Patient involvement: A must-have in medicine development, but is it being overlooked in a cost-constrained environment?

The life sciences industry is in a constant state of advancement, bringing more and more groundbreaking medicines, cutting-edge technologies, and innovative solutions to market. Amidst these rapid changes, patients remain at the heart of these scientific developments.

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In recent decades, the life sciences industry has experienced a monumental shift toward embedding patient insights and experience throughout medicine development. It has pivoted from traditional, insular decision-making to a more open, inclusive, and collaborative approach that values insights from patient communities to guide strategy. This patient-focused perspective extends right across the product life cycle from drug discovery and development to regulatory decision-making, measuring outcomes, and defining value.

Patient engagement and its involvement in drug development has been on the rise for decades. A significant leap forward was achieved in the 1980s when people affected by HIV rallied together and created an HIV advocacy movement that established the importance of patient-driven clinical trials. The HIV community’s advocacy caused a reassessment of how much evidence is needed to gain access to potentially life-saving therapies which prompted a complete overhaul in how HIV medicines were licensed.

Fast forward to 2024, embracing patient engagement has transcended from being a “nice to have” to a “must-have” if pharma companies want to deliver innovative, relevant solutions that meet patients’ needs. Regulatory and market access changes have also been powerful drivers behind the rise in patient involvement in drug development. There is now an increasing expectation that the life sciences industry will demonstrate patient engagement as part of their regulatory submissions. Both the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) have developed guidelines on patient engagement during product development and the regulatory process. Huge strides have been undertaken outside of the pharmaceutical and biotech industry by public health ecosystems (such as the UK’s NHS and research arm, NIHR) who have truly led the way with patient involvement in health and social care provision and research.

Placing patients at the heart of healthcare not only improves patient outcomes and experience, but also adds significant value to the life sciences industry. Research has shown that drugs developed using patient-centric practices have a 19% greater chance of being launched, with 87% of drugs reaching launch compared to 68% of drugs without patient-centric designs. This finding was consistent across multiple therapeutic categories including rare diseases, oncology, and neuroscience. In fact, successful clinical trial enrolment, often a make-or-break obstacle for companies, was faster for patient-centric trials, particularly in rare diseases. Involving patients in the early stages of study design has also been shown to accelerate product launch by 2.5 years and add a financial value that is more than 500 times the initial investment in patient engagement.

While a critical part of medicine development, there are multiple barriers to effective patient engagement.

Regulations and compliance

The life sciences industry is of course tightly regulated and for good reason, given its impact on the health of billions of people. Concerns about how to compliantly involve patients can be high with significant red tape, yet increasingly in-market compliance requirements are becoming clearer as regulatory bodies recognize the significant benefit of collaborative working with patients. Following local guidelines around transparency for working with patient organizations and patient advocates, having clear and accessible contractual obligations, publicly declaring the nature of relationships between industry and patient organizations, and ensuring fair remuneration for patients involved in joint projects, are some of the key areas to consider. Ensuring the right protocols are in place for working with patients from the outset can also ameliorate any accusations of conflict of interest between for-profit and non-profit partners.


Demonstrating the value of patient involvement takes time. As the impact of decisions made in phases 1 and 2 of drug development may not be seen for many years, it requires patience to demonstrate the return on engagement. However, countless instances exist of the significance of strong advocacy relationships. A good example of value delivered by patients is that of the Parent Project Muscular Dystrophy (PPMD) group which developed the first patient advocacy-initiated draft guidance for a rare disease, after guidance created by regulatory organizations failed to meet their needs. This astounding effort from patients included a coalition of more than 80 stakeholders working across nine time zones. The outcome of this project fundamentally changed the way the FDA reviews new potential therapies for all rare diseases.

Another powerful example is the vaccination of young people against the human papillomavirus (HPV) in the UK to reduce the risk of cancer. The HPV vaccine became available to adolescent girls in the UK in 2008. Despite evidence of the benefit of vaccinating boys also, access was restricted to girls only. A sustained advocacy campaign was launched to extend the national HPV vaccination program to both sexes. The campaign was a collaborative partnership of over 50 professional and patient organizations who campaigned strongly, citing sex discrimination as well as the proven health benefits. Four years after the launch of the campaign, the UK announced that boys aged between 12 and 13 in England would be vaccinated to protect them against HPV-related cancers.

While the impact for every company may not be so dramatic, the value of patient insights and strategic partnerships can be significant, benefiting both the patient community and the company. These benefits can vary widely, from improving patient experience and reducing dropouts in clinical trials, to identifying more meaningful clinical outcomes and informing patient support programs and care pathways. Companies should ensure that objectives for advocacy are identified and tracked, setting a clear strategic scorecard for all patient engagement activities and measuring progress over time.


Budget limitations can lead to delayed or insufficient patient involvement, particularly in the early stages of clinical development. However, engaging patients early and often in an authentic manner can have a profound impact on the success of a drug’s development and its acceptance by the patient community. Companies that find themselves playing catch-up and integrating patient engagement in later stages can ultimately spend more money creating a less effective treatment.


Culture significantly impacts the success of a company’s patient engagement approach. Care should be taken to evaluate employee perceptions, beliefs, and behaviors, as well as roles, company structure, processes, and the tools that organizations use to assess how these can be more patient-focused. To be successful, executive leadership must dedicate time and resources to this focus. Leaders should identify, role-model, and reward behaviors to build a patient-centric culture.

Enablers and barriers to patient-centric behaviors should be assessed and acted upon so every employee is equipped to embed patient needs in the work they are doing. Patient engagement can be fragmented, so companies need to think about how insights can be meaningfully shared across the organization to have the greatest impact.

While great strides have been made, there are still barriers to effective patient involvement which should be tackled. Only by allowing patients to guide and shape health innovation at every stage will we be able to develop medicines that precisely serve the needs of our customers and patients. 

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