Patient perceptions are key to ensure optimal treatment outcomes in rare disease

February 2018


Over recent years there has been a flurry of oral medicines developed for diseases for which previous there were only intravenous treatments. Several rare disease have been the beneficiaries of these innovations.

Doctors have long had a ‘blind spot’ for non-adherence in their own patients, especially as it is the perception of the patients regarding their medicines that is the most important factor in determining adherence. It is not in anyone’s interest if these innovations fail to achieve their potential efficacy due to poor adherence and poor persistence. Especially as these populations of patients with rare diseases do not often have much choice regarding their medication.

So what perceptions might really matter in this situation. Patients have a perception that infused medicine are more potent. Feedback of sensations related to a treatment have a direct impact on patients’ perceptions of the efficacy of a medication. This perceived efficacy is a strong predictor of adherence and satisfaction with the perceived efficacy of a treatment directly contributes to long-term continuation of that medicine (persistence).

For rare diseases, these medications are often extremely expensive. Leaving these perceptions to chance does no-one any good, and worse, risks an inappropriate payer and prescriber view that the medication does not work, due to a hidden adherence problem.