Treatment of neuropathic pain (NP) is challenging. Interest in real-world evidence (RWE) for benefit-risk assessments of NP treatments increases given the paucity of drugs showing efficacy in randomized controlled trials and restricted labels of available medicines. To provide further context, a literature review regarding regulatory use of RWE and a clinical trial registry search for randomized controlled trials over the last 10 years was carried out. Taken together, and especially for available NP treatments, there is increasing support to consider RWE when evaluating their benefit-risk profile. Examples are provided in which RWE could be used effectively for updating the product label and informing treatment recommendations. Collected and analyzed according to state-of-the-art standards, RWE can inform treatment recommendations and product label decisions.
Keywords: benefit-risk assessment; labeling guidelines; neuropathic pain; real-world evidence; treatment guidelines.
Neuropathic pain (NP) is caused by damage to the sensory part of the nervous system and is often described as burning, throbbing or shooting pain. This condition is difficult to treat and may become chronic. Before a new treatment can be approved for use, its effectiveness and safety must be shown in controlled clinical trials. Such trials are difficult to conduct in NP and often fail. Therefore, there is increasing support for the use of real-world data (routinely collected data from e.g., patient registries, electronic medical records, health insurance claims databases) to evaluate the benefits and risks of treatments. This article presents the views of three pain specialists about the value of real-word evidence in general and specifically for the evaluation of pharmacological treatments of NP.