A recent systematic literature review of randomized controlled dermatology-related trials showed that patient-reported efficacy outcomes (PROs) were mentioned in some form in only 25.6% of 125 trials between 1994 and 2001. Our research aimed to characterize the benefits of PROs in drug development in dermatology from the patient, prescriber, regulator, payer, and manufacturer perspectives using a case study approach. The case studies were identified based on the use of PROs in pivotal clinical trials for the product.
A targeted literature review was conducted in PubMed from 2004 to 2014 for six products (Atopiclair for atopic dermatitis, botulinum toxin type A for hyperhidrosis, calcipotriol plus betamethasone dipropionate gel for scalp psoriasis, pimecrolimus and tacrolimus for atopic dermatitis, and ustekinumab for psoriasis. Regulatory and health technology agency websites and publications were searched for documentation of PRO label claims and mentions.
For patients, inclusion of PROs ensured the full benefit of the product was demonstrated, including improvement in symptoms, quality of life, and/or treatment satisfaction. For prescribers, comparative trials reported PRO data information on each product’s benefits and risks and also which product was superior from the patient perspective. For regulators, for all except one of the six products, PROs were included in the product label. For payers, utility values based on PROs were used in cost-effectiveness evaluations for three of the six products. For the manufacturer, the PRO data generated label claims and many publications that allowed extensive public dissemination of product benefits.
Patient-reported assessment of the treatment impact on disease during drug development has many benefits for all stakeholders.
Benefits of patient-reported outcomes in dermatology drug development