Benefit assessment of pharmaceuticals: intensive involvement of external experts with controversial results.

12.04.2016 12:58

Experts from science and healthcare are heavily involved in the benefit assessment of new drugs. In three-quarters of the assessments, their opinions differ significantly from those of the Institute for Quality and Efficiency in Health Care (IQWiG). Furthermore, their positions are incorporated differently into the final decision of the Federal Joint Committee (G-BA) regarding the added benefit of new medications. This is shown in a study by the IGES Institute.

In the study, IGES researchers comprehensively examined the role of third-party opinions in the benefit assessment under the German Act on the Reform of the Market for Medicinal Products (AMNOG). All 136 procedures that were regularly completed between the start of benefit assessment in 2011 and August 2015 were included.

The strong interest of external experts in participating is evident in the continuously growing number of statements from medical professional societies. This number rose from an average of 1.6 to 1.8 statements per procedure between 2012 and 2015. In total, 60 different professional societies, associations, and working groups submitted statements to 112 (82 percent) of the 136 AMNOG procedures analyzed.

Discrepancy in added benefit in more than half of the procedures

The comments from the external experts focus primarily on three areas: the patient-relevant added benefit, the comparator therapy against which new drugs are compared, and the endpoints by which the added benefit is measured. Regarding added benefit, the external experts disagreed with the IQWiG in more than half (54 percent) of the cases. For the comparator therapy and the endpoints, the figures are 43 and 47 percent, respectively.

Professional societies see a higher added benefit than IQWiG and AkdÄ.

It is striking that professional societies deviate more frequently and more positively from the IQWiG assessments. In 84 percent of their statements, they recognize a greater added benefit for a new drug, while the Drug Commission of the German Medical Association (AkdÄ) only recognizes this in 18 percent.

„The extent of the differing assessments is surprising, since everyone is judging on the basis of the same data and the methods of evidence-based medicine,“ says Hans-Holger Bleß, study leader and head of health services research at the IGES Institute. „Clearly, early benefit assessment is not a mechanical process, but allows for different interpretations, even when it is supposed to be an objective classification of the patient-relevant added benefit.“

The G-BA agrees more often with the AkdÄ than with professional societies.

The G-BA (Federal Joint Committee) takes the opinions of external experts into account differently in its final decision. For example, regarding added benefit: here it agrees more frequently with the German Medical Association (AkdÄ) than with the specialist societies, in 69 percent versus 51 percent of the statements.

„With their intensive involvement, scientific experts contribute to a sound benefit assessment. Their involvement should be strengthened and made more transparent,“ says Bertram Häussler, head of the IGES Institute. He referred to international examples. In Great Britain, for instance, unlike the G-BA, the decision-making body is composed of two-thirds representatives from science and outpatient physicians.

Experts would like to see more intensive participation in the IQWiG survey.

The IQWiG itself also surveys experts as part of its assessment process. This is done via questionnaires that experts must return within five days. The IQWiG provides no information on the questions and answers or their consideration in the assessment process. As a survey conducted as part of the IGES study shows, the experts involved perceive the processing time, clarification, and use of their input as insufficient. Nevertheless, they signal a high level of interest and a willingness to participate more intensively in the assessment process.

The study „Impact of scientific opinions in the benefit assessment of pharmaceuticals“ was commissioned by the research-based pharmaceutical company Takeda.