Best practice for implementation of patient reported outcomes in real world evidence generation

Maruszczyk, Konrad ORCID: 0000-0002-0173-5020 (2024). Best practice for implementation of patient reported outcomes in real world evidence generation. University of Birmingham. Ph.D.

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Abstract

Real world evidence (RWE) plays an increasingly important role within global regulatory and reimbursement processes. RWE generation can be enhanced by collecting and using patient reported outcomes (PROs). They offer valuable insights into the long-term effectiveness, safety and tolerability of treatments from the patient's perspective. Since RWE is not limited by the constraints of randomised controlled trials (RCTs), it can provide a more generalisable picture of how therapies work in real world target populations. Additionally, collecting data during routine clinical care allows researchers to reach patient groups who might be hesitant to participate in traditional clinical trials.
However, collecting PROs in real world settings presents challenges for researchers. The doctoral research constituting this thesis aimed to identify these challenges, characterise the use of PROs in RWE generation, and seek opportunities to successfully implement PROs into real world studies. A mixed-methods approach, comprising a systematic review, quantitative analysis of current practice and qualitative interviews, was adopted to address the overall aims of this research.
Firstly, a systematic review was conducted to identify and summarise existing guidance for using PROs in RWE generation. Seven publications met eligibility criteria and were included in the review. They provided some level of guidance, addressing the following issues: PROM selection, participation and engagement, burden to health care professionals and patients, stakeholder collaboration, education and training, and implementation process. My review demonstrated that current guidance is fragmented and that no international guidelines directly address the use of PROs in RWE generation.
A quantitative analysis of ClinicalTrials.gov database records followed the review. This workstream aimed to characterise the current and past use of PROs among real world studies. Descriptions of phase IV trials were searched using an automated computer algorithm to identify studies which utilised PROs. 21% of phase IV studies between 1999 and 2021 assessed PROs. A steady increase in the utilisation of PROMs in phase IV trials has also been observed in recent years. These results suggest the potential underutilisation of PROs in phase IV trials compared to earlier phases of clinical investigations.
Finally, interviews with international stakeholders were conducted to gain deeper insights and identify challenges and opportunities for collecting and using PROs for RWE generation. Twenty-three semi-structured online interviews were conducted with patients, patient advocates, regulators, payers, clinicians, academic researchers, and industry experts. While participants acknowledged the potential of PROs in RWE generation, they also expressed mixed confidence in their value. Two types of barriers hampering the full implementation of PROs in RWE generation were identified: operational and methodological.
This doctoral research has underscored the promise of PROs in the RWE generation. Nevertheless, it also emphasised the need for further research to fully unlock their potential. Currently, a limited pool of available guidance and recommendations supports the use of PROs for RWE generation. Collaborative efforts among various stakeholders are needed to establish best practices and generate practice-changing examples of its use.

Type of Work:

Thesis (Doctorates > Ph.D.)

Award Type:

Doctorates > Ph.D.

Supervisor(s):

Supervisor(s)	Email	ORCID
Calvert, Melanie	UNSPECIFIED	orcid.org/0000-0002-1856-837X
Aiyegbusi, Olalekan L	UNSPECIFIED	orcid.org/0000-0001-9122-8251
Keeley, Thomas	UNSPECIFIED	orcid.org/0000-0003-1584-9093
McMullan, Christel	UNSPECIFIED	orcid.org/0000-0002-0878-1513