Orphan Drugs

A Thomson Reuters report claims to highlight how orphan drugs – those developed to treat rare diseases – might have big market potential for the pharmaceutical industry. 

The media and information company says its report is the first to compare the economic viability of orphan versus non-orphan drugs. Dr. Kiran Meekings, co-author of the paper with Dr. John Arrowsmith, explained that they didn’t know what to expect at the outset.

“A big unanswered question that [Thomson Reuters] clients are interested in is whether an orphan drug R&D strategy is likely to pay dividends,” she said. “Can pharmaceutical companies generate profit from orphan drugs, bearing in mind the small target populations? I was interested in testing this hypothesis using Thomson Reuters data.”

Historically, mainstream pharmaceutical companies have not focused on developing treatments for so-called orphan diseases, like Cystic fibrosis or Wilson’s disease. These diseases individually affect small groups of patients, but collectively may affect up to 25 million people in the U.S. alone. The report – which used proprietary Thomson Reuters data – purports to show that orphan drugs can generate as much lifetime revenue as non-orphan ones. When viewed in combination with factors such as shorter and more successful clinical trials, and government incentives to reduce R&D costs, Meekings and Arrowsmith hypothesize that orphan drugs might even be more profitable than non-orphan drugs.

 “I’ve had a good response both from individuals involved in rare disease R&D and the investment industry,” Meekings explained. “I’ve had many individuals pass on positive responses to the paper saying this is the data they’ve really been lacking, and it will be really useful for their strategy going forward.”

Thomson Reuters published its report in Drug Discovery Today in collaboration with Dr. Cory Williams, M.D., Ph.D., Pfizer. Meekings says:

 “The report will help stimulate interest and generate more investment for more rare diseases… For the millions of people worldwide affected by these diseases, that’s the most valuable aspect of all.”

Now let’s be clear, Thomson Reuters is trying to engage in industry “thought leadership” in order to generate business opportunities, particularly for its Life Sciences Professional Services team. Fair enough, it is in the business of making money. But is it disingenuity to claim that treating rare, tabloid-headline-of-the-week conditions should be an attractive proposition? This study plays against a complex background of public health economics and policy, where the holders of the purse strings struggle to justify spending scant funds on expensive treatments for a relatively small minority of afflicted patients, pharmaceutical companies can’t therefore justify the R&D investment, and all the while patient advocacy groups lobby hard for attention and a lifeline to help save themselves and their loved ones from sometimes tragic conditions. No doubt appealing to the almighty dollar is a somewhat cynical but, in a world where pharma companies are facing a troubled future as their blockbuster drug patents expire, is it cynicism or realism?

And is anyone out there equipped to review this study and express a view as to whether it holds together and supports the conclusions in the soundbites? Any bored Ben Goldacres floating about looking for something to do…?

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