Why medicines regulators should start a 'Drug Evidence Watch' process
BRUSSELS - New indications for existing drugs are often missed, as new evidence does not automatically gets picked up by medicine regulators. In a publication in The Lancet, we argue that regulators should consider creating a new pathway, called a Drug Evidence Watch: a process for label extension that would start as soon as new evidence emerges. This could unlock potential treatments for patients in need.
Drug regulators, such as the European Medicines Agency (EMA), are constantly looking for any danger signals arising from the use of medicines and medical devices. As soon as there is a signal that there is unexpected toxicity associated with a drug, regulators can act – ranging from issuing updated instructions for prescribers and patients, to restricting the use of a drug in a given condition or even withdrawing a drug completely.
However, the regulators are not looking for when there is a signal that a drug might be of benefit in a different disease to the one it is approved for. This means that patients may be missing safe and effective treatments using drugs that are already approved, a process called drug repurposing.
A new paper by the Anticancer Fund, together with Professor Ian Roberts of the London School of Hygiene & Tropical Medicine, and published in the leading medical journal The Lancet, draws attention to this issue. The authors highlight concrete examples showing that there is already strong evidence supporting new medical uses for some drugs, but that these uses are not currently licenced.
Today only the company that produces the drug can initiate a label extension
Furthermore, the authors point out that only the company that produces the drug can apply for approval to use the drug in a new disease. In cases where the company is not interested there is no mechanism in place to amend the approval, known as a label extension. This contrasts with the danger signals example, whereby the regulator has the power to change the drug approval without the company asking for it.
Currently, many companies do not want to file for label extensions of their drugs for new diseases because of data and/or financial concerns. The data concerns arise when the evidence of effectiveness has been produced independently of the company – yet it is the company that has to take responsibility for it in order to apply for the label extension. The financial aspects involve both the direct costs of applying for the extension, and also the indirect costs associated with producing the evidence dossier required and/or the costs of generating additional evidence. Simply put, it may not make financial sense from the company’s perspective to seek the updated approval.
We propose a solution, so patients will not miss potential treatments
The Lancet article goes further than just bringing attention to the issue – it proposes a novel solution. It suggests that a ‘Drug Evidence Watch’ process is brought into play which will look at both safety and efficacy signals for approved medicines. When data emerges suggesting a new use for an existing drug, the regulator should synthesise the evidence, working with clinicians, academics, and patients, to produce all the information required to file an application for approval in the new disease. In effect the regulator will make the process of applying for the label extension as straightforward and as low-cost as possible to induce companies to seek approval for the new diseases – ultimately to the benefit of patients and public health systems alike.