changes the nature of work. But she also has a personal connection to Transparency’s mission, having struggled with an intestinal disease for many years. “Patients are what’s missing from the equation,” she says. “I always wanted to do something like this.” Transparency also maintains a growing presence on Facebook and Twitter.
Sablinski admits Transparency’s first three drugs are not likely to be huge moneymakers. That’s because they’re all repurposed versions of generic drugs that are already on the market to treat other diseases. The MS treatment, for example, is lisinopril, an anti-hypertensive drug that has shown promise in animal studies as an immune system modulator. “We needed to prove the concept works, and a generic strategy reduces the financial risk,” he says.
Part of what Sablinski needs to prove is that his virtual, crowd-sourcing model can be used to develop drugs at a fraction of the usual cost. In the typical clinical trial, patients must frequently visit the trial sites for physical exams and the like. Sablinski believes a lot of that follow-up can be done with telemedicine—by phone and Internet. “We want the first and last visits to be actual visits, but everything in between will be happening in patients’ homes,” he says. Sablinski and his staff are out searching for “gizmos,” he says, that will allow patients to monitor their own vital signs, for example, or consult with study investigators via Skype videoconferencing.
Sablinski estimates that embracing telemedicine will allow Transparency to complete a clinical trial for about $700,000—half of what it would cost if it were farmed out to a contract research organization. “CROs make money by introducing inefficiencies into the model,” he says. “It’s mind boggling. The savings are not going to be possible across all indications, but most trials can be done with a huge complement of telemedicine.”
Transparency isn’t the only pharma company dabbling in telemedicine. Last June, Pfizer announced a virtual trial of its marketed product tolterodine tartrate (Detrol LA) for overactive bladder. The company said the goal of the trial is to prove that patients in remote locales could participate in clinical trials by using their mobile phones and the Internet to provide data to trial investigators. When Sablinski went to the FDA for preliminary discussions, he says, the agency was already inspired by Pfizer’s lead. “The FDA wants trials to be patient-centric and they have guidelines for telemedicine,” he says. “I don’t expect much pushback from the FDA.”
Sablinski, who once led clinical development and medical affairs for Novartis, is self-funding Transparency and running it when he’s not at his day job as head of clinical development at Celtic Therapeutics, a life sciences private equity firm in New York. He says he wants to prove out his model before approaching venture capitalists. At first, he struggled to persuade folks in pharma that his idea had legs, he says, but he quickly recruited some pretty impressive co-founders: Marc Foster, who commercialized a technology from MIT for FoldRx Pharmaceuticals and then sold the company to Pfizer; Martin Streeter, an e-health entrepreneur; and Lawrence Steinman, an MS specialist who is professor of neurology and neurological sciences, pediatrics, and genetics at Stanford School of Medicine.
Ultimately, Sablinski says, his goal is to use his open-source development model to rescue experimental drugs that have been shelved by cash-strapped pharmaceutical companies. “The real target is to get all these disparate assets from pharma—compounds that can’t move because they require budgets that are too high,” he says. “We will try to develop them with beautiful ideas from the crowd.”
Transparency is a long way from proving that its transparent approach to drug development will actually work, but Sablinski’s confidence is unwavering. On January 31, he announced the launch of the company during a speech he made at a conference in Philadelphia called Patient Centricity in Clinical Trials. Sablinski joked to the crowd that the pharma industry needing an official get-together to talk about how best to serve patients was akin to Detroit automakers having a meeting about driver-friendly car design. “Think about the state of the industry,” he says. “You have to have a separate meeting to talk about patient-centric clinical trials? Pharma should be driven by what patients need.”
