A team of researchers and health officials, including European Medicines Agency (EMA) Executive Director Guido Rasi, are proposing a three-step validation process to enhance the use of surrogate endpoints in regulatory and reimbursement decision-making. The issue, they say, is that poorly validated surrogate endpoints can lead to products being approved or reimbursed that have little or no benefit, and in some cases even harm patients. In recent years, drugmakers have i...

This article introduces the roles of HTA bodies within the EU member states healthcare system and provides key elements to consider for manufacturers who would like to enter this geographical market. In addition, solutions to maximize the product development strategy are discussed with the intention of bridging regulatory and market access gaps. The rapid diffusion of health technologies has presented governments with unprecedented challenges to provide high quality an...

The European Medicines Agency (EMA) says its pilot project for delivering scientific advice to sponsors in parallel with health technology assessment (HTA) bodies has been successful in helping to design efficient clinical development plans and speeding access to new drugs. Due to the success of the pilot, which concluded on Wednesday, EMA says it will continue to offer parallel scientific advice as part of its routine activities. "Our parallel scientific advice fosters...

The complicated world of drug pricing presents an array of challenges for keeping costs low in the US and EU, though European countries are increasingly employing new policies to keep price gouging in check. The 260-page report on drug pricing in Europe, released Thursday by the European Commission, looks into two policy options: external price referencing (EPR), which is predominantly a tool for medicine price control and currently employed across the region, and differ...

The path to regulatory approval can be long and arduous. It can also be especially difficult for smaller companies to navigate. But a new report finds that early input from health authorities can help make the process more straightforward. The report is based on information gathered through interviews with small- and medium-sized biotechnology companies, the European Medicines Agency (EMA) and HTA bodies. The report finds early engagement with regulatory authorities an...

The Institute of Quality and Efficiency in Healthcare (IQWiG) gave negative assessments for three recently approved cancer drugs this month. IQWiG is one of several bodies in Germany charged with conducting health technology assessments (HTAs) for newly approved drugs in Germany. The goal of these assessments is to weigh the benefit of new therapies against the costs to the country’s healthcare system. According to IQWiG’s assessment, the drugs idelalisib , sipuleucel...

In the EU, a product must clear two hurdles before it can reach patients. First, it must be approved by regulators, such as the European Medicines Agency (EMA), which determines if a product is safe and effective. Thereafter, the product is evaluated by health technology assessment (HTA) bodies, which determine if the product is suitable for reimbursement according to the standards of various countries. Now, EU regulators and reimbursement bodies are teaming up in ...

The European Commission (EC) has implemented a new set of rules for the establishment and management of an EU-wide health technology assessment (HTA) network meant to evaluate the cost-effectiveness of healthcare products. The rules come out of Article 15 of Directive 2011/24/EU ("Directive on Patients' Rights in Cross Border Healthcare"), which directed the EU to "support and facilitate cooperation and the exchange of information among Member States working within a vol...

In final draft guidance released today, the UK's National Institute for Health and Care Excellence (NICE) has recommended against the use of ruxolitinib (Jakavi, Novartis) for the treatment of disease-related splenomegaly (enlarged spleen) or its symptoms in adults with primary myelofibrosis, post polycythaemia vera myelofibrosis, or post essential thrombocythaemia myelofibrosisi. Myelofibrosis is a rare blood cancer in which the bone marrow produces too many cells too...

The UK's Medicines and Healthcare products Regulatory Agency (MHRA) has announced a new set of lofty goals that it says will catapult the regulator to the top tier of those now on the "world stage." Overview Under a five-year corporate plan, MHRA said it would focus on five areas of improvement: the role of regulation and the regulator bringing innovation safely to market strengthening surveillance safe products and secure supply in globalized industries achieving...

The UK cost containment agency, the National Institute for Health and Care Excellence (NICE), recommended on 9 April 2013 reimbursement for two in vitro diagnostic (IVD) devices for detecting epidermal growth factor receptor-tyrosine kinase (EGFR-TK) mutations in the tumors of adults with previously untreated, locally advanced or metastatic non-small-cell lung cancer (NSCLC).  The two diagnostic tests, Qiagen's Therascreen EGFR RGQ PCR Kit and Roche's Cobas EGFR ...

Even casual observers of US healthcare policy are aware of a common refrain: Costs are too high, unsustainable and a poor indicator for the quality US patients ought to be receiving. While the reasons for the high costs of healthcare are frequently and loudly debated, a new report out from the US Institute of Medicine (IOM), a venerable think tank of sorts that advises US agencies on matters of health, argues that at least some of those costs are the result of inefficient...