No Known Benefit for Patients with Hemophilia A from Treatment with Simoctocog Alfa

There is no viable data supporting the use of simoctocog alfa (Nuwiq) for hemophilia A treatment even though it has been approved since July 2014, according to a dossier examined by the German Institute for Quality and Efficiency in Health Care (IQWiG).

IQWiG evaluated whether this drug can offer benefit over the appropriate comparator therapy. The added benefits could not be obtained from the dossier, though, because the manufacturer did not submit any suitable data, the researchers said in a press release.

The manufacturer presented 2 randomized controlled trials with crossover design for the direct comparison with octocog alfa, which the researchers combed through. The primary endpoint was pharmacokinetics such as ingestion and excretion of the drug. Participants in these 2 studies received a single dose of simoctocog alfa or octocog alfa and, after a “wash out phase,” the participants received a single dose of the opposite drug they originally received. But, the IQWiG researchers don’t believe a single dose is sufficient to assess the added benefit, as hemophilia patients require lifelong treatment to prevent bleeding. The manufacturer did not report an advantage of its drug after publishing the results of these 2 studies. Additionally, the European Medicines Agency guidelines have since recommended a minimum duration of 6 months for similar hemophilia prophylaxis trials.

The pharmaceutical company did report an added benefit of simoctocog alfa based on single arm studies of the 2 drugs. Octocog alfa was only referred to in the Summaries of Product Characteristics in publicly available documents of the regulatory authorities. The pharmaceutical company did not conduct any other searches for studies, which is required for the dossier. IQWiG researchers had already found one other octocog alfa study. But, again, the data was unsuitable to determine if there was any added benefit, as it was unclear if the manufacturer had considered all of the available octocog alfa studies.

The assessment is part of an early benefit assessment mandated by the Act on the Reform of the Market for Medicinal Products, which is overseen by the German Federal Joint Committee (G BA). The G BA comprises the 4 leading umbrella organizations of the German health care system and patient representatives, and is a branch of the Federal Ministry of Health. When the dossier assessment is published, the G BA then conducts a commenting procedure, and finally makes a decision on the extent of the added benefit.