Cancelled meetings and assessment delays: HTA bodies are revising priorities in response to COVID-19

By Nicola Allen, Associate Director Email: nallen@partners4access.com The priorities of health technology assessment (HTA) bodies are evolving as health care systems around the world are seeking to manage the strain of the Covid-19 pandemic and the biopharmaceutical industry are rising to the challenge to find an effective treatment. Initially, agencies moved in-person meetings to teleconferences…

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Deadline day approaches for Israel’s market entry hopefuls

By Erfan Akbraian, Analyst Email: eakbraian@partners4access.com Majority of orphan drug manufacturers do not currently view Israel as an early launch opportunity. However, universal reimbursed access to high quality healthcare which is at par with Western European countries, combined with the expansion of the prenatal and new-born screening programmes[1] that have considerably improved diagnosis rates is…

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Evolution of payment models for cell and gene therapies in Italy

By Joanna Fernandes, Associate Consultant Email: jfernandes@partners4access.com Cell and gene therapies (CGTs) are considered one-time transformative treatments, positioned as having life-long benefits. These new therapies are challenging the traditional business model, with health technology assessment (HTA) and reimbursement processes having been established around chronic therapies. This has led to one of the biggest hurdles currently…

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Is NICE fit to review orphan drugs? UK MPs aren’t convinced

By Max Rex, Senior Analyst Email: mrex@partners4access.com Jacob Rees-Mogg, Member of Parliament (MP) for North East Somerset on one of his last days as a backbench MP, was granted an Urgent Question in Parliament on July 22nd to discuss the failure of the National Institute for Health and Care Excellence (NICE) to recommend BioMarin’s orphan…

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Germany’s first outcomes deal for CAR-T-cell therapy

By Ciaran Cassidy, Analyst Email: ccassidy@partners4access.com Novartis recently announced an innovative reimbursement scheme with the GWQ –  (a body representing a small number of insurance funds in Germany) –  for its CAR-T cell therapy, Kymriah (tisagenlecleucel). Since September 2018, Kymriah has been used to treat patients up to the age of 25 with refractory and…

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Sustainable funding of cell and gene therapies: bluebird bio puts the ball in the payer’s court

By Nader Murad, Associate Consultant Email: nmurad@partners4access.com At JPMorgan’s  37th Healthcare Conference in 2019, bluebird bio provided its strategic outlook and plans for the year where it highlighted its pipeline progress and commercial activities. bluebird’s pipeline consists of several gene therapies which have demonstrated in clinical studies a high likelihood of potentially curing severe rare…

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Portfolio access agreements – Lessons learnt from Vertex Pharma’s Orkambi

By Jack Rawson, Analyst Email: jrawson@partners4access.com Road to commercialization of Vertex’s CF portfolio The European Commission recently approved Vertex’s combination regimen Symkevi (tezacaftor / ivacaftor; marketed as Symdeko in the US). This is their third disease- modifying Cystic Fibrosis (CF) drug on the market.  The first was Kalydeco (ivacaftor) in 2012, which was the first…

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Chaotic & Unprepared: British biotech industry waits for Brexit

By Joanna Fernandes, Analyst Email: jfernandes@partners4access.com #Brexit #UKBiotech The United Kingdom’s  (UK) biopharma sector are in a precarious position, with less than 24 weeks to go, a no deal Brexit is becoming an increasing reality, forcing the industry to face a potentially, extremely difficult set of circumstances now and in the future. The pharmaceutical sector…

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LentiGlobin: a potential gamechanger in thalassaemia therapy

By Jack Rawson, Analyst Email: jrawson@partners4access.com October 2018 turned out to be a good month for β-thalassemia patients seeking new and effective treatments. The European Medicine Agency (EMA) accepted US-based biotech firm bluebird bio’s marketing authorization application (MAA). The new gene therapy – LentiGlobin – could be a gamechanger for patients with transfusion-dependent β-thalassemia (TDT).…

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