Thank you sir, may I have another.

02/22/2013 08:52 AM |

The Washington Post reports that David G. Miller, executive vice president of the International Academy of Compounding Pharmacists, "said he will support legislation requiring pharmacies that operate like drug manufacturers to register with the Food and Drug Administration and be subject to stricter standards enforced by the agency." The Post notes, "Miller and his 2,700-member group have traditionally argued that all pharmacies should fall under the purview of state pharmacy boards, not the FDA, and fought efforts in 2007 to shift primary oversight from the states to the federal government. On Thursday, Miller said he now wants to see FDA registration for what he describes as compounding manufacturers and supports giving the agency the power to enforce safety standards for these firms."

  Read More & Comment...

"Essential" -- for the time being

02/21/2013 01:43 PM |

HHS issued a final rule on Wednesday that includes language that will increase the number of drugs eligible for reimbursement by insurers under the Affordable Care Act's essential health benefits requirements. Under the final rule, insurers must cover at least one drug per therapeutic area or the same number of drugs in each category and class as specified in the state benchmark plan, whichever is greater. The rule also states that a health plan "must have procedures in place that allow an enrollee to request and gain access to clinically appropriate drugs not covered by the health plan." The language on coverage requirements for drugs is virtually identical to that included in the proposed rule in November.

In response to comments expressing concerns about the cost of the requirement, HHS said it believes the policy "reflects drug coverage in a typical employer plan and will have a negligible effect on premiums." The agency added that the policy will be a "transition" for the first two plan or policy years starting in 2014 -- when ACA is slated to come into effect -- and that it "will study and take into considerations the effects this policy, if any, have on changing typical drug coverage in the market."

  Read More & Comment...

Digital Health Reform

02/20/2013 06:31 AM |

If we want to change our national healthcare paradigm we must also change the way people learn, discuss and address healthcare issues. And that means social media.

Attention Pharmaceutical Industry: If you’re not at the table, you’re on the menu.

  Read More & Comment...

Measuring REMS

02/19/2013 08:48 AM |

If you can't measure it, then it doesn't count.

The FDA plans to draft guidances on how companies should create goals for a REMS and on the other metrics needed to determine if those goals are successfully met and a guidance on methodologies for assessing REMS. (FDA agreed in the PDUFA V commitment letter to issue a draft assessment guidance by the end of September 2014.)

An OIG review of 49 REMS, and the FDA’s reviews of those assessments states the obvious, “If FDA does not have comprehensive data to monitor the performance of REMS, it cannot ensure that the public is provided maximum protection from a drug’s known or potential risks.”

But sometimes the obvious is important to state.

According to the Pink Sheet, “FDA currently possesses no way to force sponsors to provide the needed assessment data, so OIG recommends the agency seek enforcement authority from Congress. FDA agreed that this recommendation should be pursued if an opportunity arises. That opportunity could be the reauthorization of the Animal Drug User Fee Act, which is eyed as a means to gain approval of a track and trace system.”

We’ll see if that dog hunts.

  Read More & Comment...

Social Media: Advise and Dissent

02/18/2013 09:20 AM |

When it comes to social media, the FDA wants companies to do what’s in the best interest of the physician and the patient (really!). But there’s an unfortunate disconnect – the regulatory go-forward proposition of many companies is to avoid any regulatory ambiguity. The result is a vast regulated healthcare speech  wasteland. Alas, when it comes to social media, “in compliance” and “in the best interest of the public health” are often viewed as mutually exclusive.

  Read More & Comment...

PCORI and Epigenetics: Looking Backward and Looking Forward

02/15/2013 12:42 PM |

PCORI is being propped up by syncophants in the so-called stakeholder community.  Those who claim they are advocates for patient-centered medicine should spend less time publishing and promoting the "right way" to use CER.  Instead, they should be constantly educating and reminding people of how medical progress will make CER endlessly outdated.  As the example below demonstrates,  personal epigenetics will make real-time monitoring and treatment of prostate cancer widespread.  So why are we spending millions on 'empowering' patients to decide on watchful waiting vs active surveillance or three different types of treatment?   

Personal epigenetic 'signatures' found consistent in prostate cancer patients' metastases

Discovery may help distinguish indolent from lethal cancers

In a genome-wide analysis of 13 metastatic prostate cancers, scientists at the Johns Hopkins Kimmel Cancer Center found consistent epigenetic "signatures" across all metastatic tumors in each patient. The discovery of the stable, epigenetic "marks" that sit on the nuclear DNA of cancer cells and alter gene expression, defies a prevailing belief that the marks vary so much within each individual's widespread cancers that they have little or no value as targets for therapy or as biomarkers for treatment response and predicting disease severity.

A report of the discovery, published in the Jan. 23 issue of Science Translational Medicine, describes a genomic analysis of 13 men who died of metastatic prostate cancer and whose tissue samples were collected after a rapid autopsy.

Samples from three to six metastatic sites in each of the patients and one to three samples of their normal tissue were analyzed to determine the amount of molecular marks made up of methyl groups that attach to sites along the genome in a process known as DNA methylation. The process is part of an expanding target of scientific study called epigenetics, known to help drive cell processes by regulating when and how genes are activated. Mistakes in epigenetic processes also are known to trigger or fuel cancers.

"Knowing both the genetic and epigenetic changes that happen in lethal prostate cancers can eventually help us identify the most aggressive cancers earlier and develop new therapies that target those changes," says Srinivasan Yegnasubramanian, M.D., Ph.D., assistant professor of oncology at The Johns Hopkins University School of Medicine. "But there has been an open question of whether epigenetic changes are consistently maintained across all metastatic sites of an individual's cancer."

The research team found that while methylation patterns vary from one patient to another, many methylation patterns occur "very consistently" within different metastatic sites in an individual patient. They identified more than 1,000 regions of the genome where various types of DNA methylation were consistently maintained within their 13 subjects' genomes.

"As they evolve and grow, cancer cells acquire and maintain changes that enable them to continue thriving," says Yegnasubramanian. "We know that cancer cells maintain and pass along genetic changes in the nucleus of cells across metastatic sites, and our research now shows that epigenetic changes also are maintained to nearly the same degree."

The scientists say that the consistent methylation changes they found appear to represent so-called driver changes critical to the cancer's development and could be targets for treatment. By contrast, other methylation changes found only sporadically in the metastatic sites are more likely what are called passenger changes that occur by chance and are less promising as treatment targets or biomarkers than driver changes.

"Our study shows that for prostate cancer, at least, each person develops his own path to cancer and metastasis, and we can find a signature of that path in the epigenetic marks within their tumors," says Yegnasubramanian, who envisions that certain epigenetic changes can be grouped into clusters to be used as biomarkers signaling a lethal cancer.

Yegnasubramanian and his team also plan to study how each of the driver changes work and how they influence cancer metastasis.
  Read More & Comment...

Honey I Shrunk The Medicare Drug Benefit

02/15/2013 12:34 PM |

Every once in a while there's a great piece summarizing how real progress in health care is achieved.  Here's one by Jim Capretta

http://www.economics21.org/commentary/incredible-lowering-medicare-drug-benefit-baseline
  Read More & Comment...

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

  Read More & Comment...

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

  Read More & Comment...

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.

  Read More & Comment...

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]

  Read More & Comment...

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.  

  Read More & Comment...

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.

  Read More & Comment...

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.

  Read More & Comment...

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

  Read More & Comment...