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  1. Beenthere2Hippie
    The Senate last month confirmed Dr. Robert Califf to run one of the world's most influential health agencies, the U.S. Food and Drug Administration. The 89-4 vote followed a delay by a handful of lawmakers who had turned President Obama's nomination into a proxy fight over the FDA's willingness to approve opioid pain-killers, despite a growing problem with addiction nationwide. Opioid addiction will be a major priority, Califf says, but it is just one of the items on his to-do list. As commissioner of the FDA, his responsibilities extend from the safety of the U.S. food supply to the regulation of drugs, some produced through scientific advances that were unimaginable even a few years ago.

    Prior to his appointment, Califf was professor of cardiology at Duke University School of Medicine and has led numerous landmark clinical trials and served on many pivotal committees, including one charged with developing recommendations for identifying and preventing medication errors. In his first in-depth interview since taking office, Califf spoke with U.S. News about his agency's efforts to fight the opioid epidemic, the future of cancer research and other priorities. Excerpts:

    Your nomination was held up by senators concerned about the opioid epidemic. How is FDA responding to their concerns?

    We are going to be dealing with opioids every day, until everyone's convinced that the tide has turned and the problem is receding significantly. It's a very big problem and we're going to stay hyperaware and focused on it.

    We are working in conjunction with [the U.S. Centers for Disease Control and Prevention] to support their guidelines . The fundamental issue right now is over-prescribing and FDA has a major role to say prescribe fewer opioids. Don't neglect people in significant and chronic pain, but prescribe fewer opioids.

    You've advocated the development of abuse-deterrent forms of pain-killers that make it more difficult for abusers to crush pills so that they can inject the medicine. Critics say this won't solve the problem. What's your response?

    We completely agree that if you take an abuse-deterrent formulation, it doesn't change the risk that you could get addicted. But formulations that work may still prevent some forms of abuse. It's early days. There's a lot of technology development to do. It's a very high priority for us to work with industry to come up with effective abuse- deterrent formulations.

    What is at the top of your agenda for FDA?

    The workforce. We have a tremendous workforce that has done a pretty amazing job across the ecosystem from agriculture to medical products. We're going to need to reinforce that by hiring really good people and creating the best possible environment to retain the really great people that we have. The expertise you need to be great at FDA is the same expertise needed in cutting-edge companies that are developing new medical products or health related information technology – or figuring out ways to get higher yields in agriculture with better nutrition.

    You've long been an advocate of rethinking the studies that we use to approve drugs and draw conclusions about public health. How do you see these changing?

    In the way we generate [scientific] evidence. The FDA does really well when it has high quality evidence. It's really good at making decisions that benefit the public health. When decisions have to be made without the highest quality evidence, it's not ideal. What's really great now is that – with over $50 billion invested in electronic health records and with multiple other sources of data, from social media to high-end computation to devices that are implanted or wearable – we've got an amazing opportunity to increase the evidence that will drive good decisions.

    Another way to think about this is that we've been very dependent on clinical [drug] trials as one key source of information. Clinical trials are very expensive; as we learn to use existing data sources better, the cost of clinical trials can come way down. This is also pertinent to food safety… If you think of the vast diversity and complexity of agriculture, there's no way you can inspect every product, one product at a time. But when you use high-end analytics, one can [target] the more detailed inspections to where the problems are.

    What about clinical trials specifically, the hugely expensive studies that examine the risks and benefits of medication?

    In this era, genetic and genomic information have given us the opportunity to develop targeted therapy and treat rare diseases in ways that wouldn't have been imaginable before. This also affects clinical trial design. In some cases, [involving rare diseases,] clinical trials may actually be a lot smaller and more targeted [than in the past]. In other cases, when therapy might be administered to millions of people on a global basis, they may need to be much larger and more inclusive.

    How much influence does FDA have when it comes to shaping the design of these trials?

    FDA gets to see all the trials before they're started, if they're done with the goal of gaining approval or changing the label. This is a very powerful position to be in, with a lot of responsibility. As you might imagine, people don't want to do a trial that the FDA doesn't like, if they need FDA's approval when the trial's completed.

    After decades of relative drought, there are dozens of cancer drugs in the pipeline. Have we turned a corner in cancer treatment ?

    You're right, if you went back a couple of decades, there was a lot of worry that cancer was an area where progress was slow. It was hard to find highly effective therapies. That's totally reversed. The ability to implement targeted chemotherapy and immunotherapy appears to be revolutionary. But it appears that it is not a magic bullet.

    [Cancer researcher Dr.] Bert Vogelstein at Johns Hopkins [University School of Medicine] has a tremendous lecture on this. He makes the point that we really are at war with cancer: We fire our salvo in terms of therapy, and the cancer responds by adapting and changing its tactics. Then we have to go after it again. For many kinds of cancer, we now, for the first time, have effective treatments. But…many people need additional treatment as time goes on.

    Our growing understanding of the human genome makes it possible to come up with precisely targeted therapies for many diseases, not just cancer. What does the precision medicine revolution look like from your perspective?

    It's useful to think about precision medicine and the cancer "moonshot" [Vice-President Joseph Biden's effort to accelerate research to find a cure] as being tightly integrated and overlapping. To me, of course, [the power of] genetics and genomics to help us understand the molecular basis of disease is crucial for being able to target therapies better than we could before. But it goes beyond genetics. If you look at the Precision Medicine Initiative [the White House plan to study one million people and precisely target therapies] the emerging use of wearable technology and social media allows us to understand things like patient preferences and continuously record data that we couldn't monitor before.

    One part of precision medicine would be to say, "We understand you have gene that's putting you at risk and we have developed a treatment." Another would be to say, "Now that we can look at your blood pressure [24 hours a day, seven days a week] we understand you have a pattern that's different. You need a treatment that is different." Or, "We have three different treatments for your problem, and now that we can communicate with you about your preferences, we can make the treatment more precise to achieve the risk-benefit trade-off that' s important to you."

    If you look at the Precision Medicine [million-person study] – the initial funding is underway now – it will be built along the whole spectrum from genes to patient preferences.

    How does this affect FDA?

    As you can imagine, this has major implications for FDA. As precision medicine therapies are developed, they still, like everything else, have to meet evidentiary standards to be put on the market and made available.

    Do you have to create these new standards out of whole cloth?

    In many cases, they will be less different than people think. If we were restricted by paper records, as we used to be, we might say, "Gee if you've got a targeted therapy that pertains to a small part of the population you can't possibly do a [large] clinical trial. But when everybody has an electronic health record and people can sign up on the web to be part of the Precision Medicine Initiative [research study] we may not be limited by the kinds of things that kept us from getting high-level evidence before.

    In other cases?

    There are areas that are entirely novel. One that's getting a lot of our attention now is next-generation sequencing. Up until now, one worked in a lab [and developed one test at a time]. Next-generation sequencing allows [us to decode] the entire genome all at once. Each one of us has three billion base pairs in our genome. You could say it's like doing 3 billion tests at one time on each individual person.

    It's obvious we can't regulate that one test at a time. We're working closely with the National Institutes of Health and others to develop...what's called a data commons. You put the [sequence] data in a big database—which, by the way, would have been impossible three years ago, because you need cloud computing to do it. We can look at the database and see which tests are working and which aren't. And we can label the tests appropriately, so that, when a doctor orders a test, there's a much higher chance of understanding what the result means when the test comes back. It's a whole new away of thinking about regulation, made possible by this amazing change in technology.

    Everyone's concerned about the high cost of drugs. Can FDA do anything about it?

    The fact that over 90 percent of prescriptions are now [lower-cost] generics is a major victory. it's critical for us to keep the pipeline of generics healthy.

    By Steven Sternberg - US News/March 21, 2016
    Newshawk Crew

    Author Bio

    BT2H is a retired news editor and writer from the NYC area who, for health reasons, retired to a southern US state early, and where BT2H continues to write and to post drug-related news to DF.


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