11.30.16 | Forbes.com
By Steve Brozak
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If the healthcare industry’s approach to Alzheimer’s disease were a building project, it could best be described as constructing a skyscraper from the top down–possible, yet at best impractical. Last week Eli Lilly and Company announced that the company’s Alzheimer’s drug solanezumab, which is intended to reduce amyloid plaque, failed to help Alzheimer’s patients recover or maintain their mental acuity in a late-stage trial.
It is the latest example of such a failure and it happened because Lilly made too many assumptions about Alzheimer’s disease before having an adequate diagnostic. Of course, internally we have believed Lilly has overly relied on its internal pipeline for far too long, with its CEO resisting larger M&A activity that could have derisked potential pipeline failures such as the one that occurred last week. But Lilly’s failure in this instance is indicative of the top-down approach to drug development in diseases of the central nervous system. Drugs now being developed by companies like Biogen, Inc., and the Genentech division of Roche Holding AG are likely to experience the same fate.
The lack of progress in developing an Alzheimer’s therapeutic might be President-elect Trump’s first opportunity to demonstrate a new and practical approach to treating the devastating disease that afflicted his father, Fred Trump, who passed away in 1999 after a six-year battle. Just as President Obama and Vice President Biden established a moonshot for cancer after the Vice President’s son’s death from brain cancer, President-elect Trump may decide to have his own moonshot (or Trumpshot, as we call it), directing resources and initiatives to central nervous system diseases like Alzheimer’s, which are predicted to increase the healthcare burden exponentially over the next several decades.
60 Minutes spent an entire segment this past Sunday focused on the billions of dollars companies are spending to cure Alzheimer’s and mentioned that Alzheimer’s is only definitively diagnosed by autopsy after death. What they didn’t really explain was that medicine lacks a definitive, objective diagnostic tool; diagnosis is a painstaking process where physicians must first rule out all other cognitive disorders by asking questions, testing coordination and evaluating reflexes before arriving at a diagnosis of Alzheimer’s. The lack of an Alzheimer’s diagnostic is a missing key that would enable many more researchers to approach the disease from varied vantage points.
Alzheimer’s was identified a century ago and characterized fifty years ago. Sixty to eighty percent of all dementia cases are Alzheimer’s, close to 47 million people worldwide have the disease, and 5.3 million people in the U.S. are living with Alzheimer’s at a cost of $236 billion a year. People with Alzheimer’s will not die directly from the disease but from the debilitating effects of the ailment.
Historically, diagnostics are neither lucrative nor profitable. They are low-revenue-producing products in a world where cures produce high revenue. This is primarily due to a commodity based pricing approach to diagnostics, unlike the value-based approach taken with therapeutics. But having a diagnostic is crucial in Alzheimer’s because it could enable researchers to identify early stages of the disease, providing insight into disease origin and progression. The true clinical value is not reflected in the price of most diagnostic tests. Instead, new diagnostics are priced against products and services currently on the market, usually based on decades (if not generations) old technologies. This approach to pricing provides very little incentive for private investment in newer technological approaches which could herald a new era in diagnostics. In Alzheimer’s where there are no good therapeutic options, a diagnostic could enhance the ability to develop a cure or an intervention that could transform a mortal disease into a chronic one.
Donald Trump’s campaign provides us with insight to how he could approach healthcare research. The President-elect was told at the beginning of his campaign that he had little chance of winning because people were evaluating him based on conventional political assumptions, but Trump rejected the conventional approach and pursued a different path to the White House.
Over the years, Trump has shown both financial and organizational support for causes related to combating Alzheimer’s, and this could be an opportunity to demonstrate a commitment to public/private partnerships and to alleviate suffering from the same disease that afflicted his father. Using his insights from business identifying and completing large real estate projects, a Trump administration could refocus industry efforts on diagnostics as well as cures. Good diagnostics matter in the fight against Alzheimer’s just as they have mattered in the war against cancer. In fact, by getting industry’s priorities straight by re-incentivizing a quest for better diagnostics, the entire healthcare sector could be transformed.
If research and development continue to follow Lilly’s conventional approach of reducing amyloid plaque, which is only one of several hypothetical causes of Alzheimer’s, there is every unfortunate chance of failure until we can diagnose the disease correctly. Without a diagnostic, companies developing an Alzheimer’s treatment are almost guaranteed to spend more and lose more in the end.
Along with revising the pricing models around diagnostics, significant investment of money and brainpower could advance development of a diagnostic test for Alzheimer’s before signs of the disease appear. By bolstering and advocating for more grant-based research and development of an Alzheimer’s diagnostic through the National Institutes of Health and providing incentives for businesses to develop an Alzheimer’s diagnostic, a Trump administration could blaze a new trail for the healthcare research and development model. Perhaps the recently announced prize for antimicrobial diagnostics by the NIH (modeled on the X Prize and Longitude Prize) could be replicated for the development of an Alzheimer’s diagnostic as an award and a call for action.
There is another precedent for an incentive that has worked well for the drug industry and the companies who develop new cures. The FDA offers what it calls a priority review voucher to developers of treatments for neglected diseases and rare pediatric diseases. The voucher grants the holder expedited New Drug Application review by the FDA. The voucher can be sold by the company that receives it and vouchers have been sold for hundreds of millions of dollars to help fund further research or to speed a new drug to market. A modified version of the FDA voucher program could be expanded to include the diagnostics industry.
With a growing disease as cruel to patients and the people around them as Alzheimer’s, we can neither wait nor fixate on a single-minded approach to attacking this disease. The wrong approach would be to develop only therapeutics or only diagnostics. We must do both. There is an urgency to develop better drugs. There must be an equally intense urgency to develop precise diagnostics. To be successful we must do both.