DEBRA Bulgaria and the Bulgarian Helsinki Committee are launching a “Save the butterfly children!” campaign. It advocates for the inclusion of epidermolysis bullosa in the list of disorders whose home treatment is covered by NHIF. People with this rare disease have extremely fragile and delicate skin. The usual daily activities lead to blisters and sores such as from severe burns. Pain can be reduced if special non-adhesive bandages and antibiotic creams are used. However, their monthly cost is unaffordable for the Bulgarian patients.
Support the fair demands of patients with epidermolysis bullosa by signing their online petition and sending a postcard to the health authorities. More information can be found on the campaign website.
Issue 12 of “Rare Diseases & Orphan Drugs” is already published on our website. The main topic of this edition is the outcome of the last month’s National Conference for Rare Diseases. You can check the feedback from Elena Eneva (DEBRA Bulgaria), Assoc. Prof. Margarita Genova (National Hematology Hospital/ Bulgarian Hematology Society) and Nadezhda Tsekulova (Bulgarian National Radio). In the editorial we would remind you about the requirements of the Cross-Border Healthcare Directive, as well as the so-called “Elchinov” case. Furthermore, you can get informed about osteogenesis imperfecta, access to orphan drugs in Bulgaria and health technology assessment. Have a nice reading!
The European Union Committee of Experts of Rare Diseases (EUCERD) adopted a recommendation in the area of facilitating the exchange of scientific information on orphan medicinal products. It involves the creation of an Information Flow between individual Member States and the EU that would bridge existing knowledge gaps, especially at the time of marketing authorisation. The recommendation should ultimately accelerate access to approved orphan medicinal products, while encouraging pricing and reimbursement decisions based on the value of the orphan medicinal products and promoting good medical practices throughout the EU. To read the full document, please, visit EUCERD’s website.
Ukrainian Institute of Clinical Genetics of Kharkiv National Medical University is organising a scientific and practical symposium on rare hereditary diseases. It will focus on all range of rare diseases problems, as main topic will be the integration of all achievements of modern medicine in prevention and treatment of rare hereditary diseases. The event will be held on 20-21 November 2012 in Kharhiv, Ukraine. For more information, please visit the official website of the event.
On October 8 2012 (Monday) at 11 am DEBRA Bulgaria and the Bulgarian Helsinki Committee are organising a press conference on the problems of the epidermolysis bullosa patients in Bulgaria. Epidermolysis bullosa is а rare disease that is characterised by abnormal fragility of the skin and mucous membranes. The disease starts at or soon after birth with blisters and erosions. Blisters usually result from minor mechanical trauma but may appear spontaneously.
The University of Leuven (Belgium) and the VU University Medical Centre Amsterdam (The Netherlands) are organizing on 19 November 2012 the International Conference “Newborn Screening: Challenges for Europe” in Brussels. In Europe, approximately 5 million newborns are screened yearly. European policies around newborn screening are challenges by various policy controversies, including questions to what degree newborn screening panels should be expanded, to what degree informed consent of parents should be obtained, how carrier status results should managed, and how storage and research use should be dealt with. Conference participation is free, but registration is required before 12 November through here. Further information is available on the event’s official brochure.
The European Medicines Agency (EMA) has approved NovoThirteen (catridecacog) for use within the EU for the long term prophylactic treatment of bleeding in patients with congenital factor XIII A-subunit deficiency, a form of the rare disease hemophilia. For further information, please visit EMA website.
Department “Medical Rehabilitation and Ergotherapy” at the Faculty of Medicine of the Medical University – Sofia, together with its partners is organising Rehabilitation Academy, entitled “Rehabilitation in Major Diseases of the Nervous and the Musculoskeletal Systems”. The event will take place at Hotel “Rodina” in Sofia on 15-17 November. Covered topics include advanced methods of rehabilitation, rights of patients in rehabilitation clinics, multidisciplinary rehabilitation team, training and specialisation in the field of rehabilitation. For more information, please see the official brochure of the event.
The European Commission today proposed two Regulations, which are going to ensure better quality and safety of the medical devices and in vitro diagnostic medical devices, as well as adaptation to the scientific and technological progress in this field. The medical device sector is highly innovative, particularly in Europe and has an estimated market value of around €95bn. Main elements of the proposals include: stronger supervision of independent assessment bodies by national authorities; clearer rights and responsibilities for manufacturers, importers and distributors, applying also to diagnostic services and internet sales; stricter requirements for clinical evidence, to ensure patient and consumer safety. The proposed regulations will replace the existing Directive 90/385/EEC regarding active implantable medical devices, Directive 93/42/EEC regarding medical devices and Directive 98/79/EC regarding in vitro diagnostic medical devices. For more information, please visit the Commission’s website.
In 2013 the Commission has to present a general report on experience acquired as a result of the application of the Regulation on medicinal products for pediatric use. The Regulation was seen by the legislator as a response to the absence of sufficient numbers of suitable, authorised medicinal products to treat conditions in children. The key measures were the establishment of an expert pediatric committee within the European Medicines Agency, as well as the requirement, when applying for marketing authorisation for medicines and line extensions for existing patent-protected medicines, to submit data on the use of the medicine in children in accordance with an agreed pediatric investigation plan. In preparation of this report the Commission released a public consultation document. Citizens and organisations (public and private) are welcome to contribute by 28 November 2012 at the latest. For more information on how to contribute, please read the consultation document.