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Patients with rare diseases suffer from complicated drug availability

13 November 2017 | Brno

More than a hundred experts from all over the Czech Republic, but also from abroad, visited the Fifth PharmAround Conference 2017. They discussed key topics that significantly affect patients and the entire healthcare system. These included the availability of medicines for rare diseases, public awareness, addressing the high costs associated with biological treatment or the newly drafted law on personal data processing, which will also have an impact on healthcare.


The two-day event, from 9 to 10 November, was organized in Hustopeče u Brna by the PharmAround endowment fund in cooperation with the CZECRIN research infrastructure. MUDr. Kateřina Kopečková from the University Hospital in Motol and the representative of the Czech Republic in the Commission for Rare Diseases of the European Medicines Agency focused mainly on the issue of orphan drugs. "Sometimes orphan drugs are criticized for not being robust enough to have a small number of patients. Quality of life data are often lacking because, for example, there are no specific questionnaires for patients with rare diseases. This is despite the fact that innovative medicines benefit the patient in many rare diseases. The creation of expert centers dealing with the treatment of rare diseases can help. Their aim is to help speed up market entry and enable reimbursement from insurance companies, and then collect additional clinical data, " said Kopečková. She specified that in the Czech Republic it takes an average of two years for a registered medicine to reach a patient. According to her, this is very sad and the goal of experts should be to shorten this process. "Patients with rare diseases, among other things, suffer from complicated drug availability. That is why it is important to improve access to information, " added Kopečková.


JUDr. Karel Hlaváček from the law firm Šustek & Co. pointed out that a rare disease is legally classified as any other disease. This means that those who suffer from it are not more entitled to reimbursement from insurance companies. "The law only sets out the conditions under which an innovative medicinal product can be credited with a temporary reimbursement for two years. There is a well-known case, for example, when a drug for cystic fibrosis fought for this reimbursement for up to two years, "Hlaváček described.


MUDr. Tomáš Doležal, Ph.D. from iHETA (Institute for Health Economics) pointed out that insurance companies do not pay more than 16 million crowns a year for orphans. "It simply came to our notice then. The methodology of insurance companies is a tough negotiating factor. The number of these drugs prescribed for permanent reimbursement is declining, which has an impact on patients as well, " said Doležal.


Head of the Department of Clinical Trials of the Masaryk Cancer Institute and founder of the PharmAround Endowment Fund doc. MUDr. Regina Demlova, Ph.D. She said new ways to ensure the availability of new, very expensive medicines for patients were needed: “Expert working groups should look for new approaches to further assess the clinical benefit and effectiveness of medicines to ensure that effective treatment is available to patients. In the Czech Republic, we have recently been increasingly faced with the fact that insurance companies are not willing to contribute to highly innovative medicines. The population is aging, more and more patients with serious diseases, new drugs are becoming more and more expensive, and this is also related to the increase in expenses for such sick patients, "Demlová pointed out.


MUDr. Beata Čečetková from the AIFP (Association of the Innovative Pharmaceutical Industry) pointed out the importance of recruiting patients in specialized facilities and cooperation not only between physicians or sponsors, but all those involved in clinical trials. "The implementation of clinical trials is moving to pharmaceutical development markets, especially to East Asia. Although the Czechia is a small country, the number of clinical trials is relatively high. This brings a number of benefits not only for patients, who have the opportunity to get the latest treatment, but also for the state, which saves considerable resources. However, better cooperation with patients and regional workplaces is necessary in the future, "said Čečetková.


Director of the Institute of Drug Guide PhDr. Ivana Plechatá stated that it is very important to raise awareness of the effects of drugs towards the public. Especially for seniors, medicines are sometimes a complete "maze". "We are trying to increase drug literacy, the Czechs are not doing well. Studies show that seniors receive a large amount of medication and suffer from a whole complex of diseases. At the same time, they are more prone to develop side effects. The vast majority of drugs used by seniors have not been shown to be effective at all in seniors over 65 years of age. For example, there are seniors who have been taking blood pressure lowering drugs for ten years or more, even though they are already taking them in smaller doses or not at all. Then there are the falls and fractures, "said Plechatá.


Deputy Minister of Health for Law and Legislation JUDr. Radek Policar in his lecture spoke about the GDPR (General Regulation on the Protection of Personal Data), which will enter into force on 25 May 2018 and will also affect medical practice. "The current law on personal data protection will be repealed and replaced by this new law. When someone works with personal data, they will have to have a legal reason to do so. The person whose data is involved will come first. These are the key things. However, there is no need to worry that the regulation, which is under the responsibility of the Ministry of the Interior, will bring with it a new wave of paperwork. It will be possible to apply it wherever there is an obligation to keep work data," said Policar.


Media Contact:

Ing. Andrea Křístková, coordinator of the PharmAround endowment fund, tel: +420 608 407 900


CZECRIN is the Czech national research infrastructure supporting academic clinical trials, which connects teaching hospitals, universities and scientific centers in the field of biomedicine. It is the Czech national hub of the European Consortium of Clinical Research Infrastructures (ECRIN-ERIC). Their common goal is to support the development of academic clinical trials and to improve cooperation at the national and international levels. CZECRIN is currently conducting a number of national and international academic clinical trials in many areas, including pediatrics and rare diseases.


The PharmAround Endowment Fund builds on the successful project of the Faculty of Medicine of Masaryk University. Its aim is to increase awareness and knowledge of drug life cycle issues through educational activities, especially in relation to clinical trials, pharmacovigilance or pharmacoeconomic aspects. The endowment fund organizes professional courses, workshops and conferences for doctors, teachers, researchers and students, and supports independent projects in the field of biomedicine. It pays special attention to patient and public education and promotes an open, independent exchange of views and experiences in the field of biomedicine.

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