Orphan drugs, developed to treat rare diseases, have the potential to generate as much lifetime revenue as drugs used for more common health conditions, according to a study by Thomson Reuters’ intellectual property and science business.
Life sciences researchers at Thomson Reuters found that developmental drivers such as government incentives, shorter clinical trials and high rates of regulatory success make top orphan drugs as economically viable as non-orphan ones, even though the number of patients benefitting from them is significantly smaller than those benefitting from non-orphan medicine.
"This new data gives economic validity to the importance of targeting rare diseases in the global pharmaceutical market," said Kiran Meekings, Ph.D., life sciences consultant at Thomson Reuters and co-author of the report. "Not only does such focus help those affected by rare diseases, of which there are 25 million people in the United States alone, it also furthers the aim of precision medicine and substantiates the envisioned high returns on the R&D investment, particularly for drugs with multiple orphan disease approvals."
Current estimates indicate that 250 new rare diseases are identified annually. This report provides extensive and tangible evidence that orphan drug development is an important component of biopharmaceutical R&D strategy and that a number of orphan drug therapies are already achieving blockbuster status.
"The higher value of companies that have a strong orphan drug strategy reflects the increasing degree of optimism for the sale-and-profit potential of the rare disease market," said Brian Lester, senior analyst and managing director of the life sciences group at the financial services firm Manning & Napier. "We expect the orphan disease business model to sustain a competitive edge over the traditional primary care business model in the future."
The data was aggregated by Thomson Reuters’ life sciences consultants who performed an economic analysis of orphan versus non-orphan drugs while collaborating closely with Cory Williams, MD, Ph.D., Pfizer, in reporting their findings.
Thomson Reuters compared the total value of orphan drugs from 1990 to 2030, while industry benchmarking data was provided by CMR International.