Patients Reported Outcome (PRO) data provide information on patient health that is obtained directly from the patient.
PRO measures serve to report the patient’s description of their symptoms, their satisfaction with treatment, or how a disease or treatment affects their physical, mental, emotional, spiritual, or social well-being. When reported electronically during clinical trials, electronic Patient Reported Outcomes (ePROs) bring significant and proven value to data quality, including data accuracy, legibility, attribution, and timeliness.
These benefits contribute to enhancing the overall value of clinical trial data, and as such, increase the chances of success of a clinical trial. Although a substantial body of evidence from the scientific literature has convinced the scientific community and regulators of the value of ePRO data, other benefits of incorporating electronic Clinical Outcomes Assessments (eCOAs) into clinical trials are less obvious and warrant further discussion.
In this article we describe the key role of eCOA in market access, not only through the collection of high-quality data but also through its utility in demonstrating the therapeutic value of a new molecular entity. We illustrate this through a real case study.
Beyond clinical data: how can PROs support market access for new molecules?
During clinical trials, PROs are the only way of capturing patients’ direct input regarding their feelings and symptoms. As such, PRO measures allow for a more in-depth evaluation of the patient experience in the assessment of the efficacy and safety of a new drug. Additionally, these PRO data provide a lever for market access of new treatments.
ePRO to strengthen the demonstration of therapeutic value
By capturing the patient voice directly, PROs bring depth and relevance to trial outcomes. They augment other clinical data (eg, clinical, imaging, and laboratory results) by generating additional evidence on the impact of a new treatment on patient health and quality of life. By capturing symptoms, feelings, and information on functional status, PRO data that are based on endpoints that really matter to patients help to better characterize the safety profile of a new molecular entity and improve the evaluation of efficacy . Ultimately, when properly implemented, this enhances the scientific value of the research.
ePRO to generate more solid regulatory dossiers
- Standardized and precise data collection
As regulatory authorities place particular importance on the quality of clinical data reported by patients to evaluate the therapeutic value of medications, reducing biases and errors that are associated with traditional methods has strong value. Electronic data collection of patient data through ePROs and eDiaries ensures the data reliability that is crucial to demonstrate the efficacy and safety of treatments to health authorities.
- High compliance
One of the underlying strengths of eCOA data is the high compliance rates it generates. eDiaries and ePROs have been shown to result in compliance rates as high as 90%, which makes them the best way to mitigate the threat of missing data. Electronic reporting of aspects such as symptoms, feelings, fatigue, social activity, ability to work, daily functioning, coupled with the capacity to monitor the potential need to use concomitant medication during a clinical trial, have made ePROs and eDiaries indispensable for the reliable demonstration of the value of a new treatment in regulatory submission dossiers.
eCOA to support pricing and reimbursement negotiations
Pharmacoeconomic studies are used to compare the costs and outcomes of different therapeutic interventions and are essential for pricing and reimbursement decisions. eCOA data are used in Cost-Effectiveness Analysis (CEA), which quantify whether an intervention provides a significant additional benefit compared to its extra cost. eCOA data are also used to calculate quality-adjusted life years (QALYs) scores in Cost-Utility Analysis (CUA). These analyses allow the evaluation of both the quantity and quality of life gained with a particular intervention.
CEA and CUA encourage the development of new therapies that are not only clinically effective but also economically viable. As such, eCOA data are used to influence payers’ decisions and contribute to improving reimbursement and pricing criteria.
eCOA to optimize post-marketing surveillance plans
By strengthening post-marketing data, eCOA supports post-marketing analyses. After the licensing of a new product, eCOA facilitates continuous patient monitoring, through the collection of long-term efficacy data and rare adverse events. In this way, eCOA contributes to the implementation of risk management plans.
Adding PROs into clinical trials not only provides solid data to support the therapeutic value of a new entity, but also allows detailed health economic analyses that help to determine a fair price for new therapies. By allowing the broader evaluation of patient well-being to better characterize the therapeutic benefit, and by helping to quantify the trade-offs between costs and benefits, eCOA data have become crucial to payers’ negotiations.
Case Study
The challenge: health authorities were to evaluate the cost of a new treatment compared to standard of care (vitamin K antagonists).
The data collection:
- ePRO data provided a regular follow-up on patient quality of life using EQ-5D-5L data and the SF-36 scale.
- An assessment of perceived side effects (bleeding, fatigue) to compare the new treatment with existing options was possible through daily collection of eDiary data.
The measures:
- Short term: thromboembolic and hemorrhagic events reporting through eDiaries allowed measurement of the impact of the new treatment on therapeutic adherence by patients, as well as providing help with hemorrhagic risk management.
- Longer term: the evolution of the occurrence of side effects allowed assessment of long-term patient adherence to the new treatment.
- ePROs allowed measurement of the self-perceived patient quality of life (QoL) when using the new treatment versus the standard of care.
The results:
eCOA data showed that:
📌 By reducing the associated side effects, the new treatment improved treatment adherence (+30% compared to the standard of care).
📌 The lower incidence of hemorrhagic side effects resulted in fewer hospitalizations (-20% compared to the standard of care).
📌 An improvement in quality of life was perceived by patients (versus the standard of care), which contributes to long-term maintenance of patient adherence to treatment, ultimately leading to increased efficacy.
These results justify a higher price, as the medication reduces indirect costs related to complications.
