Lack of comparative data on new therapeutic agents poses a challenge to health care systems and important ethical issues for patients and clinicians – Lancet Series calls for increased research into comparative effectiveness, both before and after market entry
Facing any new diagnosis, the first question many patients will ask is ‘which treatment will work best for me?’. There can be a bewildering range of options available but there is often a lack of clarity on how these compare to each other.
A push from regulators internationally to shorten the path of new treatments from development to clinic, has unintentionally exacerbated this situation. As a result there is less time and impetus for undertaking Comparative Effectiveness Research at all stages of a treatment’s development from initial approval onwards.
A series of three articles published in The Lancet explores the opportunities and challenges associated with Comparative Effectiveness Research. Under the leadership of researchers from the Department of Psychiatry, University of Oxford and the Oxford Health Biomedical Research Centre, the international team behind the publications has called for a paradigm shift to improve the evidence base available on new medicines. Professor Andrea Cipriani says: “Our evidence-based recommendations for regulatory agencies include improving product labels that accompany new drug and device approvals, limiting the use of programs that facilitate drug and device approvals on the basis of incomplete benefit and harm data, and promoting the use of ‘gold standard’ randomised trials with active comparators”.
The three papers in the series look at the role of comparative effectiveness research on new treatments before and after market entry and examine the implications for patients, doctors and the health care systems. Professor Cipriani continues: “After approval, we recommend that both industry and non-industry sponsors design randomised trials that evaluate a product’s net clinical benefit compared with current known effective therapy to address key decisional dilemmas. Regulators, governments, and research funders should limit the use of non-randomised studies, streamline patient recruitment and data collection, and invest in the development of collaborative research networks and data systems that reduce the complexity, cost, and waste of rigorous post-marketing research efforts.”
The researchers find that end-users are often left in the dark about where new treatments fit into clinical practice and the addition of biased industry interests can cause adverse economic and health consequences. Professor Ilina Singh explains “In the clinical relationship, trust, evidence and respect for patient autonomy are critical to support an ethical process of shared decision-making and personalise treatment. A lack of comparative evidence on benefits and harms means that clinicians are unable to provide patients with key information in the decision-making process.”
They conclude regulatory agencies need to provide the necessary incentives for generating comparative data and assuring availability of evidence that is useful for decision making.