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After Scotland says no, NICE backs Lilly’s Retsevmo for lung cancer

Eli Lilly’s RET inhibitor Retsevmo can now be prescribed on the NHS for people with RET fusion-positive advanced non-small cell lung cancer (NSCLC) in England and Wales, within the Cancer Drugs Fund (CDF). The recommendation from health technology assessment (HTA) agency NICE comes a couple of weeks after Retsevmo (selpercatinib) was cleared for use in …

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NICE backs ViiV/J&J’s six-per-year injection for HIV

People living with HIV in England and Wales now have an alternative to daily oral tablets, after NICE backed NHS use of two long-acting injectable therapies developed by ViiV Healthcare and Johnson & Johnson’s Janssen pharma unit. The cost-effectiveness watchdog has recommended the use of ViiV’s Vocabria (cabotegravir) with Janssen’s Rekambys (rilpivirine) as a combination …

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Drug-resistant epilepsy drug Ontozry backed by NICE

NHS patients in England and Wales with drug-resistant epilepsy are in line for access to another treatment option, after NICE recommended routine use of Angelini group company Arvelle Pharma’s Ontozry. The cost-effectiveness agency has backed Ontozry (cenobamate) as an option for treating focal onset seizures with or without secondary generalised seizures in adults with drug-resistant …

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NICE changes tack on ‘game changer’ rare cancer therapy Poteligeo

NICE has reversed an earlier decision not to recommend NHS use of Kyowa Kirin’s Poteligeo for two rare blood cancers, ending the threat of a north-south divide in access to the drug in the UK. The cost-effectiveness watchdog has now backed Poteligeo (mogamulizumab) for adults in England and Wales with mycosis fungoides (MF) and Sézary …

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Cautious welcome as NICE backs Forxiga for chronic kidney disease

Tens of thousands of people with chronic kidney disease in England could be eligible for treatment with AstraZeneca’s SGLT2 inhibitor Forxiga, after NICE backed NHS use of the drug in draft recommendations. AZ said that the decision is a “milestone” in the treatment of CKD, becoming the first new treatment option for patients in nearly …

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NICE uncertain on insomnia app Sleepio in draft guidance

Big Health’s Sleepio app for insomnia has been cautiously backed by NICE in preliminary guidance, although the cost-effectiveness agency says more data on longer-term use is needed. The document says that Sleepio shows promise as a treatment for insomnia in primary care that could be an alternative medicines and sleep hygiene advice, but it is …

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Seagen’s breast cancer drug Tukysa turned down by NICE

NICE has decided not to recommend Seagen’s orally-active HER2 inhibitor Tukysa for advanced breast cancer in draft guidance, saying it isn’t a cost-effective use of NHS resources. Tukysa (tucatinib) was approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA) in February as a third-line treatment for HER2-positive breast cancer, shortly after it was …

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Alnylam gets NICE backing for porphyria therapy Givlaari

UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam’s gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after its advisors issued a positive verdict on the drug. Givlaari (givosiran) has been recommended as a treatment option for people aged 12 and over with AHP, a …

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NICE changes stance on Sanofi’s Dupixent for severe asthma

18 months after saying “no”  to regular NHS funding for Sanofi and Regeneron’s Dupixent (dupilumab) for severe asthma in initial guidance, NICE has now backed the drug. The final appraisal document for Dupixent (dupilumab) has given the green light to use of the drug as an add-on maintenance therapy for people aged over 12 with …

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Scotland backs Cabometyx combo for kidney cancer, amid stalled NICE review

Patients in Scotland with newly-diagnosed advanced kidney cancer can now access a therapy combining Bristol-Myers Squibb’s immunotherapy Opdivo with Ipsen’s targeted drug Cabometyx, ahead of the rest of the UK and most other countries in the world. The Scottish Medicines Consortium (SMC) has backed Opdivo (nivolumab) plus Cabometyx (cabozantinib) as a first-line therapy for people …

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Consternation as NICE rejects Janssen MS drug Ponvory

NICE has delivered its first verdict on NHS funding of Janssen’s oral multiple sclerosis drug Ponvory, and it’s not good news for patients with the disease waiting for new treatment options. In a provisional decision, NICE has said Ponvory (ponesimod) should not be available routinely on the NHS in England and Wales for relapsing forms …

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Joy as sickle cell patients get access to Novartis’ Adakveo after NICE ruling

People living with sickle cell disease (SCD) in England and Wales will be able to get access to treatment with Novartis’ Adakveo, thanks to a special arrangement between the drugmaker and NICE. The decision makes Adakveo (crizanlizumab) the first new treatment for SCD available via the NHS for 20 years, according to the cost-effectiveness agency, …

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NICE clears NHS use of Roche’s Tecentriq in bladder cancer

NICE has recommended routine NHS access to Roche’s cancer immunotherapy Tecentriq for some bladder cancer patients, four years after it was first made available via the Cancer Drugs Fund (CDF). The PD-L1 inhibitor was initially turned down by NICE for a form of bladder cancer called urothelial carcinoma (UC), but Roche was encouraged to make …

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Keytruda backed by NICE for late-stage oesophageal cancer

MSD’s Keytruda has become the first immunotherapy cleared by NICE for previously-untreated patients with advanced oesophageal cancer, making it an option for routine NHS treatment. Keytruda (pembrolizumab) has been given the green light for use alongside platinum- and fluoropyrimidine-based chemotherapy in patients with locally advanced or metastatic oesophageal cancer that cannot be treated with surgery. …

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NICE backs BioCryst’s Orladeyo for hereditary angioedema

BioCryst Pharma’s Orladeyo has been recommended by NICE as a treatment for the rare disease hereditary angioedema (HAE) in England, Wales and Northern Ireland. Orladeyo (berotralstat) – which was approved by the EMA and MHRA in April and May, respectively – is set to become the first oral, once-daily therapy that can be used to …

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NICE backs Cosentyx’ use in thousands of children with psoriasis

NICE has recommended broadening NHS use of Novartis’ IL-17 inhibitor Cosentyx to include children aged six to 17 with severe plaque psoriasis. The cost-effectiveness agency says in draft guidance that Cosentyx (secukinumab) should be offered as an option to patients in this age bracket who either don’t respond to or cannot tolerate other systemic treatments. …

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NICE backs NHS use of Novartis’ cholesterol drug Leqvio

Novartis’ cholesterol-lowering drug Leqvio will be made available on the NHS in England and Wales, and could help to prevent up to 30,000 deaths, according to NICE. The PCSK9-targeting oligonucleotide – which is already available in Scotland – requires dosing just twice a year and has been approved by the health technology assessment (HTA) agency …

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NICE rejects J&J’ Darzalex regimen for multiple myeloma

New guidance from NICE has rejected a combination regimen based on Johnson & Johnson’s Darzalex for a group of previously-untreated patients with multiple myeloma, a type of bone marrow cancer. The draft document covers the use of Darzalex (daratumumab) as an add-on to standard induction treatment with Takeda’s Velcade (bortezomib) plus thalidomide and dexamethasone given …

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NICE says yes to Novartis’ Rydapt for rare blood disorder

Novartis’ Rydapt has become the first and only licensed treatment for rare and life-threatening blood disorder systemic mastocytosis (SM) to be cleared for routine NHS use, after getting a green light from NICE. The decision means around 170 people with advanced SM in England and Wales will be eligible for  treatment with oral, twice-daily drug, …

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UCB’s bimekizumab ‘pre-approved’ using NICE fast-track scheme

UCB’s bimekizumab hasn’t been approved for marketing in Europe yet, but has already been given the nod for NHS use in England and Wales under a new fast-track scheme introduced by NICE. The IL-17 inhibitor is the first to go through a new process aimed at accelerating appraisals for drugs deemed to be low-risk, which …

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NICE changes its mind on rare bile duct cancer drug from Incyte

Incyte’s Pemazyre has become the first targeted therapy available for NHS patients with cholangiocarcinoma or bile duct cancer, after NICE concluded it provided value for money. The green light for Pemazyre (pemigatinib) has been hailed by cholangiocarcinoma charity AMMF, which said it was a “major milestone” in the fight against the “Cinderella” cancer, which for …

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NICE rejects Orchard’s gene therapy for rare childhood disease MLD

Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. The agency said that while there was evidence of a short-term benefit with Libmeldy (atidarsagene autotemcel or OTL-200), assumptions about its long-term effects are uncertain, making it unclear whether it will offer …

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NICE backs Novartis’ Cosentyx for underdiagnosed back condition

NICE has extended the range of indications that Novartis’ Cosentyx can be prescribed for by the NHS in England, giving a green light for its use in non-radiographic axial spondyloarthritis (nr-axSpA), a form of arthritis affecting the joints of the spine. IL-17 inhibitor Cosentyx (secukinumab) is already approved by NICE to treat ankylosing spondylitis or …

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NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK. The cost-effectiveness agency’s initial assessment is that Evrysdi is simply too expensive at its current price to be provided to the roughly 1,500 people …

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Scotland backs NHS use of Alexion’s Ultomiris for rare disease aHUS

NHS patients in Scotland with the rare disease atypical haemolytic uremic syndrome (aHUS) will now be able to access treatment with Alexion’s Ultomiris, ahead of their counterparts in England and Wales.  aHUS is an ultra-rare disease that can cause progressive injury to the kidneys via damage to the walls of blood vessels and blood clots …

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NICE says no to Merck Serono’s Bavencio in bladder cancer

Merck Serono’s Bavencio is the only checkpoint inhibitor to be approved for first-line maintenance treatment of bladder cancer in Europe, but UK cost-effectiveness agency NICE says it is too expensive to justify routine NHS use.  Bavencio (avelumab) was approved in the UK earlier this year for locally advanced or metastatic urothelial carcinoma (UC) – the …

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Scotland opens door to Translarna for Duchenne muscular dystrophy

A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of PTC Therapeutics’ Translarna for the first time.  The Scottish Medicines Consortium (SMC) says that Translarna (ataluren) can be provided by NHS Scotland for a period of three years, once PTC …

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NICE backs Sobi’s Kineret for rare inflammatory disease

The UK’s cost-effectiveness agency NICE has recommended that Sobi’s Kineret can be used to treat NHS patients with Still’s disease, a rare form of arthritis affecting children and adults. The final guidance concludes that interleukin-1 receptor antagonist Kineret (anakinra) can be used as a treatment for two forms of Still’s, namely systemic juvenile idiopathic arthritis …

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Double first for Scotland as it backs Roche’s Rozlytrek, Novartis’ Zolgensma

The Scottish Medicines Consortium (SMC) has said Roche’s Rozlytrek can be used by NHS Scotland for the treatment of solid tumours with NTRK gene fusion mutations, regardless of where they occur in the body.  This is the second approval by the SMC for Rozlytrek (entrectinib), coming after the drug was cleared for use in ROS1-positive, …

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NICE says no to AZ’s Lynparza for prostate cancer

NHS patients in England will not be able to get access to AstraZeneca’s PARP inhibitor Lynparza if they have BRCA-positive advance prostate cancer, according to draft guidance from NICE. The cost-effectiveness agency is assessing use of Lynparza (olaparib) in patients with tumours that carry BRCA1 or BRCA2 mutations who have previously been treated with the …

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NICE backs broad use for Gilead/Galapagos arthritis pill Jyseleca

Patients with rheumatoid arthritis (RA) in England will be able to get treatment with Gilead Sciences and Galapagos’ JAK inhibitor Jyseleca, after it was backed by cost-effectiveness agency NICE. Jyseleca (filgotinib) has been recommended for moderate and severe active RA in patients who have responded inadequately to intensive therapy with two or more conventional disease-modifying …

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Scotland backs Roche’s Rozlytrek for ROS1 lung cancer

The Scottish Medicines Consortium (SMC) has given a green light to Roche’s Rozlytrek for a rare form of lung cancer, almost seven months after NICE backed the drug in England. Rozlytrek (entrectinib) can now be used by the NHS in Scotland as a treatment option for ROS1-positive, advanced non-small cell lung cancer (NSCLC) not previously …

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NICE backs AZ’s Forxiga for heart failure

Just a few weeks after its EU approval for heart failure, AstraZeneca’s Forxiga has been backed by NICE for this use by the NHS in England and Wales. Forxiga (dapagliflozin) – originally developed as a type 2 diabetes drug – is the first SGLT2 inhibitor to be approved for the treatment of symptomatic chronic heart …

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Okra says AI-based drug price predictor is 90% accurate

UK artificial intelligence startup Okra Technologies has launched a new software platform that it claims can take the guesswork out of the price that can be charged for new drugs, years ahead of launch. The AI system – called ValueScope – can predict the price  as well as the likely outcome of negotiations with health …

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Gene Therapy Medical Affairs

The inaugural Gene Therapy Medical Affairs Summit is dedicated to exploring the pivotal internal and external role that Medical Affairs plays in delivering gene therapies to patients. Industry pioneers from leading pharma and biotech companies at various stages of development and commercialization including Novartis Gene Therapy, Orchard Therapeutics & Bluebird Bio will share how to engage effectively with internal clinical and commercial teams as well as external HCPs, …

Gene Therapy Medical Affairs Read More »