a well-known journal (JAMA) a paper overstating the results of a trial. A rigorous investigation of all trial results carried out by the American Drug Agency (FDA) showed that the version published had distorted the trial protocol, misconstrued the results by focusing on the most favourable period of the trial and omitting the final results. Eventually the modification to the marketing authorisation was not granted but this decision took time and expertise.

Such in-depth literature search and comparative appraisal of all data is far from being achieved in the present time limit of 210 days allowed for the decision to grant marketing authorisation. Experts actually have at best two months within this time limit, and the agencies do not usually provide them with any other data than what has been selected by manufacturers.

Once evaluation of data has been carried out, the experts hand in their reports, which form the basis of the opinion given by the Committee responsible for recommending the use of the medicine. Limitations to usage, contraindications for some categories of patients (the elderly, people with liver, kidney or heart problems) are some of the difficult issues this Committee has to deal with. They show that time and thoroughness are vital elements in the decision making process, above all with this scarcity of data.