Patient-focused care is no longer a niche concept; it is increasingly central to healthcare and, crucially, the pharmaceutical landscape. With patients being directly engaged throughout the drug development process, the industry is moving towards genuine and meaningful collaboration. By partnering with patients, caregivers and the public, pharmaceutical companies can design and market treatments that address the real unmet needs that matter most to patients.
Although the many benefits of patient engagement* are clear and widely recognised, its full potential is not always realised due to the upfront time and financial commitment needed for its successful implementation. A growing body of evidence shows that patient engagement improves communication, fosters trust and supports regulatory decision‑making, leading to better market access and tangible economic benefits. In short, patient engagement is not only valuable for patients; it also makes commercial sense for pharmaceutical businesses.
*In this post, mentions of “patients” generally refer to patients and their families, carers and the public. Patient-focus and patient engagement are used interchangeably, to refer to: activities in which patients are the key focus (e.g. development of materials for patients to provide accessible information or meet patient-defined needs) and/or are actively involved in.
Patient perspectives during early product development can guide research priorities, highlight key unmet needs, refine trial concepts and ensure trial endpoints reflect patient priorities.1–6 This supports identifying new niches for market differentiation and targeting products not just to symptoms, but also to lifestyles and treatment priorities.3, 4
Patients are best placed to identify which treatment goals are most beneficial to them and can inform project-funding decisions at the earliest stages of development. Without robust patient input, products risk underperforming, or even failing, at launch.
The inhaled insulin example offers a cautionary tale: marketed for convenience, the product struggled to gain patient acceptance because dosing, maintenance and preparation were cumbersome versus modern pen‑like injectors. While early preference surveys were conducted, they were biased toward participants with favourable views and comparisons with outdated injection approaches.6 Engaging patients in a comprehensive, unbiased manner throughout the development process could have highlighted these convenience issues earlier, guiding improvements and potentially avoiding poor market reception.
When patients are involved early and consistently, every downstream step in the development process – from study design to market differentiation – is strengthened.2
At the clinical trial stage, recruitment hurdles, protocols misaligned with patient needs and participant burden can derail timelines and inflate costs. Patient organisations can catalyse trial funding through venture philanthropy,7 and patients can provide input into protocol design, consent materials and eligibility criteria to improve trial relevance and the patient experience, leading to more successful drug development.
The benefits are clear:
Listening to and involving patients in clinical trial design ultimately saves pharmaceutical companies time and money, and can lead to earlier patient access.
Patient‑focused communication improves understanding of study findings and treatment information, boosts clinicians’ confidence in discussing or prescribing new options and accelerates product adoption because patients can more easily engage in their care. Patients are increasingly seeking out information about their treatment and when accessible, high‑quality science is missing, misinformation fills the gap.
In the UK, 43% of adults struggle with health literacy, rising to 61% when numeracy is involved.11 Low health literacy is the strongest correlate of ill health, underscoring the need for accessible scientific communication.12, 13 In a 2019 study comparing study summary formats, participants reported significantly higher perceived understanding and a clear preference for video abstracts or plain‑language writing versus graphical or published abstracts.14 Articles with features like infographics or video abstracts not only help patients; they also travel further. In one analysis, articles with video abstracts had the highest average accesses per year, followed by those with a graphical plain-language summary (PLS).15 The benefits of PLSs also extend into clinical practice:
Improving patient understanding and countering misinformation in turn reduces medication‑related harm, supports adherence and helps more patients become aware of appropriate treatment options – driving home the value of producing patient-focused materials.18
Regulatory bodies increasingly expect that the evidence provided by pharmaceutical companies reflects patient priorities and demonstrates real‑world relevance.19–24 Many HTA bodies also formally recommend inclusion of health-related quality of life valuations and patient impact statements, as well as encourage patient participation in HTA decisions (see examples of key patient-focused initiatives below).3, 25 Furthermore, in the European Union, the Joint Clinical Assessment guidance explicitly suggests that patients should be involved in the assessment process.26 A shift from patient engagement to patient collaboration within the Health Economics and Outcomes Research community was also clear in the discussions taking place at ISPOR Europe 2025, with a demonstrated agreement that healthcare decision-making should be patient‑focused. It is clear that pharmaceutical companies should be engaging with patients to enhance their regulatory and HTA submissions and improve their chances of success. The task now is meaningful execution and putting promise into practice: collaborating with patients early and generating disease‑specific qualitative and quantitative evidence, so that patient experiences can truly be reflected in decisions.
For practical advice, we have previously shared our thoughts on how to use patient-focused approaches to enhance value dossiers, supporting the development of a comprehensive and compelling narrative for market access processes.
The FDA Patient-Focused Drug Development Guidance aims to incorporate the patient voice into regulatory decision-making.19
“When we engage patients, their families and caregivers, throughout the medical device clinical investigation process, we will have more patient-centric clinical investigations and ultimately timely access to innovative, safe and effective medical devices.”
– Statement on FDA efforts to encourage patient engagement in medical device clinical investigations20
The MHRA Patient Involvement Strategy 2021–25 aims to encourage pharmaceutical companies and research teams to harness the power of the patient voice.21
“When we launched our Patient Involvement Strategy in 2021, I made a commitment to ensure the patient voice was heard and understood at every step of the regulatory process.”
– MHRA Patient Involvement Strategy: An Assessment of Progress22
The EMA Engagement Framework is designed to promote patient involvement in regulatory processes throughout all stages — from early development, through benefit-risk evaluation, to post-marketing surveillance.
“EMA is committed to ensuring that the patient voice is included in the different regulatory activities of a medicine’s lifecycle, which improves the quality of and trust in the regulatory decisions and in new medicines placed onto the EU market.”
– EMA Engagement Framework23
The NICE Public Involvement Policy aims to ensure that the public can contribute to developing guidance and standards to enhance the focus and relevance of NICE’s products for those directly impacted by their recommendations.
“Involvement of patients focused on the humanity of the topic and an orientation to patient experiences.”
– Chair of NICE guideline development, on the importance of involving patients, carers and the public in discussions of independent committees and working groups developing NICE guidance24
Public trust in pharmaceutical companies is one of the biggest issues the pharmaceutical industry faces, as it can undermine participation in trials, treatment adherence and acceptance of effective interventions, such as vaccination mandates.28, 29 Failing to build that trust could have devastating impacts on the pharmaceutical industry as a whole. Patient‑focused trial design and clear health communication can build health literacy, which in turn can increase trust and loyalty among patients and HCPs, and support market success.3, 30–32
When surveyed, more than half of 12,017 patients said that knowing a company actively works with patients might positively impact their trust in said company, and 51% of doctors reported they were more likely to prescribe medications from a patient-focused company.33, 34 Furthermore, a staggering 93% of 2,346 respondents in a variety of pharmaceutical roles held the belief that the integration of a patient-focused strategy improved overall business outcomes.35
Adopting a patient‑focused approach can motivate the industry and build brand reputation while reducing treatment adoption barriers, thus connecting research to its real‑world impact and purpose.
Ultimately, innovation does not translate into impact without sustained adoption and adherence in routine care. Non-adherence can be catastrophic for some patients; in some disease conditions, more than 40% of patients experience harm due to misunderstanding, forgetting or disregarding medical advice.31
Patient engagement initiatives can ensure that new treatments are not only clinically effective but are also embraced in real-world settings. By involving patients in drug development and research communication, companies gain insight into practical barriers and preferences that shape real‑world use, resulting in higher treatment adherence.31, 36–38 Patients also experience a direct benefit, as engaged patients are more likely to follow prescriptions correctly, reducing complications, hospital visits and emergency admissions.38 Health literacy interventions, when delivered by a range of professionals using diverse methods including pictograms, workshops and online education, have been shown to improve health‑literacy‑related outcomes in older adults, improving patients’ ability to access, understand and use health information.37
From a commercial standpoint, improved treatment adherence and persistence can reinforce real‑world effectiveness and impact, protecting long‑term revenues.39
Patient engagement delivers commercial benefits across the pharmaceutical product lifecycle. It drives patient‑guided product development, accelerates clinical trial timelines, strengthens narratives for regulatory and HTA decision-making, broadens the reach of scientific communications and supports sustained adoption. Embedding patient perspectives—whether through direct engagement or by applying a consistently patient‑focused mindset—aligns decisions with real‑world needs and reinforces the commercial sustainability of pharmaceutical innovation.
Ultimately, without patient engagement, we develop less impactful treatments that lead to worse commercial and patient outcomes. Put patient needs at the heart of the pharmaceutical development process and watch both outcomes and commercial value climb.
If you would like any further information on the themes presented above, please get in touch, or visit our Patient Engagement page to learn how our expertise can benefit you. Imogen Brooks (Analyst Intern), Lucy-Paige Willows (Analyst), Sonia Shaw (Senior Analyst) and Trishna Bharadia (Patient Advocate and Patient Engagement Professional) created this article on behalf of Costello Medical. The views/opinions expressed are their own and do not necessarily reflect those of Costello Medical’s clients or affiliated partners.
