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Improving the experimental landscape
NEW YORK—With an eye on the bottom line, a new analysis of clinical research related to several FDA-approved drugs indicates that key decisions in designing a clinical trial toward a specific target have a positive impact on a product’s potential commercial success, while a separate study finds the success of a new drug is determined by patient-interactive, virtual trials performed in the privacy of their homes or offices, helping them feel more in control of their own health.
Experts from the Life Sciences Practice of the Boston-based global consulting firm Charles River Associates (CRA) presented their latest findings on targeting clinical trials toward specific patients in a poster at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Europe annual meeting in Barcelona, Spain (November 2018).
CRA analyzed clinical research programs for drugs to treat familial hypercholesterolemia, stroke prevention in atrial fibrillation and pulmonary arterial hypertension, finding that issues including patient targeting, exclusion criteria, lines of treatment, comorbidities and risk factors appear to directly correlate with different measures of success for a new drug—including price, health technology assessment (HTA) outcomes and commercial performance.
These findings indicated that payers appear to prefer higher levels of selectivity in trial design, which can allow them to more precisely assess the patient population positioned to benefit from treatment—and thus avoid the cost of a failed trial.
Leanne Finch, senior associate at CRA and one of the lead study authors, discussed the most common factors related to trial design that can affect a new drug’s chances for success.
“The study focused on the payer aspect of trial design and the impact on payer led outcomes, such as pricing and access,” Finch tells DDNews. “However, from both the doctor and patient perspective, the most crucial aspect would be relevant endpoints. Sometimes, unfortunately, there may be a mismatch between what payers perceive as relevant and what patients perceive as relevant.”
“[P]harmaceutical companies increasingly face difficulties designing their clinical trials in a way that corresponds to the needs of regulators, whilst at the same time optimizing the overall commercial success of products and minimizing costs associated with their clinical trial programs,” the CRA report states. “There is, therefore, a growing need for pharmaceutical companies to re-evaluate their trial design strategies. This study aims to assess if a broad, investment-heavy clinical trial strategy results in broader market access and improved commercial success, and to provide a set of recommendations on possible winning trial design strategies.”
The CRA study found that a clinical trial strategy that is more targeted generally yields a better market success in terms of price, HTA outcomes and commercial success than broader, bigger and less selective clinical trials. In each case investigated, the most successful product in each therapy area was the one with the more targeted approach, while a broad strategy was unlikely to pay off.
“In a broader clinical trial, this effect would be diluted due to the larger patient sample with limited potential to deliver a positive HTA recommendation,” according to CRA. “From a clinical trial design perspective, our analysis showed maximal return on initial investment can be achieved with a more targeted clinical program.”
“The effectiveness of the drug will always be the most important factor in any drug’s commercial success,” Finch says. But a well-thought through clinical trial design can stack the odds in a manufacturer’s favor. “The study we performed showed that by carefully picking your target patient population and identifying sub-populations within a therapy area, you can not only minimize the level of investment required for the trial, which is very costly, but also define a patient population that enables payers to have a better idea of ultimate budget impact, thus allowing for smoother market access and pricing negotiations and faster access of the product for patients.”
But even the most well thought-out clinical trials will fall flat if too many patients drop out early due to travel, expense, inconvenience or ambivalence.
For that reason, New York-based Transparency Life Sciences (TLS) developed its VISITOR study, designed to assess the ease of collecting clinical data directly from healthy volunteers in their homes by leveraging the Transparency Virtual Trials (TVT) digital study hub.
Top-line results, announced April 16, showed that participants were quickly able to adapt to using telemedicine devices to capture data commonly collected in clinical trials. Unlike more traditional trials, compliance in completing the virtual study visits was very high and participants reported high levels of satisfaction with the study experience.
Barbara Lindheim, founder and managing partner at BLL Partners LLC, a consultancy providing strategic communications services to the biotechnology and life-sciences industries, says TLS’ VISITOR program was primarily designed to assess participant acceptance of the TVT software platform and the telemedicine devices associated with it.
The primary endpoints were participant compliance, user experience satisfaction and data integrity, Lindheim adds. The study was successful in confirming the utility and feasibility of fielding all-digital trials with the TVT platform.
TLS co-founder and CEO Dr. Tomasz Sablinski commented, “To our knowledge, VISITOR is the first-ever clinical study integrating visit scheduling, participant screening, telemedicine and collection of safety and efficacy variables via mobile device and data management technologies.
“At TLS, we are putting patients at the center of the clinical research enterprise and reducing the burden of participation. Clinical trial sponsors benefit from greater patient engagement and retention, more representative study populations, and simplified and enhanced data capture.”
“Our TVT platform is now ready to scale to collect data globally, accommodating a variety of wearable or hand-held devices and making virtual siteless trials possible across many therapeutic areas,” he says. “We are currently collaborating with a number of sponsors interested in increasing the productivity of their development portfolios by fielding all-digital and hybrid clinical trials using our Transparency Virtual Trials platform.”
Lindheim concludes, “There is wide agreement that the current model of conducting clinical research is not working well. Enrollment and retention issues plague clinical trials, costs have skyrocketed, and timelines have lengthened. This model is not sustainable. The good news is, new digital technologies provide the opportunity to address these issues in ways that benefit patients, sponsors and the overall healthcare system. We expect that direct-to-patient digital elements will become commonplace in clinical research in the next few years, and over time, will become the norm.”