(AP)
Expediting the development and delivery of medical treatments—treatments that bring a new standard of care, or even actual cures for serious illnesses—should be a bipartisan goal, a mission of mercy that should be undertaken by both political parties. We are both involved with a new, non-profit organization, the Tomorrow’s Cures Today Foundation, which supports consumer choice and competition as one way to achieve this goal.
The world is a very different place today compared to 1962, when new federal legislation required at least two randomized control trials, in which a new drug was tested against a placebo or an alternative FDA-approved drug. Now we live in a data-intensive world, offering the capability to analyze observational data of actual drug treatment outcomes and patient health information.
We might note that time is especially critical for Lieutenant Commander (Ret.) Matt Bellina, who recently sent us an email that began: “My name is Matt. I‘m 31 years old, I’m a U.S. Navy veteran, I have a beautiful wife and two small boys, and I’m dying. I have ALS, or Lou Gehrig’s disease, and without hope for a cure, my doctors say I only have a couple of years left to live.”
The world is a very different place today compared to 1962, when new federal legislation required at least two randomized control trials, in which a new drug was tested against a placebo or an alternative FDA-approved drug. Now we live in a data-intensive world, offering the capability to analyze observational data of actual drug treatment outcomes and patient health information.
Matt has joined with other ALS patients to petition the Food and Drug Administration (FDA) to expedite the availability of a new drug, GM604, produced by the company Genervon, based in Pasadena, California. GM604 is showing truly remarkable effectiveness in clinical testing. Moreover, as of today, there is no FDA-approved drug—not one—that has stopped the progression of ALS. Given this glaring shortfall, we believe the FDA should grant “Accelerated Approval” so that patients such as Matt Bellina can gain access to GM604. Yet such Accelerated Approval is rarely given, and that’s part of the problem.
Indeed, even if the FDA cuts through its own red tape to help needy ALS patients, the broader issue still remains—the need to fix and streamline the fundamentally obsolete FDA regulatory process that puts FDA analytical wishes ahead of actual patient needs.
If the FDA were to act in the interests of real patients, and not bureaucratic abstractions, GM604 and other highly effective, but not-yet-approved, drugs would be routinely accessible as part of a new 21st century regulatory system. That is, we would have a system designed to expedite innovation for the benefit of patients today and tomorrow.
The world is a very different place today compared to 1962, when new federal legislation required at least two randomized control trials, in which a new drug was tested against a placebo or an alternative FDA-approved drug. Now we live in a data-intensive world, offering the capability to analyze observational data of actual drug treatment outcomes and patient health information. Technology firms such as Apple, Amazon, IBM, Google, and Microsoft work with insurance companies and hospitals to analyze what is now called “Big Data”—observations of medical treatments to discover what works best and for what type of patients.
Let’s imagine a new and better world which takes full advantage of this new flood of data—a world in which patients like Matt Bellina have the freedom to take the risk of using not-yet-FDA-approved drugs in order to receive potentially life-saving treatments not otherwise available. For some, being treated with a not-yet-FDA-approved drug is an unacceptable risk. However, risk depends upon an individual’s unique health condition as well as his personal preference for potential health improvement beyond that attainable from approved drugs. For ALS patients in dire straits, GM604 is not a “risky” treatment at all; it is their best current hope to stop the terrible slide from loss of muscle control, to paraplegia, to being on a ventilator—to death.
Moreover, in this better world to come, the treatment results and health details for patients such as Matt would be aggregated in ways that would help all future patients—as well as help the larger cause of medical science. That is, the results would be tabulated as observational data in an open-access database that you, and your doctor, could easily access on the Internet. This data could then be combined with clinical trial findings and other scientific research to further enable patients and their doctors to benefit from the resulting treasure trove of information.
How could this happen? The answer, of course, is Big Data. IBM’s Watson computer system is perhaps the best known computer system now taking us into this new world of analysis. Indeed, Watson-like technology is ideally suited to help us achieve Tomorrow’s Cures Today. And that’s a good thing, even if it threatens to overturn some elaborately contrived bureaucratic hierarchy in Washington, DC.
To make this new world a reality, our leaders need a vision of, yes, Tomorrow’s Cures Today. They need to see that a healthier population would also be a more prosperous population. How so? That’s because healthy people are not only personally happier, but also more economically productive.
We need legislation based on three mutually reinforcing political-scientific principles—principles that would comport with the technological vistas of Big Data.
First, we need a “free to choose” track; second, we need an open-access database; and third, we need FDA Observational Approval enacted. This alternative approval would utilize the power of today’s Big Data analytics to enable faster access to the most promising new drugs. In the Foreword in Bart Madden’s 2012 book, "Free To Choose Medicine: Better Drugs Sooner at Lower Cost," Nobel Laureate economist Vernon Smith wrote: “These three design components for patient/doctor control of medical treatment are both innovative and soundly based. With this conceptual blueprint, legislation could be crafted to promote both expanded consumer choice and the discipline of choice to the long-term benefit of society.”
Let’s see how this would work: After a drug successfully passes safety trials and shows initial effectiveness in clinical trials—that is, the early steps—a drug developer could request that their drug be available for sale on a “free to choose” track (the developer could elect also to continue on the FDA clinical trial track). As a result, patients such as Matt Bellina would be able to access innovative new drugs up to seven years earlier than waiting for a final FDA decision. For patients given only a few years—or months—to live, seven years sooner could spell life, not death.
Under our proposal, a patient’s doctor would be required to submit treatment results and medical information such as a patient’s genetic data to the open-access database. Doctors and patients would get real-time updates about the benefits and side effects of any “free to choose” drug and be able to make informed decisions about an early use of these new drugs versus approved drugs.
We might bear in mind that clinical trials involve patients who are mostly similar. On the other hand, because the “free to choose” option would be available to everyone, new insights would be obtained about how a drug performs for a far broader range of patients. These insights would better inform the biopharmaceutical industry, leading, in turn, to better allocation of research funds and faster innovation.
Some drug developers, especially small companies, would want to provide “free to choose” drugs in order to quickly demonstrate that their new drugs work and, in so doing, significantly enhance their ability to raise needed capital. Nevertheless, we need an added incentive that benefits both patients who need insurance reimbursement as well as drug developers seeking FDA recognition of their drugs’ safety and effectiveness. We need Congress to authorize an alternative drug approval mechanism—that is, FDA Observational Approval based on the safety and effectiveness demonstrated by observational data posted on the open-access database.
This movement to liberate medicine—the movement to apply Big Data and provide freedom of choice in medical treatment—may save your life.
No wonder, then, that it has become a political movement of sorts, enjoying tremendous popular support. Fourteen states have passed “Right To Try” legislation which allows terminally-ill patients the right to use an unapproved drug (and assumes also that drug developers agree to participate). The three principles of “free to choose” medicine described above extend the pioneering accomplishments of Right To Try proponents by providing incentives for patients, doctors, drug developers, data analysis firms, venture capital firms, and insurance companies—all to participate in improving the system of developing and delivering new drugs.
That’s why so many of us have come together in support of freedom of choice in medical treatment. Patients already benefit from a limited version of this new paradigm, since doctors are already free to prescribe any approved drug “off label” to treat medical conditions for which the drug was not tested. Extending this freedom as described would result in accelerated innovation, benefiting patients in the same way that consumer choice and competition benefits the private sector.
It is time for Congress to introduce Free To Choose Medicine legislation so that Tomorrow’s Cures Today becomes a reality. If our leaders can pull together for our common good, we are all likely to live healthier, longer, and more productive lives.
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