Early benefit assessment: Informative RCTs are also feasible in later lines of oncological treatment

In two early benefit assessments, the Institute for Quality and Efficiency in Health Care (IQWiG) investigated whether the oncological drugs/drug combinations under consideration offer patients an added benefit compared with the previous treatment options, even after several pretreatments (known as “treatment lines”).

The (German-language) reports were published in November 2023; an English translation in March 2024.

The answer is “yes” in both cases: For the combination of trifluridine/tipiracil with bevacizumab in adults with metastatic colorectal cancer who have already received two cancer therapies, the Institute found an indication of a non-quantifiable, but at least considerable, added benefit compared with the appropriate comparator therapy. The most striking finding was the increase in overall survival. And for sacituzumab govitecan in patients with unresectable HER2-negative breast cancer in the fourth line of treatment, there is a hint of a minor added benefit compared with the appropriate comparator therapy. This combination offers advantages to patients particularly in terms of health-related quality of life.

Drug manufacturers conducted randomized controlled trials

These findings were only possible thanks to two randomized controlled trials (RCTs), each of which involved over 90 study centres worldwide. “This is doubly pleasing,” says Volker Vervölgyi, Head of the Oncology Division in IQWiG’s Drug Assessment Department. On the one hand, it shows that it is certainly possible to conduct RCTs in third and fourth-line treatment and to also recruit enough people to obtain informative results. On the other, the results open up new treatment perspectives for patients and their physicians.”

G-BA decides on the extent of added benefit

The dossier assessments are part of the early benefit assessment according to the Act on the Reform of the Market for Medicinal Products (AMNOG) supervised by the Federal Joint Committee (G-BA). After publication of the dossier assessments, the G-BA conducts commenting procedures and makes final decisions on the extent of the added benefit.

You can find an overview of the results of IQWiG’s benefit assessments in the following English extracts. In addition, the website informedhealth.org published by IQWiG provides easily understandable information on this benefit assessment.