| 27 February 2017

Breaking the access deadlock to leave no one behind!


To mark Rare Disease Day 2017, Rare Disease Europe EURORDIS hosted a Multi-Stakeholder Symposium to initiate an unprecedented cooperative process to ensure patients’ access to rare disease medicines. This year’s event was held on the 22nd and 23rd of February 2017 in Brussels and was the second year of the important event, which brought together 400 participants from patient groups, industry, policy as well as science and medicine.

The opening session focused on Improving Patients’ Access to Rare Disease Therapies and provided a unique opportunity for patient advocates from four different countries and disease areas to question six industry representatives and market analysts on how they believe stakeholders can better engage to reduce costs.

As a questioner Ms Avril Daly, CEO of Retina International asked how the emergence of European Reference Networks (ERNs) might result in the reduction of costs for emerging therapies for rare diseases. Ms Daly highlighted the fact that ERNs, which will launch on March 1st, have the potential to develop patient registers that include biological and clinical data that can drive the design of advanced clinical trials and accelerate patients’ recruitment. She emphasised the particular opportunity to  register all patients consulting a medical expert in a centre of expertise and having a minimum common datasets as well as standard operating procedures, full interoperability between national centres of expertise as well as the opportunity to develop clinical endpoints and biomarkers, as their validation at the international level. This, along with the rapidly growing possibilities of bioinformatics, will drive major changes on how future clinical trials will be conducted faster and, surely this could only lead to a significant cost reduction in the development of treatment?

It was agreed that the ERN development would lead to much better integration of the process and this would have benefit in improving the research enviornment and quickening the pace of innovation. The US example of Cystic Fibrosis which utilised a well networked system in collaboration with patient groups was cited as a good example of how treatments can be expedited. On the question of how this would make therapies cheaper, it was seen as a move in the right direction. However the issue of early engagement with patients and development of real world evidence were seen as critically important in reducing risk and cost.

The issues that exist in market access between the United States and Europe were also addressed. There were concerns expressed about a centralised system of drug approval through the European Medicines Agency that is ‘derailed’ by the need for countries to perform a Health Technology Assessment  (HTA )prior to reimbursement in each member state. The possibility of a European HTA system was addressed and is being explored.

Retina International was concerned that the issue of regulation with respect to advanced therapies is often left behind in such discussions. This is of particular interest to our community and  must be addressed internationally. With almost 50% of the audience coming from outside of Europe the importance of working together on critical issues such as market access as a global community was clearly endorsed.

After the two days of intense discussion it was agreed that there is an appetite between patient advocates, payers, health technology assessment authorities, academics, and medical professionals to work in an open environment to find ways to improve patients’ access to rare disease medicines. It was well presented that even though ever-advancing science and technology now create rapidly growing opportunities to develop more innovative medicines, the rare disease community is still facing problems accessing these medicines because of their affordability.

The system must change to ensure that medicines are immediately accessible to patients, affordable and sustainable for national healthcare systems. The system should create a predictable and sustainable environment for companies and investors.

Yann Le Cam, Chief Executive Officer, EURORDIS, commented, “We will not take no for an answer. EURORDIS is calling for radical change. This change will not be easy but is urgently needed to translate innovation into health benefits for patients. Improving access can only be achieved by establishing a collaborative multi-stakeholder structured approach. Our goal is to leave no one behind”

He continued, “Collectively, we should aim for the goal of a three to fivefold increase in the number of rare disease medicines approved by the European Medicines Agency (EMA) per year by 2025 and those medicines available at one third to one fifth of the price.

A one text paper on the issue will now be developed by drafters and contributors accross all stakeholder groups. Retina International will keep its members fully updated on progress. 

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