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…

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…

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…

Rare Diseases: A New Purpose

How drug repurposing could help drive orphan drug access if pharma can accept the risks By Nader Murad, Senior Analyst Email: nmurad@partners4access.com The US National Institutes of Health describes drug repurposing as “studying drugs that are already approved to treat one disease or condition to see if they are safe and effective for treating other…

P4A to present Cell & Gene Therapy insights at WODC 2018

Partners4Access, a leading market access consultancy specializing in orphan drugs, cell and gene therapy today announced its participation at the World Orphan Drug Congress, an industry conference that brings together rare disease stakeholders under a common platform. The three-day event is being held between November 6-8, 2018 in Barcelona, Spain. P4A’s Sophie Schmitz, Managing Partner…

Cancer treatments: A brief history

February 4 was World Cancer Day and here at Partners4Access, we are having a look back at how cancer treatments have evolved through the years and where they are likely to go in the future. First came surgery In the 18th century, surgery was hailed as the first effective cancer treatment. The discovery of anesthesia…