Outcomes Data for Reimbursement

Tampa—Despite the difficulties pharmaceutical manufacturers face in getting drugs approved, the FDA does not require them to submit outcomes data. During a session at the AMCP meeting, 2 speakers mentioned that information could be helpful in determining the value of drugs and help payers make reimbursement decisions.

Nathan White, CPC, director, NucleusX Market Access, said outcomes research that the FDA does not mandate includes cost reductions, healthcare resource utilization, quality of life analysis, and patient satisfaction. He added that manufacturers could benefit from this data because of crowded drug classes and high costs for clinical development. If companies can prove their products are superior to competitors, payers may give them preference on their formularies. Meanwhile, payers need to control drug and medical costs and can benefit when knowing which drugs offer the best value.

During the fall of 2013, NucleusX Market Access and Percipient, LLC, worked together on a primary research project to determine payer attitudes on outcomes data. NucleusX Market Access helps companies with their pricing strategies and reimbursement, while Percipient, LLC, is a managed care consulting firm that works with pharmaceutical, biotechnology, and device manufacturers.

The researchers conducted interviews with 20 pharmacy and medical directors at commercial payers, 4 advisors to health technology assessment committees at payers, and 5 health economics and outcomes research and managed market stakeholders at manufacturers. Dr. White and Katya Svoboda, MBA, MPH, partner, Percipient, LLC, presented the results.

Of the national payers, 80% said they considered outcomes data important when drugs were launched compared with 53% of regional payers. Within the past 2 years, Ms. Svoboda said most payers increased their expectations of companies providing outcomes data after product approvals, although they did not expect outcomes data before drugs were approved. According to the survey respondents, outcomes data is most helpful in high-volume and high-cost therapeutic categories.

Ms. Svoboda said payers were skeptical of manufacturer-supplied data and recommended pharmaceutical companies make the study results more applicable and actionable, conduct head-to-head trials, use third parties to conduct research, improve transparency, standardize measures, and publish results in peer-reviewed journals.

When conducting the value assessments of drugs, payers said the following study end points were most helpful: head-to-head comparisons, utilization of healthcare resources, final versus surrogate end points, safety data, economic data, indirect comparisons, and quality of life indicators.

Although patient-reported outcomes are not typically assessed, Ms. Svoboda said a few drugs have been approved based on patient-reported outcomes end points, including imatinib for chronic myeloid leukemia, milnacipran hydrochloride for fibromyalgia, duloxetine for generalized anxiety disorder, and tofacitinib for rheumatoid arthritis.

The survey found that pharmaceutical manufacturers understood that outcomes research could help them demonstrate value and differentiate themselves from competitors, but they also mentioned that conducting the research could lead to delays in approvals and FDA deadlines, higher costs for complex studies, and an increase the risks of negative data.

Survey respondents also predicted more manufacturers would develop outcomes data for new drugs because of payers’ drug management techniques, closed formularies, crowded therapeutic categories, and improvements in technology to collect the data. Meanwhile, they also mentioned that payers would require more outcomes data because accountable care organizations are taking on more risk, technical integration of data is becoming more popular, costs of new drugs are escalating, and treatment alternatives are increasing.