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Posted 10 September 2015 | By Nick Paul Taylor
Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news.
The European Medicines Agency (EMA) has brought together a consortium of regulators, patients, academics and industry representatives to establish accelerated development pathways. Over the next 30 months, the collective will work toward creating a model that favors continuous evaluation and gradual expansions of patient access over binary approval and reimbursement decisions.
EMA is working with 31 other international groups, including Novartis, Pfizer and Sanofi, on the plan, dubbed ADAPT SMART. The broad concept of moving away from the traditional model in which a sponsor gathers a full Phase III dataset, applies for regulatory approval and then starts attempting to win reimbursement being explored by ADAPT SMART is well established. EMA’s own adaptive pathways pilot project is among the initiatives with a similar objective.
ADAPT SMART aims to bring together the organizations working on these separate projects and have them collaborate on the creation of Medicines Adaptive Pathways to Patients (MAPPs). The United Kingdom’s National Institute for Health and Care Excellence (NICE) and Massachusetts Institute of Technology, which are respectively involved with the Early Access to Medicines Scheme and the New Drug Development Paradigms initiative, are among the partners in ADAPT SMART.
EMA is pairing these groups with pharma companies, patients and other bodies to work on a model that meets all of their needs. Such diversity is central to the effort. “Diverse stakeholders, including payers, patients, practitioners, regulators, and industry, must be aligned in a truly cross-functional, cross sector engagement of continuous evaluation of the performance over the entire lifecycle of a new therapy,” Solange Rohou, director of Regulatory Affairs at AstraZeneca, said in a statement.
Rohou is co-leading one of four work packages unveiled to date. The initiative gives AstraZeneca and NICE oversight of a program looking at the generation of evidence throughout the product lifecycle. Specifically, the partners will assess the potential and problems of biomarkers, patient-reported outcomes, registries and other sources of data. The partners will also develop an analysis to show gaps in the generation of such evidence, both now and in the future.
The other three work packages bring together representatives of Bristol-Myers Squibb, the Centre for the Advancement of Sustainable Medical Innovation and other organizations to investigate how to design MAPP pathways, how researchers, regulators and clinicians can adopt adaptive decision making and the organization of the overarching ADAPT SMART program. The Innovative Medicines Initiative 2 has provided funding for ADAPT SMART.
ADAPT SMART Release, NICE Statement, EMA Workshop, Work Packages
Roche CEO Severin Schwan has branded the United Kingdom’s cancer drug system “stupid.” Schwan made the statements following the decision to drop certain Roche products from the Cancer Drugs Fund (CDF), a move that contributed to a rising tide of resentment against the UK’s reimbursement process.
The CDF has stopped paying for Roche’s Kadcyla in patients with breast cancer, and Avastin in certain other indications. Officials at the CDF view such adjustments as unavoidable given that its spending has increased rapidly and exceeded its budget at a time when healthcare departments in the UK are under pressure to control their costs. For Schwan, such cuts are an example of cost considerations taking precedence over value.
“It’s stupid from a cost point of view not to look at the overall cost of healthcare,” Schwan said at a media briefing attended by Reuters. “This is really sad for patients and I have no understanding for this decision whatsoever. There is a fundamental flaw in how the UK operates when it comes to pricing for medicine.” A diverse range of organizations, including the National Health Service (NHS), agree that the CDF is flawed but for different reasons than Schwan.
In February, researchers at the University of York calculated that the money the CDF spends to gain one quality-adjusted life year (QALY) would deliver five QALYs if it was used in other areas of the NHS. This discrepancy between the QALYs associated with expensive, end-of-life cancer drugs and other medical interventions was part of the reason for removing certain oncology drugs in the CDF from the value calculations used by NICE. The UK is now working on a longer-term solution.
NICE showed it is willing to reimburse innovative new cancer drugs this week when it signed off on the use of Merck’s anti-PD-1 checkpoint inhibitor Keytruda. The positive decision came after Merck agreed to offer the NHS an undisclosed discount compared to the $150,000 it costs to treat a patient for a year in the United States. Patients in the UK have been receiving Keytruda through the Early Access to Medicines Scheme since March. NICE’s backing secures its long-term future.
The industry will be watching uptake of the drug closely. NHS’ use of branded medicines increased by 2.4% in the second quarter of 2015, half the rate seen in the previous year. The slowing growth occurred despite the industry paying $634 million to support the use of branded medicines in the first half of 2015, more than it contributed in all of 2014. The Association of the British Pharmaceutical Industry (ABPI) said it was “deeply concerned” by the trends.
Reuters, The Financial Times, ABPI Statement
The United Kingdom Medicines and Healthcare Products Regulatory Agency’s Yellow Card mobile app has been downloaded more than 1,000 times. Since being made available in mid-July, the app has been used to report 27 suspected adverse drug reactions.
Around 70% of the 1,037 downloads to date have been for Apple devices, with phones and tablets running on Google's Android operating system accounting for the rest. MHRA is pitching the app as both an alternative, possibly more convenient, way to communicate suspected adverse events and a tool for keeping track of safety issues relating to specific medicines.
The data communication goes both ways. MHRA is analyzing the lists of drugs that users have signed up to receive notifications about to gain an insight into which medicines are of particular interest or concern. The app is available to anyone so the public can use it to keep tabs on the safety of medicines they are taking. Similarly, physicians can use it to track drugs they prescribe frequently.
The European Directorate for the Quality of Medicines and Healthcare (EDQM) has released a new edition of its Guide to the Quality and Safety of Tissues and Cells for Human Application. EDQM, which published the first edition of the text in 2013, has updated the document to bring it up to date with quality control and risk mitigation when dealing with human tissues. Press Release
EMA has added Immunocore’s lead product to its adaptive pathways pilot program. The decision gives Immunocore a chance to win a conditional approval for the use of IMCgp100 in patients with metastatic uveal melanoma on the basis of a limited dataset. The company can then apply to upgrade to a full approval once it has completed a Phase II trial. Press Release
The UK’s National Institute for Biological Standards and Control (NIBSC) has named Dr. Christian Schneider as its director. Schneider, who currently works at the Danish Health and Medicines Authority, will replace Dr. Stephen Inglis as the leader of the biologics quality testing organization in January. Press Release
Tags: European Regulatory Roundup, EU Regulatory Roundup, Regulatory Roundup
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