So much has changed in the clinical research space in the last 20 years. Shifting regulations and growing pressures to cut costs while shortening time to market has had a cascading impact on every phase of the development lifecycle – especially Early Clinical Development (ECD).

In the early 2000s, the ECD/Phase I space was booming, as pharmaceutical companies believed in pushing as many molecules as possible through Phase I studies hoping to get potential molecules for further development. But as the cost of development skyrocketed this model began to lose its appeal. According to ISR’s 2016 Phase I Study Trends report, 64 percent of companies cite the cost of research as a leading external force that could cause them to conduct fewer Phase I studies in 2020.

In response, biopharma companies became more selective with a targeted approach, pushing fewer molecules into ECD.

Traditionally, these Phase I studies have been conducted entirely in healthy volunteers – unless the drug was too toxic to warrant their participation. But in an effort to cut time from the development lifecycle, many biopharma companies are pursuing hybrid models, in which both healthy volunteers and patients participate in these early phase trials. We’ve seen the numbers steadily shift from predominantly healthy volunteers, to more hybrid trials in recent years. According to the ISR report, almost one-fourth of Phase I studies will include a combination of healthy volunteers and patients by 2020, up from 17 percent today.

These hybrid models address the growing complexity of the molecules being developed, and the need to deliver more efficient drug development cycles. By involving patients in early phase trials, biopharma companies can begin tracking the impact of the drug on the targeted population to more rapidly develop early proof of concept.

The challenge of course is getting patients to participate.

The elusive Phase I trial patient

Because the goal of a Phase I trial is to get safety and pharmacokinetic data of the drug with less tangible benefits to subjects than later phase trials, there is little upside for patients to take part. That isn’t to say that recruiting patients for this level of research can’t be done, but pharma companies need to recognize the multiple challenges they will face recruiting for hybrid studies, and choose clinical research partners who have experience running Phase I trials in both healthy volunteers and  patients, which isn’t always an easy combination to find.

When trials only involve healthy volunteers, they can usually be conducted in a single site by staff who are well-versed in operating such trials. But hybrid studies are different and require partners who are adept at recruiting both healthy subjects and targeted patients for small rapidly-delivered Phase I trials. That means choosing a CRO that can tap into an established global network of sites to recruit and conduct early phase studies in the targeted patient population. They also need to be sure that these sites have the necessary infrastructure, staff and training to accommodate the needs of patients and are managed with the appropriate level of oversight to safely and efficiently deliver ECD/Phase I studies.

Biopharma companies should also note that in most cases, hybrid or patient-based early phase trials may require multiple sites, and can take more time to recruit than one involving only healthy volunteers.

When biopharma companies are vetting partners to run these trials, they should look for organizations that have demonstrated ECD/Phase I experience and expertise. Specifically, they should consider the partner’s experience conducting successful early phase trials, expertise in the specific disease category, access to high class sites and patient populations and an understanding of what it takes to  successfully, ethically and safely deliver Phase I trials.

As hybrid and patient-based studies become a more common aspect of the early clinical development process, having partners who can execute the unique requirements of these projects is vital. Building these relationships today can help biopharma companies generate the most value from their early phase studies, and more rapidly bring new drugs to market.

Topics in this blog post: Early Clinical Development