Bad 'Precision' Medicine — If Nobody Knows How It Works, Sometimes It Doesn't

The endeavor known as precision medicine, which Obama singled out in his State of the Union Address, may sound futuristic, but its been around long enough for people to have screwed it up, and badly. One of the worst medical scandals this century started with cancer researchers at Duke promising something that sounded a little too good to be true and ended with retracted papers, dashed patient hopes and lawsuits.

But precision medicine is obviously moving forward. To learn more about it, and what lessons the past has to offer, I caught up with Keith Baggerly, whose dogged investigations uncovered the problem with the Duke project. Baggerly is a professor in the Department of Bioinformatics and Computational Biology and Division of Quantitative Sciences at UT MD Anderson Cancer Center. (He is also a witness in a pending lawsuit filed by patients and their families.)

Though precision medicine has different meanings, medical researchers tend to use that term or personalized medicine to refer to the use of individual DNA differences in tailoring treatments to patients. The strategy is being driven by advances in the ability to quickly and cheaply read the sequences of code characters in DNA and by the growing use of big data to find patterns. As described in this Philadelphia Inquirer story, a number of big data cancer initiatives are gathering momentum.

The dream of precision medicine has been particularly tantalizing for cancer treatment, since cancer cells are just ordinary cells with broken DNA mutations that change the cells instructions and cause them to run amok.

And so, in 2006, cancer researchers around the word took notice when a team led by Dr. Anil Potti at Duke claimed in the prestigious journal Nature Medicine that theyd created a highly complex mathematical system that could assess a given patients tumor and determine from its genetic make-up exactly which drugs would give that patient the best odds of survival. While investigations have revealed fraud on the part of Anil Potti, many other people made mistakes in ignoring whistle blowers and allowing the technique to be used on cancer patients in a clinical trial.

While some avenues of precision medicine could lead to new, prohibitively expensive drugs used for rare subsets of patient, the Duke technique promised to chart the best course among existing treatments said Baggerly.

It would be based on the DNA in individual patients tumors. And it didnt just apply to one kind of cancer but to cancers across the board. Instead of telling a patient there was a 70% chance a drug would work to kill her tumor, he said, they could find out ahead of time if she was in the other 30% and prescribe an alternative course of treatment.

Doctors were excited and thought if the system worked, they owed it to their own patients to adopt a form of it, he said. Several groups asked Baggerly to look into it. One danger with the approach, he said, was that it was impossible to know how the technique worked. The data were so big they were measuring thousands of things per patient and there was this perception that the analysis of such data sets would be complex, he said. In most medical tests, theres some understanding of how they work. Thats true in some of the early advances in precision medicine. In some cases of melanoma, for example, theres a break in a particular gene called BRAF, and drugs that target cells with that broken gene. Theres a mechanistic understanding of how it all works.

But with the Duke project, he said, nobody has a good intuition of what 50 or 60 things are doing at once. And so there was no way for intuition to tell anyone whether it worked at all. When Baggerly started to re-analyze how the Duke researchers created the system in the first place, it didnt work. Was he using the system wrong or was there something wrong with the system?

As he investigated further, he found egregious errors that should have prevented it from working. The team had relied on cancer cell samples that had various degrees of resistance to an array of drugs. Those had been mislabeled. Some were reversed, so that the cells that were most resistant were labelled as the least.

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Bad 'Precision' Medicine -- If Nobody Knows How It Works, Sometimes It Doesn't

UnitedHealth's $43 Billion Exit From Fee-For-Service Medicine

Continuing the health insurance industrys march further away from fee-for-service medicine, UnitedHealth Group UnitedHealth Group (UNH) executives said this week they will increase value-based payments to doctors and hospitals by 20 percent this year to north of $43 billion.

UnitedHealth, considered a barometer for the health insurance industry given its size, is rapidly departing from the traditional fee-for-service approach that can lead to overtreatment and unnecessary medical tests and procedures. Value-based pay is tied to health outcomes, performance and quality of care provided.

We are expecting about a 20% increase in the concentration of value-based reimbursement, Dan Schumacher, chief financial officer of UnitedHealths UnitedHealthcare subsidiary, told Wall Street analysts on the companys fourth-quarter and 2014 full-year earnings call earlier this week. We ended the year at about $36 billion of spend in value-based arrangements and were looking to drive that north of $43 billion in 2015.

UnitedHealths pronouncements are in keeping with its previously stated commitment to increase payments that are tied to value-based arrangements to $65 billion by the end of 2018.

Value-based payments come in a variety of forms. They include: pay-for -performance programs, patient-centered medical homes and accountable care organizations, a rapidly emerging care delivery system that rewards doctors and hospitals for working together to improve quality and rein in costs. UnitedHealth said it is generating 1 percent to 6 percent in savings from its various value-based reimbursement approaches.

Once rolled out by commercial and government insurers on a pilot basis, they are quickly becoming the norm. The Centers for Medicare and Medicaid Services said 20 percent of its payments are no longer fee-for-service based for providers reimbursed by the Medicare health insurance program for the elderly, a spokesman confirmed to Forbes this week.

As insurance companies report their fourth quarter earnings in the next two weeks, look for Aetna Aetna (AET), Cigna Cigna (CI), Humana (HUM) and others to provide updates on their value-based contracting for this year.

The structure actually drives volume towards the better providers that enter into these performance contracts, UnitedHealth chief executive Stephen Hemsley said earlier this week. Were progressing these contracts into more sophisticated forms where theyre actually taking on even greater performance responsibility over time. Wondering how the move away from fee-for-service medicine will affect your health care? The Forbes eBookInside Obamacare: The Fix For Americas Ailing Health Care Systemanswers that question and more. Available nowat AmazonandApple.

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UnitedHealth's $43 Billion Exit From Fee-For-Service Medicine

Penn Medicine Opens Pavilion for Advanced Care, Integrating Critical Care Specialties and Expanding Penn Presbyterian …

Features of the new PAC will include:

Three state-of-the-art critical care units including a designated Heart and Vascular critical care unit, and PPMCs first neurosurgical and neurocritical care inpatient units increasing bed capacity by as many as 36 beds. A 16-bed increase in capacity in the new Emergency Department, in addition to a new 5-bay Rapid Assessment Treatment area designed to quickly and more accurately triage emergency patients. 24/7 eye injury treatment in the Emergency Department A new concourse that provides a consolidated Pre-Admissions Testing and Medical Imaging services, including the most advanced CT and MRI technology, digital X-rays, ultrasound and flouroscopy A new surgical suite that provides a bridge to the second floor of PPMC, and includes a new 30-bed Short Procedure Unit for outpatient surgeries such as hernia repairs, gallbladder removals, or eye and ear procedures. A new inpatient therapy gym An outdoor space which serves as both a healing garden and a common outdoor space for eating, and gathering.

Over the last decade, Penn Medicine has made numerous investments in people, facilities, and patient care that have strengthened our commitment to our patients, staff and our multiple missions of clinical care, research and teaching, said Ralph Muller, CEO of the University of Pennsylvania Health System. With this latest endeavor, Penn Medicine has the resources and in place to effectively elevate our care processes and provide better value to both our patients and payers.

Planning for the Pavilion for Advanced Care has involved work by hundreds of staff and leaders spanning 37 unique departments and divisions across Penn Medicine during the three-year planning process for the new facility.

This has been a momentous year for Penn Presbyterian, said Michele Volpe, executive director of PPMC. Beginning with the opening of Penn Medicine University City in August 2013 which now houses many of Presbyterians outpatient services and as we approach the final stages of the transition to the PAC, Presbyterian is now poised to deliver the most advanced medical care to some of our most vulnerable and critically ill patients.

Transitioning the Level I Regional Resource Trauma Center Penn Medicines Trauma Program treats more than 2,200 patients with life-threatening injuries per year. These injuries include those resulting from severe falls, motor vehicle and motorcycle collisions, injuries associated with violent crime, including gunshot wounds and stabbings. The new trauma center includes upgrades to the overall design and efficiency of caring for these critically injured patients, including:

A new oversized helipad on the roof of the PAC, equipped with self-cleaning and snow-melting technology, and to an elevator that takes the PennSTAR flight team from the helipad to the OR or Trauma resuscitation unit in seconds The John Paul Pryor, MD, FACS, Shock Trauma and Resuscitation (STAR) Unit: a state-of-the-art, 5-bay trauma resuscitation area and the largest known design dedicated to trauma resuscitation, which facilitates immediate access to Corridor of Life critical care treatment areas, including ceiling-mounted CT and MRI imaging and X-rays. Designated operating rooms, elevators and pathways for trauma patients and providers, allowing the quickest care when every minute counts toward the chances of survival

The Emergency Department at the Hospital of the University of Pennsylvania (HUP) will continue to be a full-service ED, equipped and staffed to handle more than 60,000 visits each year. HUP will also remain Penn Medicine's home for specialty emergency services such as the most advanced cardiac resuscitation techniques, hyperbaric medicine for carbon monoxide poisoning, and medical toxicology expertise for poisoning and adverse effects of drugs.

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Penn Medicine Opens Pavilion for Advanced Care, Integrating Critical Care Specialties and Expanding Penn Presbyterian ...

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