Question

How should policymakers and providers make decisions when faced with a dearth of evidence? Do you prefer a more cautious approach that does not approve procedures or drugs until evidence is available or a more aggressive approach that encourages experimentation and use of treatments that appear to be effective? What about medical care for children, who are generally excluded from clinical and research trials for ethical reasons?

Answer 1

EBP is a discourse or set of methods which informs the policy process, rather than aiming to directly affect the eventual goals of the policy. It advocates a more rational, rigorous and systematic approach. The pursuit of EBP is based on the premise that policy decisions should be better informed by available evidence and should include rational analysis. This is because policy which is based on systematic evidence is seen to produce better outcomes. The approach has also come to incorporate evidence-based practices.

There are lots of different stages to the policymaking process and at each juncture different evidence is needed. Therefore evidence does not merely enter the policymaking process at one point. The challenge is to analyse the conditions that facilitate evidence-informed policymaking (Nutley, 2003) and translate these conditions into practical tools for the governments of developing countries. Despite the challenges to creating an EBP approach, there is a general consensus that a more evidence-based approach to policy would be a positive development. We have also identified some important considerations. It is clear from the literature that:

• policy should be informed by a wide breadth of evidence, not just hard research. Key issues include the quality, credibility, relevance and the cost of the policy;

• evidence is needed, and in different ways, at a number of different points of the policy cycle;

• time constraints will affect the mechanisms available to mobilize evidence – urgent issues require different approaches than processes to develop strategic policy directions. This paper continues by suggesting a number of tools which can be used to make policy more evidence based. The tools are presented on the assumption that the reader is a progressive policymaker in a developing country, who is interested in developing policy which is more evidence based. Undoubtedly the development arena is distinct and therefore there are a number of reasons why the tools are not directly transferable. This is a work in progress and thus this is only a preliminary version of a paper, which is likely to change and develop based on comment and further testing.

b.

the introduction of new drugs because approving a bad drug (Type I error) has more severe consequences for the FDA than does failing to approve a good drug (Type II error). In the former case at least some victims are identifiable and the New York Times writes stories about them and how they died because the FDA failed. In the latter case, when the FDA fails to approve a good drug, people die but the bodies are buried in an invisible graveyard.

The result is important especially because in a number of respects, Montazerhodjat and Lo underestimate the costs of FDA conservatism. Most importantly, the authors are optimizing at the clinical trial stage assuming that the supply of drugs available to be tested is fixed. Larger trials, however, are more expensive and the greater the expense of FDA trials the fewer new drugs will be developed. Thus, a conservative FDA reduces the flow of new drugs to be tested. In a sense, failing to approve a good drug has two costs, the opportunity cost of lives that could have been saved and the cost of reducing the incentive to invest in R&D. In contrast, approving a bad drug while still an error at least has the advantage of helping to incentivize R&D (similarly, a subsidy to R&D incentivizes R&D in a sense mostly by covering the costs of failed ventures).

The bottom line, however, is that even without taking into account these further points, Montazerhodjat and Lo find that the FDA is far too conservative especially for severe diseases. FDA regulations may appear to be creating safe and effective drugs but they are also creating a deadly caution.

C

In recent decades, advancesin biomedical research have, each year, helped to save or lengthen the lives of tens of thousands of children around the world, prevent or reduce illness or disability in many more, and improve the quality of life for countless others. Beyond the infants, children, and adolescents directly affected, the benefits of research extend to the families, friends, and communities who love and care for them. Since the 1950s, for example, researchers have created vaccines against polio, measles, mumps, and a number of other childhood infections that have dramatically cut deaths, disability, and discomfort from these diseases. Children and their families have also benefited from research demonstrating the harm or ineffectiveness of what were once standard therapies, for instance, high-dose oxygen for premature infants.

Despite these advances, pediatricians and others have argued that infants, children, and adolescents have not shared equally with adults in advances in biomedicine. In particular, many drugs with potential pediatric uses have not been tested in studies that include children. These drugs may still be prescribed for children based on physicians' judgment about how data from studies with adults might be extrapolated to children. Because children differ physiologically from adults in myriad ways that can affect how drugs work in the body, extrapolation based on adult drug doses and children's weight or age can be dangerous and lead to underdosing, overdosing, or specific adverse effects not evident in adults.

• Well-designed and well-executed clinical research involving children is essential to improve the health of future children—and future adults—in the United States and worldwide. Children should not be routinely excluded from clinical studies. No subgroups of children should be either unduly burdened as research participants or unduly excluded from involvement.
• A robust system for protecting human research participants in general is a necessary foundation for protecting child research participants in particular. An efficiently administered, effectively performing system with adequate resources must, however, commit additional resources and attention to meet ethical and legal standards for protecting infants, children, and adolescents who participate in research.
• Effective implementation of policies to protect child participants in research requires appropriate expertise in child health at all stages in the design, review, and conduct of such research. This expertise includes knowledge of infant, child, and adolescent physiology and development as well as awareness of the unique scientific, psychosocial, and ethical requirements and challenges of pediatric clinical care and research.