Why cures are so hard to come by
ONE OF eight Americans over 65 develops Alzheimer’s disease. Roughly one in five Americans over 60 is living with diabetes. These and a long list of poorly treated diseases cost society hundreds of billions of dollars a year. Since many of us will develop severe illnesses that could be treated by effective medicines, why are we not discovering better drugs? The reasons are simple: there are ever greater impediments to making new drugs. Pharmaceutical research and development is becoming too costly, lengthy, and risky.
It may be surprising to many that an investment in discovering new drugs is an increasingly dicey proposition. After all, walking through Cambridge, you see the vibrant academic centers of Harvard and MIT, many new academic and industrial research buildings, and a seemingly thriving biopharmaceutical community. But look beneath the surface in Boston and beyond, and one quickly discerns growing barriers to discovering and developing important new drugs.
Start first with the overall expense. Developing a new drug in the United States costs a staggering amount — well over $1 billion. In addition, taking a drug from initial idea all the way to approval by the US Food and Drug Administration typically takes well over 10 years. And finally, it is estimated that fewer than one in 100 drug leads that start the rigorous process of testing in cells, then animals, then in patients will ever reach the market.
How are the increasing costs, the lengthening timelines, and the growing risk profile influencing investments in drugs discovery? Take a look at Wall Street investors, pharmaceutical companies, and small biotechs. The price-earnings ratios of large pharmaceutical companies show that average investors are one-third as interested in investing in drug discovery as they were just 10 years ago.
In the case of pharmaceutical companies, the reaction has been predictable. Pharmaceutical companies are busy merging and significantly reducing research and development expenditures, or diversifying away from pharmaceutical drug discovery. Finally, many biotech companies in Boston and across the country are reducing headcount or shutting down, often unable to raise financing to support drug research and development. Remember, a biotech needs to raise well in excess of $1 billion to sell its first product.
Most of us will develop a serious illness at some point. If we don’t make investment in drug discovery more attractive, odds are an effective drug will not be there when we develop Alzheimer’s or diabetes.
We could improve the odds of developing effective new drugs by lessening the impediments. The lengthy and risky nature of drug discovery is based in part on ever-expanding regulatory requirements. As an example, guidelines from the Food and Drug Administration ask for multiple studies in many hundreds of patients each for extended periods to get a new Alzheimer’s drug approved. In practice, this step alone can cost several hundreds of millions of dollars and take many years. The hurdle for new drug approval for many diseases is higher today than it was 20 years ago.
Society would benefit from faster, less expensive access to important new drugs, especially for devastating diseases with no good current therapies, like Alzheimer’s. First, we could apply lessons learned during the AIDS crisis, when drug regulators authorized more rapid clinical trial and approval timelines for a disease with no good therapies. This was achieved by accepting indications of efficacy in smaller, shorter trials, and following patients closely over longer periods of time after approval of the drug.
Second, we might increase public support of drug discovery via the National Institutes of Health and local initiatives such as the Massachusetts Life Sciences Center. Third, some of the labor- and cost-intensive initial steps in drug discovery could increasingly be shifted to countries such as India and China. There are no easy solutions to decreasing the current disincentives for drug discovery, but these approaches could facilitate the development of more and better drugs for serious illnesses.
Christoph Westphal, a guest columnist, is a biotech entrepreneur and a partner at Longwood Founders Fund.