The Growth Of Phase IV Trials And The Need To Demonstrate Effectiveness Using Electronic Patient Reported Outcomes

Patient centricity is now at the forefront of drug development, from early understanding of Burden of Disease through to Comparative Effectiveness Research and the use of Real World Data to generate evidence on the patients’ perspective of outcomes and quality of life. With the rise in patient centricity, we see the growth for the need of Patient Reported Outcomes (PRO) and associated technologies allowing these outcomes to be collected and leveraged to meet the needs of multiple stakeholders including Patients, Patient Advocacy Groups, Payors and Pharma.

Rapid growth in the development of innovative therapies and medicinal products over the past several decades has seen a rise in the development of Advanced Therapy Medicinal Products (ATMPs), including Gene and Cell Therapies as well as other complex molecules, and with this there has been fast changes to regulatory requirements. Today, convincing regulators of a product's safety and efficacy to obtain marketing authorisation is no longer the only barrier to ensuring accessibility to medicines for patients.

Pharmaceutical companies must now satisfy the value perceptions of multiple stakeholders, including payers, to attain market access for products. Regulatory approval is an essential and necessary step in market authorisation, but Market Access is no longer guaranteed1. This paper therefore seeks to address and describe the growing need to satisfy the value perception of multiple stakeholders and the importance of Late Phase trials capturing Clinical Outcome Assessments (COA) and specifically electronic Patient Reported Outcomes (ePRO) in relation to this.