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Patient insight-led drug development: Ensuring patients are at the centre of clinical trial design

The journey of drug development is a step-by-step process, offering numerous opportunities to harness invaluable patient insights and shape future treatments to best align with their needs. Although patient-centricity is well-established in healthcare, it is critical that we, as industry professionals, integrate these perspectives into clinical decision-making to advance the patient experience of illness.

The first step in ensuring clinical trials are patient-focused and accessible to all is gathering diverse patient feedback. An effective way to hear and understand patient views is through building partnerships that foster trust between patients and pharmaceutical companies. Specific approaches for collaboration include focus group discussions with local patient communities, surveys, and exit interviews. (1,2) By conducting engagement activities, healthcare providers can collect extensive patient insights throughout the clinical development lifecycle. This real-world data may include patients' experiences with the study design, the progression of their condition, their quality of life, any burdens related to their treatment, disease, or the clinical trial, their treatment preferences, and their perspectives on the benefit-risk ratio of the treatment. (3-6)

Patients' unique expertise can be incorporated into clinical decisions related to the development of novel therapies and diagnostics. Healthcare professionals face a range of decisions in clinical settings, such as designing the trial protocol, selecting the treatment arm, assessing health and safety risks, determining which biomarkers to evaluate, and identifying clinical outcome assessments, among others. (7-9) These medical decisions often lack a clear ‘right or wrong’ answer, as what works for one patient may not work for another. Therefore, clinicians must carefully weigh the advantages and disadvantages of each option to ensure that patient needs and values are considered inclusively. (10) Fortunately, technological advancements have resulted in the creation of clinical decision support systems: ever-evolving tools that empower clinicians to make better-informed decisions in both clinical development and patient care. (11)

Implementing patient-guided decision-making in clinical development plans has profound benefits for all stakeholders. Placing patients at the core of treatment development not only encourages true patient-centricity in our healthcare systems, leading to better patient-reported health outcomes, but also offers various advantages for pharmaceutical companies. Firstly, incorporating the patient voice into healthcare research positively impacts clinical trials by enhancing patient recruitment and retention while aligning research aims and study endpoints with patient priorities, ultimately resulting in the development of therapies that effectively address their needs. Furthermore, adopting patient-led decision-making in clinical trials boosts the financial gains of pharma companies; the likelihood of successfully launching an investigational drug is greater, the time to market is reduced, and there is an overall enhanced return on investment. (12)

As healthcare professionals, it is our responsibility to ensure that patients’ lived experiences are considered in clinical decision-making. Future research should, therefore, focus on developing a systematic approach to involve patients seamlessly in clinical development plans. Some progress has already been made, as demonstrated by TransCelerate Biopharma Inc.’s Patient Experience Initiative, which explored frameworks to promote early patient collaboration in the drug development lifecycle. Nevertheless, there is still a long way to go. (13)

At Synthesis, our team of specialist medical writers is committed to driving meaningful change for patients by ensuring their voices are amplified across the industry and translated into tangible action. Contact us at info@synthesishealth.co.uk to learn how we can support your partnerships with patients and their advocates throughout the entire drug development process, providing valuable insights to guide your decision-making in clinical settings.

References

  1. Patient Engagement: Technical Series on Safer Primary Care, World Health Organization 2016 https://iris.who.int/bitstream/handle/10665/252269/9789241511629-eng.pdf

  2. Matza LS, Stewart KD, Landó LF, Patel H, Boye KS. Exit Interviews Examining the Patient Experience in Clinical Trials of Tirzepatide for Treatment of Type 2 Diabetes. Patient. 2022;15(3):367-377. doi:10.1007/s40271-022-00578-8

  3. Lucas R, Wang SF, Riley J, Pepper J, Cowie M. Patient experience in clinical trials: results of a survey. Eur J Heart Fail. 2018;20(3):612-614. doi:10.1002/ejhf.1105

  4. How can the value of patient engagement be quantified and measured? https://pharmaphorum.com/patients/how-can-value-patient-engagement-be-quantified-and-measured

  5. Kleykamp BA, Dworkin RH, Turk DC, et al. Benefit-risk assessment and reporting in clinical trials of chronic pain treatments: IMMPACT recommendations. Pain. 2022;163(6):1006-1018. doi:10.1097/j.pain.0000000000002475

  6. Masri HE, McGuire TM, Dalais C, van Driel M, Benham H, Hollingworth SA. Patient-based benefit-risk assessment of medicines: development, refinement, and validation of a content search strategy to retrieve relevant studies. J Med Libr Assoc. 2022;110(2):185-204. doi:10.5195/jmla.2022.1306

  7. Choodari-Oskooei B, Thwin SS, Blenkinsop A, Widmer M, Althabe F, Parmar MK. Treatment selection in multi-arm multi-stage designs: With application to a postpartum haemorrhage trial. Clin Trials. 2023;20(1):71-80. doi:10.1177/17407745221136527

  8. Risk Considerations, Queen Mary University of London https://www.jrmo.org.uk/performing-research/conducting-research-with-human-participants-outside-the-nhs/risk-considerations/

  9. Califf RM. Biomarker definitions and their applications. Exp Biol Med (Maywood). 2018;243(3):213-221. doi:10.1177/1535370217750088

  10. Krist AH, Tong ST, Aycock RA, Longo DR. Engaging Patients in Decision-Making and Behavior Change to Promote Prevention. Stud Health Technol Inform. 2017;240:284-302.

  11. Chen Z, Liang N, Zhang H, et al. Harnessing the power of clinical decision support systems: challenges and opportunities. Open Heart. 2023;10(2):e002432. Published 2023 Nov 28. doi:10.1136/openhrt-2023-002432

  12. Levitan B, Getz K, Eisenstein EL, et al. Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI's Patient Groups and Clinical Trials Project. Ther Innov Regul Sci. 2018;52(2):220-229. doi:10.1177/2168479017716715

  13. TransCelerate Biopharma Toolkits Core Team, Elmer M, Florek C, et al. Amplifying the Voice of the Patient in Clinical Research: Development of Toolkits for Use in Designing and Conducting Patient-Centered Clinical Studies. Ther Innov Regul Sci. 2020;54(6):1489-1500. doi:10.1007/s43441-020-00176-6