Orphan Drug Adoption the More Regulation the Better

New survey research indicates that a country with a strong regulatory commitment for advancing drugs for rare diseases does better in getting orphan drugs to patients than countries with lesser regulatory frameworks.

In his study, ‘The Effect of Market-Based Economic Factors on the Adoption of Orphan Drugs Across Multiple Countries,’ John Matthews, Associate Director of Project Management at Merck examined the relationship between how market-based a country is, and how many patients with rare disease actually receive approved treatments for their disorder. Contrary to the widely held belief that the more free-market a country is, the more likely drugs will be adopted, Matthews found that countries with more regulation actually did a better job of getting orphan drugs into the hands of patients. In other words, in examining data from 13 orphan drugs across US, Germany, UK, Spain, and France, the US was actually the least successful in providing access.

Using the Index of Economic Freedoms and the Economic Freedoms of the World Index, the study used  business, labor, and trade restrictions; government spending; prevalence of corruption; taxation rates, and other economic factors to assess how free-market these countries were at the time of study. While Matthews initially hypothesized that ‘Countries with more market-based economies will be associated with a greater adoption of orphan drugs’, with adoption defined as ‘purchase of an orphan drug’, he noted that the exact opposite was true. “Based off of general economics, the literature out there suggests countries with greater economic freedoms are more efficient markets. But perhaps those mechanisms are not at work in the orphan drug market. No one has examined this issue from an orphan drug perspective, until now,” he said.

Matthews looks to study this issue in greater detail, examining reimbursement systems across these different markets and how they affect orphan drug adoption. Additionally, his study points out the fact that there is no central organization that represents the views of patient groups, and thereby there is no hard data on how they affect access. “There are several organizations aspiring to this role, including the Genetic Alliance, and the National Organization for Rare Disorders (NORD), but I don’t think anyone’s assuming a coordination role. There’s a lot of anecdotal information on patient groups’ wielding their clout to increase drug adoption, but no empirical research discussing observable impacts a patient advocacy group can have on adoption,” he explained. The patient movement in the US has been more robust in its influence, with its close connection to the needs and actions of patients, contrasted with the EU’s more paternalistic approach, focused on governments’ invention to sponsor groups to suit patient needs. Clearly, while patients in the US have a particular flair for advocacy, the relationship between this effort and its ability to move the access needle warrants closer examination.

Matthews does not believe a study looking at newer datasets from a broader range of countries and orphan drugs will produce different results. At the end of the day, he explains, “The results of this study basically say that greater government regulations are able to support better delivery of these medicines. Intervention is an option that can work.”

‘The Effect of Market-Based Economic Factors on the Adoption of Orphan Drugs Across Multiple Countries’ by John T. Matthews, MBA, PhD, and Lucas Glass, MS