Rethinking the Orphan Regulation

Europe risks undercutting its best asset for treating millions of rare cancer sufferers, says Stéphane Lassignardie, VP for Region North & Oncology at AbbVie Europe.
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By AbbVie

19 May 2022

Over 30 million Europeans suffer from one of 8,000 life-threatening or debilitating rare diseases. Thankfully, their situation has improved since the adoption of the EU Orphan Regulation just over 20 years ago. This pioneering legislation provides predictable incentives and protections that enable the best medical minds to work on treatment solutions. There were just eight authorised rare disease medicines before the Orphan Regulation, now there are over 200, and many more are on the way, making it a true EU success story.

The Orphan Regulation is, however, due an update. The Commission will propose revisions at the end of this year, marking an important opportunity for both the industry and patients to address blind spots. But two proposals currently being discussed could prove detrimental to the regulation and consequently limit the future treatment options for rare disease patients.

The first relates to a potential lowering of the prevalence thresholds – that ultimately determine which treatment receives financial incentives to research and develop – below the current five patients out of 10,000. This could mean that incentives, a proven method of stimulating treatment development, would no longer apply to diseases that are just below the current threshold. This could result in, for example, EU patients afflicted with diffuse large B-cell lymphoma, an aggressive cancer which has prevalence of 4.5/10,000, facing the prospect of few or no new treatment options.

“With 25 percent of all cancer cases in Europe being rare cancers, we can see just how important the maintaining and enhancing of the Orphan Regulation can be for so many”

The second revision relates to the removal of market exclusivity – a key incentive of the Orphan Regulation – should pharmaceutical companies not launch medicines in most or all countries. Companies operate in highly regulated markets and cannot alone decide when medicines reach patients – this comes down to national pricing and reimbursement processes. Putting the entire onus on companies without addressing national factors would therefore create a lack of predictable incentives, discouraging investment and ultimately new research.

That said, AbbVie, like all members of the European Federation of Pharmaceutical Industries and Associations (EFPIA), has committed to speeding up patient access by filing for pricing and reimbursement as fast as possible in all EU countries, and within a maximum of two years after market authorisation. This is a substantial step that could go even further towards addressing access and availability inequalities if governments meet industry halfway with their own commitment to speed up the pricing and reimbursement process.

In addition, the Commission could consider smart solutions to stimulate research for orphan drugs, such as transferable exclusivity extensions, which encourage research into promising avenues regardless of their economic viability – crucial for (ultra) rare diseases.

The EU’s Beating Cancer Plan, unveiled early last year, has lofty ambitions. With 25 percent of all cancer cases in Europe being rare cancers, we can see just how important the maintaining and enhancing of the Orphan Regulation can be for so many.


This content was commissioned by AbbVie and produced by Dods Impact

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