When pharmaceutical companies win regulatory approval for a drug to be sold – a long and expensive journey that requires successful phase 1, 2 and 3 clinical trials – their work is far from over.
They are increasingly required by the Food and Drug Administration to monitor how safe and effective the drug is when taken by large numbers of patients over longer periods. This real-world monitoring, known as postmarketing surveillance, is done through phase 4 clinical trials, which are time-consuming and expensive.
Reducing the time and cost of postmarketing surveillance is a major aim of TARGET PharmaSolutions, a clinical data company that was spun out of the University of North Carolina at Chapel Hill.
“Any time there is inefficiency in a system, there is an opportunity,” says Meg Powell, Pharm.D., TARGET’s president, chief executive officer and co-founder. (Powell is a Statesville native who graduated from the North Carolina School of Science and Math and the UNC pharmacy school before earning an MBA from Stanford University.)
TARGET’s solution is to create “collaborative communities” of stakeholders around diseases to generate more powerful clinical data and better insights about the natural history of diseases, including treatment outcomes.
“By bringing everyone together, it eliminates a lot of redundancy amongst companies and academic researchers that are all looking at similar questions,” Powell says. “It also provides a much larger cohort of patients than any one stakeholder could put together themselves and therefore can answer critical questions such as real-world comparative safety and effectiveness analyses that may have otherwise gone unanswered.”
TARGET also maintains a biorepository of patient tissues that can be matched with patient-reported outcomes, providing unique data and insights compared to typical patient registries, Powell says.
While the company’s main focus is on postmarketing surveillance, it also works with pharmaceutical partners that haven’t yet launched therapies. For example, TARGET says its data and services can help companies conduct clinical pharmacology studies or decide which customer segments to target for future clinical trials.
The basis for the company’s formation in February 2015 was a successful case study of the collaborative-communities clinical trial model for hepatitis C by researchers Michael Fried, M.D., of UNC and David Nelson, M.D., of the University of Florida, both of whom subsequently became TARGET co-founders and scientific advisors.
That study, called HCV-TARGET, enrolled more than 10,000 hepatitis C patients at 58 sites across North America and Europe.
“Most notably, HCV-TARGET was able to bring all the key stakeholders together around one large clinical study and data set,” Powell says. “HCV-TARGET demonstrated that the TARGET model brings significant efficiency when a cohort is large enough to answer questions from all the stakeholders including pharma/biotech companies, regulatory agencies, payers, key academic thought leaders, and patient advocacy groups to advance resulting in the entire field moving forward.”
The company says data insights from the study have enabled pharmaceutical sponsors to expand drug labels, helped other companies mitigate their phase 4 commitments, informed treatment guidelines, been used in payer dossiers and spurred at least 21 publications by 90 authors.
TARGET has since replicated the collaborative-communities model for other diseases. It is enrolling patients in its communities for nonalcoholic steatohepatitis, or fatty liver disease; primary biliary cholangitis, a disease of the liver’s bile ducts; and hepatocellular carcinoma, the most common type of liver cancer.
TARGET will also begin enrolling patients for irritable bowel disease by mid-year and expects to announce a fifth community by the end of the year, Powell says.
“Our model is disease specific, not drug specific,” she explains. “Therefore, we do not do any work with investigational agents. We are, however, able to generate systematic data about how drugs are used in the real world, both on and off label. As a result, insights derived from our data has been used by pharma partners to plan their individual phase 1, 2 and 3 studies.
“By looking through a disease state lens instead of a specific therapeutic lens, we are able to answer a much broader set of questions around real-world comparative effectiveness and safety,” she adds. “Our keen focus on data quality and completeness results in a high level of reliability and credibility.”
TARGET has entered the postmarketing surveillance business at an opportune time. Amendments to the federal Prescription Drug User Fee Act in the last decade have emphasized real-world utility of drugs, leading to more phase 4 trials funded by fees from pharmaceutical companies. New spending on these trials worldwide has grown by more than $1 billion in the last five years, TARGET says on its website.
The company has attracted four commercial partnerships – two with Bristol-Myers Squibb and two with Intercept Pharmaceuticals – and expects to announce additional partnerships soon, Powell says.
As its client base and workload grow, TARGET expects to hire about five more employees in the next few months, mainly in its clinical operations group. The company has 20 employees and works regularly with six consultants.
TARGET is backed by a Series A funding round completed in 2016 and led by Raleigh-based REX Health Ventures, the venture capital arm of UNC REX Healthcare, with participation by VentureSouth of Greenville, S.C., and local angel investors.
TARGET was awarded a $500,000 Strategic Growth Loan by the North Carolina Biotechnology Center in 2016. Although the company hasn’t yet used the loan, it has benefitted from other Center services.
“Even before we raised our Series A, the Center was very helpful in providing guidance to refine our business model,” Powell says. “In addition, we have leveraged the Biotech Center’s library for detailed data, and as we continue to increase our team, we are leveraging resources for finding key employees.”
TARGET is also an active portfolio company of the Durham-based Blackstone Entrepreneurs Network, which provides mentoring and advice.
(C) N.C. Biotechnology Center