The summer is over and European health policymakers are back to face a packed EU policy and legislative agenda. First up: the long-awaited review of the Orphan Medicinal Products (‘Orphan’) Regulation that aims to foster the development and availability of medicines for patients with rare diseases. The anticipated reopening of the Orphan Regulation is part of a longer term effort by EU Member States to reform the Intellectual Property incentives framework for the pharmaceutical sector.
The stakes are high for all stakeholders involved, as key components of the existing legislative framework incentivizing research and development of orphan medicines are under scrutiny and up for discussion. The post-COVID-19 political environment increased public and political awareness of health and, as countries try to recover from economic hardship and expensive recovery plans, issues of cost and pricing are driving the debate.
The current Orphan Regulation was adopted over twenty years ago; obviously, much has changed since then. Rapid developments in digital technologies have empowered European rare disease communities to become better connected and more integrated. The question being asked in the review is: How many more and better medicines has the Orphan Regulation helped deliver to these communities to help them treat their rare diseases?
Agreeing on the success of the Orphan Regulation has turned out to be difficult and controversial, for a number of reasons that are set out in the EU Orphan Regulation evaluation study that was released by the European Commission last month. It all comes down to two currently unanswered questions about ‘what success looks like’. Firstly: how are we measuring success? And secondly: what is the price we are willing to pay for this success? Both questions cannot be addressed by the Orphan Regulation alone.
Regarding the first question, the evaluation study flagged a lack of accessible and comparable data to measure the impact of incentives. Our current understanding relies heavily on the information processed by the European Medicines Agency (EMA) which, falling at the front end of the market access process, is more about ‘intent’ (e.g. what disease does it aim to treat) and not so much about ‘impact’ (e.g. net effect of additional costs and cost-savings). Here we get into the interplay of market authorisation and reimbursement processes, which goes beyond the remit of the Orphan Regulation. However, questions about measuring success could perhaps be addressed by building in obligations for specific effectiveness and efficiency data tracking and sharing. Linking this to the objectives for the desired European Health Data Space, intended to foster the exchange of different kinds of health data in Europe, could enable the development of European-wide Key Performance Indicators for the effectiveness of orphan incentives
That brings us to the second question: who should pay what price for incentivizing innovation? This, of course, is a well-known ‘hot-potato’, but considering medical science is becoming more and more specialized, the evaluation study seems to point towards a need for greater flexibility to allow for more tailored incentive models. However, more flexibility on the incentives side would need to be matched by more flexible pricing models to better align the upstream R&D process with the downstream patient access process. The right framework for incentives to drive research to areas of unmet medical need cannot be obtained by EU legislation alone, as it needs to take into account the interrelation between EU legislation and national competences related to value and affordability. If this is not fully understood by all stakeholders, the debate on the Orphan Regulation will be distorted as EU competences in health are mixed-up and confused.
The current Orphan Regulation was written at a time when nobody could have foreseen how medical science and digital technologies would transform health. The need for future-proofing the Regulation is clear, but it will be important for policymakers to not only zoom in on the issue of incentives, but at the same time zoom out to reach a more common understanding of what metrics to use for defining value and tracking outcomes for the development and bringing to market of orphan medicines in Europe.