Why understanding all aspects of the patient journey is imperative to successful clinical research

By Rosamund Round

For patients living with unmet medical needs, the development of effective therapies is critical. To ensure solutions deliver the most meaningful benefits possible, it is the responsibility of sponsors and contract research organisations to develop a complete understanding of any given condition and the literature, as well as seek an array of expert opinions. And yet, even this comprehensive approach does not provide the full picture without involvement from the ultimate end users – the patients themselves.

Patients are the experts on their disease. They understand treatment hurdles and can express which outcomes are most meaningful to them, which may not always align with those deemed to be of greatest importance by clinicians and industry. Only by including patient insights across the entire process can we efficiently mitigate risk while navigating the incredibly complex environment of drug development.

But what benefits will sponsors see by including patient perspectives in their decision-making? From informing asset planning and endpoint selection to medical communications and market access strategies, the advantages of these insights are endless.

Understanding all aspects of the patient journey is imperative to successful clinical research. However, accomplishing this requires early and continuous discussions between healthcare providers, patients and care partners to fully incorporate their voices into the drug development process. This includes learning more about patient treatment goals, their most challenging symptoms, the impact of existing treatment side effects, considerations for the whole family unit and practical barriers to trial participation, each critical to patient decision-making. Members of Parexel’s Patient Advisory Council tell us that half of the challenge of being in a trial is medical and the other half is practical. Understanding these practicalities is therefore critical to planning and implementing an effective clinical trial that proactively addresses as many barriers as possible on both fronts.

Additionally, during a study, various issues from participants must be addressed by investigators and healthcare professionals (HCPs) to maintain engagement. From asking for updated information on their disease to new data and treatment options for managing adverse events, and requests for support services, communication must be handled with care. But with continuous feedback between participants, investigators and HCPs, these issues can not only be anticipated but handled proactively, minimising, if not eliminating, interruptions in study engagement.

By seeking a true understanding of patient needs, we can not only inform each step of a sponsor’s trial design – addressing challenges earlier, allocating resources more effectively and minimising waste – but develop therapies that better meet patient needs and improve overall outcomes. Through building trust and engagement with the patient community, sponsors can incorporate these learnings from the start to advance treatments and patient access in the long term.

Products that address the unmet needs of patients may be granted accelerated regulatory review and reimbursement. Without consensus that defines these needs among regulators, payers, providers and patients, these various players can be left with conflicting priorities. However, implementing an evidence-generation plan early in the development process can fulfil the needs of these different audiences.

For example, patient-reported outcomes (PROs) are a critical tool to measure how patients feel and function without any external interpretation. While many companies invest both time and effort into collecting PROs, this information often does not appear on product labels. As a result, it’s not readily available to US providers and patients. In fact, a recent study analysing clinical review documents from the US Food and Drug Administration (FDA) for 164 novel orphan drugs and biologics approved from 2017-2022 found that just 13% of products were labelled with PRO data, even though 63% of sponsors collected it during pivotal efficacy trials.

But why does this occur? Ultimately, selecting relevant PRO tools and collecting and analysing the data with sufficient rigour for regulators can be a challenge. For example, PRO data is often aimed at the European Medical Association (EMA) rather than the FDA, which, according to a recent report, allows more PRO data on product labels because it is ‘more likely to accept data from open-label studies and broad concepts such as health-related quality of life’. Other sponsors may gather the data primarily to support European HTA agencies’ reimbursement decisions.

Still, this analysis suggests that PRO data is not utilised efficiently and needs to be more widely disseminated to patients. To ensure the scientific rigour of PROs, sponsors should consider three key practices: validate the PRO and define a ‘clinically meaningful’ threshold for change; adopt a sound, prespecified statistical analysis plan from the start; as well as designing blinded, controlled trials whenever possible. Taking these steps can help drive more holistic, patient-focused therapies.

In recent years, new guidance from the FDA and other international regulators has urged sponsors to focus on recruiting a trial population that is representative of the study population. In addition to the scientific and safety benefits of ensuring that the medication is efficacious for those who will eventually take the drug, it also helps to drive health equity. With varying access to healthcare around the world, for some, clinical trial participation is a care option that helps them receive medical treatment that may otherwise not be readily available to them. Research shows that there are a number of communities underrepresented in trials, including those from certain racial or ethnic groups, transgender patients, the elderly, those of lower socio-economic status, pregnant and breastfeeding individuals, those with disabilities and more. So, considering diversity from the earliest stages of trial design is essential for our societal health.

Incorporating a diversity programme successfully does not necessarily require significant time and investment, but when adopted early in development, sponsors can save significant long-term costs. It can help keep timelines on track, prevent costly rework and delays in market access.

Further, by accommodating the needs of all patients through deep engagement and addressing practical barriers like transportation and childcare, as well as considerations of culturally sensitive study design, such as translated materials based on the needs of the local community – we can increase engagement, minimise dropouts and facilitate an environment in which all patients are able to access research. For example, Parexel recently supported a phase 3 study for a severe autoimmune disease with the aim of maximising recruitment from African-American and Latino communities with a high disease incidence. As part of a two-pronged strategy to engage patients and sites, patient education tools with diverse representation in imagery and appropriate health literacy terminology were deployed. Transportation and childcare support were offered to patients, as well as engagement with sites through clinical enrolment managers, who map referral pathways and share best practices for their local communities. Ultimately, the study recruited 38% of study participants from ethnically and racially diverse backgrounds, exceeding the FDA’s 20% diversity target for the condition and the industry average of 12%.

Addressing inclusion in clinical trials therefore not only helps patients, but also has a significant benefit for sponsors, potentially impacting reimbursement, market uptake and overall innovation. It’s really a win-win situation.

Patient-guided drug development isn’t simply a one-time sit-down with patient advocates or an inspirational quote on a website. To achieve success, a methodical approach is recommended, which takes place from the very start of planning.

People living with rare, complex and chronic diseases each have a story to tell. In our experience, patients are delighted to share their experiences, needs and views, when they feel that they are genuinely valued and that the outcome of their communication is incorporated into study planning to help other patients. Every time I speak with a patient, I learn something new and, as an industry of scientists, it is critical to stay curious and adjust our methodology as part of continuous improvement in our approach.

By building inclusive communication strategies, effectively using patient-reported outcome data and prioritising patient inclusion, stakeholders can gain valuable insights from an early stage to ultimately optimise drug development. Making use of these patient insights, sponsors can develop commercially viable products that meet patient needs, bringing them to market swiftly to help those who need it most.

Rosamund Round is VP of Patient Engagement at Parexel