Effective Use of Health Technology Assessment to Maximise Market Access: Start Early and Update Often

We all know that the face of drug discovery and the market for new pharmaceutical products (and medical devices/diagnostic technologies) is changing. The era of the blockbuster appears to be over, the cost of bringing a new product to market has skyrocketed and when a new product is eventually licensed, sceptical payers are increasingly demanding evidence, not only of the product's efficacy, but also its value for money. This demand on the part of payers for evidence of value has led to an increasing need for health technology assessment to support reimbursement and market access strategies. The drug development pyramid (see Figure 1) is a familiar concept to those working in clinical research. With so many candidate substances screened for development just to support the launch of a single product, it would be tempting to see it as folly to embark on a formal assessment of potential value for a product prior to launch – before it has even been shown whether the product works. However, as the quote above suggests, to wait for successful demonstration of efficacy before thinking about demonstrating value, risks coming to market totally unprepared, with the consequent danger that the successfully licensed product will fail to 'hit the ground running.'

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Variations and Similarities in Health Technology Assessment and Market Access in Europe, North America and Asia

Health systems have always had to make decisions about the value of new ways of managing patients. Here 'value' should be taken as meaning the extent to which new interventions offer improvements in patients' health at an acceptable cost. This reflects the fact that the vast majority of health care systems, now and in the past, experience some limit on the resources they have available to devote to patient care. But it is only over the last 30 years or so that systems have been explicit about these market access decisions and, more importantly, sought to inform them through scientific research. This type of research can be broadly defined as health technology assessment (HTA), although it consists of many types of activity and a bewildering set of terms including evidence-based medicine, outcomes trials, patient reported outcomes, comparative effectiveness and cost effectiveness. In general, HTA can be thought of as a constellation of scientific research activities which relate to the effects of health care interventions, relative to appropriate alternatives, as used routinely in health care systems. Although there is a wide array of 'effects' of interest, these are generally characterised by a focus on the patient's ultimate experience of changes in health and by the resource implications of interventions.

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Key Elements of Observational Studies that Make Major Contributions to the Literature

Successful observational studies often yield multiple abstracts and manuscripts, as befits their multifaceted goals. Unsuccessful observational studies sometimes fade away with nothing more than an archived final report that was never even presented to the investigators. While publications are by no means the only measure of success in an observational study, the presentation of abstracts and the later publication of peer-reviewed manuscripts are among the most visible and lasting markers for success.

As a statistician with a long history of co-authoring results from observational studies, I am often asked which statistical tools are critical for observational as opposed to randomized studies. While observational study statisticians do have their own specialized statistical tools, success has much more to do with process than with statistical methodology.

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Profile

Paul Quarterman - Executive Vice President - Oxford Outcomes - An ICON plc Company

I lead the Oxford Outcomes team which is spread across offices in the US, UK and Canada with a focus on development strategy, business administration and coordination of a lively and collegiate leadership team.

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Did you know?

ICON has expanded its Late Phase & Outcomes Research capabilities through the acquisition of Oxford Outcomes, a leading international health outcomes consultancy.

Together with Oxford Outcomes, ICON offers a broader suite of services in Late Phase and Outcomes Research supporting the Pharmaceutical, Biotechnology, Consumer Medicine, Medical Device and Diagnostics industries.

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