Orphan drug reimbursement should consider more than cost, says Eurordis
This article was originally published in SRA
The European rare disease association Eurordis has called on drug reimbursement authorities to bear in mind that while orphan medicines may be expensive, there are relatively few of them and they will be taken by very small numbers of patients1.
Eurordis is concerned that as cash-strapped authorities increasingly rely on health technology assessments to guide their decisions on the cost:benefit and reimbursement of new drugs, orphans might stand out as particularly expensive and so be denied coverage.
Individually, of course, it costs more to treat a rare disease than one that affects large numbers of people because of the high cost of orphan drugs. However, this cost is generally offset by the small number of treatments available and the rarity of the disease, the organisation says in a position paper.
Nine years after the implementation of the orphan drug regulation in Europe, 577 drugs had been designated as orphans, but just 57 had been approved for marketing. Data from the US and Europe so far suggest that about 100 new orphan drugs will be approved over the next ten years, equating on average to ten products a year, according to Eurordis. There will, therefore, be “no tidal wave of new orphan drugs flooding the market” as some have claimed, it asserts.
Moreover, the numbers of patients taking orphan drugs will always be low because these diseases concern a limited number of individuals in the general population: in Europe, for a drug to qualify as an orphan, the disease it treats must affect no more than five in 10,000 people.
In any case, the actual numbers of patients taking an orphan medicine are much lower, for a number of reasons. For example, the drug may not be effective in all patients with a particular disease, or there might be only limited access to specialist diagnostic and treatment centres.
Also to be borne in mind is that not treating a rare disease might actually generate more costs than treating it, in the form of frequent hospitalisations, time off work, etc, the Eurordis paper adds.
The organisation wants funding bodies to take all these factors into account when deciding whether to reimburse particular orphan products. “The overall cost of treating complications and frequent hospitalisations, together with the cost to society of the limitations in activity of non-treated patients due to serious, chronic and disabling diseases, should be thrown into the equation in order to have a more realistic view of the burden of orphan drugs in public health budgets,” says the paper’s author, Dr François Faurisson.
References
1. Eurordis Statement, Orphan Drugs: rising to the challenge to ensure a better future for 30 million patients in Europe, October 2009, www.eurordis.org/IMG/pdf/Statement_Future_of_Orphan_Drugs_14_October_09.pdf