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A Problem Diagnosed
By Peter J. Pitts
Molecular diagnostics are what make medicines personal. Diagnostics are how drugs can be made safer through safe use. They make the “four rights” (right medicine for the right patient in the right dose at the right time) possible. And the four rights lead to lower costs through better outcomes.
Case in point: Warfarin, which is the most widely used anti-coagulant medication in the world. Prescribed to more than 2 million people a year to prevent blood clots, heart attacks, and strokes, patients can display markedly different responses to the drug. Doses vary enormously between individuals, so achieving the correct dose is critical. Patients who receive too high a dose are at risk of severe bleeding, while those who receive too low a dose may remain at risk of life-threatening blood clots. Thanks to molecular diagnostics that are now called out in the amended U.S. Food and Drug Administration label, physicians will prevent 85,000 serious bleeding events and 17,000 strokes annually—and that’s just in the United States. And this “safer use” is estimated to save $1.1 billion annually. And that’s the mid-range.
But diagnostics are in trouble. And that trouble comes in the form of skittish reimbursement and ambiguous regulation.
On the reimbursement front, many payers aren’t ready to accept the up front expense, even though the longer-term savings can be substantial.
Case in point: Herceptin, which is used to treat a specific form of breast cancer fueled by an excess of a growth factor known as HER2. Studies show that testing for HER2 breast cancer delivers savings that are 65 times its cost. (For a very powerful presentation on the economics of the HER2 test and molecular diagnostics in general see Genzyme’s Mara Aspinall’s presentation. In short, reimbursement should be based on value rather than activity. This is an essential paradigm shift.
Clarity and predictability are required on the regulatory front. The FDA approved the molecular diagnostic for warfarin based on a broad range of published literature together with the results of a study, conducted by the manufacturer, on hundreds of DNA samples. But guidances on diagnostic approvals are vague as is the pathway. To reinforce the agency’s commitment to personalized medicine, the FDA should embrace ever greater clarity and commitment to diagnostic tool review. This should be a top priority of the agency’s Critical Path initiative, which is designed to modernize the process through which products move from discovery to the market.
Unless and until the reimbursement and regulatory issues are addressed, investment in developing these tools will languish, patients will needlessly suffer, and our healthcare system will continue to be burdened by unnecessary costs.
The Critical Path Institute (created in 2005 by the University of Arizona and the FDA to standardize an approach to validating medical products) is working on a solution, a “Good Housekeeping Seal of Approval” for diagnostics. C-PATH plans to launch its United States Diagnostic Standards in the coming months.
“The United States Diagnostic Standards will offer a voluntary certification for laboratory and pathology diagnostics, much like the Underwriters Laboratories certification for many tools and equipment. Companies already submit their tests to data test sites for evaluation prior to FDA submission,” says Jeffrey Cossman, chief scientific officer of C-Path. “This would take the place of a data test site.”
This speaks directly to the contentious issue of partnership between the FDA, industry and academia. No one entity can do it alone. This is a core philosophy that will, no doubt be vigorously debated in Congress—and by the next FDA commissioner. We need a stated policy of pragmatic partnerships.
If the popular culture clarion call is for “safer drugs,” then the path forward shouldn’t include beating up Big Pharma or reversing FDA preemption authority—it’s via molecular diagnostics.
This message is finally reaching a mass audience. Consider the recent USA Today story, “Genetic testing boosts efficacy in cancer care,” which reports that “tailoring cancer therapies to fit a person’s genetic makeup could spare thousands of patients from harmful side effects and save millions of dollars a year.”
Personalized medicine is not about denying care. It’s about providing “the four rights” and can, indeed must, be both cost-effective and patient-centric.
Nobody said it's going to be easy.
Peter J. Pitts is President of the Center for Medicine in the Public Interest and a former FDA Associate Commissioner. |
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