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The Evolution of Patient Power

Writer's picture: Clarinda CerejoClarinda Cerejo

Updated: Nov 4, 2024


Introduction

I was 25 when I was diagnosed with a debilitating rare disease called neuromyelitis optica spectrum disorder (NMOSD).

As a science communication professional trying to make sense of the bizarre feelings in my body and the overwhelm in my mind, my recourse was to look for what the research had to say about my condition. But research for my rare disease was hard to come by, and doctors who were not comfortable saying they didn’t know enough chose to dismiss my questions.

Without realising it, the first time I advocated for myself as a patient was when, at 25, I bypassed those dismissive doctors, sought out one of the country’s leading experts in the field, and went to him with a stack of research papers, demanding that he speak to me as an intelligent being who could understand research, treatment choices, probabilities, and outcomes. This experience set me on an unexpected path; little did I know that my medical journey that began 15 years ago coincided with the broad timeline when the concept of ‘patient centricity’ started taking off in the pharma industry.

 

The emergence of patient power

Although campaigning for patient-centred medicine began with the pioneering advocates who emerged throughout the HIV/AIDS crisis of the 1980s,[1] before 2010 or so, pharma companies only had to engage with healthcare professionals (HCPs) to see their products receive the desired market penetration.[2] Once HCPs were convinced, the treatment would be prescribed, and patients wouldn’t dream of challenging this decision because ‘the doctor knew best’.

But a wave of change was coming. What was once an exceptional level of involvement with research and care started to become commonplace, as the Internet, social media, and smartphone technology began to give patients easier access to medical information than ever before.[3] Just like the 25-year-old girl in India, they were asking questions, they were seeking treatment options, they were forming groups with other patients online, they were telling doctors that it was their body and their choice, and they were keen to share their lived experiences and priorities so that new drugs or studies might better meet their needs. Some advocates even sought technical training to elevate their advisory capabilities further.

The power balance was shifting towards patients, and pharma had to start seeing them in a new light – not as passive recipients of medicines, but as essential stakeholders in the research and development of new treatments.

 

Evolution and evidence

In 2006, the UK government published a new national health research strategy, calling for patients to be “involved in all stages of the research process: priority setting; defining research outcomes; selecting research methodology; patient recruitment; interpretation of findings and dissemination of results.”[4]

By 2008, the term ‘patient centricity’ had become popular in medical literature, with over 500 uses of the term in PubMed.[5] In 2012, Leonard Kish, a health information technology consultant, made the bold statement, “Patient engagement is the blockbuster drug of the century.”[6] Around the same time, the US Food and Drug administration (FDA) introduced the Patient-Focused Drug Development (PFDD) initiative, officially recognizing the importance of the patient voice in pharma.[7]

In 2014, the European Medicines Agency (EMA) launched a pilot project through which patients would be involved in risk-benefit assessment of medicines in its Committee for Medicinal Products for Human Use (CHMP).[8] In 2016, the Japan Pharmaceutical Manufacturers Association (JPMA) started a taskforce for “Drug Development with Patient Voices.”[9]

Between 2010 and 2020, ‘patient centricity’ and ‘patient engagement’ became popular industry buzzwords both online and in conference halls the world over. As per a search of PubMed, the number of papers published containing the term “patient engagement” increased from 72 between 2000 and 2010 to 2710 between 2010 and 2020 (and thousands more since then)

People were asking: What is patient engagement? Why is patient engagement important? What are some strategies to empower and engage patients? And the big question – what is the return on investment for patient engagement? (If you’re curious to hear the answers to these questions, check out the ‘Voice 2 Value’ studies on the ‘LEARN’ area of the ISPEP Hub)

Gradually, all stakeholders (including funders and regulators) joined the patient engagement revolution. In 2021, the Pharmaceuticals and Medical Devices Agency (PMDA), the regulatory authority for pharmaceutical affairs in Japan, published a Guidance on Patient Participation to help embed patient voices in its operations.[10] In 2022, the International Coalition of Medicines Regulatory Authorities (ICMRA), supported by the EMA, US FDA, and Health Canada, published a joint statement on international collaboration for real-world evidence (RWE) generation to support regulatory decision-making.[11] Also in 2022, funders, regulators, and research organizations in the UK signed a novel shared commitment to improve public involvement in research.[12]

As these milestones passed, patient advocacy groups were rising to the occasion and occupying a larger share of voice in pharma. As a result, emerging data began to tell a story that reflected the strong, measurable, and irrefutable return-on-investment that is achieved when patients are engaged in the drug development process – for example:

  • Research questions, hypotheses, interventions, medical technologies, and outcomes become more relevant and usable for patients[13]

  • Research design becomes more appropriate, inclusive, and sensitive, with higher trial satisfaction ratings[13,14]

  • Patient-centric trials have shorter recruitment times (4 months vs 7 months on average for the first 100 participants)[15]

  • New medicines are 19% more likely to receive marketing approval when trials are co-designed with patients[15]

As the power of patients and the value they add became more obvious globally, I was studying patient engagement for my thought leadership role in a medical communications agency. It had been 12 years since my diagnosis, and the more I read and wrote about the value of patient engagement, the more strongly I felt that my career background and my lived experience were together steering me towards a career dedicated to patient engagement. In 2022, I quit my job to explore my new calling. In 2023, I completed the EUPATI Patient Expert Training Programme. Then 2024 brought something totally new…

 

Where are we now?

This year, over 700 diverse patient engagement professionals from all over the world – including patients, patient advocates, industry representatives, and solution providers – have come together in non-competitive spirit to form the International Society for Patient Engagement Professionals (ISPEP), a first-of-its-kind professional society for all those who bridge the gap between patients and pharma. I was excited to join this Society as an early member, and my hope is that through the work of ISPEP and the broader patient engagement community, the power of patients will be recognized yet further, with patient authorship on research papers and patients as members of regulatory and health technology assessment (HTA) committees becoming standard practice.

Before the rise of patient power, there was little support for the value of patients’ unique lived experiences in informing pharmaceutical development. However, with years of expertise and evidence now behind us, patients and industry partners today frequently work side by side. Indeed, the launch of ISPEP shows us how far patient engagement has come; now, the world is ready for patients, industry executives, solution providers, and more to come together in a non-hierarchical, united, global community – a community where patient engagement is not only practised, but also systematically elevated.

ISPEP is about to show just how far patient power can take us. Are you ready?


References

  1. Active involvement of patients in drug research, evaluation, and commercialization – European Perspective. Houÿez. 2004. The Journal of Ambulatory Care Management https://journals.lww.com/ambulatorycaremanagement/abstract/2004/04000/active_involvement_of_patients_in_drug_research,.10.aspx 

  2. Factors influencing prescribing decisions of physicians: A review. Davari et al. 2018. Ethiopian Journal of Health Sciences.  https://www.ajol.info/index.php/ejhs/article/view/186869 

  3. A systematic review of healthcare applications for smartphones. Mosa et al. 2012. BMC Medical Informatics and Decision Making. https://bmcmedinformdecismak.biomedcentral.com/articles/10.1186/1472-6947-12-67 

  4. Policy paper - Best research for best health: a new national health research strategy. Gov.UK. 2006. https://www.gov.uk/government/publications/best-research-for-best-health-a-new-national-health-research-strategy 

  5. The evolution of patient engagement. Society for Clinical Research Sites. 2021. https://myscrs.org/wp-content/uploads/2021/03/2021.01-Patient-Engagement.pdf

  6. Patient engagement is the blockbuster drug of the century. Forbes. 2012. https://www.forbes.com/sites/davechase/2012/09/09/patient-engagement-is-the-blockbuster-drug-of-the-century/#7a2d2f8d5638 

  7. FDA-led Patient-Focused Drug Development (PFDD) public meetings. US Food and Drug Administration. https://www.fda.gov/industry/prescription-drug-user-fee-amendments/fda-led-patient-focused-drug-development-pfdd-public-meetings#:~:text=In%202012%2C%20the%20U.S.%20Food,and%20their%20currently%20available%20treatments.

  8. Partnering With Patients in the Development and Lifecycle of Medicines: A call for a action. Hoos et al. 2015. Therapeutic Innovation & Regulatory Science. https://pmc.ncbi.nlm.nih.gov/articles/PMC4616907/

  9. Enhancing patient engagement in clinical trials by pharmaceutical companies and patients in Japan. Kitamura. 202. DIA Global Forum. https://globalforum.diaglobal.org/issue/july-2021/enhancing-patient-engagement-in-clinical-trials-by-pharmaceutical-companies-and-patients-in-japan/ 

  10. Pharmaceuticals and Medical Devices Agency Guidance on Patient Participation. 2021. https://www.pmda.go.jp/files/000243407.pdf 

  11. ICMRA statement on international collaboration to enable real-world evidence (RWE) for regulatory decision-making. 2022. https://icmra.info/drupal/sites/default/files/2022-07/icmra_statement_on_rwe.pdf 

  12. Shared commitment to public involvement. National Institute for Health and Care Research. 2022. https://www.nihr.ac.uk/shared-commitment-public-involvement

  13. Evaluating the “return on patient engagement initiatives” in medicines research and development: A literature review. Vat et al. 2020. Health Expectations. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6978865/ 

  14. Patient engagement initiatives in clinical trials: Recent trends and implications. Sine et al. 2021. Therapeutic Innovation & Regulatory Science. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8182995/  

  15. The Innovation Imperative: The future of drug development. Part I: Research methods and findings. The Economist Intelligence Unit. 2019. https://druginnovation.eiu.com/wp-content/uploads/2019/05/Parexel-innovations-in-drug-development-part-1_V14.pdf  

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