News
Conference "Real clinical practice. Possible and real"
China changes approach to access and financing of innovative drugs
Russia expanded the list of rare orphan diseases for the fourth time in 2026
Brazil Evaluates Multiple Technologies for PNH Treatment: An Example of an Integrated Approach to HTA
Russia is expanding its neonatal screening program for rare diseases.
Two child deaths have cast doubt on China's accelerated gene therapy research.
Первая терапия атаксии Фридрейха столкнулась с отказами в возмещении
Saudi Arabia is introducing a new accelerated route for orphan drugs.
FDA Proposes to Reduce Regulatory Burden in the Development of Platform-Based Gene Therapy Using Genome Editing
Patient Organisations in Europe Submit Comments on the First Joint Clinical Assessment Report under the HTA Regulation
The First Joint Clinical Assessment Report Published in the EU under the New HTA Regulation
Resolution of the III CIS Orphan Forum: Establishment of the BRICS Orphan Consortium and Development of International Cooperation in the Field of Rare Diseases
Following the Forum on May 25–26, 2026, a Resolution was adopted, agreed upon with the participants of the CIS Orphan Consortium. The document consolidates the key proposals developed over two days of work by experts from 21 countries — members of the Commonwealth of Independent States (CIS), BRICS, and partner states.
The III CIS Orphan Forum has come to an end: the Commonwealth countries are entering into cooperation with BRICS
The creation of the BRICS Orphan Consortium was announced at the III CIS Orphan Forum
🧬 IQVIA published a report on the sustainability of biosimilar markets in Europe.
In the UAE, for the first time outside the USA, an adult patient with SMA has been administered the gene therapy Itvisma
Chiesi acquires KalVista: the first oral drug for the treatment of attacks of hereditary angioedema
Following the results of the II Orphan Forum of the CIS countries, an article was published in The BRICS Health Journal
Registration is now open for the webinar "Orphan Drug Status: Regulatory Approaches and Practices of EAEU Countries"
The CIS Orphan Consortium and the National Institute for Health Research of Iran signed a cooperation agreement
The first gene therapy to restore hearing: A historic FDA decision and a new challenge for healthcare systems