Author: Melissa Newara, Senior Director, Subject Matter Expertise
As drug development unfolds, a new frontier in clinical trial design is taking shape. Competition is stiff. Being first-to-market matters more than ever. And as participant expectations shift, weaving patient centricity into the fabric of clinical trials becomes increasingly important.
Patient Reported Outcomes (PRO) help to incorporate the “patient voice” into drug development and evaluation – a key priority for the US Food and Drug
Administration.[1] PROs achieve this by capturing myriad outcomes directly from participants, without interpretation by clinicians, that would typically be excluded from traditional clinical trial endpoints.
Yet, not all methods of PRO collection are created equal, and the path you choose to venture during the early phases will have lasting repercussions as trials progress. Read on to discover how harnessing ePRO in early-phase trials can boost patient centricity, create more cost-effective operations, and help generate better data faster.
Driving Patient Centricity
Medrio ePRO uniquely ensures the secure collection of PRO from any location on any device. This helps sponsors to better understand participants’ experiences by gaining direct insights from participants. This boosts patient centricity, which isn’t just good news from an ethics standpoint by enabling the delivery of improved patient care but makes commercial sense too.
Unlike paper-based PRO diaries, which are inconvenient for participants and sites, subject to error and misinterpretation, and easy to forget, ePRO keeps participants more engaged by placing the engagement in the palm of their hands and mobile device. The result: Participant burden reduces while compliance and retention rates soar.
Understanding the participant experience early on – while reviewing dosing and other critical components – is vital. This is especially important for early-phase oncology trials, where improvements in participants’ quality of life and any additional lifespan provided by study drugs are key measures.
The Cost Conundrum
By early 2020, sponsors reported that approximately 65 percent of their studies[2] included ePRO or e-diaries. However, ePRO is commonly associated with later-phase trials and is often (incorrectly) considered cost-prohibitive for small, early-phase studies.
It’s essential to understand the origins of this misconception to derive the most value from ePRO during the early stages of research and obtain a competitive advantage in the process. The reality is that there are many hidden costs of paper, which include costs associated with storage, distribution, data quality, patient compliance, data accuracy, transcription, source data verification and delay when studies conclude.
To analyze the true study lifecycle cost/benefits of ePRO v paper, download our recent eBook: Facts and Misconceptions about ePRO Cost-Effectiveness.
Better Data Faster
Experts argue that incorporating PROs into early-phase trials can:
- work to optimize PRO measures[3]
- lead to more rigorous and systematic assessment of PROs in later-phase trials[4]
- differentiate products with similar efficacy but different tolerability profiles
- support early identification of efficacy and safety profiles to inform the recommended phase II dose[5]
However, this can only be meaningfully achieved if data quality is prioritized, which isn’t a strength of paper PRO collection. Paper PRO collection increases the likelihood of protected health information becoming subject to transcription errors, form misplacements, and data inaccuracies. This can be detrimental to the success of smaller studies, including early phase and rare disease trials.
In contrast, by allowing patients to report from anywhere at any time, ePRO increases participation. Flexible, automated notifications, robust survey tools, and EDC integration provide sponsors with more secure, accurate, and complete data faster.
This facilitates a competitive advantage for sponsors of early-phase studies by allowing them to prioritize data quality and scale up more quickly and efficiently. Likewise, sponsors are empowered to make more informed treatment decisions that support enhanced quality of life and better health outcomes for participants.
References:
[1] Chalasani M, Vaidya P, Mullin T. (2018, April 02). Enhancing the incorporation of the patient’s voice in drug development and evaluation Research Involvement and Engagement. Retrieved February 07, 2023
[2] Lamberti, M. J., Smith, Z., Dirks, A., Caruana, T., Mitchell, T., & Getz, K. A. (2022, July 27). The Impact of Decentralized and Hybrid Trials on Sponsor and CRO Collaborations. Applied Clinical Trials Online. Retrieved September 15, 2022.
[3] Basch E, Dueck AC. Patient-reported outcome measurement in drug discovery: a tool to improve accuracy and completeness of efficacy and safety data. Expert Opin Drug Discov. 2016;11(8):753–8.
[4] Wagner LI, Wenzel L, Shaw E, Cella D. Patient-reported outcomes in phase II cancer clinical trials: lessons learned and future directions. J Clin Oncol. 2007;25(32):5058–62.
[5] Fiteni F, Ray IL, Ousmen A, Isambert N, Anota A, Bonnetain F. Health-related quality of life as an endpoint in oncology phase I trials: a systematic review. BMC Cancer. 2019;19(1):361.
