by Pritha

Sep 12, 2017

European Reference Networks: Bring optimal care to people living with rare diseases

European Reference Networks (ERNs), funded by the European Commission bring together 900 healthcare stakeholders from all across the European Union and Norway to work together to increase understanding of the nature history and treatment of a host of rare conditions, from bone disorders to blood diseases, from childhood cancer to immunodeficiencies.

ERNs are intended to improve the health outcomes and equitable access to high quality care for people living with rare diseases and rare cancers.  A key benefit of ERNs for patients will be accelerated diagnosis of rare diseases and a corresponding reduction in the number of undiagnosed or misdiagnosed patients, a key challenge for this community.

EURORDIS established e-PAGs (European Patient Advocacy Groups) for each disease group.  E-PAGs are a key stakeholder that will shape and govern these networks in partnership with specialist physicians, ensuring they deliver truly patient-centric care and treatment.

As ERNs evolve, we look forward to discussions around cross border reimbursement, innovations in IT development and rollout, patient and patient advocate engagement and the role of industry in governance and participation.  In each case, ERNs have the opportunity to create new standards of excellence while drawing on best practice from other parts of the scientific and medical community.  We encourage ERN leaders to save valuable time by drawing freely lessons learned outside of rare diseases as well as the experience and preferences of patients and their advocates.

The upcoming EURORDIS’ Round Table of Companies meeting on September 26, focused on ERNs, will convene stakeholders to examine the role of industry. Industry’s involvement is essential and look forward to hearing more about how to clearly define rules of engagement, especially around transparency in decision making.  Look for our next blog for an update on these discussions.

We whole-heartedly support and applaud the European Commission’s support for ERNs and look forward to still more added value from European collaboration.  We also support EURORDIS is its consistent persistence in establishing the e-PAGS to ensure the patient voice is pivotal to the conversation. Momentum is essential and all stakeholders need to work together to move towards one common goal: Ensuring patients are treated and have access to the best care, regardless of geographic location.  Patients are waiting.

VOZ’s Mark Krueger, MPH, president, and Ellen Coleman, MPH, senior vice president, will be in attendance at the EURORDIS’s Round Table of Companies on September 26.  If you wish to meet with them to discuss more about ERNs and other cross border access issues


Categories: Innovation Policy Rare Disease Stakeholder Forums

About The Author [ Pritha Kuchaculla ]

Pritha Kuchaculla, MPH, is an associate vice president at VOZ. Her work is focused on building the firm’s rare disease portfolio, helping companies integrate the patient voice into all activities from discovery through launch. Her counsel brings value to her clients and organizations and most importantly to the patients and families they serve. An example of this is Pritha’s work in Duchenne muscular dystrophy that helped to drive the execution and submission of the first-ever patient-initiated guidance for a rare disease to the US Food and Drug Administration to accelerate development and review of potential therapies.