Issues, challenges and opportunities for economic evaluations of orphan drugs in rare diseases: an umbrella review

25 May 2024 - There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. ...

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NICE and NHS England announce plan to enable innovative tech to be adopted quicker

23 May 2024 - NHS patients in England will benefit from faster access to the most innovative and cutting-edge medical technologies ...

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Setmelanotide acetate for the treatment of patients with obesity and hyperphagia in Bardet-Biedl syndrome (final guidance)

22 May 2024 - NICE has published final evidence-based recommendations on the use of setmelanotide acetate (Imcivree) for treating obesity ...

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Sirolimus for the treatment of patients with facial angiofibroma caused by tuberous sclerosis complex

22 May 2024 - NICE is unable to make a recommendation on the use of sirolimus (Hyftor) for the treatment of ...

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Efanesoctocog alfa for the treatment and prevention of bleeding episodes in patients with haemophilia A

17 May 2024 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...

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Tisagenlecleucel for the treatment of patients with relapsed or refractory B-cell acute lymphoblastic leukaemia

15 May 2024 - NICE has published final evidence-based recommendations on the use of tisagenlecleucel (Kymriah) for the treatment of ...

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Acceptability of using real world data to estimate relative treatment effects in health technology assessments: barriers and future steps

15 May 2024 - Evidence about the comparative effects of new treatments is typically collected in randomised controlled trials (RCTs). In ...

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Selinexor in combination with bortezomib and dexamethasone for the treatment of patients with relapsed/refractory multiple myeloma (final guidance)

15 May 2024 - NICE has published final evidence-based recommendations on the use of selinexor (Nexpovio) in combination with bortezomib ...

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Atogepant monohydrate for the prevention of migraine

15 May 2024 - NICE has published final evidence-based recommendations on atogepant monohydrate (Aquipta) for preventing migraine in adults. ...

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First ever life-saving treatment for rare heart condition available on the NHS

13 May 2024 - NHS patients with a life-threatening heart condition are set to benefit from a cutting-edge new medicine ...

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Remdesivir and the combination of tixagevimab and cilgavimab for the treatment of patients with COVID-19 disease (final guidance)

8 May 2024 - NICE has published final evidence-based recommendations on the use of remdesivir (Veklury) and tixagevimab and cilgavimab ...

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Tests could lead to fewer people having unnecessary chemotherapy after surgery for early breast cancer

10 May 2024 - More people with early breast cancer could be spared chemotherapy and therefore avoid the side effects associated ...

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Selinexor with dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma after 4 or more treatments

8 May 2024 - NICE has published evidence-based recommendations on the use of selinexor (Nexpovio) in combination with dexamethasone for ...

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Improved deal signals NICE recommendation of sickle cell treatment voxelotor

3 May 2024 - Around 4,000 people with sickle cell disease could benefit from a new treatment recommended by NICE ...

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NHS rolls out ‘life-changing’ treatment for thousands with sickle cell disease

3 May 2024 - Thousands of patients with sickle cell disorder are to be offered a new “life-changing” treatment on ...

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