Time for civil society to take action to influence EU regulations on medicines for children and rare diseases – EURACTIV.com

The revision of the EU legislation on medicines for rare diseases and for children is one of the key actions of the Pharmaceutical Strategy for Europe. Civil society now has a unique window of opportunity to propose concrete recommendations and improve the Orphan and Paediatric Regulations, writes Ingrid Stenstadvold Ross.

Ingrid Stenstadvold Ross, an expert in health policy and strategy and secretary-general of the Norwegian Cancer Society, is the Chair of European Fair Pricing Network (EFPN).

After twenty years of being locked, the Paediatric (1901/2006) and Orphan Regulations (141/2000) are being opened. This is the golden opportunity that civil society organizations have been waiting for. Let me explain why.

EU rules to incentivize the development of medicines for children and for people suffering from rare diseases have been in place for decades. Several shortcomings need to be addressed to ensure timely access to affordable, effective, life-saving medicines addressing the specific needs of these two populations.

95% of rare diseases still do not have any approved treatment options

Just so you understand the implication of 95% of patients with a rare disease not having any treatment options. Most people who are diagnosed with cancer, face very hard times. If you get diagnosed with a rare cancer, one which has less than 6 new cases per 100,000 persons per year, you are worse off than others.

First of all, it takes a lot more time to get the right diagnosis. A rare cancer can be difficult to diagnose. The longer the time for an accurate diagnosis, the longer one has to wait for adequate treatment. And due to its rare nature, there is generally less known about the biology of a rare cancer.

We can’t start to imagine what it is like to first hear, after a long period of time, that you have a rare cancer, and that 95% of rare diseases have no approved treatment. We can and must do more for these patients, and we have to step up and improve these Regulations.

Academia can play an important role, if we let them

The system currently seems to be designed for the industry, the small and medium-sized enterprises and the consultants. I strongly believe that academia, whose research is often at the source of novel methodologies and early discoveries that result in life-saving treatments, can and should play a bigger role.

Rare diseases by definition affect a small number of patients and are, therefore, perceived as an unattractive commercial market. Why not give non-commercial parties, such as academia and NGOs a bigger role in drug development?

Like for example an academic pharma setting. Inventions and discoveries come pouring out of universities and in non-commercial settings. To encourage the development of treatments for rare diseases, the European Commission should generously support academia developing orphan medicines.

Incentives – what is worth incentivizing

How I understand it, the goal of this regulation is to ensure a sustainable ecosystem for Orphan Medical Products that reach all EU patients. This means that it should be attractive to research and develop these medical products. Hence, professional assistance and incentives for academic and non-commercial researchers must be delivered.

It also means that new innovations with a benefit and added-value actually reach patients. We, as a team of cancer societies under the umbrella of the European Fair Pricing Network (EFPN), set an even higher ambition. We want all European patients to be able to access them. For that, affordability of medicines and a decent return on investment is needed.

Nothing is free, but innovations shouldn’t cost a fortune

This brings me to my last, but maybe my most important point. It is not a solution but an approach and a way of thinking.

Incentives in the current system are not always proportionate. For example, they are not proportionate to the costs required to put a specific medicine on the market or the return-on-investment they provide. There is also no (annual) report on drug- specific revenues. There is too much opacity. In a nutshell, the current system can use some fair doses of transparency.

Incentives should be higher where the unmet need is greater. Incentives should be valued better when they reach all patients. Not only available, but actually reaching them. For that, transparency is needed. And let’s be frank, we are talking about rare diseases – less than 30.000 new cases a year in Europe. Can we really not deliver for them?

Join our policy dialogue!

I would be delighted to hear your opinion on which gaps and opportunities your organizations identify in the Orphan and Pediatric Regulations, and make use of our Joint Policy Recommendations co-created with other civil society organisations.

Join our policy dialogue to share your views and discuss possible solutions.


Agenda: https://bit.ly/3szm31Q

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