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Orphan drugs: amendment needed

 Editorial  The European orphan drug regulation needs to be amended, to ensure that it better serves patients' interests, and that the public resources invested benefit public health rather than private interests.

In 2000, the European Union adopted a regulation to encourage pharmaceutical companies to bring to the market orphan drugs, i.e. drugs to treat rare diseases (see two examples in the May 2021 issue of Prescrire International: “Gilteritinib and acute leukaemia”, and “Ciclosporin eye drops in severe vernal keratoconjunctivitis”) (1). By 2006, the limitations of this regulation, and its abuse by some pharmaceutical companies, were already evident (2). A number of evaluations published in 2020 have confirmed these shortcomings (3).

The European regulation on orphan drugs was intended to encourage pharmaceutical companies to develop drugs for situations which they would otherwise have considered insufficiently profitable, and thereby give more patients access to treatment. But nothing in this regulation prevents pharmaceutical companies from setting the price of orphan drugs so high that they become inaccessible for many patients, while still bringing in huge profits for drug companies.

Back in 2006, the case of imatinib (Glivec°) showed that a drug could generate considerable sales revenue (almost $6 billion in total) by successively stacking up new indications, each benefiting from orphan drug status (2). By 2019, total revenues from Glivec° had exceeded €43 billion. And 10 other orphan drugs had each generated total revenues of over €10 billion (3).

In the face of growing criticism and the fact that many patients in Europe do not have access to orphan drugs, the European Commission conducted an audit of this policy (4). It welcomed the fact that 142 orphan drugs had been brought to the market since 2000, and that over 6 million patients had benefited from earlier access to these treatments as a result of the regulation. But it also acknowledged numerous limitations: only about 20 of the 131 orphan drugs still on the market would not have been marketed without this regulation; access to orphan drugs is greater in Europe’s wealthiest countries; too many pharmaceutical companies focus on specific cancers and ignore the rarest diseases; and some companies take advantage of the regulation, mainly by stacking up a series of extremely narrow indications for the same drug. The Commission is therefore proposing the revision of certain rules in order to limit abuse and refocus the policy on neglected rare diseases.

Let’s hope that the Commission will not delay in decisively amending the European orphan drug regulation, to ensure that it better serves patients’ interests and that the public resources invested benefit public health rather than private interests.

©Prescrire 1 May 2021

1- Prescrire Rédaction “Médicaments orphelins. Le règlement européen enfin adopté” Rev Prescrire 2000; 20 (206): 382-383.
2- Prescrire Rédaction “Médicaments pour des maladies rares: bilan contrasté en Europe” Rev Prescrire 2006; 26 (277): 780-787.
3- Marselis D and Hordijk L “From blockbuster to “nichebuster”: how a flawed legislation helped create a new profit model for the drug industry” BMJ 2020; 370: m2983: 5 pages.
4- European Commission “Joint evaluation of Regulation (EC) No 1901/2006 of the European Parliament […] on orphan medicinal products” 11 August 2020: 109 pages

Source: "Orphan drugs: amendment needed" Prescrire International 2021; 30 (226): 115. Free

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For more information:

"Gilteritinib - Xospata°. Acute
leukaemia with a FLT3 gene
mutation: an additional option
for relapsed or refractory
Prescrire Int 2021;
30 (226): 117.
Pdf, subscribers only

"Ciclosporin eye drops
(Verkazia°) in severe
vernal keratoconjunctivitis"
(Mai 2021)

See also:

"Orphan drugs: affordability
is at risk due to abuse of
orphan drug incentives"
Advancing healthcare policy
(April 2019)

"Drugs to treat rare
diseases: abuses spell
risks for patients
(January 2018)

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