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Christoph Westphal

Pharma needs US help

Important drugs will be slow to market if agencies don’t coordinate

By Christoph Westphal
February 7, 2011

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THE VAST majority of prescription drugs are discovered and developed by pharmaceutical and biotech companies, not by academic labs. Nevertheless, companies depend on vibrant academic research to feed the early stage of their new drugs pipeline — which is funded largely via the National Institutes of Health. At the late stage of the new drugs pipeline, the Food and Drug Administration weighs risks and benefits before deciding whether to grant approval to market a new medicine.

The development of important new drugs is most at risk at the late stage of the drugs pipeline. In an ever-shifting regulatory environment, it is hard for companies to understand exactly what might be needed to gain approval for their medicines. This increases the risk of drug discovery investments for companies, and reduces the attractiveness for public markets to invest in research-based drug companies.

One need only look at the FDA’s recent rejection of drugs to treat obesity on the grounds of unclear benefits measured against risk in order to see examples of such regulatory uncertainty. It would be helpful to patients suffering from poorly treated diseases, and to the investment required to bring such medicines to the market, to bring all the stakeholders to the table (the NIH, industry, and the FDA) in supporting the most innovative and promising new drugs in mid- and late-stage trials.

Despite vast increases in government and industry spending on research and drug discovery, fewer important new drugs are being approved now than before. From 1995 to 2004, roughly 30 new drugs were approved by the FDA every year. From 2005 to 2010, the average number of yearly approvals was down by about 33 percent. Only 20 new drugs a year are now approved by the FDA. What could we do to combat this trend?

To improve drug discovery efforts at the early stage, Francis Collins, the director of the NIH, is creating a new National Center for Advancing Translational Sciences, with a goal of investing up to $1 billion on improved tools to speed academic discoveries from the lab into animal and human testing. While this is a laudable undertaking and is likely to help early stage drug discovery efforts, an immediate focus on the late stage of the drug development pipeline, by advancing the most promising drugs quickly through human trials, should be undertaken in parallel.

A focus on improving the late stage of the new drugs pipeline is likely to be accomplished only via a close collaboration with the NIH and the FDA on the one hand and the pharmaceutical and biotech industries on the other. Think of it this way: the new NIH center may help to bring more new drugs to market — but in 12 years at the earliest. Twelve years and $1 billion are a minimum estimate for the time and money required to move an important discovery from an academic lab through human testing to the market.

But many patients with Alzheimer’s or incurable cancers want important new drugs to reach the market sooner. The only way to jumpstart the approval of important new drugs within the next dozen years requires increased funding for the study of the most important new drugs in animal and early human testing — now. Industry is cutting back such funding, given recent poor returns. Hence, government support is required.

The NIH, the FDA, and the drug industry all share a primary mission of bringing important new drugs to patients in need. Given this common interest, the creation of a fund similar in size to the budget of the National Center for Advancing Translational Sciences, $1 billion, seems prudent. The NIH and the FDA would jointly staff a small granting committee (perhaps consisting only of the FDA and NIH heads), which would determine how to allocate these funds to augment direct investment by companies.

Criteria for investment should include the transformational potential of these medicines and their scientific and clinical innovativeness. This would integrate the early stage and late stage thinking of the NIH and FDA together with industry in supporting the creation of more important medicines in the near term. Including the FDA (the gatekeeper at the late stages of drug development) early in investment decisions should improve coordination throughout the long and expensive drug development path. This investment by the government in important new drugs could be repaid when these important new drugs are successfully approved and marketed. This could represent a win for patients, industry, and the government.

Christoph Westphal is a biotech entrepreneur and a contributor to the Globe. He can be reached at www.twitter.com/drcwestphal.