Do regulatory agencies approve new medicines earlier and with less data today compared to before? This is a question that has been raised during recent years, but is there some truth in this? The basis for regulatory approval of a new drug is that positive benefit-risk profile has been proven. This decision is based on the Agency´s review of the products quality, efficacy and safety documentation. Today, many of the new products are developed to treat patients with diseases for which there are no or rather ineffective treatments available. So, regulatory decisionmakers must balance the need for evidence versus the need for new treatments for a potentially serious and potentially life-threatening disease. It is just not possible to collect data from thousands of patients when the new treatment is targeting a small patient population in which large scale clinical trials are not doable.

New medicines are by default be approved with some uncertainty, but the important question is when there is sufficient information to confidently take the benefit-risk decision. What “unknowns” can be or must be answered after approval when the product is in use? This is the challenging decision regulators must make. Building more knowledge in the larger patient population following the approval, not limited to the inclusion criteria in the Phase III studies is therefore the key element in successful drug development. The legislation offers important tools for regulators to reassure that information is gathered for a long time after the approval either through specific studies or through active surveillance activities. Registries, in which the result of the treatment is documented, are of specific interest.

Thus, it is a misconception that regulators have changed their “standards”. But a larger uncertainty will be accepted when the potential benefit of the product is high. It is however true that today’s scientific landscape comes with new challenges not only for the regulators but also for HTA-agencies and payors. Therefore, it is important that these decision-making bodies increase their collaboration and provide solutions for how knowledge and information can be shared for the benefit of the patient. To answer the question, regulators must navigate the new research landscape and still make firm benefit-risk decision, while at the same time develop the regulatory toolbox. And we must not forget the upside; new treatments are developed for patients in great need for effective medicines. That is good!