Utilizing all available data sources to optimize clinical trial design and planning, as well as adopting novel trial formats, such as decentralized trials, telehealth and mobile health applications, can get us closer to that holy grail: conducting a fast, successful clinical trial – and even reduce cost.
Conducting a fast, successful clinical trial is the holy grail of drug development: the ultimate but very elusive goal.
The statistics around drug development, especially clinical trials are sobering. A study conducted by the Institute for Safe Medication Practices (ISMP) found that the median cost of pivotal phase III trials was US$48?million. In addition to overall cost, the study also looked at cost drivers and found that the largest single cost driver was the number of patients required for the study (average estimated cost per patient: US$41,413) followed by the number of trial clinic visits (estimated median cost of each patient visit to the study clinic: US$3,685). Their model showed that trial costs rose exponentially with these two variables.
To make trial recruiting even more difficult, more than 60% of patients don’t meet the eligibility criteria of any given study and a comparable percentage declines participation leading to delays in 80% of trials with 20% being delayed for six months or more.
No wonder then, that drug developers are looking for ways to streamline clinical trials and to improve the factors they can control.
Let’s take a closer look at the controllable factors.
Working with the Right Sites and Principal Investigators
Site and PI selection are critical because they heavily impact how quickly and successfully patients can be enrolled. Sites should have relevant patient populations and experienced PIs who not only see these patients but also have referral networks they can tap into. Quick enrollment avoids delays — another costly factor — as well as underpowered studies, which have limited statistical power and only allow the sponsors to draw limited or no conclusions and, worst, might expose participants to risks without providing meaningful knowledge.
Conduct Trial with Diverse Patient Populations
Increasing diversity of patient cohort, e.g. to include more women, racial and ethnic minorities, older patients and other underrepresented groups is not only an industry commitment but, as of November 2020, FDA guidance. While FDA guidance documents are not legally binding, they have to be taken seriously, because, unsurprisingly, the FDA tends to follow them. Increasing diversity of trial participants relies on a detailed understanding of where underserved communities with high incidence rates live so site can be selected accordingly.
The ability to identify healthcare providers that work within these underserved communities, understand their concerns and have earned their trust is important. These healthcare providers are uniquely positioned to successfully enroll underserved patient populations if provided with convincing arguments how participation benefits their patients.
Understand the Competitive Landscape and Trends
Understanding the environment in which a trial happens is critical to success. Having answers to questions like “Are there similar or competing trials ongoing and where?”, “Are our preferred PIs involved?”,” What successful and failed trials have been conducted in the past?”, “If they failed, why?” can help streamline design and plan of clinical trials, avoid delays, and hone in on relevant patient populations, site and PIs.
Comprehensive, Integrated Data Facilitate Study Planning
Common to all of these controllable factors is, that the more data a trial sponsor has, the more proactive they can be in addressing them. Relevant data streams include:
- up-to-date information on clinical trials,
- claims and referral data,
- detailed profiles of prospective PIs, e.g. clinical trial experience, current trials they are involved with, referral network, number and impact of their publications as well as their payment history, which can raise flags about engagement with a competitor.
- comprehensive site information, e.g. research impact, comparison to similar institutions, size, referral network, and patient populations they serve.
However, siloed data streams don’t solve the problem either. Disparate data streams need to be integrated and incorporated with the proprietary information the company has collected over time to create a single source of truth that stakeholders across the organization can utilize.
Decentralized Trials Address Key Cost Driver
With the number of clinic visits per patient being one of the key cost drivers of a clinical study, reducing that number can significantly lower the cost of a trial. Telehealth visits cost about half of clinic visits and health monitoring using wearables, while incurring the cost of the electronic devices, save personnel time which more than makes up for these expenses.
What’s more, frequent clinic visits are often a deterrent for patients, especially those who live far away, are very sick or can’t take time off for economic reasons. Reducing the burden of trial participation by offering telehealth, mobile health or in person visit in a closer, decentralized facility can therefore contribute to faster and more successful enrollment and retention and with that reduce clinical trial cost.
Data and Decentralization – Two Key Factors for Successful Clinical Trials
Clinical trials will always be time-consuming and expensive as drugs and medical devices need to prove their efficacy and safety in a large group of patients over long periods of time. However, utilizing all available data sources to optimize clinical trial design and plan as well as adopting novel trial formats, such as decentralized trials, telehealth and mobile health applications, can get us closer to that holy grail: conducting a fast, successful clinical trial – and even reduce cost.