Challenges and opportunities for the development of OMPs in Europe

Maria Vittoria Di Sangro

Orphan Medicinal Products (OMPs) are used for the diagnosis, prevention and treatment of rare diseases. Although their prevalence is less than 5 cases per 10,000 inhabitants, there are about 5,000 different types of rare diseases and over 30 million people affected by these diseases in Europe (between 2 and 3 million in Italy alone). These diseases are highly complex, partially due to the limited availability of clinical and scientific knowledge in this field. Furthermore, due to the low prevalence of rare diseases, the medicinal products used for their treatment are not commercially attractive under normal market conditions.
However, these products can make the difference between life and death for patients and, therefore, should be governed by an ethical rather than a political imperative.

OMPs have been subject to specific legislative initiatives, to offset the risk of development failure but also the small size of their target population. Europe set up a unified policy for orphan drugs for all Member States, by adopting the EC Regulation 141/2000. Thanks to this Regulation, pharmaceutical companies that invest in the research and development of these drugs benefit from tax reductions on the evaluation procedure, scientific and protocol assistance, and enjoy a ten-year period of market exclusivity following the marketing authorization.

This regulatory framework has definitely boosted investment in the development of orphan drugs by pharmaceutical companies. According to EMA data, the orphan drug status designations between 2000 and 2018 increased by 15% each year, reaching 2,233 at the end of 2019. Since the adoption of the European Regulation, 23 Member States have adopted a national plan for rare diseases.

The Regulation was truly a game changer and significantly transformed the lives of patients and their families, improving outcomes in terms of health and quality of life, and proving to be crucial for developing treatment options for previously untreatable conditions. The introduction of the Regulation has also led to further developments in medical knowledge and promoted an ecosystem favorable to research, innovation and investment across Europe.

In the European Pharmaceutical Strategy, presented on 25 November 2020, an Inception Impact Assessment was planned in order to investigate the possible impact of some changes to the European regulations concerning medicines for rare diseases and pediatrics. In recent years, there have been several reasons that have led to an increase in the control regarding incentives. These involve the commercial success of some treatments for rare diseases raising questions about the appropriateness of use of incentives in the sector and whether these incentives should be reduced, health budgets being under increasing pressure and national health services seeing market exclusivity and incentives as one of the reasons for high health expenditure, and scientific progress leading to the personalization of medicine, which is perceived as increasingly expensive.

Undoubtedly, the current regulatory framework has some shortcomings. Issues arise from the insufficient stimulus provided in the context of unmet needs, partly relating to the fact that the regulations have not proved flexible enough to support scientific developments, and that some procedures have proved inefficient. However, the main problem to be solved is the differences in terms of treatment access among Member States. Unfortunately, it is not possible to solve these problems at a supranational level as they depend on national policies and lie outside the scope of the European Regulation.

Despite the hypothesis of a revision at European level, it is therefore important that each Member State activates an extensive debate in order to comply with national specificities. The discussion needs to take into account the contribution that patients, experts and the industry can make.

The proposed revision involves various risks. There is a risk of regressing towards a situation of no protection, due to economic considerations that apply to the short term, but which would not be any more valid in the medium and long term, in terms of additional expenditure for national health services.

However, the re-opening of the legislation review is a process that lasts several years and involves a climate of uncertainty to the detriment of patients and investment decisions for the development of new treatments. Moreover, there is a concrete risk that a review could lead to a weakening of the regulatory framework, with negative effects on the level of innovation in treatments for rare diseases, without actually creating added value in access to care.

In order to be ready for the potential reopening of legislation procedures, various European players – under the leadership of EURORDIS and EUCOPE – have set up the European Expert Group on Orphan Drug Incentives (OD Expert Group). This multidisciplinary group aims to become a source of innovative ideas and potential solutions that could be an input to the evaluation of the regulation for OMPs. The group’s approach, which combines the different stakeholders’ perspectives, allows for a complete overview of how the development of orphan drugs takes place in Europe and is suited to the intent of understanding how policy-makers can assist in this process.

The challenges for rare diseases and the development of orphan drugs are varied and complex, hence international cooperation is essential to continue in the right direction and make treatment options available to a growing number of patients, given that these therapies can be life changing.


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