BIO Deutschland Criticises the Retroactive Benefit Assessment for Orphan Drugs

BIO Deutschland has criticised that medication for rare diseases (so-called orphan drugs) will only be exempt from benefit assessment up to a turnover of €50 million. The criticism stems from the fact that all of these drugs have proved their additional benefits in accordance with the European regulations on orphan drugs. A law on the New Regulation of the Medication Market in the Statutory Health Insurance was passed by the Bundestag on 11 November.

The motion for an amendment from the coalition parties was only discussed conclusively in the Bundestag’s Committee on Health on 8 November. According to this, orphan drugs are to be fundamentally exempt from the planned renewed national evaluation of their benefit. However, they must still prove this benefit at a later stage if a “statutory health insurance turnover” of 50 million euro at pharmacy retail prices (equivalent to a net turnover of the company at the manufacturer’s sales prices of around €33 million, that is, not counting pharmacies’ and wholesale margins, VAT and manufacturer’s tax) is surpassed and if the Joint Federal Committee requests that the pharmaceutical entrepreneur do so.

“The benefit does not depend on whether a drug for rare diseases has an annual turnover of €50 million or not,” said Robert Schupp, Chairman of BIO Deutschland’s Working Group on Health Policy. He pointed out that it was not apparent how anything should be changed simply by the fact that a drug was now sold more frequently and therefore achieved a higher turnover. “The benefit depends on the proven efficacy of the drug and not on its turnover,” he added.

Besides, drugs for rare diseases are granted orphan drug status only if they can prove that they either have a significant benefit in comparison with available alternative therapies, or that there is not yet a satisfactory method for the diagnosis, prevention or treatment of the disease in question in accordance with European Regulation EC No. 141/2000. This is checked by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Association (EMA), that is, a committee made up of specialists from all member states, representatives of patients, and representatives of medical science. Furthermore, orphan drugs are subject to the same licensing process as all other medications. They must be centrally licensed by the EMA, which checks the required quality, harmlessness and efficacy of the drug, using the presented and requested documents and studies.

“Orphan drug status is subject to strict inspections – there is no room for abuse here,” Peter Heinrich, Chairman of the Board of BIO Deutschland, made clear. He emphasised that the planned retroactive benefit assessment in cases of an annual turnover of more than €50 million would slow down innovations, as this limit does not only represent a meaningless bureaucratic obstacle, but also causes harm – the incentives to conduct research will be reduced. “The patients concerned will be the ones to suffer. They have a rare disease, rely on innovative medications, and have previously benefited from the promotion of research in this field,” Schupp added.

The authorities in Germany (the Joint Federal Committee and the Institute for Quality and Efficiency in Health Care (IQWIG) have three months’ time following the new draft law to produce a benefit assessment based on exactly the same data that was already available to the COMP and EMA. A decision that differs in terms of content from the findings of the COMP and EMA is therefore not possible. Moreover, the Joint Federal Committee is legally bound to the results of these inspections by the COMP and EMA – different evaluations are thus not possible in legal terms.

BIO Deutschland therefore urges that orphan drugs that have already proved their additional benefit in accordance with European regulations be exempt from the planned benefit assessment without restrictions and irrespective of turnover.

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