While drugs and devices will actually be used by patients, the focus of the drug development process has more often been on the requirements of regulatory bodies, the needs of the healthcare professionals and the income for the shareholders. This drives a focus on patient safety, clinical trial outcomes, patient confidentiality and return on investment, with patients receiving only limited information. This makes their only role in the study that of participant. [1] However, as patients are the real end users of the drugs and devices, drug developers need to put them at the heart of the study – this is patient-centricity.
Patient centricity, which is relevant to both drug development and healthcare, can be defined as: ‘Putting the patient first in an open and sustained engagement of the patient to respectfully and compassionately achieve the best experience and outcome for that person and their family.’ [1] This is not only about doing the right thing – which is of course vitally important – it’s also about making clinical trials more efficient and cost-effective. More efficient clinical trials mean that drugs are more likely to get through the regulatory process and onto the market, therefore improving the return on investment for biopharma companies. Drugs developed with patient-centricity in mind are more likely to be taken by patients, therefore bettering their short- and long-term outcomes. These improved outcomes help society and make healthcare systems more cost-effective.
Improving patient-centricity through clinical trial design
Getting drugs through clinical trials is a major hurdle in the route to market, as the failure rates for candidates passing through phase I to III studies can be as high as 90%. [2] One of the reasons that trials fail is participant retention, as up to 40% of participants can drop out before the trial is completed. [3] This increases the number, length or size of trials required to get sufficient high-quality data for approval.
There are a number of approaches to improving the patient-centricity in clinical trials: decentralising studies, using adaptive clinical trial designs, optimising dosing, leveraging personalised medicine and incorporating quality of life endpoints. [4]
One of the challenges to recruiting patients to clinical trials in the first place, or to retaining them through to trial completion and follow up, is the number of site visits required and the location of the sites. The time to travel to central clinical trial sites may be long, sites may be hard to access for people who rely on public transport, and there are additional challenges for older people, neurodiverse people, people from the LGBTQIA+ community and people from ethnically minoritised groups. Moving to virtual clinical trials takes away many of these barriers. Virtual studies reduce costs by cutting the need for travel reimbursement, decreasing the investment required in sites and staff and speeding up participant enrolment. Better recruitment and retention means a reduced risk of trials having to be expanded or repeated. [4-10]
The randomised controlled trial (RCT) has been the gold standard for drug development for many years. In these, patients are randomised to a treatment or control arm. However, the inclusion and exclusion criteria are often very strict, resulting in low levels of diversity in the study population, as well as data that may not represent the general population. The studies can also be lengthy, increasing the risk of participant drop-out. [11-13] Adaptive clinical trials, where researchers conduct interim data analyses throughout the study and make changes based on the results, are more flexible. As an example, a decision may be made to drop an ineffective treatment, reduce patient visits or finish a trial earlier, making the experience easier for participants. [4, 12]
Dose-finding clinical trials, particularly in oncology, can involve patients receiving the maximum tolerated dose (MTD) of a drug, which may mean that patients experience severe side effects. By optimising the dosing, patients have a better experience and researchers can still learn about the drug efficacy. [4]
Personalised medicine – treatments tailored to a patient’s genetic makeup – allows participants to receive therapeutics that ...
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