Market Access & Reimbursement

After an initial funding rejection last year, CSL Behring’s gene therapy for hemophilia B is to be made available to English patients under the Innovative Medicines Fund via a “first-of-its-kind” agreement. Hemgenix is also the subject of a funding deal in Denmark.

The R&D-based pharma industry has welcomed the “more careful and considered approach” taken by EU ministers to the European Commission’s proposed compulsory licensing system, but says there is room for further improvement.

The significantly scaled back version of the Treat and Reduce Obesity Act is expected to cost the Part D program $1.7bn over 10 years.

The Council of the EU says it has produced a “well balanced” text in response to the proposals on tackling medicine shortages in the pharma reform package, and has flagged up a number of areas where it wants more clarity.

Two new pieces of guidance, from the EMA and the HTA coordination group respectively, should help companies understand which drugs must undergo an EU-wide joint clinical assessment and how to notify the relevant authorities about them.

The European Commission has published draft procedures for how the European Medicines Agency is to share information relating to joint clinical assessments with the Member State Coordination Group on HTA.

EU policymakers have the chance to adopt legislation that establishes a strong and predictable framework of incentives for R&D into new medicines, particularly those that require riskier investments, says CSL Behring’s Lutz Bonacker.

EU citizens can access medical treatments in countries other than their own through cross-border health care regulations – but these legislations were not built with advanced therapy medicinal products (ATMPs) in mind, experts warn.

As well as differences of opinion over the “incentives” part of the EU legislative overhaul, the latest meeting of health ministers revealed uncertainties about whether to use incentives or obligations to improve medicines access and how to define “unmet medical needs."

The US Medicare agency is not moving forward with a proposal to cut payments for drugs with accelerated approval status pending further discussions with the FDA about whether new regulatory authorities have addressed concerns about timely completion of confirmatory trials.

England’s NICE should factor in the impact of patent expiry on future drug prices when analyzing the cost effectiveness of new medicines and should also take account of societal benefits, a group of health economists has said.

With the 4 July UK general election less than two weeks away, the Pink Sheet looks at the manifestos published by the major political parties and finds pledges to drive innovation, boost clinical trial activity and speed up regulatory procedures.

Drug maker pursuing novel approach to establishing that contract pharmacies are improperly taking ‘title’ to 340B-discounted drugs, leading to diversion, which is prohibited by law.

In a sign of difficult negotiations to come on the European Commission’s pharma reform package, the presidency of the Council of the EU has drawn up its own set of proposals on regulatory data protection and market exclusivity that differ in several respects from those of the commission and the European Parliament.

Cystic fibrosis patients in England will be able to access Vertex’s three combination therapies after the company announced an extended reimbursement agreement with the National Health Service and a positive opinion from the country’s HTA body, NICE.

Launched in April 2014, the UK’s Early Access to Medicines Scheme has delivered pre-approval patient access to many treatments for life-threatening and seriously debilitating conditions. But after 10 years of the EAMS, the pharmaceutical industry wants new guidance and says the scheme should evolve so that companies can make the most of the opportunity it offers in the future.