​Romania is one of the last countries in the EU in terms of the share of reimbursement of innovative medicines (below 25%), and the time that passes from European approval until they are available in Romania (on average 899 days) is a challenge for public health. I .

Access to innovative treatment methods “too late in Romania”

“Access to innovative treatments in Romania is extremely delayed, although there have been many updates to the list of innovative drugs since 2014. New treatments, which are also beginning to appear in Romania, require early, correct diagnosis, good monitoring and improved neonatal screening,” said Dorica Dan, President of the National Alliance for Rare Diseases of Romania during the event “Access of Romanian Patients with Rare Diseases diseases and rare types of cancer to innovative treatment methods.”

“As the last years of the pandemic have shown, medical and pharmaceutical innovations are an important factor for a health care system adapted to the challenges of our age. However, to be effective, innovations must reach those who need them in a timely and predictable manner. Despite progress in access to innovative medicines, as also revealed in the WAIT 2021 study (performed by IQVIA for EFPIA), Romania continues to rank low – compared to the rest of the EU Member States – in terms of the share of reimbursement of innovative medicines (below 25%) and duration after European approval during which they are available to Romanian patients (899 days on average). This has a negative impact both from the point of view of health and socio-economic indicators. ARPIM believes that fair, predictable and as soon as possible access (maximum one year after approval in Europe) to innovative medicines and drug policies to stimulate innovation are necessary, and is open to partnerships with patient associations, decision makers and any other stakeholders to determine the best solutions in this regard.” – says Ioana Bianchi, Director of External Relations of the Romanian Association of International Medicines Manufacturers.

“More and more diseases are becoming rare as a result of the increasing ability to decipher the biological mechanisms by which they occur. Genomic medicine and other omic sciences are helping us increasingly better characterize diseases that we knew were rare, but they are also allowing us to classify classic diseases like cancer on the basis of a high molecular profile. This complexity should not scare us, because it actually means better chances for patients. On two conditions: that an accurate diagnosis (or, in some cases, screening and an accurate diagnosis) can be formulated fully and in a timely manner, and that patients are quickly provided with access to the necessary therapy with ever better efficacy. The personalization of screening, diagnosis and treatment must be accompanied by the personalization of access to innovation, precisely in order to maximize the potential,” says Dr. Marius Geanta, president of the Center for Innovation in Medicine.

The first step to gaining access to effective and innovative treatments is neonatal screening, a program aimed at presymptomatic detection through testing soon after birth for treatable but clinically undetectable conditions. Currently, in Romania we have only two rare diseases that are included in the neonatal screening program. At the opposite pole is Italy, where newborn screening is included in the national health care program and checks for 44 rare diseases.

According to the materials of the university conference. Dr. Raluca Ioana Teleanu, head of the pediatric neurology clinic at the Dr. Victor Homoiu Children’s Clinical Hospital in Bucharest, newborn screening “is not intended to diagnose a specific disease, but to identify in the population those subjects who have a higher risk of developing such a disease and allows early appointment of appropriate therapy, prevention of long-term disability or premature death.”

EURORDIS, an organization representing more than 1,000 rare disease patient organizations in 74 countries, recommends newborn screening for more efficient and accurate early diagnosis, especially for diseases that can be intervened and treated or have supportive care. Neonatal screening should also be organized as a system with clearly defined roles and responsibilities, as well as accountability and communication mechanisms, integrated into the national health system of each member state.

A rare type of cancer affects less than 6 in 100,000 people

A rare cancer is defined as a cancer that affects fewer than 6 people per 100,000 per year.

It is estimated that 5.1 million people in Europe are living with rare cancers and that there are more than 200 types of rare cancers grouped into 12 ‘families’. (source: RARECARENet).

In Europe, 650,000 rare cancers occur annually.

There are no patient registries in Romania, but it is estimated that 25,000 people are diagnosed with rare cancers each year, and one million patients have rare diseases.

The webinar “Access of Romanian patients with rare diseases and rare cancers to innovative treatments” is part of a series of six webinars that will take place until the end of the year, united under the concept of “Sharing the experience 2022” and had as guests:

• Dorica Dan, President of the National Alliance of Rare Diseases from Romania

• Gulchyn Humus, head of the EURORDIS research and policy project

• Dr. Ioana Bianchi, ARPIM Director of External Relations

• Dr. Raluka Ioana Teleanu, Children’s Clinical Hospital “Dr. Viktor Gomoyu”

• Dr. Marius Geanta, Center for Innovation in Medicine.

“Share the experience 2022” is a project organized by the National Alliance of Rare Diseases of Romania and the Romanian Association of Rare Cancers.