Orphan medicinal products: onwards and upwards

The Orphan Medicinal Products regulation delivered impressive results for rare disease patients, but the pending revision can move it up a gear, argues Stelios Kympouropoulos
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By Stelios Kympouropoulos

Stelios Kympouropoulos (EL, EPP) is a member of Parliament’s EMPL and PETI Committees, the SANT Subcommittee and substitute in the ENVI Committee.

28 Feb 2022

The Orphan Medicinal Products (OMPs) Regulation and the Paediatric Regulation, first adopted in 2001 and 2006 respectively, partly overlap. This is because many diseases that affect only children are rare, and rare diseases often affect children. Both regulations have introduced obligations, incentives and rewards to address the market failure to provide medicinal products for these patients. Since their introduction, these regulations have fostered research in the EU to the benefit of rare disease patients. 

In fact, during the 20 years of the regulation, the number of OMPs has increased from eight to 182, an outstanding achievement. However, this is not yet enough, as there are more than 6000 rare diseases and 95 per cent of these still do not have an authorised treatment option.

“During the 20 years of the regulation, the number of Orphan Medicinal Products has increased from eight to 182”

At the same time, regrettably, for that remaining 5 per cent of diseases that do have a treatment, the patient journey is far from simple. Moreover, the Paediatric Regulation has been largely beneficial to children, but not sufficiently effective in certain therapeutic areas, particularly paediatric oncology. Therefore, the revision of these two pieces of legislations could be beneficial for addressing these deficiencies. 

Personally, I support that any such a revision should first aim to define the unknowns and gaps in patients’ needs. We need to address the insufficiencies in the development of medicines for the 95 per cent of rare diseases that still have no available treatment option. The revision should also consider the fact that patients are unevenly distributed across this 95 per cent, as only 400 out of the 6000 rare diseases account for 98 per cent of rare disease patients.

This means that if the 182 OMPs are doubled in the coming years (following the advanced development race of the previous years and through to possible new incentives that may be introduced), then 89 per cent of the patients will have the opportunity to have access to a treatment. 

Nevertheless, even if a treatment is available, not all OMPs are equally accessible to patients in all EU countries. The revision of the regulations should thus aim to ensure availability and timely access to all orphan and paediatric medicines across all Member States.

Although the access is primarily a Member State competence - as are State rare diseases policies, plans and strategies vary greatly across the EU - the authorisation of orphan and paediatric medicines is fully harmonised at EU level. Nevertheless, national pricing and reimbursement decisions can determine whether a patient can actually receive a medicine.

Consequently, I consider it essential that the revision aims at strengthening the cooperation between the stakeholders, the companies and the Member States, to boost funding and research into medicinal products for rare diseases.

“We need to address the insufficiencies in the development of medicines for the 95 per cent of rare diseases that still have no available treatment option”

We should support a common European strategy that encourages Member States to protect the commercial interest of the companies’ investing in the production of orphan drugs, thus making the OMPs more easily accessible in the market.Companies that might lose commercial interest in a product should be encouraged to offer it for transfer to another company rather than withdrawing it, thereby guaranteeing market continuity. 

We need to understand that investment in rare disease patients is an investment in society. Rare diseases may affect 30 million EU citizens, a number that is significant. In addition, even though some rare diseases are incurable, their early treatment can lead to an excellent quality of life for the patients.

This is why I support new-born screenings being available in all Member States. All of the above would contribute to improving the social inclusion of these patients (in education, employment opportunities, etc.) and secure them a more independent future. 

Finally, the revision of the regulations should aim to ensure that the legislation is fit to make the most of technological and scientific advances - including advanced therapies - and innovative clinical trials designs. At present, not all EU countries have the resources, the expertise or the specialised centres to diagnose and treat all rare diseases.

The EU should take the lead in developing better technologies and models to provide faster access to treatments for all patients across borders. Personally, I would be honoured if the current medicinal products I use were made in the EU. I would like for all the rare disease patients in the EU, especially children, to be able to feel proud for living in a society that supports their access to a high level of human health protection. We need to make sure that rare diseases patients are treated equally to any other patient. 

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