Bringing clinical trials into the home

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Lowering the costs of drug development is more important than ever; with the increasing pressure on drug pricing and billions in funding poured into small biotechs, competition is fierce. While the latest reports have estimated the cost to bring a single drug onto the market at $ 2.7B, a look at the R&D expenditure of big pharmaceutical companies, and the numbers of their approved drugs, implies that even this estimation is pretty optimistic. Optimising clinical trials is key to solving these issues, as they count for over 80% of the costs of drug development.

“The amount of wasted resources in traditional clinical trials is staggering. From underperforming sites, to screening failures and patient drop-outs; all end up causing trial delays and an enormous increase in cost. This means that there are great opportunities for innovation” – Tarek Roustom, V-Bio Ventures

The early bird that went hungry: Pfizer and the REMOTE trial

In recent years, pharmaceutical companies have showed an increased interest adding more agility and efficiency to clinical trials by bringing the trial to the patient. Back in 2011, Pfizer courageously initiated their ambitious REMOTE trial: the first randomized clinical trial where patients could participate entirely from home. Using a string of technological tools, Pfizer aimed to conduct all aspects of the trials remotely: patient recruitment, screening, identity verification, consent signing, drug delivery and data reporting. Realizing that their expectations were too lofty, Pfizer halted the trial in 2012 due to a lack of participants.

Picking up where Pfizer left off

Taking the lessons of the Pfizer trial into account, a growing number of start-ups, CROs, and big enterprises have continued down this path; they have developed tools to enable the success of the ‘site-less’ clinical trial model. Most of these companies are offering specialized technical products designed to tackle one aspect of the trial at a time. However, some companies like Medidata, ClinPal, Medable etc. are offering more holistic platforms that cover the trials from start to finish: from patient recruitment, education and screening, to site management and data collection. Their mobile platforms decrease the number of patient travels to the clinical trial site, while facilitating the collection of reliable and structured electronic datapoints. However, not all indications are amenable for remote trials without any involvement of medically trained staff.

Companies who manage to integrate the latest technology and regulatory requirements into a patient-oriented platform are likely to attract keen attention from pharma industry and life science investors alike. – Tarek Roustom, V-Bio Ventures

Some companies, like Science 37, are offering fully site-less clinical trials even for those indications requiring physical interaction with patients. They do this by combining a fully-fledged mobile platform with telemedicine and home visits by nurses. With over $70Mil in funding, the 4-year-old start-up Science 37 has succeeded in setting up collaborations with Novartis, Sanofi and UCB, to run a number of home-based trials in various indications including neuroscience, dermatology and even oncology.

The benefits of a remote trial

While some may argue that the expense of sending nurses to patients’ homes, and having doctors available for telemedicine consultations, will not lead to lower trial costs, remote trials provide many other advantages that nonetheless result in huge savings for the pharma sponsor. A remote trial model significantly improves convenience for participating patients and, therefore, increases the number of potential study participants. This speeds up the recruitment process and ultimately shortens the duration of the clinical trial itself. Furthermore, remote trials provide a more diverse and representative patient population, and improve patient compliance and retention, which has been a major factor for trial delays and failures worldwide.

The rise of digital biomarkers and passive monitoring

The amount of wasted resources in traditional clinical trials is staggering… there are great opportunities for innovation. – Tarek Roustom, V-Bio Ventures

In the pursuit of innovating clinical trial efficiency, the increasing usage of consumer wearables and tracking apps offers an unprecedented opportunity to obtain relevant data passively, continuously and at low cost. This sort of electronic monitoring is particularly valuable in medical conditions that tend to fluctuate throughout the day, like many neurological and cardiological disorders. This trend has triggered a rise in start-ups developing digital biomarkers. The hope is that this new technology will some day change the way we conduct trials and monitor efficacy of experimental drugs.

Tarek Roustom, MD and Junior Analyst at V-Bio Ventures, says: “The growing acceptance of telemedicine combined with the advancements in medical devices and wearables have the potential to make clinical trials cheaper, faster and much more convenient for patients. The space is far from mature yet and many start-up companies are all trying to build a customer base. Those companies who manage to integrate the latest technology and regulatory requirements into a patient-oriented platform are likely to attract keen attention from pharma industry and life science investors alike.”