Outcomes research

The mean versus the gene: will national healthcare systems ever adopt personalised medicine?

Jul 26, 2008
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By John F P Bridges

While outcomes for any given treatment differ significantly among patients, national healthcare systems continue to take a top down population perspective in reviewing not only epidemiologic data but to evaluating the effectiveness and cost-effectiveness of new medicines.

This top down approach, coupled with a growing need for cost containment, has recently caused many governments to institutionalise these practices through health technology assessment institutions. The purpose of these agencies is to promote better quality or value for money in the healthcare system; but this has led to medicines and technologies either being considered good (in other words, good for all) or they are deemed bad and are blacklisted.

Whether it be data from a randomised control trial, a comparative effectiveness study or a cost-effectiveness study, the focus is on the average patient’s health outcomes, where all individuals are treated equally (or to be more correct, identically). Variation is something that we consider only when it comes to statistical inference, viz. does the average effect differ from zero.

This is a major over simplification since patients do vary: their needs vary; their preferences vary; their circumstances vary and, most important, their outcomes vary. Even if you wanted to treat individuals equally on ideological grounds, these top down approaches ignore the risks and uncertainties in medical decision making. For example, rather than understanding risk in clinical trials, we attempt to make it go away by demanding larger and larger trials (a movement away from the individual, towards the population).

As technology progresses, we are increasingly aware that the variation in benefits and adverse consequences of many healthcare interventions are predictable. As our knowledge of genetics and proteinomics expands, our ability to predict these events grows exponentially. This has prompted many healthcare innovators to develop diagnostics to tailor medicines for particular patients – in what is often referred to as personalised medicine. Governments everywhere have been eager to support these start-ups, almost to the point of frenzy and with little accountability.

One key problem exists, however, in that these new technologies are incompatible with the fundamental principles of many national healthcare systems and with the top down evaluation that has been implemented. Hence a bottleneck is occurring – ironically with government playing a dual role of promoting and rationing medical technology. In order to alleviate this bottleneck, we either have to address the funding crisis through personalised approaches to healthcare finance (The Netherlands and Switzerland have attempted this) or we need to stop wasting money on research and development of personalised medicine technologies that are unlikely to be funded in the future.

Essentially this means that healthcare systems need to decide whether they want to focus on the mean (the average effects of medicine) or on the gene (by accommodating personalised medicine.

John Bridges, PhD, is Assistant Professor, Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Senior Fellow at the Center for Medicine in the Public Interest and founding editor, The Patient: Patient-Centered Outcomes Research.

You need to know the global issues in health economics

Dec 24, 2007
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By Robert Hand (robert.hand@rxcomms.com)

As a health economist and outcomes researcher, you may work primarily within a single country. Naturally, you know the economic, regulatory, and management issues of that country. However, with globalisation, you need increasingly to be aware of the corresponding conditions around the world.

Why? Individual countries or cooperative groups of countries can differ markedly in how they regulate pharmaceutical development and marketing, control the pricing of pharmaceuticals and other aspects of healthcare, and pay for healthcare. All these factors significantly affect health economics.

Most pharmaceutical and healthcare device companies already have to adapt their operations to do business across borders. Often, their research involves studies conducted in two or more countries. Of course, once a new product or service is developed and approved, the manufacturer has to contend with marketing it in different countries.

As a researcher, how can you keep abreast of the varying international environments? One place to start is with “Pharmacoconomic Guidelines Around the World,” at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) website.

Derived from an article by Tony YH Tam and Marilyn Dix Smith in ISPOR Connections (volume 10, number 4, August 2004), the webpage presents guidelines that can be used as standards for preparing studies for inclusion in an application for reimbursement, guides for designing and conducting studies, or templates for evaluating economic study report.

The current set includes 28 guidelines from 23 countries, grouped as Eastern Europe, Western Europe, Northern Europe, Southern Europe, Northern America, Eastern Asia, and Oceania. Guidelines are presented as hyperlinks, full text (as copyright permission allows), or publication reference.

Among the guidelines are 21 pharmacoeconomic guidelines, six submission guidelines, and one guideline for journal publication.

The webpage also includes a comparative table of 32 key features of the guidelines. These include the type, title, and year of the document, main policy objectives, standard reporting formats included, target audience, and the various analytical factors used.

What is the value of outcomes research?

Dec 24, 2007
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By Kevin Frick (kevin.frick@rxcomms.com)

It is ironic that we should have to answer the question: “what is the value of outcomes research?” when outcomes research is a key component of establishing the value of healthcare.

Despite the seemingly obvious answer, i.e. “we could not possibly establish the value of healthcare without outcomes research”, there is reason to pause. The meaning of the value of healthcare may not be entirely obvious, single works of outcomes research do not always provide all the information necessary to establish value, and the use of value assessment in real world policy-making is not always apparent.

What is the meaning of “value”? Critically, the value of healthcare depends on the perspective of the decision-maker who is assessing the value. For the government, the value may be in limiting expenditures for a programme such as Medicare or a National Health Service. Government decision-makers may also be directly concerned about the health of the population. For an employer, the value of healthcare may be in limiting workplace absenteeism. For an individual in the population, the value may be a combination of saving money, providing for a family, greater functionality, and enjoying life in general.

Outcomes research can document all these meanings of value, but a single study will rarely establish savings in medical care claims, changes in absenteeism, and changes in other aspects of quality of life. However, taken as a whole, multiple studies of outcomes provide information that can be critical for formulating policy. For decision makers to use the information in the process of making policy, those performing outcomes research must demonstrate not only the importance of their own work but the importance of the set of work into which their work fits. A coherent literature review that integrates a study into the extant literature is useful for achieving this objective.

Thus, outcomes research has value when each study, viewed in combination with other works, is useful in establishing the value of healthcare from multiple perspectives so that more informed policy can be made for the optimal allocation of healthcare resources.

The “other drug costs” – Part 1: non-adherence

Oct 22, 2007
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By Mary Gabb (mary.gabb@rxcomms.com)

The term “adherence” (or sometimes “compliance”) – the extent to which patients take their treatment as prescribed – has been understood to be a nagging clinical issue, but is now seen as a major cost driver in many therapeutic areas.

A recent report from the National Council on Patient Information and Education (NCPIE) in the United States reveals depressing statistics: 49% of those polled had forgotten to take a prescribed medication, 31% had not filled a prescription they were given, 29% had stopped taking a medicine before the supply ran out, and 24% had taken less than the recommended dosage.

A recent report in the Archives of Internal Medicine documents our failed efforts to improve adherence. Only about half of randomised controlled trials designed to improve medication adherence showed any consistent improvement in patient adherence, and less than one third of the studies demonstrated improvement in at least one clinical outcome. Non-adherence has even been referred to as the “other drug problem”.

How does the issue of adherence/compliance affect the field of health economics? The International Society for Pharmacoeconomic Outcomes and Research (ISPOR) has created the ISPOR Medication Compliance and Persistence Special Interest Group to study non-adherence in pharmacoeconomic evaluations.

JoAnne LaFleur, Research Assistant Professor at the Pharmacotherapy Outcomes Research Center, University of Utah College of Pharmacy (Salt Lake City, UT, USA) is a member of the ISPOR special interest group on adherence/compliance. She says there has been a surge in the last five years in measuring the cost outcomes of poor adherence.

She explains that medication costs are often much less expensive than the surgical or hospitalisation costs if the medications are not taken as directed and the clinical sequelae manifest. LaFleur also says that, in the US, a consistent problem has been that, with a few exceptions, the myriad health insurance plans don’t invest in programmes to improve adherence, in part because their pharmacy budgets are separate from the medical budgets. In fact, management of pharmacy coverage is often outsourced to other companies called pharmacy benefit managers. Thus, each group (pharmacy versus medical benefits) is not considering the ramifications of their decisions on the total healthcare cost, but rather only on their specific budgets.

According to David Nash, MD, MBA, Chairman of the Department of Health Policy at Jefferson Medical College (Philadelphia, PA, USA), who recently reviewed the Archives study, the only techniques that seem to have any impact on improving adherence are “once-a-day dosing and a rigorous reminder system to patients”.

For health economists, Dr. Nash sees three main issues regarding adherence and health economics:

  • a lack of programmatic progress in improving adherence, as evidenced in the Archives-published study and the NCPIE report
  • a lack of understanding of the downstream costs of poor adherence, which are “formidable”
  • the effect of co-payment (or “cost sharing”) on adherence.

In our next HOC article on adherence, we will discuss some of the challenges to including adherence in cost outcomes analyses, including effect of co-payment on adherence, as well as the use of accurate models of adherence in cost outcomes analyses – how closely do they reflect real life data?

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