Misuse of cost containment policies threatens patient access to Orphan Medicines

23-Oct-2007

EuropaBio welcomed the 8th EPPOSI Workshop on Partnering for Rare Disease Therapy Development in Copenhagen (Denmark) on 18-19 October. Organised by the European Platform for Patients' Organisations, Science & Industry (EPPOSI), the workshop discussed the application of Health Technology Assessment (HTA) to orphan drugs, prevalence and the forecasts models of "Orphan" medicines - those intended for the treatment of rare diseases. The workshop gathered European representatives of patient groups, academia, industry, regulators and politicians to proactively address the reality of access to orphan medicines.

Today more than 470 products have been designated in the EU as potential treatments for rare conditions, of which more than 30 are already allowed on the European market, but they are not all being made available to patients in Europe.EuropaBio continues to urge authorities to ensure timely and effective access to treatments for rare diseases; this should be achieved by evaluating the broader societal and individual value of orphan drugs.

"Several factors need to be solved simultaneously in order to ensure that rare disease patients have access to treatments. Cooperative dialogue among all interested parties is key to overcome the existing hurdles," said Andrea Rappagliosi, Chairman of EuropaBio's Healthcare Council, speaking from the conference. He continued, "access to treatments remains a governmental policy decision within the broader framework of European and national healthcare priority setting. Health Technology Assessments can and should be used to better inform these decisions, taking into account the medical, social, economical and ethical benefits that these therapies brining to patients and society at large. This is the challenge that governments, regulators, payers, reimbursers, and industry must address to ensure that patients suffering from rare diseases will get the right drug at the right moment."

https://www.bionity.com/en/news/72286/misuse-of-cost-containment-policies-threatens-patient-access-to-orphan-medicines.html