Contract services think outside the box

Virtual clinical trials, nimble CDMOs say speed is key

Helping get treatments to market faster while lowering costs is a mantra for contract service organizations, who are seeing a proliferation of smaller drug companies without Big Pharma budgets seeking the competitive advantage of being early to market. Solutions such as crowdsourced clinical trial protocols, virtual digital patient data collection, and broad-based services from smaller, nimbler partners are helping drug developers get to market quicker and at lower cost.

Transparency Life Sciences, for example, is the first all-digital clinical development services company—essentially removing “clinical sites” from “clinical services.” The company harnesses crowdsourcing and mobile health technology to advance biopharma drug candidates through clinical trials with unprecedented patient relevance and efficiency. The model is scalable and is designed to increase R&D productivity while producing more informative data.

A major downside of the current clinical trial model is the high cost of collecting data at a traditional site such as a hospital or clinic.

“We’ve really stripped it down to the metal,” says Marc Foster, COO of Transparency Life Sciences. “It’s extremely expensive; you’re loading the per-patient cost of a trial with the high fixed costs of an institution,” he adds. This is particularly true in therapeutic areas such as central nervous system (CNS), autoimmune disorders, chronic cancers, and many rare diseases. “A lot of this data can be collected using mobile health solutions and telemedicine,” Foster says. Study data, such as blood pressure, weight, and pulse rate, can be collected in a patient’s home or workplace with digital technologies, specifically telemedicine devices and virtual video visit technology—eliminating or reducing the need for site visits. The model facilitates recruitment and enables obtaining a more representative patient population.”

The Transparency Life Sciences approach is well-suited for Phase 2 trials, generally ranging from 100–300 patients. “Digital solutions make sense for a trial of that size,” Foster says.

Phase II is also when companies begin to experiment with end point selection, which digital solutions can facilitate. The company also conducts Phase IV trials—generating data to support marketing and reimbursement by confirming new drugs continue to produce the stated therapeutic benefits over time. Not all indications currently work well with Transparency’s digital data collection model. “Stroke and acute anti-infectives, both involving hospitalization, are not ideal for us,” Foster says “But we can help design those trials. Part of our three-part digital platform is protocol crowdsourcing, to solicit input globally from patients and relevant professionals. But for trial execution, we ideally focus on trials that can be decentralized using telemedicine and mobile health.”

FDA response has been encouraging, Foster says. For example, Transparency Life Sciences worked on behalf of a small sponsor to complete multiple rounds of crowdsourcing for the design of a trial for in pulmonary sarcoidosis. “We submitted the resulting protocol and an IND to the FDA,” Foster says. “It calls for 180 out of 200 patients in the study to be measured completely remotely, with no interaction with the study staff at the hospital. FDA gave the go-ahead without delay. That was a big signal from a regulatory perspective that the FDA is open to this kind of innovation, and trying to push data collection out to the patient’s home The FDA Commissioner has recently voiced support for wider use of digital approaches.”

Transparency Life Sciences is a digital trials pioneer launched seven years ago and is now in a period of rapid growth, as interest in virtual trials continues to accelerate.

Breaking down barriers

Speed and nimbleness is equally important before the clinic. The funding environment is strong, leading to a proliferation of companies pursuing niche or orphan indications, and whoever reaches the market first has a competitive advantage.

“Lots of new companies are coming into existence and the virtual biotech model is more common than ever,” says Dan Smithey, CEO, of Serán Bioscience. “For virtual companies trying to take a new biology into the clinic as fast as possible, almost all of their development needs are outsourced. That includes early chemistries, analytical development, pre-clinical work, non-clinical toxicology, all the way up to formulation. There’s a big need for companies that can do broad-based services.”

Serán partnered with Cascade Custom Chemistry in [year?] to form SeránCascade, a contract development and manufacturing organization (CDMO) specifically focused on GMP manufacturing of APIs. Smithey says the partnership aims tackle the "almost arbitrary disconnect" between formulation and chemistry. "If you come from a process or medicinal chemistry mindset versus a more CMC [chemistry, manufacturing and control], focus, the perspective is very different. Removing that barrier in thinking would make both much more efficient."

Recent growth is validating this thinking. Serán has boosted its headcount and added 15,000 square feet of manufacturing space. It also aims to reach Phase 3 capability in the next 12 months, Smithey says. "A lot of our early-phase clients want to move fast, and they also want access to both chemistry and formulation capabilities—as well as the associated analytical, regulatory and quality support that goes with that.  In the past 18 months we've been seeing a lot of interest in that offering."

While many large CDMOs are effective, they can be large, bureaucratic, and inflexible—opposite their customers. "Early-phase companies trying to get into the clinic for the first time, their whole business model is based on flexibility,” Smithey says. “They have to be fast and respond to data as it comes in. For them, working with a large CDMO can be very frustrating." Together, Serán and Cascade have less than 100 people and their organizational structures are largely flat. "We are able to respond exceedingly fast when something does change," Smithey says. "We understand that change is part of the business that we are in, so we embrace it."  Being able to respond quickly and efficiently also requires good science, Smithey says. "You can't establish that sort of relationship with customers unless you're data-driven. Every decision we make is based on good, hard data. This helps reducing risk in decision making.

Looking ahead, Smithey does expect the creation rate of new biotechnology companies to slow. "It's pretty high right now, and the industry is cyclical. We are making sure that we become the go-to partner, whether the creation rate is high or not." He also expects gene therapy to be the "next great frontier" of outsourcing. "There's a huge unmet need for that right now. We are working out the best way for us to be a part of that."

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Contract services think outside the box
Helping get treatments to market faster while lowering costs is a mantra for contract service organizations, who are seeing a proliferation of smaller drug companies without Big Pharma budgets seeking the competitive advantage of being early to market.