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The first Polish workshop on rare diseases, addressing medical, legal, ethical, and economic aspects of rare disease health care, was held in Warsaw on Friday, 16 December 2005. Organized by EU project (QLG1-CT-2002-90358) – the CoE PERFECT workshop was affiliated with the Children’s Memorial Health Institute (CMHI), the largest paediatric research centre and hospital for children with chronic, severe and rare diseases in Poland.
Some 200 participants took part, including representatives from government, public insurance and pharmaceutical companies, paediatric hospital directors, public health specialists, and paediatricians involved mostly in metabolic medicine and clinical genetics.
Invited lecturers, divided into panels focusing on medical, legal & ethical, and economic issues, spoke on the issue of providing appropriate health service for children with rare disorders - achievements, difficulties, and future improvements in Poland and Europe. During the medical panel, Ségolène Aymé (Orphanet, France), in her lecture on Rare disease action plans in Europe, presented various research programmes and projects in Europe, such as Orphanet and the French National Plan for Rare Diseases. She proposed practical ways in which Poland could be included in the European cooperation and network of research on rare disorders, such as joining Orphanet, which Poland has done. Other lectures were on The Polish registry of congenital malformation as a tool for rare disease identification by Anna Latos-Bielenska, Eurocat Poland), and the Polish experience in enzymatic replacement therapy (Anna Tylki-Szymanska, CMHI). For the ethical/legal panel, Jordi Llinares Garcia (EMEA) presented a lecture on Orphan medical products – registration procedure, safety issues. Marian Filar (Torun, Poland) stated in his lecture on Law-abiding medical personnel: an issue of limited services, negligence that doctors are almost solely responsible for the appropriate management delivered to patients with rare disorders. There were also lectures on Medical products for paediatric use - new EU regulation (Marek Migdal, CMHI); Which industry-sponsored paediatric research programs can be expected to be triggered by the EU paediatric regulation? (Klaus Rose, Hoffman-La Roche, Switzerland; and Ethical conduct of clinical research involving children (Bogumila Milewska-Bobula, CMHI).
The economics panel included lectures on Cost, benefits, and effectiveness of rare disease diagnostic programmes and treatment – Polish experiences (Zbigniew Kulaga, CMHI); Priority setting in health care policy: how should rare diseases be treated with limited sources? (Krzysztof Landa, Krakow, Poland); and Publicly founded health care provisions – hard decisions facing the politicians (Jacek Ruszkowski, Warsaw, Poland). The last session of the conference was an interactive presentation entitled, Can we afford rare disease treatment? The session was chaired by Maciej Piróg, Director of The Children’s Memorial Health Institute. Discussion was led by Richard Grenda (CMHI). Two presenters, Marek Migda and Zbigniew Kulaga, discussed health care financing issues, with special focus on rare diseases.
The audience had the opportunity to vote on questions posed by Professor Grenda after an exchange of data and views by presenters. Concerning the financing of health care in Poland, the majority of the audience voted for increased funding (some even suggested the EU average as the necessary level of health care financing in Poland).
Next, the definition of rare diseases was discussed: different definitions in different parts of the world add confusion to the subject of rare diseases; furthermore, some illnesses, which are not regarded as rare diseases (e.g., malaria in Europe) do fulfill criteria for rare disease definition. The audience voted for an ultra-rare disease definition (such as is used in the UK) as more precise and better reflecting the character of rare diseases.
Subsequently, the issue of accepting treatments not conforming with Evidence-Based Medicine was discussed. Lack of data (and evidence) on efficacy and safety from sufficiently big populations and observations is a major limitation. The audience, however, voted for continuing treatments with pharmaceutical products which were accepted after studies with a limited number of patients.
Almost half of the audience voted for some kind of economic assessment of interventions in the area of rare diseases, which, in turn, is in line with the view that health outcomes (regardless of frequency of disease) be valued equally. The audience expressed the view that rare disease treatment should be financed via the state budget and that resources should be increased in Poland, yet it was agreed (52% of votes) that it is not ethical to allocate huge resources (especially with a limited budget) to interventions for which positive results have not yet been established (29% voted this as ethical; 19% answered “I don’t know”).
Overall, the workshop was considered an important and successful contribution to improving the organisation of rare disorder management in Poland.
For more information, contact CoE PERFECT Coordinator, Professor Ewa Pronicka
