Exponent's John Doyle Offers Insight to Pharmaceutical Commerce

October 27, 2022

Exponent Health Sciences VP discusses value-based agreements for orphan drugs

John Doyle, Dr.P.H., M.P.H, Exponent group vice president and principal scientist, was recently interviewed for an article in Pharmaceutical Commerce, a popular pharmaceutical industry periodical written for biopharma company decision-makers focusing on business processes and the technologies involved in bringing approved drugs to market.

"Betting the House on Orphan Drugs" covers the rising interest of biopharma companies — large and small — in rare-disease drugs, otherwise known as orphan drugs. One focus of the article is on the use of value-based agreements (also known as outcomes-based agreements). These contracts aim to reduce economic risk by linking payer coverage and reimbursement directly to real-world clinical outcomes. They continue to grow in popularity, and many stakeholders agree that they make particularly good sense in the orphan drug marketplace.

With over 25 years of experience conducting research in areas such as pharmacoepidemiology, health economics and outcomes, and real-word evidence (RWD), Dr. Doyle explains how two pharmaceutical companies benefited from using value-based agreements when they recently launched their orphan drugs. In both instances, the contracts allowed for refunds to payers if the drugs failed to provide a determined outcome to patients, thereby sharing financial risk in the performance of the drug.

While such agreements among biopharma companies, payers, and others offer a promising option to share risk more equitably, creating them produces yet another challenging hurdle for drug developers.


"All parties must decide what are the most essential metrics, endpoints, or surrogate endpoints to demonstrate clinical success," Dr. Doyle explains, "and then carry out modeling and simulation (i.e., epidemiological, actuarial, economic), collecting and analyzing many forms of RWD to design the novel contract terms and manage them over time."


Along with Dr. Doyle's contributions, the article also features insight from other key figures in the industry, including Ela Lourido, vice president of biopharma services for Biologics by McKesson, and Scott Swain, director of regulatory sciences and real-world evidence for Cardinal Health.

To read more about this growing trend among biopharma companies, go to "Betting the House on Orphan Drugs."