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European Pharmaceutical Review
NOVEMBER 27, 2023
The National Institute for Health and Care Excellence (NICE) has recommended enzyme replacement therapy sebelipase alfa (Kanuma ® ▼) in final draft guidance. This ultra-rare metabolic disease occurs due to lysosomal acid lipase (LAL) enzyme deficiency. How does the enzyme replacement therapy work?
European Pharmaceutical Review
DECEMBER 12, 2022
According to the National Institute for Health and Care Excellence (NICE), an individualised approach should be adopted when treating women with HRT. Researchers have considered it to be associated with fewer risks compared to synthetic hormones. The SMC approval of Utrogestan is limited within NHS Scotland.
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European Pharmaceutical Review
NOVEMBER 27, 2023
The National Institute for Health and Care Excellence (NICE) has recommended enzyme replacement therapy sebelipase alfa (Kanuma ® ▼) in final draft guidance. This ultra-rare metabolic disease occurs due to lysosomal acid lipase (LAL) enzyme deficiency. How does the enzyme replacement therapy work?
European Pharmaceutical Review
DECEMBER 12, 2022
According to the National Institute for Health and Care Excellence (NICE), an individualised approach should be adopted when treating women with HRT. Researchers have considered it to be associated with fewer risks compared to synthetic hormones. The SMC approval of Utrogestan is limited within NHS Scotland.
pharmaphorum
JUNE 30, 2021
The UK innovation passport adds an additional layer however, as it involves input from the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC), meaning that NHS funding deliberations are bundled into the review.
European Pharmaceutical Review
APRIL 12, 2023
In November 2022, the Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Passport was awarded to Boehringer Ingelheim’s investigational BI 907828 in de-differentiated liposarcoma (DDLPS), a rare and aggressive oncology indication for which there has been no new first-line treatment in over forty years.
European Pharmaceutical Review
SEPTEMBER 22, 2022
Natural history information also describes the current standard of care or emergent care, which may alter disease morbidity and mortality. Rare disease drug development poses unique challenges that can be overcome by using real-world evidence (RWE). Benefits of historical controls.
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