The Feds Need to Start Supporting Early-Stage Drug Development
The U.S. Congress is on track to fund a pivotal element of healthcare reform, a $50 million dollar down payment on the $500 million dollar Cures Acceleration Network (CAN) initiative. This is a critically important element of the landmark new law. It’s also an opportunity Congress should seize to refocus the National Institutes of Health toward an essential part of its mission—fostering drug development for the improvement of human health. The Cures Acceleration Network has the potential to radiate as much benefit to patients as healthcare reform itself.
CAN is the brainchild of Senator Arlen Specter of Pennsylvania, who himself has twice fought off lymphoma. Specter pressed for the CAN allocation to focus vital dollars at the weakest point in drug development, the translational gap. This is a laudable goal.
In the most generous terms, drug development in America is static. In 1982, the pharmaceutical industry produced 28 genuinely new therapeutics. In 2009, the number was 26. Over that near thirty year time span, the costs of flat-line output have soared from $14 billion to $80 billion in current inflation-adjusted dollars (NIH and pharmaceutical industry spending combined.) What’s more revealing is historical analysis of the quantity of drug candidates entering Phase 1. In 1986, there were 1,618 compounds entering clinical trials at the FDA’s Center for Drug Evaluation and Research (CDER). In 2008, the number grew to 2,039, but this largely reflects a reallocation of numerous biologics products from the FDA’s Center for Biologics Evaluation and Research to CDER. The resource-bloated system is heading toward infinite inefficiency.
Meanwhile, the chronically ill wait for better drugs—real people like Specter, confronting cancer, multiple sclerosis, Parkinson’s… and the list goes on. Anomalous as it is, patients, taxpayers and lawmakers alike are imbued with the belief that new and better drugs are bursting from the pipeline. And why shouldn’t they be, media reports daily tout promising scientific and medical breakthroughs.
The National Institutes of Health is the largest public-sector source of funding for medical research in the world. To be clear, its mission is both to support scientific research for the advancement of human knowledge, and to improve human health and reduce the burdens of illness and disability. Toward this end, the NIH budget has hovered around $30 billion for the last few years. A glimpse into one particular disease area—autoimmune diseases like type 1 diabetes, rheumatoid arthritis, and multiple sclerosis—shows that the NIH provided $879 million in grant funding in 2009. But less than 15 percent of this was directed at new therapies or clinical trials.
The exclusion of biotechnology companies from NIH funding mechanisms was particularly extreme with the 2009 stimulus program. Last year the NIH received an additional $10 billion via the American Recovery and Reinvestment Act. Again, only a fraction was directed at genuine new drug development. Washington State received 406 ARRA grants from NIH totaling $182M. Only five of the 406—0.6 percent of the total Washington State NIH ARRA allocation—were provided to businesses. The remaining 401 grants all went to universities and not-for-profits and was primarily directed at basic research. Furthermore, the process was largely noncompetitive. 57 percent (232) of the approved ARRA grants were administrative supplements to existing grants that did not involve peer review or other competitive process.
In excluding biotechnology companies, a critical engine of both new drug development and job growth in Washington State, small businesses were effectively shut out of the stimulus process.
The current NIH granting process is impeding its stated mission of accelerating cures. Academic research institutions that receive the bulk of federal dollars largely conduct basic scientific research. While important work, academic entities are not designed, nor are they focused on developing drugs. Further, in the last decade, the pharmaceutical industry has precipitously divested itself from early stage research and development. Small startup and early stage biotechnology companies are virtually alone in doing this vital translational work.
If the federal government is genuinely driven to accelerate the development of high-need new drugs, it must adjust its funding ratios to bolster early stage drug development programs and allow private sector companies to compete on a level playing field. The Cures Acceleration Network is a good place to start.
[Editor's Note: Kineta's Meg O'Conor Bannecker co-authored this post.]