Findings from a recent Economist Intelligence Unit (EIU) report¹ indicate that the use of patient-centric clinical trials—that is, trials that are designed and conducted with the patient perspective in mind—can accelerate enrollment, increase the likelihood of drug launch, and ultimately facilitate inclusion in payer formularies. Yet, patient-centric trials have not been widely adopted (they represent only 5.2% of Phase II and Phase III clinical trials conducted globally from 2012-2017), and adoption rates are particularly low in China.¹

In China in particular, there is a significant opportunity to expand the use of patient-centric trials. The Chinese clinical research market is growing thanks to regulatory reform, the Government’s interest in healthcare and patient demand for better medical care and medicines. Patient centricity in clinical trials may help support innovation and the introduction of novel medicines to the Chinese market and to patients who need them.

Focused on the development of innovative new therapies to improve the world’s health, Parexel recently organized a roundtable discussion in Shanghai on patient-centric approaches and how various stakeholders can more effectively bring the patient into the drug development process. Participants from pharmaceutical and biotech companies, hospitals, a government agency, a research institute, and a patient advocacy group (PAG) discussed topics that included policy, culture, processes, technology, and other enablers of patient-centric clinical trials.

This article illustrates both the opportunities and the challenges surrounding patient centricity and patient-centric trials in China based on this roundtable discussion and related considerations, including Parexel’s own experience.

What is patient centricity?

There is no universally accepted definition of patient centricity, though most sponsors describe it in terms of putting the patient at the heart or center of clinical research.  The EIU report defines a “patient-centric trial” as one that involves patients in its design and execution. ¹ The ultimate aim of a patient-centric trial is to make it as easy as possible for patients to learn about and participate in clinical trials by reducing barriers, and to generate clinical data that reflect the real needs of patients and not only those perceived by doctors and pharmaceutical companies. In turn, this can have a positive impact on recruitment, retention, patient experience, compliance, and, therefore, budget and timelines.

Around the globe, experience with patient-centric trials is growing. Adoption of these innovative trials, however, is still in its infancy. Today, 38% of patient-centric trials are conducted in the US, while 34% are performed in the EU. In China, just 5% of trials are patient-centric.¹ While barriers remain, enabling factors also exist to improve adoption.

Two case studies illustrate how sponsors are overcoming barriers to adoption across the globe.

Case study 1: Phase II, Serious Neuropsychiatric Condition (Parexel, data on file)

Situation

The sponsor wanted to take a patient-centric perspective on protocol design by eliciting feedback from patients and caregivers. The sponsor was faced with two options:

a) A 12-month study with a compulsory lumbar puncture (LP)—resulting in a lower visit burden, but with an uncomfortable procedure.

Or

b) An 18-month study with a choice for sites between LP and PET scan—the result being a higher visit burden but with the potential to avoid the difficult procedure and with an option for a 12-month extension study if the drug was efficacious.

Challenge

The sponsor was uncertain on which approach was preferable to patients to ensure compliance with the protocol.

Solution

Parexel deployed Patient Centric Protocol Optimization (PCPO) methodology and found that

patients, caregivers, and site staff overwhelmingly preferred the longer duration study with the higher visit burden, in order to avoid the difficult procedure and the subsequent stress and anxiety that it would cause in this vulnerable population, in addition to the potential to participate in the extension study. The sponsor was, therefore, able to make an informed choice about its final protocol to ensure a truly patient-centric approach.

Case study 2: Phase III, Serious Pediatric Neurological Condition (Parexel, data on file)

Situation

The study was designed to explore a possible treatment for very young children with a serious neurological condition. Our client was asked by the FDA to include in their protocol design a requirement for three 48-hour or 72-hour videos of EEG monitoring in the hospital.

Challenge

The sponsor was concerned that recruitment of patients into the trial would be unachievable due to the time commitment required from parents and their children.

Solution

Parexel deployed two of the four possible elements of the PCPO service:

1. One was a caregiver survey, which identified and directly surveyed parents of eligible children to understand the current burden of treatment, their potential interest in a clinical trial, and the possible impact of 72-hour video EEG monitoring.
2. The other was a Patient Burden Analysis (PBA), which surveyed expert nurses who were asked to provide feedback on the study design.

Surprisingly, the caregiver and PBA surveys showed that the visit burden was in line with the standard of care for patients. The majority were hospitalized around three times per year for approximately three days at a time.

An unexpected finding was that the highest barrier to participation cited by the majority of parents was lack of childcare for their other children while they stayed with their participating child for longer visits.

As a result, the sponsor was given recommendations to help sites overcome the childcare situation as part of their recruitment and retention plan, such as on-site childcare facilities and scheduling these longer visits on weekends.

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