A WHO report, entitled "Access to new medicines in Europe: technical review of policy initiatives, opportunities for collaboration and research", illustrates the challenges faced by the national health systems in the WHO European Region where only a handful of countries are able to appraise the cost–effectiveness of new drugs. The report makes a special mention of orphan drugs as well as the cross border initiatives benefiting rare disease patients.

This study furnishes information on the methods used by the health authorities of 27 European countries when dealing with high spending on new medicines. The report emphasises that there is a lack of transparency in the process of negotiating the prices of drugs between the governments and the pharmaceutical companies. Additionally the supply and prices of new medicines are often fixed in framework agreements between governments and pharmaceuticals.

The report outlines possible policy directions and choices that may help governments to reduce high prices when introducing new drugs and recommend strengthening collaboration and transparency in policy-making as well as cooperation between governments, regulators and drug companies. Particular focus is given to chronic care, specialty medicines and rare diseases (orphan drugs).

More information: WHO report, OrphaNews 


Mayo Clinic Proceedings has published a guidance document on obtaining informed consent from paediatric population to participate in a biorepository. Practices have become more complex, the task of developing appropriate informed consent practices has become more challenging. Written on behalf of the Consent, Education, Regulation, and Consultation Workgroup of the eMERGE Network, this document addresses the unique issues that arise for biorepositories that aim to collect samples from paediatric participants as a guidance for investigators and institutional review board (IRB) members in the United States regulatory context. The evolving roles of parents and children in making decisions related to research participation as children mature and the role of the IRB are the two main issues that are addressed in this article around which the recommendations are made. The document also presents guidance on a variety of paediatric-specific consent issues that arise frequently in the development of biorepositories.

More information: eMERGE Network, Article, OrphaNews

thal day 2015

8th of May marks the international thalassaemia day aiming to raise awareness of the disease. On this special day, every year, the Thalassaemia International Federation (TIF) focuses on a different theme that intervenes with the quality and appropriate treatment of patients and impacts their quality of life. This year's theme is "Enhancing partnership towards patient-centred health systems: good health adds life to years!".

For more information, visit the dedicated 8th of May website created by the TIF.


One of the challenges involved in sharing bioresources (biological samples, data, and databases) is the lack of structural guidance to correctly recognise and trace their use especially in publications. An article published in BMC Medicine proposes, a guideline for reporting bioresource use in research articles, named CoBRA (Citation of BioResources in journal Articles). Developed by the members of the journal editors subgroup of the Bioresource Research Impact Factor (BRIF), CoBRA provides a citation system where “each individual bioresource that is used to perform a study and that is mentioned in the Methods section should be cited as an individual “reference [BIORESOURCE]” according to a delineated format”. Additionally, the European Association of Science Editors has adopted BRIF’s suggestion to incorporate statements on biobanks in the Methods section of their guidelines. According to the authors: “The endorsement and the adoption of the CoBRA guideline by authors, editors, researchers, and bioresource policy stakeholders is the first necessary step to achieve these goals and is essential to enhance transparency in health research”.

More information: BMC Medicine, OrphaNews

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Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications. Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, and damage to body organs. One medication used to treat sickle cell disease is hydroxyurea.

Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs). Hydroxyurea has been given to many HbSC patients but HbSC patients were not included in the large clinical trials used to test hydroxyurea in SCD, so we don't know as much about how HbSC patients respond to hydroxyurea.

The purpose of this research study is to see if hydroxyurea, a medication given to many children with the most common type of sickle cell, HbSS, helps children who have HbSC, by giving a questionaire when the medication is started, and then each month at a clinic visit. The questionaire, called the PedsQLTM 3.0 Sickle Cell Disease Module, measures quality of life. Thia study will also evaluate how hydroxyurea changes laboratory test numbers, and blood thickness.

More information: clinicaltrials.gov, ITHANET Clinical Trials

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