
Zolgensma’s ‘Day One’ access scheme – can Novartis’s early access plan succeed?
By Max Rex, Associate Consultant Email: [email protected] Zolgensma, Novartis’s blockbuster gene therapy for the treatment of spinal muscular atrophy (SMA), was officially given the green

Cancelled meetings and assessment delays: HTA bodies are revising priorities in response to COVID-19
By Nicola Allen, Associate Director Email: [email protected] The priorities of health technology assessment (HTA) bodies are evolving as health care systems around the world are

Deadline day approaches for Israel’s market entry hopefuls
By Erfan Akbraian, Analyst Email: [email protected] Majority of orphan drug manufacturers do not currently view Israel as an early launch opportunity. However, universal reimbursed access

Evolution of payment models for cell and gene therapies in Italy
By Joanna Fernandes, Associate Consultant Email: [email protected] Cell and gene therapies (CGTs) are considered one-time transformative treatments, positioned as having life-long benefits. These new therapies

Is NICE fit to review orphan drugs? UK MPs aren’t convinced
By Max Rex, Senior Analyst Email: [email protected] Jacob Rees-Mogg, Member of Parliament (MP) for North East Somerset on one of his last days as a

Germany’s first outcomes deal for CAR-T-cell therapy
By Ciaran Cassidy, Analyst Email: [email protected] Novartis recently announced an innovative reimbursement scheme with the GWQ – (a body representing a small number of insurance

Sustainable funding of cell and gene therapies: bluebird bio puts the ball in the payer’s court
By Nader Murad, Associate Consultant Email: [email protected] At JPMorgan’s 37th Healthcare Conference in 2019, bluebird bio provided its strategic outlook and plans for the year

Portfolio access agreements – Lessons learnt from Vertex Pharma’s Orkambi
By Jack Rawson, Analyst Email: [email protected] Road to commercialization of Vertex’s CF portfolio The European Commission recently approved Vertex’s combination regimen Symkevi (tezacaftor / ivacaftor;

Chaotic & Unprepared: British biotech industry waits for Brexit
By Joanna Fernandes, Analyst Email: [email protected] #Brexit #UKBiotech The United Kingdom’s (UK) biopharma sector are in a precarious position, with less than 24 weeks to