Seventeen years ago, Ronald Reagan penned what some have called his "Long Goodbye": a letter to the American people announcing that he had been diagnosed with Alzheimer's disease. He poignantly thanked readers for allowing him to serve as president. "I only wish there was some way I could spare Nancy from this painful experience," he wrote.
Since then, Reagan's public goodbye has been echoed in private by thousands upon thousands of parents and spouses: the painful recognition that Alzheimer's currently has no cure, accompanied by a hope that someday, medicine will advance to the point where the disease can be prevented, managed or even cured.
Today, as we recognize Alzheimer's prominence, what is the state of the medicine -- are we any closer to fulfilling Reagan's wish?
The question is an important one: Alzheimer's is expected to affect one out of every 85 people globally by 2050. The well-documented costs of Alzheimer's ranges from the deeply personal -- I can still remember my confusion, as a 7 year old, when my grandfather didn't know who I was -- to the economic, with some experts estimating social costs of approximately $100 billion per year in the U.S. alone.
One of the more promising lines of research involves "biologics" -- a comparatively new category of medicine that, in contrast to drugs like Lipitor that are chemical-based, are derived from living tissue. Scientists are just beginning to understand and appreciate the full potential of biologics, which have promising applications for diseases as intractable and diverse as cancer, arthritis -- and potentially Alzheimer's. (For instance, a biologic called Enbrel has showed signs of promise in treating this disease.)
But medical advances require a fertile innovative environment, with room for competition and a supportive regulatory structure. Will new government policies nurture a competitive, creative atmosphere resulting in breakthrough treatments -- or stymie future innovation?
The FDA is wrestling with that question now. Not only does the agency have to set clear standards for the development of biologic treatments, but the inevitable development of their imitative counterparts. The core issue pertains to "biosimilars" -- what one might initially think of as being akin to a "generic biologic" (an imitative drug not made by the original drug company), but which, unlike generic chemical drugs, are not truly identical: just as no two living things are precisely alike, drugs extracted from living tissue can be similar at best, but never the same.
As we all know, generics are often far cheaper than brand name drugs. But, unlike chemical drugs, the process for developing imitative biosimilars is not clear. The question for the FDA is how to safely permit a "pathway" to biosimilars by companies who want to create similar versions of a biologic, but -- because they didn't invest in the initial research, which can run into the hundreds of millions -- can market the drug for cheaper.
A few key points come to mind. First, it's important to put patient safety first. By definition, there will be small differences between the proposed biosimilar and original biologic. The only way to ensure that these don't result in patient harm is to subject the biosimilar to similarly rigorous clinical testing as the original biologic.
Second, the U.S. has the advantage of not needing to reinvent the wheel: The European Union created a regulatory pathway for biosimilars in 2003, and that process can serve as a model for the U.S. to improve on.
Third, we've learned valuable -- sometimes, painful -- lessons from the traditional pharmaceutical industry. Heard of counterfeit medicines? "Track and trace" is the surveillance of a drug's performance and supply chain -- a process that helps identify and prevent fakes, and also monitors the medicine for unexpected adverse reactions. Before biosimilars are permitted in the U.S. market, a good traceability system should be in place that includes distinctive labeling, tracking codes and a way to report adverse medical events.
Today, too many families face President Reagan's "long goodbye" -- and too many Alzheimers' victims know, even as the disease begins to rob them of their memories, of the pain their families will face. A biologic medicine that will shorten or even eliminate this long goodbye is a reasonable hope and goal for our generation. But it requires a holistic approach to the innovation of not only new biologic medicines, but biosimilar medicines as well, that puts patient safety first.
John Horton, president of LegitScript in Portland, is the former Associate Deputy Director at the U.S. Office of National Drug Control Policy where he was the primary drafter of the Administration's National Synthetic Drug Control Strategy and co-drafted the President's National Drug Control Strategy from 2003 until 2007.
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