News
🧬 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
Bismarck or Beveridge? Does the financing model determine health system outcomes?
April 23–24 | St. Petersburg
New drug price regulation policy in the US and its impact on willingness to pay thresholds
The FDA has approved Avlayah for the treatment of neurological manifestations of Hunter syndrome
Monitoring patient-reported outcomes is becoming the new norm in clinical trials
Russia has updated its list of rare diseases
The CIS Orphan Consortium has joined Rare Diseases International.
FDA launches new mechanism to expedite development of individualized targeted therapies for ultra-rare diseases
A new rare disease, El-Hattab–Schmidts syndrome, has been established in global clinical genetics.
China updates regulations for implementing the Medicines Regulation Law
The US has extended the Priority Review Voucher (PRV) program for rare pediatric diseases.
Sechenov University discussed the possibilities of innovative treatment methods for children with severe and rare diseases.
An expert review under the auspices of the CIS Orphan Consortium was published in the Journal of Health Development.
The CIS Orphan Consortium continues its series of webinars on MCDA