Blogs
Driving patient access for treatments targeting rare genetic disorders in the Middle East
By Ciaran Cassidy, S Analyst Email: [email protected] At the World Orphan Drug Conference (WODC) this year Dr Maryam Matar presented on the success of the
Under the microscope: Orphan Drug incentives in Europe
By Chloe Sheppard, Analyst Email: [email protected] The European Commission is imminently expected to publish a report evaluating the pros and cons of the EU Orphan
France to place restrictions on Nominative ATU
By Erfan Akbraian, Analyst Email: [email protected] France’s National Agency for the Safety of Medicines and Health Products (ANSM)1 governs the early access program called autorisation
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
