The Slippery Slope of Price Controls

02/15/2013 11:03 AM |

June 2003, "I happen to be a proponent of a single-payer universal health care program."  -- Barack Obama

Calling for the revocation of the Kennedy/Daschle Non-Interference Clause is the Drug Importation canard of the Obama Administration. – a faux policy that pretends to offer an easy solution to a complex problem. Populism may "sell" but it doesn't solve – and it may be a stalking horse for something more ominous.

During Tuesday’s State of the Union the President said, “We’ll reduce taxpayer subsidies to prescription drug companies.”

And on Thursday (and right on cue), a “coalition of liberal groups” (according to an article in Politico), launched a campaign to allow Uncle Sam to negotiate Medicare drug prices, claiming it will “save billions of federal dollars every year.”

Senator Amy Klobuchar (D/MN) recently introduced a bill (S. 117) to empower the Health and Human Services Department to negotiate for lower drug prices. "This is a matter of fairness for our seniors, who deserve affordable prices for their prescription drugs, and it is a matter of fairness for America's taxpayers, who deserve less waste in our system," she said.

Senator Klobuchar should pay closer attention to the numbers.

The non-partisan Congressional Budget Office (CBO) has found that Part D plans “have secured rebates somewhat larger than the average rebates observed in commercial health plans.”

And the Medicare Trustees report that “many brand-name prescription drugs carry substantial rebates, often as much as 20-30 percent and that on average, across all program spending, rebate levels have increased in each year of the program.

Is the argument that Uncle Sam could do better?

According to the CBO (in 2004), revoking the Kennedy/Dascle 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 2007 after two years of experience with bids in the program, the CBO found that striking noninterference “would have a negligible effect on federal spending because … the Secretary would be unable to negotiate prices across the broad range of covered Part D drugs that are more favorable than those obtained by PDPs under current law.”

In 2009 after even further program experience, 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.”

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 the words of USA Today (America’s vox populi) “Government price negotiation could leave people without drugs that manufacturers decide aren't sufficiently profitable under the plan … With that kind of clout, government might try to dictate prices, not just negotiate them. This could leave people without drugs that manufacturers decide aren't sufficiently profitable under the plan. The VA plan illustrates the point. It offers 1,300 drugs, compared with 4,300 available under Part D, prompting more than one-third of retired veterans to enroll in Medicare drug plans."

Is revoking the Kennedy/Daschle Non-Interference Clause the President’s next move towards what he has previously said is his preferred policy solution – a single payer system? It’s a question worth asking.

  Read More & Comment...

The Danger of False Profits

02/14/2013 05:46 AM |

Just in time for Valentine’s Day comes the Institute of Medicine’s new report, Countering the Problem of Falsified and Substandard Drugs.

Some relevant findings include:

 

·      Congress should fund the FDA to do a national track and trace system, possibly using California as a model.

·      The 50 states should adopt NABP's standards for wholesaler licensing (and revocation of license). 

·      The FDA should publish a list of wholesalers who have lost their licenses from states to, in effect, create a de facto national wholesaler blacklist.

·      NIST should setup a repository of counterfeit detection technologies.

 

Lots of good information.

More good information (and insights) will be at hand at the February 28^th conference, The Danger of False Profits: The Threat of Counterfeits to the Public Health. Jointly presented by the Center for Medicine in the Public Interest and the Institute for Policy Innovation, this event will feature speakers iSenior Counselor to the FDA Commissioner John Taylor, Jeff Gren, Director, Office of Health and Consumer Goods, U.S. Department of Commerce, John Clark, Vice President & Chief Security Officer, Pfizer, Michael Maves of Project Hope, Former Executive Vice President and CEO, American Medical Association, Gaurvika Nayyar Research Fellow and Analyst, National Institute of Health, among others.

Event Details:

Thursday, February 28, 2013

9:00 am - 2:00 pm

Reserve Officers Association Headquarters, 5th Floor

One Constitution Ave NE, Washington DC

*Complimentary lunch will be provided

 

Questions? Please contact Erin Humiston at (972) 874-5139, or erin@ipi.org

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Promises to keep

02/13/2013 08:55 AM |

President Obama on Medicare,

“We’ll reduce taxpayer subsidies to prescription drug companies and ask more from the wealthiest seniors.”

Does that mean another ill-considered assault on the Kennedy/Daschle Non-Interference Clause and a call for Medicare means-testing? Stay tuned.

“Our government shouldn’t make promises we cannot keep.”

Like "If you like your health care plan, you can keep your health care plan.”

Hm.

“But we must keep the promises we’ve already made.”

Like 12 years of data exclusivity for biologics?

Just asking.

He that promises most will perform least.

-- Gaelic Proverb

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Another Dick at CDER

02/12/2013 12:41 PM |

CDER Staff:
 
I am pleased to announce that Richard (Rich) A. Moscicki (Mo-shis-ke), M.D., a nationally recognized expert in clinical research and development, has been selected as deputy center director for science operations.
 
With CDER now over 4,000 employees, Dr. Moscicki, in his newly established role, will join Bob Temple, deputy director for clinical science, and Doug Throckmorton, deputy director for regulatory programs, in providing leadership and overall direction to Center activities to ensure that we accomplish our mission most effectively.
 
Dr. Moscicki currently serves as senior vice president (SVP), Head of Clinical Development at Genzyme Corporation. He joined Genzyme in 1992 as medical director and became the chief medical officer and SVP of biomedical and regulatory affairs in 1996 -- holding that post until 2011.
 
Over the past two decades, Dr. Moscicki has been responsible for worldwide global regulatory and pharmacovigilance matters, as well as all aspects of clinical research and medical affairs for the company. He is known to be inclusive in his management style and is credited with implementing solid business practices to sustain the company’s long-term operations.
 
As deputy director for science operations, Dr. Moscicki will share in executive direction of Center operations and provide leadership in overseeing the development, implementation, and direction of our programs.
 
Dr. Moscicki received his medical degree from Northwestern University Medical School. He is board certified in internal medicine, diagnostic and laboratory immunology, and allergy and immunology. He completed his residency with a focus on immunology, followed by a four-year fellowship at Massachusetts General Hospital (MGH) in immunology and immunopathology. He remains on staff at MGH and on the faculty of Harvard Medical School.
 
His medical, academic, clinical, and regulatory knowledge and expertise – coupled with his strong leadership and organizational management qualifications – make him the ideal candidate for this important position.
 
Please join me in welcoming Dr. Moscicki. He respects the work that we do and is looking forward to bringing his skills and experience to our Center to assist us in advancing the Agency’s mission.
 
Janet Woodcock  Read More & Comment...

CBO thumbs up for Part D’s downward revision

02/12/2013 09:21 AM |

Less is more.

The CBO report outlining its 2013-2023 projections is not pretty reading -- but there is one exception:

“The largest downward revision in the current baseline is for spending for Medicare’s Part D.” (This citation appears on page 57 of the report.)

The actual data table can be found here.

The ever-accumulating evidence shows that Part D is succeeding beyond all expectations, delivering needed prescription drugs to Medicare beneficiaries for less money than anyone expected—driven by strong competition among plans.

Note to President Obama, if it ain’t broke, don’t fix it.

Smart partnership between government and the free market works.

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Israeli Anti-Viral Decoy Wins First Prize at Singularity FutureMed

02/11/2013 12:55 PM |

 

3D bioprinter and “virus traps” take top startup honors at FutureMed pitch contest

February 10, 2013 7:58 am by Deanna Pogorelc | 2 Comments

As you’d expect at an event called FutureMed, disruption ruled this week’s event, and Saturday’s startup pitch contest was no exception.

Of the 27 companies that participated, the two that wowed the panel of investor judges the most were seriously outside-of-the-box ideas.

For their “startup to watch” award, the judges chose a company that made a splash in the tech world last fall when Peter Thiel’s foundation put $350,000 behind its novel idea to make more sustainable meat by 3D printing it. That company is ModernMeadow, co-founded and pitched by Andras Forgacs, who’s also one of the co-founders behind the human tissue-focused 3D bioprinting company Organovo.

It was a compelling presentation with some mind-blowing statistics and visuals depicting theresources required to produce a quarter-pound burger. ModernMeadow’s business model also includes a cultured leather product.

The overall startup winner, Vecoy Nanomedicines (Vecoy standing for virus + decoy), is focused on creating a new way to address one of the biggest unmet needs in medicine by outwitting viruses that cause infection.

The Israeli-based company is working on a therapy that uses tailor-made “virus traps” that mimic human cells and cause viruses to attack them. When they attack, the viruses get locked into the decoys and annihilate themselves before they can reach the real human cells, CEO Erez Livneh said. He noted that this new approach to fighting viruses may have the potential to lower the viral load in patients with HIV/AIDS, hepatitis B and C and other infections.

[Disrupt image from BigStock Photos]

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What Celgene's Pomalyst Tells Us About Medical Progress

02/11/2013 12:40 PM |

Here's what the pundits will say about Pomalyst,  an immunomodulating drug developed by Celgene for patients who have a form of multiple myeloma (MM) that has mutated around other treatments (this is from the well-known peer-reviewed medical publication, The Daily Beast)

“So far, most new (cancer) drugs offer only marginal extensions of life and few cures. “


Now a look at the facts and where Pomalyst fits in the progress we are making against MM.  Pomalyst was studied in people with MM who had no other options.  As in,  their MM was no longer responding to other drugs and they were going to die.  

Clinical trials showed that median overall survival for people getting Pomalyst was 13.7 months.   


Another study looked at response of patients who were no longer responding to other drugs and whose MM had already come back:

Clinical trials showed halfway into the study that nearly 50 percent of people receiving Pomalyst responded rapidly (as in the MM stopped progressing) and 78 percent had a  median surival time of between 3-6 months.   Patients receiving Pomalyst were seven times more likely to respond (and live) then those who received an old MM drug. 

Now overall survival is the average time half of all patients in a study will live after treatment.   So a median survival of 13.7 months  means  that after 13.7 months, 50% of people with that condition would be alive, and 50% would have passed away.   Which also means that many people in the trials are living much longer than 13.7.   Or in the other study, 3-6 months.   

Several studies have shown  that the median survival of people diagnosed with MM has gone from 3 years in 1997 to up to  20 years.  The first MM drug was introduced in 1998.  Two others were introduced in 2003 (Velcade) and Revlimid (2005) thereafter.    W hen age is controlled for, that  means an increasing number of people with MM can expect to have the same life expectancy as people who do not have the disease.  And we can't measure the full impact of these drugs let alone Pomalyst in a precise manner.   

But we can say that MM drugs have increased average life expectancy more rapidly among MM patients than it has average LE in the population as a whole. 

And we can say that just as a few HIV drugs introduced over a 5 year period dramatically saved lives, so too have a handful of drugs for MM turned an incurable disease into a controllable condition in less than a decade.  And these drugs work because they target specific disease mechanisms in patients for whom they work the best and offer the most hope.   The averages are indicators of profound and rapid gains among more and more people.  

If we had bought the story about marginal benefits 10 years ago and delayed and dragged out approval and use of other cancer drugs, where would we be today?  If we accepted the arrogant assertion that "most new (cancer) drugs offer only marginal extensions of life and few cures.“ how may more people would be dead?   Let me go out on a limb and say that while not all new products are useful and effective,  the current effort to control health care costs is biased and shaped by this naysaying approach to innovation, an approach delays access to new medicines -- indeed an approach that subsidizes a industry of underachieving social scientists to study average cost effectiveness in an era of personalized medicine --  assures more people will die who did not have to or want to.    Recently, the National Pharmaceuetical Council held a conference on The Myth of the Average Patient..  All well and good.  But how about a conference on what CER will cost people seeking better medicines for cancer and Alzheimer's in terms of lives saved.   My late colleague John Vernon and I did a lot of work in this area but somehow the "stakeholders" always ignore the impact of adding CER to the rate of innovation and the number of lives saved.   Why is industry and patient groups silent about the impact of CER on medical progress?  What gives? 

Finally,  why does it take so long to conduct clinical studies.  In both of the trials mentioned above, response was immediate and highly effective.  (Less than 6 months in most cases).  Can't we find other ways to confirm what works and what doesn't?  Here's Eric Topol on this important topic..

We have this big thing about evidence-based medicine and, of course, the sanctimonious randomized, placebo-controlled clinical trial. Well, that's great if one can do that, but often we're talking about needing thousands, if not tens of thousands, of patients for these types of clinical trials. And things are changing so fast with respect to medicine and, for example, genomically guided interventions that it's going to become increasingly difficult to justify these very large clinical trials.


We are not just standing in the way of medical progress, we are putting up obstacles based on outdated science and sanctimony.  

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Katz: Unjammer

02/11/2013 08:42 AM |

Herding Katz.

The departure of Rusty Katz as FDA’s Director of the Neurology Products Division is an inflection point in the agency’s 21st century direction. Will his replacement continue his philosophy that the FDA must be a partner in innovation – or will political considerations turn back the clock?

Following Katz at FDA is an important analysis of the situation.

  Read More & Comment...

Tooting our own horn

02/08/2013 07:56 AM |

It ain’t bragging if you can do it.

Pleased and proud to report that, according to the Healthcare 100 ranking of the world’s top blogs on health and medicine, Drugwonks is tied for #5. That puts us ahead of the health blog of both the New York Times and the Wall Street Journal.

Thank you for your support – and pass the word!
  Read More & Comment...

Social Media Thought of the Day

02/07/2013 02:17 PM |

Can strategic use of social media put the pharmaceutical industry back in the public health business?

  Read More & Comment...

Part D all the time

02/06/2013 08:08 AM |

From the pages of the Des Moines Register (and hello Senator Grassley):

Changes to Part D won't save Medicare

By: Peter Pitts

With a debt-ceiling showdown just around the corner, Republican lawmakers are insisting upon more government spending cuts in exchange for raising the country’s borrowing limit.

President Obama says the debt ceiling shouldn’t be used as a negotiating tool. But it’s inevitable that additional cuts will be seriously considered. And, unfortunately, lawmakers are likely to take aim at Medicare Part D, the highly successful prescription drug benefit.

This approach is misguided. Cuts to Medicare Part D would have devastating consequences for seniors and taxpayers.

Created in 2006, Medicare Part D has helped ensure that America’s seniors have access to prescription drugs. Today, nearly 47 million Americans are eligible for Part D.

The program was built to rely on market mechanisms. Under Part D, seniors choose from a wide variety of drug coverage plans — in fact, there are now over 1,000 different plans available across the country. With so many insurers competing for seniors’ business, they have a strong incentive to keep costs low and benefits generous.

This fierce competition has kept expenses down for both seniors and the government. Overall, Part D has cost $435 billion, or 43 percent less than original estimates predicted — practically unheard of for a government program.

Part D monthly premiums will again average just $30 in 2013, according to officials at the U.S. Department of Health and Human Services. That number has held basically steady for four years, and premiums are up only 2.5 percent since 2006.

Part D rates are even more impressive when compared to what is happening in the regular health insurance market. The typical premium for an employer-sponsored family health plan rose 4 percent from 2011 to 2012 and increased 9 percent the year before, according to the Kaiser Family Foundation.

Seniors, moreover, are pleased with their Part D coverage. A new poll sponsored by Medicare Today found that 90 percent of enrollees are satisfied — up 12 points from 78 percent in 2006.

Still, many Democrats are willing to compromise this highly successful program. Their proposal would require drug companies to pay a so-called “rebate” to the government for every medication sold to a Medicare Part D participant who also qualifies for Medicaid, the government health program for the poor. There are about six million of these dual-eligible seniors.

The Obama administration believes that its plan to overhaul Medicare spending will save nearly $600 billion, with 42 percent of those savings coming from the Part D drug rebates.

But this scheme won’t work the way the president intends. It will drive up health care costs for a large portion of Medicare beneficiaries.

Just look at what is already happening in our Medicaid program. Drug companies are required by law to sell their wares to Medicaid at below-market prices, and consequently they have been forced to raise prices elsewhere. In other words, the proposed Part D rebate program would effectively levy a new tax on every senior who is not eligible for Medicaid.

Indeed, two independent studies have estimated that the rebate plan will raise premiums for traditional Medicare enrollees by 20 to 50 percent.

Higher premiums are a big concern for most Part D beneficiaries. According to the Medicare Today poll, 84 percent of enrollees were worried that a Part D restructuring would increase their out-of-pocket drug costs, while 53 percent were afraid it would cause them to cut back or stop taking their medicines altogether.

Our government health programs are still in serious need of comprehensive reform. Unfortunately, the president continues to attack the very part of the system that is most financially sound. Changes to Medicare Part D will raise only minimal revenue — at the expense of seniors.

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Fools Gold(acre)

02/05/2013 08:21 AM |

Sins of omission are seldom fun, and (per Health Care’s Trick Coin, NYT, 2/3/13), Ben Goldacre makes a number of serious mistakes – specifically the claim that the Clinicaltrials.gov registration requirement in FDAAA isn't implemented and that only full transparency/publication of clinical trial will address the issue. As the former senior government official in charge of clinicaltrials.gov, it’s important to look at the facts – and the numbers.

In 2000, the National Institutes of Health (NIH) launched ClinicalTrials.gov to provide public access to information on clinical studies.  Although it initially contained information primarily on NIH-funded research, it has been expanded to include both publicly and privately supported clinical research. 

Since the launch of the site, it has been enhanced to significantly increase data sharing. The ClinicalTrials.gov database includes information on nearly 140,000 clinical trials in all 50 states and 182 countries. 

Is anyone accessing this wealth of information? Yes!  The NIH reported last year that ClinicalTrials.gov “receives more than 95 million page views per month and 60,000 unique visitors daily.

Facts do not cease to exist because they are ignored. Mr. Goldacre should realize that reality, although sometimes inconvenient to ones argument, remains reality.

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First do no reading

02/04/2013 08:28 AM |

Great BioCentury lead article by Steve Usdin, Debating Limited Use.

Usdin writes:

FDA is asking the public to consider whether it should supplement the current all-or-nothing approval structure with a system that would couple approval of selected new drugs with measures designed to discourage off-label use.

The agency says it could approve drugs for conditions such as antibiotic-resistant infections and life-threatening obesity based on small, fast trials if it were confident use would be limited to well-defined populations.

There is little controversy about approving drugs based on relatively small studies that demonstrate high levels of efficacy in tightly targeted populations. But FDA’s suggestion that it could work with physicians and payers to limit use of a marketed drug in the absence of documented safety concerns is controversial.

To say the least.

Patient and medical groups support the concept of a limited-use pathway, and the President’s Council of Advisors on Science and Technology (PCAST) has endorsed the idea. PCAST’s recommendation to create a Special Medical Use (SMU) pathway was drafted in close consultation with senior FDA officials and reflects the agency’s thinking.

But, whether or not you believe the FDA has the legal authority for a “new” pathway, Janet Woodcock nails the problem dead on:

“We can indicate things for small populations, but often we are very concerned there might be a temptation to use them much more broadly.”

There are many important issues, let’s discuss two.

First – does the FDA have the authority, the resources, and the desire to direct the practice of medicine? Can the agency really do anything other than suggest, via labeling, how physicians should prescribe approved medicines? No doubt they can deter pharmaceutical companies from detailing or even discussing off-label use via prior agreement (and Caronia notwithstanding). Whether or not doctors are “tempted,” however, is another matter entirely. Is the FDA going to “ban” certain scientific publications or conference presentations? Is the FDA going to add a new tenet to the Hippocratic oath, “First do no reading?”

 It’s a slippery slope indeed.

Woodcock added that FDA is not seeking to ban off-label prescribing of drugs approved through a new limited use pathway. The agency is interested in exploring whether payers might play a role in reducing inappropriate use, she said.

More power to the payers? Is that something we want?

Next is the issue of whether such expedited approval pathways are in the best interests of the sponsor. While there’s no doubt getting new treatments for serious and life-threatening diseases to market more swiftly is a good thing, is the threat of having a product license revoked after a “test” period in the sponsor’s best interest? Isn’t it a penny-wise, pound-foolish proposition? It depends. What is certain is that such decisions should not be driven by financial considerations of biotech venture capitalists or the panjandrums of Wall Street.

Naïve? Perhaps -- but none-the-less disturbing.

Is FDA PCAST-ing it’s net too wide?

Whenever there is authority, there is a natural inclination to disobedience.

-- Thomas Haliburton

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340B -- or not to be?

02/02/2013 11:25 AM |

A group of six Congressional Republicans sent a letter to HHS's Health Resources and Services Administration seeking details on the agency's audits of the eligibility of healthcare entities for the 340B drug discount program. The program requires manufacturers to give deep discounts on outpatient drugs to hospitals and clinics that bear the brunt of providing healthcare to low income and other special populations.

HRSA began the audits in FY12 after a 2011 report from the Government Accountability Office said HRSA's oversight of the 340B program was "inadequate to provide reasonable assurance that covered entities and drug manufacturers are in compliance" with the program's requirements. According to the letter, during 2012 HRSA decertified about 250 entities from the 340B program. The agency said it recertified more than 3,800 entities during the same time period.

The lawmakers are seeking information on audits that HRSA is currently conducting. The lawmakers also are seeking information on the decertified entities, including whether the agency plans to take legal action or recoup any ineligible 340B discounts.

The letter was sent by Sens. Chuck Grassley (R-Iowa), Lamar Alexander (R-Tenn.), Orrin Hatch (R-Utah) and Michael Enzi (R-Wyo.), as well as Reps. Joe Pitts (R-Pa.) and Bill Cassidy (R-La.). The lawmakers asked for a written response by Feb. 14. HRSA said it is working on a response.

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The impact of pay for performance on physicians

01/31/2013 03:49 AM |

Dr. Thomas Santo on pay-for-performance:

Clearly, the practice of medicine, perhaps better described as the delivery of healthcare, is undergoing a radical, permanent change, at least for the foreseeable future. After all, we are only a few weeks into the first year of ObamaCare, and already HHC, one of the largest hospital corporations in the country, has decided to hold their physician employees financially responsible for the mandatory changes outlined by CMS. The complexity of these changes makes their adoption into current practice a daunting task for many physicians.

The simple fact is, that the future of medicine looks very different than it did when many of today’s doctors started medical school, even for those who have graduated in recent years.  For those who entered the field to treat patients, cure disease and make a real difference in the lives of others, the current tasks that consume much of their time and energy are carrying them further and further away from that goal. The optimists in the field would like to believe that pay-for-performance is simply a resetting of the status quo, which will take considerable time and effort, but ultimately benefit our profession and our patients in the long term.

I am hesitant to agree with this view for two reasons, among others. First, I believe that many physicians are already at or near capacity in terms of their ability to deliver care, both in terms of organizational resources, and personal time and energy. Many individual and small-group practices simply do not have the resources to understand and implement the constantly changing mandates from CMS. Therefore, when you hand them a forever evolving checklist of arbitrary measures that they must follow in order to be reimbursed properly, you only increase confusion and frustration, and do little to impact productivity or foster a system that delivers better care.

Secondly, for any physician who sees their reimbursement cut by providing “sub-optimal care”, as deemed by CMS, what is to stop them from refusing to serve their sickest, most chronically ill and frequently hospitalized patients? In so doing, they raise the “quality” of care they provide, and lower the cost at the same time (they will not be responsible for those sick patients when CMS evaluates them the next year). Such a reaction to pay-for-performance would only further accelerate a current trend in medicine, which is seeing many physicians refuse to accept new Medicare patients.

It is impossible to forecast how pay-for-performance will ultimately impact the quality of care we provide, and the cost at which we do so.  But one thing is certain; the job of physicians providing that care becomes more difficult every day.

Read the full piece here.


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A Price Above Rubies

01/31/2013 01:43 AM |

Small is Big.

Bob Temple said he "would like to see more" comparative effectiveness trials for symptomatic treatments to help identify potential responders. In particular, Temple said trials that randomize non-responders to a drug to receive both that drug and an alternative drug have "a lot of potential" but are almost never done.

"For example, you could take people who don't respond to a migraine drug and randomize them back to a new migraine drug, the old migraine drug the one they didn't respond to, and a placebo. If it's true that there are individual differences that are important, then the new drug ought to be able to show its advantage over the drug that didn't work," he wrote in a post on FDA's From a Clinical Perspective blog.

When asked whether institutional review boards (IRBs) would approve such trials, Temple said there is no ethical issue in an informed patient. He added that such trials would not be done for indications where "not working would be dangerous."

This seems to jive with what Dr. Joe Selby (PCORI’s executive director) told me the other day – that he believes PCORI should focus on funding “narrow” clinical trials aimed at smaller, more targeted populations, that  PCORI's mission is to ensure that CER helps to advance the cause of personalized medicine and “the right treatment for the right patient at the right time.” Bravo.

If Joe Selby can be the Good Witch of Comparative Effectiveness Research, so much the better. I think he’d look lovely in ruby slippers.

As would Bob Temple.

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Consumer Reports Recommendations Will Kill Cancer Patients

01/30/2013 03:14 PM |

From Consumer Reports, the people who  promote one-size fits all medicine to boost circulation comes this killer app (literally): 

"YONKERS, N.Y., Jan. 30, 2013 /PRNewswire-USNewswire/ — In its March issue, Consumer Reports shines a light on oversold cancer screenings that might confuse rather than clarify. The report evaluates eleven cancer screenings, finding that eight should be avoided.

The report, available online at www.ConsumerReports.org and wherever magazines are sold, notes that the Ratings are for people who are not at high risk and without signs or symptoms of cancer

Screening tests for cervical, colon, and breast cancers are the most effective tests available, according to Consumer Reports’ first Ratings of cancer-screening tests.  But most people shouldn’t waste their time on screenings for bladder, lung, oral, ovarian, prostate, pancreatic, skin, and testicular cancers.  The Ratings are based mainly on evidence-based reviews from the U.S. Preventive Services Task Force, an independent group supported by the Department of Health and Human Services."

Oversold?  And given that cancer is now the leading form of death, and that treatments are increasingly targeted to cancer causing mutations that can be detected earlier, who is not at risk? Should we wait until Obamacare autocrats determine what tests should be paid for?   Will people actually pay Consumer Reports to peddle medical malpractice? 


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Arabian Mights

01/30/2013 10:35 AM |

I’m in Riyadh, Saudi Arabia where I just spoke at the first Saudi Quality Forum (sponsored by King Saud University). I also had the opportunity to meet with the leadership of the Saudi Food and Drug Authority (SFDA) -- which is also the national reimbursement agency. (And no product gets licensed without first concluding price negotiations.)

And you thought FDA swings a big stick.

My suggestion to their executives is that they form an Office of Policy and Planning – and that one of the first items on the agenda be chairing a regular meeting of industry (both innovator and generic) and academia to address issues of common interest including API and excipient quality, biosimilars (specifically the issues of biosimilarity and interchangeability), 21st century pharmacovigilance, generic interchangability, and other issues that fill the Arabian regulatory gulf -- like re-registration.

The SFDA aspires to be not only the gold standard for the region, but also a world-class regulatory agency. But to do so they must recognize their responsibility to be both regulator and colleague to industry and an innovation enabler. Sound familiar?

My favorite sound-bite from the conference came courtesy of the SFDA’s Mohammed Dahhas (Executive Director of Inspection and Law Enforcement, Drug Sector): On the issue of quality by design, “Quality is the magic key.”

My favorite new concept came from Accenture’s Pierre Anhoury, who introduced the conference attendees to notion of “value vigilance” – a new way to view post-approval value-based reimbursement via pharmacovigilance, quality of life, therapeutic benefit, and pharmaco-economic impact. Maybe we should call it Phase 4.1.

(Have a look at Anhoury’s new white paper on the topic, Approval is Nothing:  Why Pharmaceutical Companies Need a Value Management Operating Model.)

Wouldn’t it be NICE to bury the QALY in the sands of Saudi?

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If We won't Take Our Money Why would We Take Our Medicine?

01/29/2013 09:15 PM |

Americans have failed to claim tens of billions of dollars in tax refunds, interest payments and bank accounts. So here's a question: if we are not motivated to claim money that is ours, what does that say about how hard it is to encourage people to take medicine when we need it? http://money.cnn.com/2013/01/24/pf/unclaimed-money/index.html   Read More & Comment...

Cycle For Survival

01/28/2013 11:58 AM |

Michal Goodman, the daughter of a good friend of mine was recently diagnosed with a rare cancer called Synovial Sarcoma and has been undergoing treatment at Memorial Sloan Kettering Cancer Center.  She is scheduled for a major and, G-d willing, successful surgery this Thursday, January 31, 2013.  David (Michal's dad) sent around an email I'd like to share..

"We are thankful to all of you who have prayed for Michal and expressed your support in other ways.  Here is another opportunity for those interested.
On March 3, 2013 both our sons-in-law, Ben Coleman (Michal’s husband) and  Sean Wasserman, will be participating in Cycle for Survival, the national, indoor team cycling event that has raised more than $20 million in support of life-changing research on rare cancers at Memorial Sloan-Kettering Cancer Center, one of the world's premiere cancer research institutions. 

100% of the funds raised through this ride go directly to MSKCC's research of rare cancer, like Synovial Sarcoma, making us feel very personally connected to this cause. With your help, we can move closer to finding out how to cure cancers like the one Michal is battling right now.

The 2012 ride donations were already put to work in twenty-eight different MSK labs and clinics – targeting a wide range of rare cancers.  This progress – and the hope it brings to patients and their families – is only possible because of thoughtful contributions from people like you!  We are asking you to join the battle and make a gift to support our ride against cancer.  The link below will bring you to our fundraising page, where you can make a tax-deductible online donation."

http://mskcc.convio.net/site/TR/CycleforSurvival/AG_Cycle_Event?pg=team&fr_id=1818&team_id=26100
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