What is patient voice?

One of the central tenets of patient centricity is that patients’ own insights and experiences have value and should be listened to and acted upon by healthcare systems, providers, and the pharmaceutical industry. At Prime Global, we sum this up as the desire to ‘amplify the patient voice’.

It’s a term we hear a lot, ‘Patient Voice’, from industry and wider healthcare – it is the name of a website where patients in the UK can provide feedback on care providers, a large project run by the Food and Drug Administration (FDA) in the US, an Australian initiative to incorporate patient perspective in the health system, and so on. As a result, ‘patient voice’ has the potential to be lost as a ‘buzzword’. Can we look more deeply at the value of the patient voice to see its true worth? 

It can be a useful exercise to reflect on this and pressure test the concept by finding real cases – just what value is there to pharma and patients in amplifying the patient voice? What evidence is there that engaging with and listening to patients can expedite approval of treatments, or improve access to a product, service, or device? Is there any way to measure the influence and impact of the patient voice? 

The problem of N=1

In 2003, medical sociology professor Lindsay Prior wrote, “For the most part, lay people are no experts. They are, for example, rarely skilled in matters of (medical) fact gathering, or in the business of diagnosis. What is more, they can often be plain wrong about the causes, course and management of common forms of disease and illness.” (1) Through a more recent patient-centric lens, this is quite a confronting read. It’s easy to put this down to changing times and trends, but we can still see instances of this viewpoint occurring.  

A blog released in 2021 in the Postgraduate Medical Journal, for example, wrote of an interesting interaction between an advisor and student at the University Medical Centre of Utrecht, who had just finished listening to a patient keynote speaker. When asked by the advisor what they thought about the moving presentation, the student replied, “I don’t know. The only thing I can think of is N=1.” She went on to explain that her teaching had led her to view individual patient stories as ‘always biased’. (2) 

So, how do we understand the value of the “N=1” patient insight? The good news is that there is plenty of evidence to demonstrate the value of the patient voice, not just for each individual patient but for the systems and industries that those patients are engaging with. For example, a 2013 study demonstrated that patient experience, as recorded in National Health Service (NHS) settings in the UK, is positively associated with clinical effectiveness and patient safety, concluding that clinicians should resist sidelining patient experience as “too subjective or mood oriented, divorced from the ‘real’ clinical work of measuring safety and effectiveness.” (3) 

For Pharma, it’s in the industry’s interest to engage with patients to ensure they’re health- and trial-literate, so that the most informed voice possible can be provided by patients. This has implications on trial design, product development and launch, and post-launch issues such as adherence. All such aspects have cost implications attached, which can be alleviated by effective engagement. And this is where the ‘voice to value’ equation is most demonstrable. (4) 

Hearing the patient voice

There are many ways in which the patient voice can be heard and amplified. Some of these are direct, face to face interactions, while others are closer to raw data gathering en masse. At research and development level, we see tools such as patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs) adding depth and colour to the clinical datapoints by capturing data about hard-to-measure things such as headaches, fatigue, depression, anxiety, etc. PROM use is still limited, however (5), and their usage alone often puts too much emphasis on symptoms without a wider context. This is where PREMs offer some promise, with a more experiential angle. (6) 

Medical and commercial pharmaceutical divisions, meanwhile, employ tools such as patient advisory boards, and patient case studies at congresses and live events.  

Big data, wielded well, can represent the ‘voices’ of many patient populations and sub-populations. ‘Citizen science’ is a way to involve large numbers of people in research, such as using a Fitbit to collect activity data, then develop products around them that bring the greatest benefits. (7) ‘Self-tracking’ devices, worn by an individual, are hugely popular and collect health data that can often then be sold to pharmaceutical companies and used to develop new health programs or initiatives – proving that N=1 research can, when collated, provide real value.  

Examples from pharma and healthcare

Let’s look at some specific examples where pharmaceutical and healthcare organisations have embraced the concept of ‘amplifying the patient voice’ and seen benefits.  

In 2014, headlines were made when the FDA (and their Project Patient Voice scheme) suggested a novel primary endpoint to support migraine drug approvals: allowing patients to identify their most bothersome migraine-associated symptoms beyond pain. This can be viewed as an effort to incorporate the patient voice to recognise the varied presence of symptoms and truly emphasise patient-focused drug development. (8)  

Several pharmaceutical companies, meanwhile, have set up formal internal systems for including the patient voice in their activities. Roche have a ‘Patient Partners’ system of working with patients and patient groups, which aims to work in partnership “at each stage of drug development and care delivery.” (9) In Huntington’s Disease, for example, Patient Partners directly contribute to the guidance and strategy of the HD disease research programme. (10) This included seeking patient input on all aspects of study protocol. Another key ‘voice amplification’ action by Roche is the funding and organising of The International Experience Exchange with Patient Organisations (IEEPO), a global platform of engagement for the patient community. IEEPO reflects the belief that patients and patient groups need a platform for equal dialogue, to share their voices with each other as well as industry.  

In 2017, AstraZeneca launched a global patient partnership programme to engage patients in drug development. It uses patient advisory boards to ‘make science work for patients.’ (11) The programme covers many aspects, from drug development to disease management solutions and patient education. “It has fundamentally changed the way our researchers think about developing medicines,” says Helena Chung of AZ. (12) 

Merck and Pfizer are among others following a similar approach, getting patient input via workshops or advisory boards before protocols and trial designs are completed. 

Patient feedback can, clearly, lead to some important changes in trial design, which could result in improvements to recruitment and retention. For example, patients involved in MSD’s advanced cancer programme highlighted that a proposed comparator drug had already been tried by many patients earlier in their disease and so wouldn’t want to risk being randomised to it in an upcoming trial. (12) 

Demonstrating value in Research and Development

It is in the area of trial recruitment and retention that we often most clearly see the demonstrable value of integrating the patient voice into the industry’s activity, by helping to identify ways to overcome barriers to enrolment and retention. It has been estimated that a US$100,000 investment in patient engagement in the design of a trial could produce a probable value 500 times that figure. (12)  

Pfizer have demonstrated the value of incorporating patients into the designing of trials, reportedly cutting recruitment times by 37%; reduced time = reduced cost. (13) By involving patients in trial design, they are “making them far more interesting and attractive for people to get involved in”. Furthermore, they return the data back to the participants after the trial, which will encourage them to be involved in more activities and improve health literacy. 

We can find tangible examples of times where listening to patients aided trial retention, too. In 2019, Janssen had patients carry out mock visits to trial sites, which highlighted the need to shorten the length of patients’ visits and provide transportation. It worked – a year into the trial and no one had dropped out, compared with an average attrition rate of around 18%. (14) 

With 80% of trials facing difficulties with recruitment and around 30% of participants dropping out during a trial, there is clearly a need to make protocols more comfortable and relevant to patients. this can be done by listening to patients and implementing the required changes. (15) (16) 

Ultimately, the value of doing so is immense for drug developers. The Tufts Center for the Study of Drug Development (CSDD) ran a simulation to find out what would happen if a single change in trial protocol was made: asking for patient input to support design feasibility, as well as improving recruitment and retention. While this would cost the researcher $100,000, the payoff could equate to an increase in the value of the drug under development by more than $60 million over its commercial lifetime. (17) 

Reduced time to get drugs to market

Incorporating the patient voice at early stages can speed up the time to market. (4)(18) As well as higher chances of ethical approval, drugs developed with patient-centric trial design have been shown to  have 19% more Phase II and Phase III likelihood of launch compared with drugs developed in “standard” trials, and  are also more likely to be adopted by payers. (19) (20) 

Interestingly, however, only 5.2% of Phase II and Phase III trials actually use a patient-centric approach, suggesting there are still a lot of opportunities to benefit in this area. (21) 

Downstream value

Early patient engagement can have benefit downstream by saving money and/or time at the clinical and commercial stages of drug development.  Including patient insights in study planning can be done for as little as $10,000 – a drop in the ocean compared with the protection these insights provide against factors that could delay a trial by months. (22)  

Continuing the patient engagement model through to market access, patient support programmes may improve adherence by up to 29% and cut disease-related medical costs by 35%. (23) 

In a 2015 survey, 86% of pharma executives agreed with the statement “a focus on patient centricity is the best route to future profitability”. (24) However, as we have seen there is still room for improvement in the number of trials being developed with patients.  

The onus is not only on the pharmaceutical industry to amplify the patient voice. Patient advocacy groups play an incredibly important role in elevating patient needs and perspectives for both pharma and healthcare and have had demonstrable success in advocating for the changes they want to see. For example, when MS drug Mavenclad was not reimbursed for prescription, patient advocates lobbied regulators and managed to achieve reimbursement for the drug.  

Conclusion

Responsibility for amplifying the patient voice rests on many shoulders and is recognised as important by many stakeholders. Healthcare systems and pharmaceutical companies, patient advocacy groups, and many dedicated organisations such as Patient Focused Medicines Development (PFMD), Clinical Trials Transformation Initiative (CTTI), FDA, European Medicines Agency (EMA), and so on.  

Open and respectful discussions with patients and patient organisations can lead to an exchange of ideas that can result in better trial design, more relevant endpoints and outcome measures, and improved recruitment and retention of a more diverse patient population. Not only can amplifying the patient voice in this way lead to drugs that better address patient needs, improving outcomes and patients’ experiences on those therapies, but it will also demonstrably lead to more value – both monetary and reputational – for the companies involved.   

To hear more from the Prime Patient team, contact PatientEngagement@primeglobalpeople.com

Recommended blog post: How to make 2022 the best year yet for patient engagement 

References

1. Prior L. Belief, knowledge and expertise: the emergence of the lay expert in medical sociology. Sociol Health Illn. 2003;25:41-57.
2. Cox C. The patient voice: a biased or valuable source of information? Postgraduate Medical Journal, BMJ. May 2017.
3. Doyle C, Lennox L, Bell D. A systematic review of evidence on the links between patient experience and clinical safety and effectiveness. BMJ Open 2013;3:e001570. 
4. Kersey O. Oncology R&D: the patient insights we’re still missing, and how to catch up. PharmaPhorum Oncology. 2021. [Accessed at: https://deep-dive.pharmaphorum.com/magazine/oncology-2021/oncology-rd-patient-engagement/] 
5. Sacristán JA, Aguarón A, Avendaño-Solá C, et al. Patient involvement in clinical research: why, when, and how. Patient Prefer Adherence. 2016;10:631-640. Published 2016 Apr 27.
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7. Wiggins A & Wilbanks J. The Rise of Citizen Science in Health and Biomedical Research, The American Journal of Bioethics, 19:8, 3-14. 2019. 
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10. Roche. [Accessed at: https://www.roche.com/stories/elevating-the-patient-voice-in-huntingtons-disease] 
11. Patient Partnership Program. AstraZeneca. [Accessed at: https://www.azpatientpartners.com/] 
12. Wilkinson E. Are patients the missing piece in the drug development puzzle? The Pharmaceutical Journal. 2018. [Accessed at: https://pharmaceutical-journal.com/article/feature/are-patients-the-missing-piece-in-the-drug-development-puzzle] 
13. Pfizer. Patient Engagement and Involvement in Research and Development. 2019. [Accessed at: https://cdn.pfizer.com/pfizercom/Patient-Engagement-and-Involvement-in-RnD-FINAL-09.19.pdf] 
14. Brown C. Patient-Centric Approach in Clinical Trial Design: Involve Patients Early. 2020. [Accessed at: https://www.anjusoftware.com/about/all-news/insights/patient-centric-approach] 
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17. 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. Therapeutic Innovation & Regulatory Science. 2018;52(2):220-229. 
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19. Whitepaper. The Innovation Imperative: The Future of Drug Development Part 1: Research Methods and Findings. A report by The Economist Intelligence Unit. 2019. [Accessed at: https://druginnovation.eiu.com/wp-content/uploads/2019/05/Parexel-innovations-in-drug-development-part-1_V14.pdf] 
20. du Plessis D, Sake J-K, Halling K, Morgan J, Georgieva A, Bertelsen N. Patient Centricity and Pharmaceutical Companies: Is It Feasible? Therapeutic Innovation & Regulatory Science. 2017;51(4):460-467. 
21. Round R, Williams K. Achieving Patient Centricity in Clinical Trials: Uncovering the Mystery. Clinical Researcher. Nov 2018; 32 (9). 
22. Finnegan G. New medicines 19% more likely to launch when trials are co-designed with patients. PFMD.org. 2019. [Accessed at: https://patientfocusedmedicine.org/new-medicines-19-more-likely-to-launch-when-trials-are-co-designed-with-patients/] 
23. Brixner D, Rubin DT, Mease P, Mittal M, Liu H, Davis M, Ganguli A, Fendrick AM. Patient Support Program Increased Medication Adherence with Lower Total Health Care Costs Despite Increased Drug Spending. J Manag Care Spec Pharm. 2019 Jul;25(7):770-779. 
24. How patient-centric is the pharma industry? PDD innovation. 2016. [Accessed at: https://www.pddinnovation.com/how-patient-centric-is-the-pharma-industry/]