Carole Longson

From Adaptive Licensing to Adaptive Pathways: Delivering a Flexible Life-Span Approach to Bring New Drugs to Patients

The concept of adaptive licensing (AL) has met with considerable interest. Yet some remain skeptical about its feasibility. Others argue that the focus and name of AL should be broadened. Against this background of ongoing debate, we examine the environmental changes that will likely make adaptive pathways the preferred approach in the future. The key drivers include: growing patient demand for timely access to promising therapies, emerging science leading to fragmentation of treatment populations, rising payer influence on product accessibility, and pressure on pharma/investors to ensure sustainability of drug development. We also discuss a number of environmental changes that will enable an adaptive paradigm. A life‐span approach to bringing innovation to patients is expected to help address the perceived access vs. evidence trade‐off, help de‐risk drug development, and lead to better outcomes for patients.

Accelerated Access to Innovative Medicines for Patients in need

There is broad agreement among health‐care stakeholders that more must be done to ensure that patients have timely access to new and innovative medicines. Assuming that industry will continue to develop such medicines at a sustainable rate, regulators and payers become the gatekeepers. Regulators, starting in the late 1980s/early 1990s, and, more recently, payers have implemented a variety of early‐access pathways or initiatives, and this practice is continuing even today. This article describes the specific approaches that have been taken in four economically developed regions, reviews their success rates, and suggests possible new directions.

10 years of NICE: still growing and still controversial

The National Institute for Health and Clinical Excellence (NICE) will have existed for 10 years on April 1, 2009. Over the past decade, the institutes methodological approach to the development of guidance and assessment of the value of health-care interventions has received international interest and acclaim. Furthermore, individual decisions, in particular those made on new cancer drugs, have generated enormous controversy. An early example was the appraisal of irinotecan and oxaliplatin for colorectal cancer in 2002. In 2003, NICE described the rationale behind its decision making. The 10th anniversary of the institute provides an opportunity to review some of the key issues affecting cancer appraisals and to explain the development of other NICE guidance programmes relevant to the provision of cancer services.

The National Institute for Health and Clinical Excellence and Its Role in Assessing the Value of New Cancer Treatments in England and Wales

The boundaries of medical science in the treatment of cancer are constantly extending. Developments of existing treatments, innovative approaches, new discoveries, and more targeted therapeutic options are translating into practice. With advances come increasing costs, often of a magnitude that stretches finite financial resources. When decisions about funding are made on behalf of a population, standardized processes and methods are needed in order to produce robust guidance in a fair, consistent, and transparent way. The challenges of making these difficult decisions are brought into particularly stark relief when potentially life-extending treatments for patients with a short life expectancy are appraised. The National Institute for Health and Clinical Excellence (NICE) produces guidance on the clinical- and cost-effectiveness of medicines compared with current standard practice. Approximately 40% of the technologies appraised by NICE are indicated for cancer, and the majority of these are pharmaceuticals, mostly biological agents. This article provides an overview of the current role of NICE in making new technologies for cancer available in England and Wales. This includes a summary of experiences with end-of-life treatments and the supplementary advice regarding such treatments that was issued by NICE to its decision-making committees in 2009. Clin Cancer Res; 17(15); 4930–5. ©2011 AACR.

At the Center of Health Care Policy Making: The Use of Health Technology Assessment at NICE

The UK’s National Institute for Health and Clinical Excellence (NICE), a world leader in health technology assessment (HTA), sits at the interface of a policy environment where everything is urgent and consensus between policy makers and stakeholders is sometimes difficult to attain. The majority of stakeholder challenges to NICE’s use of HTA concern the interpretation of evidence and the methodological rules applied by the appraisal committees. We discuss the most significant issues: choice of comparators; evidence synthesis and indirect comparison; parameter selection, especially for the valuation of quality of life; extrapolation beyond clinical trial data; and the level of the cost-effectiveness threshold.

How can we get high quality routine data to monitor the safety of devices and procedures

Following recent problems with some medical devices, Bruce Campbell, Andrew Stainthorpe, and Carole Longson suggest some pragmatic steps to improve safety data

Prasugrel for the treatment of acute coronary syndromes with percutaneous coronary intervention: NICE technology appraisal guidance

Prasugrel (Efient, Eli Lilly) is an oral inhibitor of platelet activation and aggregation. Prasugrel, coadministered with acetylsalicylic acid, has marketing authorisation for the prevention of atherothrombotic events in patients with acute coronary syndrome undergoing percutaneous coronary intervention (PCI). National Institute for Health and Clinical Excellence (NICE) technology appraisal guidance on the use of prasugrel for patients undergoing PCI was published in October 2009.1 Prasugrel, in combination with aspirin, is recommended as an option for patients when: The following sections present evidence on use of prasugrel and an overview of the independent advisory Appraisal Committees considerations in developing its guidance for NICE. The importance of expert and stakeholder involvement in the development of NICE guidance is also explained. Prasugrel was considered under the NICE single technology appraisal process.2 In this process, the manufacturer provides an evidence submission, which comprises a report (structured according to a template set by NICE) and economic model. The structured report sets out the evidence base for the drug, and the economic model assesses its cost-effectiveness compared to current standard practice – in this case the use of clopidogrel. The manufacturers submission, therefore, forms the core evidence source for the Appraisal Committees considerations. In addition, an academic group, the Evidence Review Group (in this case, the Liverpool Reviews and Implementation Group, University of Liverpool) is commissioned to critique the manufacturers submission, and produce a report of its findings.3 The Evidence Review Group may also undertake exploratory analyses using the submitted materials and report these to the Appraisal Committee. The Appraisal Committee developed recommendations for the use of prasugrel in two stages. First, it met to consider the available evidence and the …

Health Technology Assessment in the Context of Adaptive Pathways for Medicines in Europe: Challenges and Opportunities

Adaptive pathways for medicines have gained momentum and, in Europe, adaptive pathways have recently been introduced into the European Medicines Agency (EMA) processes after a successful 2‐year pilot. Although the concept, as initially proposed, contained several elements that would have required regulatory reforms, the adaptive pathways program has developed a more pragmatic scope (Box 1). In this article, we explore the main challenges and opportunities adaptive pathways pose from a European health technology assessment (HTA) perspective.

Understanding the Role and Evidence Expectations of Health Technology Assessment and Coverage/Payer Bodies What Are They Looking for, and How and Why Does This Differ From What Regulators Require?

Pharmaceutical companies have a good understanding of the needs and requirements of regulatory bodies, but the evidence expectations of health technology assessment (HTA) and coverage/payer bodies are less well understood and addressed. This paper seeks to improve this understanding by providing an overview of the expectations of HTA and coverage/payer bodies, explaining how and why these differ from those of regulators, and describing the extent and limitations of work on harmonization. The article goes on to describe ways in which HTA and coverage/payer bodies’ expectations can be addressed, and to encourage industry to interact with HTA and coverage/payer bodies to increase mutual understanding and hence promote more efficient development of and access to innovative medicines.

Antimicrobial resistance: a light at the end of the tunnel?

• Users may freely distribute the URL that is used to identify this publication. • Users may download and/or print one copy of the publication from the University of Birmingham research portal for the purpose of private study or non-commercial research. • User may use extracts from the document in line with the concept of ‘fair dealing’ under the Copyright, Designs and Patents Act 1988 (?) • Users may not further distribute the material nor use it for the purposes of commercial gain.