Analysis: Crusades to repair broken drug R&D one disease at a time
It's a well-known problem: Pharma companies' research spending has gone up over the past decade or so without an equivalent increase in new drug approvals. Yet as Bloomberg writes in a feature on the issue, individuals have stepped up to reform the broken system in which companies usher new therapies to patients. The results seem mixed.
Take Scott Johnson's crusade to speed up development of therapies that reverse multiple sclerosis. In 2002, the entrepreneur and MS patient formed the Myelin Repair Foundation, which has raised $45 million since 2004 to fund labs that share data and collaborate rather than compete to advance new treatments, Bloomberg reports. Now Cleveland Clinic investigators are recruiting patients for a study of one of the treatments from one of the foundation's efforts.
Johnson might just beat expectations for how long it will take to bring a myelin-repair therapy to patients with MS, which causes progressive nerve damage and for which there are no approved treatments to reverse the effects of the disease. However, history shows that speeding up the pace of drug development is infinitely difficult. According to PhRMA figures cited by Bloomberg, pharma companies boosted spending on R&D from $50.3 billion in 2001 to $80.4 billion in 2011, but the number of new drug approvals remained flat about roughly 20 to 30 per year.
Disease-focused nonprofits have picked up some victories with approvals of drugs supported with their funds. The Cystic Fibrosis Foundation supported Vertex Pharmaceuticals' ($VRTX) game-changing CF therapy Kalydeco, which the FDA approved this year as the first drug to treat the underlying cause of the lung-damaging disease for a subset of patients. And the MPN Research Foundation, which backs research of treatments for certain blood cancers, and one of the drugs supported through its efforts gained an approval last year, Bloomberg reports.
"I thought we'd have all these diseases fixed in my lifetime, and then I thought it would be fixed in my children's lifetime," Robert Rosen, founder of MPN Research Foundation, said, as quoted by the news service. "And now I don't know anymore."
In fact, no amount of money seems to help expedite the pace of translating discoveries into approved therapies. But foundations and collaborative approaches have made an undeniably positive impact on driving new treatments toward the market.
- get more in Bloomberg's article
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Report urges new tactics to end drug-development drought
R&D execs continue the global search for efficiency, open innovation
Nonprofit poised to reap rewards from Vertex's pioneering CF drug