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Dr. Robert Califf named FDA Deputy Commissioner for Medical Products and Tobacco

U.S. Food and Drug Administration Commissioner Margaret A. Hamburg, M.D., today appointed Robert Califf, M.D., a recognized global leader in cardiology, clinical research, and medical economics, as FDA Deputy Commissioner for Medical Products and Tobacco.  

In this position, Dr. Califf will provide executive leadership to the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, the Center for Devices and Radiological Healthand the Center for Tobacco Products. He will also oversee the Office of Special Medical Programs in the Office of the Commissioner. Dr. Califf will play a critical role in providing high-level advice and policy direction on the agency’s medical product and tobacco priorities and will manage cross-cutting clinical, scientific and regulatory initiatives in several key areas for the agency, including personalized medicine, orphan drugs, pediatric science, and the advisory committee system.

“I am delighted to announce this important addition to FDA’s senior leadership team,” said FDA Commissioner Margaret A. Hamburg, M.D. “Dr. Califf’s deep knowledge and experience in the areas of medicine and clinical research will enable the agency to capitalize on, and improve upon, the significant advances we’ve made in medical product development and regulation over the last few years.”

Dr. Califf is currently serving as vice chancellor of clinical and translational research at Duke University. Other prominent roles during his tenure at Duke include director of the Duke Translational Medicine Institute (DTMI), and professor of medicine in the Division of Cardiology at the Duke University Medical Center in Durham, North Carolina. Before serving as director of DTMI, he was the founding director of the Duke Clinical Research Institute, the world’s largest academic research organization. Dr. Califf is recognized by the Institute for Scientific Information as one of the top 10 most cited medical authors, with more than 1,200 peer-reviewed publications.

During his career, Dr. Califf has led many landmark clinical studies, and is a nationally and internationally recognized expert in cardiovascular medicine, health outcomes research, health care quality, and clinical research. He is one of our nation’s leaders in the growing field of translational research, which is key to ensuring that advances in science translate into medical care. He was a member of the Institute of Medicine (IOM) committees that recommended Medicare coverage of clinical trials and the removal of ephedra from the market and of the IOM’s Committee on Identifying and Preventing Medication Errors. In addition, he served as a member of the FDA Cardiorenal Advisory Panel and FDA Science Board’s Subcommittee on Science and Technology. Currently, he is a member of the IOM Policy Committee and liaison to the Forum in Drug Discovery, Development, and Translation.

Dr. Califf will join the FDA in late February.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human drugs, including vaccines and other biological products for human use, veterinary drugs, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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For a balanced look at the current debate over opioids, check out A Delicate Balance: The challenge of treating pain—and stopping prescription drug abuse—in America. It’s timely, comprehensive, science-based sponsored content in The Atlantic and well worth a read.

ABOUT THIS SERIES

Pain exists. It’s pervasive. And, yet, the drugs prescribed for its treatment are often abused. A Delicate Balance explores several approaches to helping achieve the public health goal of stopping prescription opioid abuse while effectively treating people who live with chronic pain.

In his State of the Union address, President Obama proposed a federal initiative to accelerate the development of precision medicine and ensure that everyone would have “access to treatments that deliver the right treatment at the right time.” 

To illustrate his point, the President invited Bill Elder, a 27-year-old medical student with a rare form of cystic fibrosis to sit with First Lady Michelle Obama during his speech.    Mr. Elder has been using Kalydeco, which, as the president pointed out “has reversed a disease once thought unstoppable” by turning off the genetic mutation causing his disease.

But Kalydeco, is NOT easily available under most health plans.  Ask Chloe Jones, a 14-year-old Arkansan with the same type of cystic fibrosis mutation.   Arkansas’ Medicaid agency has refused to give her Kalydeco.  Instead she has to first fail to respond to older, less-expensive therapies that treat the symptoms but not the underlying cause.   (Kalydeco costs about $200,000 a year.) In other words, she has to get sicker and a step closer to death.

Sadly, Chloe is not alone.  She and hundreds of thousands of other patients are denied the precision medicines the president celebrates. Instead of ensuring access to precision medicines for the right patient at the right time, pharmacy benefit management companies  (PBMs) that put together drug formularies for health plans are only offering drugs from biotech firms that give them biggest cash discount. And then insurers are requiring kids like Chloe to get sicker or pay thousands of dollars before getting targeted treatments.

After the rebates are harvested by PBMs by excluding precision meds, health plans require patients to pay thousands of dollars out of pocket to get the one’s that are left.  Managed Care magazine points to a study by Avalere Health that revealed “many insurers require copayments of 10% to 40% for 19 classes of medications for patients with chronic conditions. More than 60% of silver plans put all covered medications for patients with multiple sclerosis, rheumatoid arthritis, Crohn’s disease, and certain cancers in the highest formulary tier.”

While most plans cap out of pocket costs, most consumers can’t afford thousands of dollars upfront.   As a result, patients don’t take the medicines that could save their lives.  Health insurers and PBMs know it and profit from it.

On top of all that, health plans and PBMs don't pay for the genetic tests that match people to the right medicines.  They want randomized clinical trials to demonstrate accuracy know it will take years to set up studies and get results. 

The orchestrated outrage over drug prices, led by John Rother and his AHIP funded National Coaltion on Health is nothing but part of cynical plan to extort rebates and limit access to precision medicines.  It is noteworthy that AHIP, and the PBMs have contributed nothing to the discussion around the bi-partisan 21st Century Cures Initiative.  If they cared about the cost of medicines, they would propose ways to reduce the time and money needed to develop and adopt precision medicine.   The same goes for critics who say nothing about the value of medicines and is not an advocate for 21st century cures.  

In fact, the pay for play schemes indicate that PBMs and insurers can only profit from precision medicine by rebates.   As Eric Topol points out in his new book "The Patient Will See You Now", the health data now controlled by payors and physicians will be generated and controlled by patients themselves.  Using smartphones patients will be able to find the right treatment for them based on their genetic and clinical data.   Where's the  need for middlemen at that point?  PBMs are the Blockbuster Video and Tower Record Stores of the healthcare system.  NO ONE will miss them when they are gone. 

We need to hasten the creative destruction of PBMs.    We can start by replacing fail first or step therapy approaches with value-based precision therapy.   And we should elimiinate the discriminatory practice to placing precision medicines in the most expensive formlary tiers and the coo-insurance costing thousands.    Finally, we need to expand the use of new medicines in the real world.  

Ultimately, the rebate and co-pay schemes plump up profits to prop up an outdated business model. PBMs and insurers are skimming off billions of dollars with drug benefit designs that target the most chronically ill and vulnerable patients. No wonder, patient groups have sued health plans for discrimination.  Change can't come fast enough.  

Are opioids “bad?” Certainly they can be addictive and that fact can’t be understated. That’s precisely why they are controlled substances. And still nascent abuse deterrent technologies are helping to further decrease the opportunities for improper use. But the value of opioids is, when used as directed, they are highly effective in combatting the scourge of pain.

The truth, of course, is that opioids aren’t bad. The problem is that they’re not perfect nor are they perfectly safe – just like 100% of prescription drugs on the market. “Safe” is a relative term. Opioids are safe when they are used as directed. Are chemotherapy drugs “bad” because of their horrible side effects? Of course not. Why – because the alternative is far worse. So too the case with opioids. For both physicians and the tens of millions of Americans with chronic pain, the absence of opioids would be disaster.

That’s why the FDA and other pertinent constituencies view the future of pain medication through the lens of “safe use.” How can we enhance appropriate prescribing, dispensing, and patient behavior? Abuse deterrent technologies are part of the answer, but better physician, pharmacist, and patient education must be another pillar. The FDA has made this a policy priority. FDA Commissioner Hamburg has called for “Improving appropriate prescribing by physicians and use by patients through educational materials required as a part of a risk mitigation strategy for extended-release and long-acting opioids.”  Now it's also time for the DEA to work with the FDA to develop smart policies for specific education as a must-have for prescribing rights.

Alas, there are too many pundits, politicians, and self-anointed citizen advocates who are keen to focus on placing blame. It’s a savvy strategy for media attention but does little to advance the public health. There’s no value in fixing the blame for medicines that aren’t 100% safe. No medicine is 100% safe. That cynical approach just leverages ignorance to produce anger. There’s tremendous value in fixing the problem – through advancing the safe use of opioids – a crucial weapon in the armamentarium against pain. 
When it comes to medicine, who should make decisions about safety and efficacy – and on what evidence should those choices be made? At present, the Food & Drug Administration has the responsibility to determine approvals and labeling language based on a scientific review of the evidence. Should this authority be ceded to the tort bar?

Consider the recent spate of litigation against the manufacturers of opioid pain medications. One example is the City of Chicago’s lawsuit against multiple manufacturers of opioid pain treatments. In the United States District Court for the Northern District of Illinois (Eastern Division), the City of Chicago’s First Amended Complaint (“FAC”) seeks to limit the ability of Chicago doctors to treat the chronic, non-cancer pain of patients in the manner doctors deem most appropriate. Although the Food and Drug Administration has approved certain opioid pain medications for the treatment of chronic non-cancer pain, the FAC seeks to deprive patients and doctors of that treatment choice by having six lay jurors determine that “the use of opioids to treat chronic pain is not ‘medically necessary’ or ‘reasonably required’ in that their risks do not exceed their benefits.”

The FDA has determined that opioids serve an important public health role: “When prescribed and used properly, opioids can effectively manage pain and alleviate suffering—clearly a public health priority. Chronic pain is a serious and growing health problem: it affects millions of Americans; contributes greatly to national rates of morbidity, mortality, and disability; and is rising in prevalence. At the same time, there is no dispute that opioids pose significant public health risks: Opioids also have grave risks, the most well-known of which include addiction, overdose, even death. The labeling for these products contains prominent warnings about these risks. Moreover, the boxed warning states that all patients should be routinely monitored for signs of misuse, abuse, and addiction.

In September 2013, the FDA ruled on a citizen’s petition filed by a group of clinicians, researchers, and health officials called Physicians for Responsible Opioid Prescribing (“PROP”). Like the Chicago FAC, the Petition directly challenged the use of opioids for “chronic non-cancer pain.” PROP contended that the “long-term safety and effectiveness of managing [chronic non-cancer pain] with opioids has not been established,” and requested that the FDA, inter alia, impose a “maximum duration of 90-days for continuous (daily) use for non-cancer pain.” The FDA carefully reviewed the Petition and more than 1900 related comments. The agency assessed the relevant literature. It held a two-day public hearing at which it received “over 600 comments” and dozens of experts and concerned citizens testified. The FDA noted that “the majority of comments” “opposed PROP’s requests” and that “many professional societies,” including the American Medical Association, “did not support the Petition and stated that the data cited by PROP did not support PROP’s requests.” After completing a 14-month review, the FDA determined that opioids should continue to be available for the treatment of chronic pain, while also directing further study and certain labeling changes for some opioid drugs. Significantly, after being presented with the same assertions as those now alleged in the Amended Complaint, the FDA made two findings directly at odds with the underlying premises that form the cornerstones of the FAC.

But the lawsuits keep coming. The FDA has the authority, the ability, the means, the mission, and the mandate to manage the health care risks and benefits inherent in the products it regulates on behalf of the American public.

A more balanced legal system will occur only when elected officials determine the time has come for real tort reform, as it affects pharmaceutical companies.  But that day is likely very far off. Healthcare leaders must devote their most aggressive efforts toward reform.

Maybe when our elected officials understand that it's the health of their constituents versus the pocketbooks of lawyers, our public servants will finally get serious on tort reform.

Bach to the Future

  • 01.20.2015

Time and again, the dangerous idiots of healthcare reform claim that there's no difference between so-called "me-too" drugs, and that they're simply a way for drug companies to turn a quick profit. The New York Times,in an editorial, claimed that "the nation is wasting billions of dollars on heavily marketed drugs that have never proved themselves in head-to-head competition against cheaper competitors." Wrong.

A recent op-ed in the New York Times, (Why Drugs Cost So Much, NYT, January 14, 2015) by Peter Bach is only the latest in a series of such bandwagon drivel – and he should know better.

The truth is that despite the assertions in and by the New York Times, different drugs are indeed different, even if you describe them as "me-too" medications. Likewise, even though every brand of store-bought peanut butter contains peanuts, oil, sugar, and salt, no one would argue that Skippy and JIF taste exactly the same.

Time and again, different medicines have proved themselves where it counts - in the bodies and biochemistries of patients. No two patients are alike, and the more options available, the more likely doctors are to find what works best for each patient. A few points to consider:

* When patients have access to more effective medications, their overall health improves, even as their overall medical expenses go down. That, in turn, reduces national health-care spending and boosts the economy. Value must be measured in patient outcomes.

* Healthcare innovation saves lives, saves money, promotes economic growth, and provides hope for hundreds of millions of people (both patients and care-givers) in the United States and around the world.

* If we do not support the development of new medicines through timely licensing and fair pricing, innovation will be stopped in its tracks – and that is not an acceptable public health outcome.

* Regulators can be partners in innovation three ways: Through robust oversight, through active collaboration, and, most importantly, by being an innovation enabler.

 The deviation between value and pricing in a distinction with a difference – and while both are important, it is value that’s the higher priority since driving patient outcomes is the higher calling (and better long-term economic investment).

After all, as Yale economist William Nordhaus has written, "The social productivity of health care spending might be many times that of other spending.”

Luckily, America's doctors, drug researchers and patients are smarter than that. Just like there aren't any "me-too" patients, there aren't any "me-too" drugs. And if cars and peanut butter are going to be personalized, then medicine is too important not to be.

Peter Bach yet again tries to make the case that new drugs are too expensive.

He writes: "The price of drugs is so unfair and mystifying that publlic often pays a high price when the identical drug is available at a fraction of the cost."

Actually, he didn't write that.  It's from another New York Times article: " Drug Prices Here Held Inequitable. "   It was written 40 years ago.  Burks, E. (1965) Drug Prices Here Held Inequitable. The New York Times Retrieved from https://secure.pqarchiver.com/

What he did write is another variation on this very old theme: "We can free insurers and government programs from the requirement to include all expensive drugs in their plans as we explain to the public that some drugs are not effective enough to justify their price. If we do this, we can be confident that manufacturers will lower their prices to ensure their ability to sell their products. Or we can piggyback on the gumption of bolder countries, and demand that policy makers set drug prices in the United States equal to those of Western Europe. Either approach would be vastly superior to the situation we have today."

First the facts.  

1. Nearly 90 percent of all brand drugs are discounted.  Companies pay rebates to Medicare, Medicaid, and the Veterans health system.    In the private sector (including Obamacare plans) drug firms pay rebates to pharmacy benefit management companies to get preferred listing.  

2. Bach is incorrect in claiming insurers have to cover every ‘expensive’ drug.  Express Scripts has 66 products on its 2015 formulary exclusion list, compared with 48 in 2014. CVS Caremark’s 2015 list has 95 products, including 72 carryovers from the 2014 edition.  

He loves this idea.  But what about patients?

From Managed Care Magazine:  

"The problem with this approach is that by adding more tiers health plans have confused employees and made some medications more expensive — the antithesis of a value-based strategy. They also have retained cost-sharing mechanisms that make high-priced drugs unaffordable for many patients, Vogenberg says. A 25% copayment on a drug priced at $1,000 a day costs a patient $1,750 each week or more than $7,000 per month. Many patients simply cannot afford their medications, even though commercial plan members often use copayment assistance programs from pharmaceutical manufacturers and can avoid such high cost-sharing amounts, says Brenda Motheral, RPh, MBA, PhD, president of Artemetrx, a specialty drug management consultant.

Bach is silent as PBMs pocket rebates while at the same time sticking patients with the higher costs of meds.


3.  Compared to Europe and Canada, on average Americans then pay slightly more for new medicines  in exchange for faster and broader access.   On average we get twice the new medicines about a year and a half earlier with fewer restrictions.  When all is said and done, using Euro-prices would save a grant total of 8.6 billion.. less than one third of one percent of total health care spending."

4.  Contrary to Bach, Americans also pay less out of pocket for medicines than in Europe. On average, out of pocket costs are about $800.  

4.   European speand more on cancer care as a percentage of health care and less on cancer drugs.  

Nowhere in Bach's article does he mention the value of broader, faster access to new medicines.   Let's stick with the European comparision.

Cancer mortality rates in the US are lower than most anywhere in Europe for every major cancer.  And five year survival rates (adjusted for lag time and incidence) are much higher and increase faster.  That doesn't include the life years lost when people with advanced forms of cancer die waiting a year and half for a new medicine or have strict limits place on access.    

American get broader access to better drugs that save money at about the same price as medicines elsewhere.  Price controls and PBM restrictions on access to new drugs actually increases cost and makes people sicker.  And probably let a lot of people die waiting for new medcines.  If a new medicine adds 3 months on average to the life of a cancer patient it would mean that each year (based on 590,000 cancer deaths a year) someone is denied access robs people with cancer of about 145000 life years annually. 


Back claims other systems are vastly superior.  The evidence suggests otherwise.  Cutting drug prices does kill people. 

The gain in pain is plainly in the main.

Abuse of opiate-based prescription painkillers such as oxycodone and morphine peaked around 2010-2011 and now may be on the decline in the United States, according to an analysis of databases designed to track illicit use of the drugs.

New laws, programs and policies, such as prescription tracking systems and the reformulation of oxycodone to make it harder to abuse, may be combining to reverse the once-growing trend, researchers said.

"I think we're at an inflection point and we're starting to turn this steamship around," said Dr. G. Caleb Alexander, co-director of the Johns Hopkins’ Center for Drug Safety and Effectiveness, who was not involved in the research.

The new study, published in the New England Journal of Medicine, looked at data collected between 2002 and 2013 from substance-abuse treatment centers, poison centers, college students and drug-diversion investigators.

There were "large increases in the rates of opioid diversion and abuse from 2002 to 2010, but then the rates flattened or decreased from 2011 through 2013. The rate of opioid-related deaths rose and fell in a similar pattern," write the study authors, led by Dr. Richard Dart of the Rocky Mountain Poison and Drug Center at the Denver Health and Hospital Authority in Colorado.

The full NEJM article can be found here.

Now we need to continue to move (and act) beyond the rhetoric and pursue additional solutions. And at the top of the list is the foundation of the Hamburg Manifesto -- prescriber education.

E&C Show and Tell

  • 01.14.2015

Per BioCentury, Reps. Fred Upton (R-Mich.) and Diana DeGette (D-Colo.) previewed on Tuesday the goals of legislation they plan to release this month as part of their 21st Century Cures initiative.


In commentary published by CNN, the legislators wrote that their bill will seek to “modernize clinical trials to streamline the approval of drugs and devices,” in part by reducing paperwork and promoting adaptive trials. It will also help FDA “better integrate the patient perspective into the regulatory process,” including using public-private partnerships to strengthen science around biomarkers and patient-reported outcomes.


The bill's sponsors also aim to promote “better access to and sharing of information such as genomic and other clinical data to foster more collaboration among researchers," and to invest in programs for young scientists.
Upton -- the chair of the U.S. House's Energy & Commerce Committee -- and DeGette also plan to “incentivize new drugs and devices for unmet medical needs” by “streamlining the premarket process while establishing mechanisms to better capture real world evidence post-market." They also said they will examine incentives, including “exclusivity or simplifying the reimbursement process,” to stimulate the development of new drugs and devices for unmet medical needs.

More as more develops.

Brill not so bril

  • 01.13.2015

The review of Steven Brill’s “What Ails Us” (NYT Book Review, January 11, 2015), refers to the pharmaceutical industry’s back-room negotiations to "gut" comparative effectiveness under Obamacare. The truth is that the Recovery Act of 2009 provided $1.1 billion for patient-centered health research (also known as comparative effectiveness research) – and that’s only part of the story.

What Mr. Brill is actually referring to is the administration’s “deal” with the pharmaceutical industry to leave in place what is known as “the Non-Interference Clause,” which prohibits the Federal government from negotiating drug prices for (among other things) the highly successful Medicare Part D drug program. It’s important to note that the Non-Interference Clause was the brainchild (during the Clinton years) of Senators Tom Daschle and Ted Kennedy – hardly candidates for the Tea Party Hall of Fame.

According to the Congressional Budget Office (in 2004), revoking the Kennedy/Daschle Non-Interference Clause, “would have a negligible effect on federal spending because CBO estimates that substantial savings will be obtained by the private plans and that the Secretary would not be able to negotiate prices that further reduce federal spending to a significant degree. Because they will be at substantial financial risk, private plans will have strong incentives to negotiate price discounts, both to control their own costs in providing the drug benefit and to attract enrollees with low premiums and cost-sharing requirements.”

In 2009 the CBO reiterated its previous views, stating that they, “still believe that granting the Secretary of HHS additional authority to negotiate for lower drug prices would have little, if any, effect on prices for the same reason that my predecessors have explained, which is that…private drug plans are already negotiating drug prices.” Unlike other healthcare benefits, Part D is also cost-effective for taxpayers. In 2014 the CBO reported Part D drug spending was 45 percent below original cost projections. 

Importantly, the CBO says that no further savings are possible unless the government restricts beneficiary access to medicines or establishes market-distorting price interventions. In other words, price controls lead to choice controls.

CMPI

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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