Under the microscope: Orphan Drug incentives in Europe

By Chloe Sheppard, Analyst Email: csheppard@partners4access.com The European Commission is imminently expected to publish a report evaluating the pros and cons of the EU Orphan and Paediatric Legislation introduced 20 years ago. The regulation has so far had success towards its primary goal to incentivise the development of orphan medicinal products (OMPs) for patients living…

France to place restrictions on Nominative ATU

By Erfan Akbraian, Analyst Email: eakbraian@partners4access.com France’s National Agency for the Safety of Medicines and Health Products (ANSM)1 governs the early access program called autorisation temporaire d’utilisation (ATU). The ANSM are expected to implement changes set by the French social security law to the nominative ATU program later this year to ease the cost burden…

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…

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…

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…

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…

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…

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…