FDA favors Orphan Drugs

Over the last few years there’s been a big increase in Big Pharma’s interest in rare diseases. With Genzyme’s success with drugs like Myozyme helping light the way, giants like GlaxoSmithKline, Pfizer, Merck and Novartis have been organizing their own rare disease drug shops. And the FDA’s more flexible attitude in how it judges the data from clinical trials for these drugs – as well as the 7 years of marketing exclusivity they earn along with some hefty tax credits – hasn’t hurt.

In a new study, the National Organization for Rare Diseases decided to put the FDA’s professed flexibility to the test, examining 135 non-cancer orphan drugs which had been approved by the agency. And the FDA comes out with a clap on the back, earning NORD’s seal of approval for being willing to demonstrate its flexibility in 90 of those rare drug programs. Writes NORD: “The study supports the FDA assertion that it exercises flexibility when reviewing applications for orphan drugs.”

The study also notes that the FDA can demonstrate its flexibility in a number of ways, most notably by regulators’ willingness to accept a far more limited set of clinical trial data for an orphan drug, including instances where a single trial was enough to warrant an approval. In 58 cases regulators were willing to customize the approval process to the therapy.

The Wall Street Journal notes that not everyone is happy with the FDA’s approach to these orphan drugs. Public Citizen’s Sidney Wolfe, an outspoken critic of the pharma industry, had this to say: “There are all kinds of other benefits already written into law. Why should the standard of approval be different if you have a rare disease than for non-rare diseases?”

NORD does want the FDA to go a step further in spelling out its approach to orphan indications.

“It is time for that policy to be clearly enunciated as a formal FDA policy, and for FDA medical reviewers to incorporate and recognize this flexibility in a systematic way into their evaluations of each new therapy in development and under FDA review for Americans with any rare disease.”

– see the study from NORD
– read the article from The Wall Street Journal

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Stanford Investigators Match Old Drugs With New Medical Uses

A small crew of researchers at Stanford University School of Medicine has come up with an ingenious new method to screen a large slate of approved drugs for compounds that have the potential to be repurposed for use against other diseases as well. The investigators used an approach they likened to online dating services, matching a symptom of a disease against the known actions of drugs to find the right medical mate.

That work led the researchers to two approved drugs which subsequently demonstrated positive attributes in animal studies, including an ulcer drug from GlaxoSmithKline which may prove promising for lung cancer. And while the animal data is just enough to glean insights that can direct developers into promising new fields, the work fits neatly into new federal initiatives designed to find a shortcut to get cheaper new meds into the marketplace.

“If there are additional uses for drugs coming off patents, one could be thinking creatively, how do we find the value in that,” Stanford’s Atul Butte told Bloomberg. Butte’s team produced two reports on their work for Science Translational Medicine. Altogether the work identified a trove of 1,000 drugs that have the potential to be redirected against new targets.

Off-patent drugs are typically not worth much. But investigators responded warmly to the new studies, noting that anyone interested in advancing the work would already have years of safety data in hand, offering an opportunity for mounting new programs at a fraction of the cost of developing a new drug. Still, without clear commercial potential, it’s a field that would likely have to be supported by federal grants rather than investors’ cash–unless the drugs are also reformulated.

– see the story from the Wall Street Journal
– read the Bloomberg article

 

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