Medicine: Dispute over “orphan drugs” against rare diseases – health

The construction is original and was intended as a treat for the pharmaceutical industry. Since it is economically risky to develop drugs to treat rare diseases, the incentive has been increased: In 2011, the benefit assessment that is customary for other drugs was no longer applied to these drugs. With the approval of the “orphan drugs” (from English orphan, “the orphan”) at European level, an “additional benefit” compared to the previously available drugs was automatically assumed – without corresponding data or comparative studies having to be submitted. A regular benefit assessment is only necessary when the drug exceeds annual sales of 50 million euros. To this end, the Institute for Quality and Efficiency in Health Care (IQWiG) prepares reports, and the Federal Joint Committee (G-BA) decides on this basis whether there is any benefit at all and, if so, to what extent.

An analysis by IQWiG now shows that the “fictitious” additional benefit of orphan drugs assumed upon market entry is not confirmed in more than half of the cases. For all orphan drugs that have undergone a regular benefit assessment process since 2011 because they have reached the sales threshold, the Cologne Institute evaluated whether the additional benefit actually existed.

A total of 41 assessments were examined for which the classification as orphan drug had been carried out since 2011 – as well as a regular benefit assessment later. These 41 ratings are spread across 20 orphan drug substances, as some drugs have been approved for multiple indications. These included drugs against cancer, metabolic disorders and hereditary diseases such as cystic fibrosis. With 22 evaluations – this corresponds to 54 percent – the regular benefit assessment showed that the additional benefit attested with the approval could not be derived from the previous data.

“This has consequences for the quality of patient care,” says Thomas Kaiser, who heads drug evaluation at IQWiG. “New drugs are given preference in these cases without a database. Patients then place great hope in a new drug for which it becomes clear only years later that there is no evidence that it is superior to an existing treatment option.”

The evaluations by IQWiG also show that the previously granted privilege of additional benefit is often only corrected after years – or not at all: It took an average of three years until the benefit assessment was made due to the turnover threshold of 50 million euros – in some cases it took it nine years. In 66 percent of the cases, the pharmaceutical companies did not provide any new data during this time to support the alleged benefits. And in some cases the predicate of additional benefit was not withdrawn at all because the drug did not achieve sales and the regular benefit assessment was never carried out.

The total number of people affected is high, even if little is known about the individual diseases

In order to end the imbalance, politicians are called upon: “It is time to abolish the privilege of additional benefits for orphan drugs,” says Jürgen Windeler, head of IQWiG. “Orphan drugs should also undergo a regular early benefit assessment versus the ACT when they enter the market.” After all, the 98-page IQWiG analysis showed “a mismanagement of orphan drugs”. It would not be possible that in a good half of the cases the stated additional benefit is not confirmed and, on the other hand, orphan drugs that represent real added value are not recognized as such from the start. “In this way, drugs remain in the supply and are privileged, of which one does not know whether they have an additional benefit,” complains Windeler. “Then we don’t know which ones are good and which ones are bad – and other tried-and-tested drugs that are already on the market are discredited and put on hold by the approval of orphan drugs.”

While the term “rare diseases” sounds like a niche phenomenon, the dimension is substantial. A disease is considered rare if it affects no more than five in 10,000 people. Extrapolated to the almost 450 million inhabitants of the EU that would be up to 225,000 people affected per illness, in Germany up to 40,000. But since there are more than 6,000 different “rare illnesses”, the total number of people affected is high, even if the individual Diseases are not so well known. According to the Ministry of Health, an estimated four million people live with a rare disease in Germany alone, and up to 30 million in the EU.

In view of such dimensions, the mismanagement of orphan drugs not only has a detrimental effect on patient care, but also on drug spending. Orphan drugs for rare types of cancer are major cost drivers for statutory health insurance. The prices may be justified for agents that bring real additional benefit to patients – not for those with no additional benefit. “Carefully differentiating here is therefore overdue for several reasons,” says Windeler.

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