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Latest From Francesca Bruce
Patient access schemes have helped the Scottish Medicines Consortium recommend rare disease products, including Regeneron/Sanofi’s Libtayo for skin cancer.
In an exclusive interview, senior NICE director Meindert Boysen reveals the best advice he ever received and how companies whose products are under review can improve their chances of success.
Criticism of Novartis/Avexis’s controversial lottery style managed access program for Zolgensma has come largely from Europe, while other countries in Latin America and Asia have been far more receptive.
Novartis’s Luxturna and Kyowa Kirin’s Crysvita are the first drugs to be evaluated through Scotland’s new ultra-orphan process.
Genetic Alliance UK says companies should be clear about how they price rare disease treatments and that the government should explain how it assesses affordability.
An unusual managed access program for Zolgensma designed by Novartis and AveXis has been attacked on ethical grounds, with critics claiming it does not properly allot the gene therapy to patients most in need. The companies have rejected the criticism, saying the program is fair and equitable.