Facts and Drug Facts

09/21/2012 08:07 AM |

A research letter published in JAMA says “closer attention” should be paid to the lack of risk information in advertising for the OTC switch versions of prescription drugs. Research sponsor CVS Caremark says OTC ads should convey the same information as Rx ads.

Facts are facts – Drug Facts, to be precise. And, no doubt, when prescription drugs become available over-the-counter advertisements are less likely to tell consumers about the potential harms and side effects.

But how little is too little – and how much is too much?

To say (as many now are) that OTC ads should carry the same warnings as their Rx brethren is to assume that Rx warnings are useful. When it comes to, for example, the so-called “Brief Summary,” that’s an open question (and that’s being charitable).  More of what the consumer doesn’t understand isn’t the solution.

Is it the FDA’s responsibility to figure this out? Before we answer that, perhaps a more direct question is, does the FDA have the social science chops to do so? With all due respect to Kit Aikin and crew – this issue will be best resolved through the joint efforts of industry and agency.

And one size may not fit all.

Beyond BTC, it’ll be interesting to see how various Rx-to-OTC applications address the question of consumer education.

Ladies and Gentlemen of industry – it’s time to step up to the plate.

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When It Comes To Beer, Size Doesn't Matter To Bloomberg

09/20/2012 05:27 PM |

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An Ugly Way To Get Insurance

09/20/2012 10:43 AM |

From The New York Post

An ugly way to get insurance
By ROBERT GOLDBERG
Last Updated: 11:23 PM, September 19, 2012
Posted: 11:10 PM, September 19, 2012

The good news: More Americans have health insurance. The bad news: It’s because they don’t have jobs.

ObamaCare supporters hail the drop in the number of uninsured, announced by the Census Bureau last week, as a sign of the new law’s success. In fact, it’s a sign of continued job-market decline and of how many of us have to depend on government programs for far too long.

Nearly 1.4 million more people had health insurance in 2011 than in 2010 — but that includes nearly 800,000 who gained coverage despite not working at all.

In short, the rise comes mainly from more Americans being forced into safety-net programs by declining incomes and reduced job opportunities.

Look at this trend another way: In 2011, the number of people covered by Medicaid jumped 2.3 million, while Medicare saw 2 million new enrollees.

And only 575,000 of those new Medicare cases were people turning 65. Most of the other 1.5 million is associated with the exponential growth in people becoming Medicare-eligible because they’ve filed for Social Security disability coverage.

By comparison, the number of people gaining health insurance via work rose only 730,000.

ObamaCare fans also claim that much of the increase in coverage came from the Obama law’s mandate that young adults can stay on their parents’ health plans until they turn 25.

Indeed, the administration boasted earlier this year that 3 million young adults got insurance that way. But the Census Bureau report shows that health coverage for people in that age bracket rose by only 540,000.

And it’s clear that ObamaCare was not responsible for much of even that increase. Past Census reports show the share of 19- to 25- year-olds on Medicaid or Medicare doubling from 2000 to 2011. That long-term trend — not the ObamaCare mandate — plainly accounts for a good chunk of the rise in coverage of these younger folks.

Slice it another way. The total increase in health coverage for ages 18-24 in 2011 was 825,000. Nearly 331,000 of that was from employer-based coverage. More than 220,000 was from Medicaid enrollment.

So, at most, that’s 247,000 from ObamaCare’s under-25 mandate.

And that gain comes at a hard-to-measure cost: The price of forcing insurers to cover under-25s on their parents’ policies is higher premiums for other people.

Let’s be clear: Medicaid, the Children’s Health Insurance Program and Medicare make health insurance affordable for tens of million of Americans. But that safety net, in place before ObamaCare was enacted, is supposed to be a temporary source of support when we need it, not a permanent solution.

And a jump in the number of Americans who have to use that safety net is nothing to brag about.

Robert Goldberg is vice president of the Center for Medicine in the Public Interest.



 http://www.nypost.com/p/news/opinion/opedcolumnists/an_ugly_way_to_get_insurance_etNZJPm74YKgP0euAZJWSM#ixzz271N2B2m9  Read More & Comment...

Transplendent TransCelerate

09/20/2012 07:52 AM |

From the pages of Medical Marketing & Media …

Drugs elude cookie-cutter approval process

A powerhouse mix of pharmaceutical companies including Abbott, AstraZeneca, Boehringer Ingelheim and Bristol-Myers Squibb has launched a non-profit tasked with making it easier for companies to bring news drugs to market.

The initiative, called TransCelerate BioPharma, is headed up by J&J alum Garry Neil. Its first project focuses on clinical trial execution and will include creating an investigator site, developing clinical data standards and establishing a comparator drug supply model, according to a statement. More tangible details were not forthcoming, other than the comment that the goal is to help speed drugs to market, but the news release quoted FDA Director Janet Woodcock as saying the collaboration “has the promise to lead to new paradigms and cost savings in drug development, all of which would strengthen the industry.”

In other words, it appears to be a project that tries to pin down the very thing that has roiled industry watchers when it comes to the FDA's review process: predictability and its seeming absence. Yet experts have told MM&M in the past that uniform standards won't create an if-then scenario in which the FDA will approve drugs based on meeting the demands of a checklist.

“Predictability assumes that you can approve drugs in a cookie-cutter kind of fashion and you just can't do that,” Dr. Steven Nissen, a frequent member of FDA advisory panels and chairman of the Cleveland Clinic's cardiology department, told MM&M. “It's always going to be a nuanced decision. It's always going to be a careful evaluation of benefit vs. risks,” he added.

The agency has come under a mixed review, with critics saying the FDA is approving drugs without adequately considering a drug's risks. Advocacy group Public Citizen is suing the agency over its approval of the Alzheimer's drug Aricept 23, an approval which its deputy director of the Health Research Group Michael A. Carome told MM&M was made even though the agency acknowledged that the drug didn't meet the standards the regulators demanded even before it came up for review. The drug, at a higher dose of the 5- and 10-mg versions which have gone generic, has  been linked to nausea, vomiting and dizziness, but with little benefit, according to Carome and other critics.

“This represents an extreme example of the FDA's failure to properly weigh the evidence and only approve drugs when there's clear evidence [that] benefit outweighs the risk,” Carome said.

Nissen, who was not part of the Aricept 23 review and does not specialize in Alzheimer's, said that the risk-benefit equation had a degree of flex that depends on who's doing the calculation.

“I think the most salient example is cancer drugs. You're dealing with a disorder with often a lethal disorder. These drugs that are used to treat cancer are pretty toxic, but the disease is a pretty lethal disease,” he said.

Former FDA Associate Commissioner Peter Pitts said the FDA gets knocked in part because it doesn't do a very good job at explaining how the drug review and approval process works.

Among the steps is a clinical trial design that the FDA approves before it's kicked off. Pitts noted that hitting all the right design notes doesn't guarantee a panel's endorsement if the results raise questions.

“Those questions need to be answered.” Pitts highlighted two other components of the FDA's decision-making process which includes basing its decisions on “the twin pillars of safety and efficacy,” a balance he said is important because “you can't look at safety independent of benefit.” He also noted that the committees are comprised of experts that see data differently, which can add nuance to discussions.

But nuance or heated argument doesn't always mean pushback. The May debate over the blood thinner Xarelto was a contentious committee hearing, in which panelists took serious issue with the quality of the data presented. The drug passed with a panel endorsement despite the attacks on missing patients and an inability to state just how many patients may have died during the clinical trials.

Adding to the fuzziness is that what constitutes acceptable risk is also subject to change. Recent examples: weight-loss drugs Qsymia and Belviq which failed to clear the FDA the first time they were up to review, which was before the CDC declared obesity an epidemic.

Pitts said one way to provide continuity would be to use the same requirements for drugs that have the same purpose and said that this industry push is not about lower standards, but about making smart investments. He said ambiguity in that regard is "not fair and that's not good science." He said it could also help within the wider context of creating a single standard that can be used for both international and domestic regulatory review.

Such a change will not remove risk, and both Pitts and Nissen said results can't simply be accepted because panelists didn't know what to ask before results roll in. Even when a clinical trial meets expectations and has the FDA's go-ahead, Nissen noted that new risks are bound to surface because clinical trials are little more than real-world approximations. “You have done a series of studies with a drug in a few thousand people and the drug may be used in a million . . . there is no way to be absolutely certain whether a drug is going to be helpful or harmful,” he explained.

Nissen said he understands the drug industry's frustration with the case-by-case approach, but he said it can't be avoided. “It's about their investment and their bottom line and I completely understand that but the FDA has a different mission and that mission is to protect public health.”

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white house celebrates talk like a pirate day

09/19/2012 11:01 PM |

http://www.freep.com/article/20120919/FEATURES01/120919055/International-Talk-Like-A-Pirate-Day-10th-anniversary?odyssey=nav%7Chead  Read More & Comment...

Occupy Pharmalot

09/19/2012 10:09 AM |

Two items for your consideration:

EC Does It

BioCentury reminds us that the House Energy and Commerce Committee is expected to vote this week on a bill that would allow FDA to collect newly enacted generic drug user fees, a committee spokesperson told BioCentury. The bill does not include a provision that would allow FDA to collect new biosimilar user fees.

Last week, the House of Representatives passed a continuing resolution that would extend funding at current levels for government operations but omitted technical language that would have allowed FDA to benefit from an increase in drug and medical device user fees and to spend the newly enacted user fees for biosimilars and generics.

FDA had planned to use $299 million from new generic drug user fees in FY13 to reduce huge application backlogs, increase postmarket safety oversight, and ramp up inspections of generic drug manufacturers outside the U.S.

Occupy Pharmalot

I was pleased to offer a guest op-ed on Ed Silverman’s Pharmalot blog.  Here’s a link.  Of particular interest is the comments section.  Have a look and see how the other 47% lives.

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The Fifth Element

09/18/2012 07:19 AM |

The Patient Centered Outcomes Research Institute began accepting proposals for up to $96 million in grants for research projects addressing four of its five priorities.

The fifth element, the one it’s ignoring, however, is the most important.

The four priorities are: comparing alternative prevention, diagnosis and treatment options; improving healthcare systems; comparing communication approaches to providing comparative effectiveness research information; and addressing potential differences in prevention, diagnosis or treatment effectiveness, or preferred clinical outcomes across patient populations.

(Earlier this month, on BioCentury This Week, PCORI Executive Director Joe Selby told that low back pain, uterine fibroids and depression could be early targets for comparative effectiveness research.)

What’s not being addressed, is PCORI’s fifth priority, improving the nation's capacity to conduct patient-centered outcomes research by building data infrastructure, improving analytic methods and training researchers, patients and other stakeholders to participate in the research.

Hello! That’s the most urgent need but, alas the least agenda-driven for those whose ultimate goal is a US NICE. After all, why fund programs to advance a science-based understanding of patient outcomes data when you can fund “research” programs to find the best way to “communicate” about comparative effectiveness.

Say it ain’t so Joe.

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(Postage) Stamp of Disapproval

09/17/2012 07:38 AM |

Since the end of 2011 the FDA has sent 11 warning letters to companies in seven different countries that were exporting drugs to the U.S. after having let their required registration and listing expire.

The warning letter recipients also failed to respond to letters FDA sent them last year notifying them that they were not registered or listed for imports into the U.S., according to nearly identical language in the warning letters.

“FDA has established an ongoing program to identify drug manufacturing firms that have not complied with registration and listing requirements and to notify such firms of their ostensible noncompliance,” the agency said. “FDA has issued warning letters to some of the firms that have not responded to this notification and are out of compliance with registration and listing requirements. These warning letters have targeted firms whose lack of compliance has the greatest potential impact on FDA's regulatory mission.”

 FDA’s focus on compliance at foreign drug manufacturing facilities has been sharper since the tainted heparin scandal in 2008, and new provisions in the recently passed user fee legislation is designed to reduce the number of unregistered facilities -- particularly for generic drugs.

But that was before sequestration.

We’ll see.

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Expanding the Scope of What's Possible

09/14/2012 07:54 AM |

“The convergence of two powerful forces is driving today’s medical innovation to the top of the national agenda:  The urgent need for medical breakthroughs and the unprecedented opportunities created by recent advances in science.”

So said El Lilly & Company’s Grand Poobah John Lechleiter at yesterday’s “Advancing Medical Innovation” summit in Washington, DC. (The event was co-sponsored by Lilly and the Washington Post.)  I was pleased to attend.

More Lechleiter:

“Our only hope of breaking our of the crisis is through innovation that changes the terms of the trade-offs we must make and expands the scope of what’s possible.”

The Hoosier Honcho then offered a very potent example of what he meant:

“In the early 1950s, the cost of polio care in the US was predicted to be $100 billion by the year 2000 but, thanks to the advent in 1954 of the polio vaccine, the cost of treating polio in the US in the year 2000 was $100 million.”

In other words – the prediction was off by 99.9% because of game changing innovation.  Can we do that again? We’d better try – and try hard.

But Lechleiter offered a crucial caveat, “Realizing the benefits of medical innovation requires an ecosystem where innovation can thrive.”

Are we there yet?

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Thursday news

09/13/2012 10:46 AM |

Betsy McCaughey on ObamaCare’s hospital cuts:

President Obama is wooing seniors with promises to protect Medicare as they've known it. On the defensive because of the $716 billion his health care law takes from Medicare, Obama assures seniors he's cutting payments to hospitals and other providers, not their benefits. 

Don't be bamboozled. It's illogical to think that reducing what a hospital is paid to treat seniors won't harm their care. A mountain of scientific evidence proves the cuts will worsen the chance that an elderly patient survives a hospital stay and goes home. It’s reasonable to conclude that tens of thousands of seniors will die needlessly each year.

Under ObamaaCare, hospitals, hospice care, dialysis centers, and nursing homes will be paid less to care for the same number of seniors than if the health law had not been  enacted. Payments to doctors will also be cut.

Read more here.
 
 
Politico on the Hatch Act violation by HHS Secretary Sebelius:

INDEPENDENT COUNSEL: SEBELIUS CROSSED THE LINE - Limits on political maneuvering by public officials working on the taxpayers' dime are nebulous, byzantine and often blurred. But the Office of Special Counsel - an independent prosecuting authority - has concluded that HHS Secretary Kathleen Sebelius broke the law when she exhorted a North Carolina crowd last February to reelect her boss, President Barack Obama, during what was supposed to be an official visit. The determination - which OSC said was an isolated incident - creates an immediate headache for the Obama administration on a day that Census data showed a dip in uninsured Americans, partly because of the Affordable Care Act.

--From the OSC report: "[Sebelius] noted that North Carolina is critical in the next election and emphasized that it is 'hugely important to make sure we reelect the president.' These statements were made in Secretary Sebelius's official capacity and therefore violated the Hatch Act's prohibition against using official authority or influence to affect the results of an election."

--Sebelius admitted she shouldn't have injected politics into an official event but pointed to her office's immediate decision to reclassify the event as a campaign stop and to reimburse the U.S. Treasury for her travel. More telling, though, was her explanation for the lapse: "As I have also explained, keeping the roles straight can be a difficult task, particularly on mixed trips that involve both campaign and official stops on the same day."

Read more here.



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OTC and DTC -- Whither Fair Balance?

09/13/2012 06:42 AM |

Much important discussion of late about possible new categories for Rx-to-OTC switching – namely erectile dysfunction medicines and statins.  But how does this impact consumer information? Consider the impact of the FDA-to-FTC switch.

The FDA requires prescription drug advertising to provide consumers with a "fair balance" of risks and benefits. The FTC, on the other hand, holds drug advertisements to the same standards as other consumer products, requiring a "reasonable" standard of truthfulness.

What’s the impact on how these products are presented to consumers?

According to a new study, when prescription drugs become available over-the-counter, advertisements for the medications are less likely to tell consumers about the potential harms and side effects. This according to Dr. Jeremy Greene, an associate professor in the history of medicine department and the department of medicine at Johns Hopkins University (study published in the September 12th issue of the Journal of the American Medical Association).

Greene and his colleagues analyzed print and broadcast advertisements for four commonly used drugs that were heavily marketed to consumers as prescription drugs and then approved for sale over-the-counter.

The drugs included loratadine (brand name: Claritin, sold over-the-counter since 2002), omeprazole (brand name: Prilosec, went over-the-counter in 2004), orlistat (brand name: Alli, Xenical, sold over-the-counter since 2007), and cetirizine (brand name: Zyrtec, sold over-the-counter since 2008).

When the drugs were available only by prescription, 70 percent of the ads mentioned potential harms. After the drugs were available over-the-counter, only 11 percent did, the investigators found.

After drugs became available over-the-counter, only about half of print and broadcast advertisements mentioned a drug's generic name, compared to 94 percent of ads when drugs were prescription-only. Knowing a drug's generic name can help consumers make sure they're not taking more than one medication that has it as a component, risking overdose, Greene explained.

Greene believes that the FDA should be given authority to regulate marketing of over-the-counter drugs, or perhaps the FTC should adopt guidelines similar to what the FDA requires.

Rather than asking either underfunded agency to take on more work (for which they are neither staffed nor suited), perhaps OTC advertisers should consider what they could do to better educate consumers. After all, a key FDA consideration in approving an Rx-to-OTC switch is whether consumers understand key communication objectives of the label, relating to directions for use, contraindications, in-use warnings and precautions.

Knowledge is power.

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Court to FDA: A picture won't be worth 1000 words

09/12/2012 07:38 AM |

A very interesting post from pointoflaw.com:

FDA's "graphic" cigarette warnings struck down

The FDA has suffered another setback in its relentless campaign to turn every cigarette pack into a "mini-billboard" for its anti-smoking agenda. The US Court of Appeals for the DC Circuit has upheld a lower court decision striking down the agency's rules that would require cigarette makers to include certain government-approved "graphic warnings" against smoking. Nothing too extreme, mind you, just a man blowing smoke out of a tracheotomy hole and similar pictures.

As reported last November, U.S. District Judge Richard Leon initially granted a preliminary injunction against the FDA rules. In February of this year, he issued a final ruling striking down the graphic warning requirement as unconstitutional "compelled speech." The DC Circuit affirmed on August 24.

The Supreme Court has long recognized that the First Amendment right to say what you want would be meaningless if the government could force you to say things you don't want. In Wooley v. Maynard, for example, the Court affirmed that Jehovah's Witnesses in New Hampshire could not be forced to use license plates with the State's motto: Live Free or Die.

Although commercial speech often merits less protection under existing precedents, there is clearly a liberty problem with forcing manufacturers to do everything possible to dissuade potential customers from buying their product. As the DC Circuit points out, manufacturers have been compelled to include certain information on labeling or other advertising if the information is (1) strictly factual, and (2) without the information, the company's advertising would be misleading. In this case, however, the FDA doesn't argue that current cigarette labels are misleading -- they include all the textual warnings. The FDA just thinks that the packages aren't scary enough; thus, they would require that 50 percent of the front and back panels of every cigarette pack contain pictures, e.g., of women crying, small children, and the guy with the tracheotomy. As the DC Circuit concluded, the images do not convey factual information, but are "unabashed attempts to evoke emotion (and perhaps embarrassment) and browbeat customers into quitting.

And yet, the government would have the courts review its rules under the weakest form of scrutiny available. By the FDA's logic, the government could dictate that every stick of butter be wrapped in images of open-heart surgery, that every candy bar be emblazoned with pictures of rotting teeth, and every sugary drink carry images of obese children -- and these rules would be virtually unreviewable.

The FDA could not even produce evidence that the graphic images would be effective -- the agency estimated a mere 0.088 decrease in smoking rates as a result of the shock-and-awe campaign. Ultimately, the agency seems to want the graphic warnings because "everybody else is doing it." The FDA cited a "strong worldwide consensus" based on the actions of various countries, including Mongolia, Venezuela, Singapore, and Iran. " It is worth noting," the Court said, "that the constitutions of these countries do not necessarily protect individual liberties as stringently as does the United States Constitution." You can say that again.

The case is R.J. Reynolds Tobacco Co. v. FDA, No. 11-5332, slip op. (DC Cir. Aug. 24, 2012).

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9/11

09/11/2012 03:10 PM |

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More to do. More to know. More to manage.

09/11/2012 07:46 AM |

From the pages of Medical Marketing & Media …

Amid misspent billions, the promise of personalized medicine

The Institute of Medicine has released a report putting the cost of unnecessary medical care at $750 billion a year and counting. What does it mean for pharmas? Depends on how you read it. 

The IOM determined that three quarters of a trillion dollars (yes, that's trillion, with a ‘t') in health spending was wasted in 2009 – around 30% of all US healthcare spending – due to unnecessary services rendered, excessive administrative costs, fraud and other causes. Unnecessary services accounts for the largest chunk of that number, at around $210 billion, followed by “excess administrative costs,” at $190 billion, and then “inefficiently delivered care,” including mistakes and the unnecessary use of pricey providers, at $130 billion. “Prices that are too high,” defined as “services and products beyond competitive benchmarks,” costs $105 billion, while fraud runs $75 billion and “Missed prevention opportunities” $55 million. 

Expressed through a different, and starker, metric, IOM noted estimates that around 75,000 deaths might have been averted in 2005 had all states delivered care as well as the best-performing ones. 

While the report, “Best Care at Lower Cost: The Path to Continuously Learning Health Care in America,” doesn't have much to say about costs associated with drugs and biologics, specifically, it could provide fodder for advocates of European-style cost curbing for federal programs, but it also adds urgency to the movement towards personalized medicine. 

“The slippery slope to comparative effectiveness is there,” says Peter Pitts of the Center for Medicine in the Public Interest. He worries that a price control scheme like the UK's NICE could be implemented in the name of efficiency, but also sees the report's findings dovetailing nicely with the drug industry's move towards more narrowly targeted drugs, biologics and diagnostics. 

“If the idea is to get the right treatment to the right patient at the right dose as early in the cycle as possible, even if that sometimes means a more expensive drug rather than a generic, that's going to save money over the “fail your way to success” route [of cycling through multiple treatments until one works],” said Pitts.

The IOM sees several big factors impeding efficiency in US care delivery – chief among them, the lack of price transparency and collaboration between across providers, institutions and other players. And then there's the sheer complexity of modern healthcare, made all the more daunting by Big Data and the rapidly multiplying number of available therapies and procedures.  

“The US healthcare system is characterized by more to do, more to know and more to manage than at any time in history,” says the report.

As medical technology has progressed, the volume of information available to practitioners has multiplied exponentially. The number of journals and other research publications has more than tripled over the past four decades, from 200,000 in 1970 to 750,000 in 2010, the report notes. 

The IOM is an influential body of the National Academy of Sciences whose pronouncements can have far-reaching effects on regulators and policymakers. In recent years, the group has taken on thorny topics like consumer advertising of prescription drugs, CME and industry influence on the practice of medicine.    Read More & Comment...

A November Vote That Matters: Rare Disease Advocate of the Year

09/10/2012 02:12 PM |

The EveryLife Foundation for Rare Diseases is seeking nominations for it's Rare Voice Awards honoree.   You can go the website of this courageous coalition and vote until September 21st.    

There are many patient advocacy organizations.  Most are more interested in getting a "seat at the table" of the Washington policy community.    A few, like Everylife Foundation for Rare Diseases, are focused on removing the barriers to faster and broader access.  The group was the leading voice for reforms that accelerate the development of drugs for rare disease..  You can join it's movement for medical progress by voting for a Rare Voice Award honoree.   Here's a link to the nomination page: http://rarevoiceawards.org/nominate/

The number of deserving awardess are growing.   Just nominating someone can shine a light on their efforts.   


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Post marketing enhancements and clinical trial advancements

09/10/2012 07:45 AM |

OPQ, I see you

BioCentury reports that the FDA is considering the creation of a new office to oversee quality throughout the lifecycle of a drug.

According to CDER Director, Dr. Janet Woodcock, the new Office of Pharmaceutical Quality would take on some of the functions currently within the Office of Pharmaceutical Science, as well as some functions from the Office of Manufacturing and Product Quality in the Office of Compliance. Woodcock said it is imperative that CDER "have a drug quality program as robust as those programs we presently have for drug efficacy and drug safety."

Woodcock also proposed to elevate the Office of Generic Drugs to a "super" office as a result of the new user fee program for generics. OGD Director Greg Geba would continue to lead the office and would report to Woodcock.

And speaking of adaptive clinical trials

Exciting story in the New York Times about advances in treatment for squamous cell lung cancer and, “a new type of treatment in which drugs are tailored to match the genetic abnormality in each patient.”

The study is part of the Cancer Genome Atlas, a large project by the National Institutes of Health to examine genetic abnormalities in cancer. The study of squamous cell lung cancer is the second genetic analysis of a common cancer, coming on the heels of a study of colon cancer.

“As a result, the usual way of testing drugs by giving them to everyone with a particular type of cancer no longer makes sense. So researchers are planning a new type of testing program for squamous cell cancer that will match the major genetic abnormality in each patient with a drug designed to attack it, a harbinger of what many say will be the future of cancer research.”

“The old way of doing clinical trials where patients are only tied together by the organ where their cancer originated, those days are passing,” said Dr. Mace Rothenberg, senior vice president of Pfizer oncology.

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Compounding the Problem

09/07/2012 08:28 AM |

A judge in the U.S. District Court for the District of Columbia has dismissed KV Pharmaceuticals suit against the FDA over the company's preterm birth drug Makena hydroxyprogesterone caproate.

After developing an FDA approved version of a drug that was in danger of becoming obsolete, KV promptly – and without warning – decided to charge $1500 per dose. The company was rightly criticized for the sudden jump in price and did the right thing by cutting the retail price of the drug to $680 and offering the drug at a reduced price to a wide range of organizations.

And yet the FDA decided to allow pharmacists to continue compounding even though KV developed the drug to put an end to the risk associated with compounding products.  (FYI -- The FDA is currently Investigating bacteria that sickened 19 people at Alabama hospitals and may have killed nine and has turned up at compounding pharmacy in Alabama. And there are many other examples.)

Is this a victory for the public health?

KV filed suit against the FDA, alleging that the agency's policy of non-enforcement for compounded hydroxyprogesterone caproate products violated KV's right to market exclusivity for Makena under the Orphan Drug Act.

No dice.

Judge Army Berman said KV's allegations relate to FDA's discretionary enforcement activities and are therefore "unreviewable claims." She said in her ruling that the "case is fundamentally an effort to get the court to direct and oversee the FDA's enforcement activities, and that it cannot do." We agree with Judge Berman -- but it's a dangerous precedent.

Last month, KV filed for Chapter 11 bankruptcy because it has been "unable to realize the full value" of Makena, citing FDA's lack of enforcement of the market exclusivity.

Is this a victory for pregnant women?

Certainly the FDA has the authority to ensure the availability of products and indirectly considers affordability.  It did so when it kept generic asthma inhalers on the market for several years rather than forcing them off to comply with an EPA requirement to remove inhalers powered by CFCs. I was at the agency at the time and the issue of inner city access was hotly debated.

That was (IMHO) a considered and organized action based on much thought and many meetings with stakeholders. The KV action (IMHO) was a reaction to media hype and political pressure.  The FDA even got involved in a little demagoguery when it cited the fact that the NIH had provided KV with support to conduct clinical trials as the agency’s reason to allow the compounding. Given that logic, should every drug developed in cooperation with the NIH or based on NIH research be denied market exclusivity?

Remember Plan B and remember that politics and FDA decisions should never be compounded   Read More & Comment...

Sequestration = Castration

09/06/2012 08:10 AM |

If the White House mandates the sequestration of the FDA budget the hurt will be shouldered by the public health.

PDUFA dollars cover reviews – but what about all of the other non-user fee items the FDA has on its plate – like advancing regulatory science and combatting counterfeiting, and developing standards for biosimilars and enhancing the safety and security of our food supply – to name only a few?

Sequestration = Castration of the FDA's mission.

Less is not more.

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Biomarker For Depression And it's Treatment

09/05/2012 02:13 PM |

Image: Jiri Hodan The Best Science Writing Online 2012 Showcasing more than fifty of the most provocative, original, and significant online essays from 2011, The Best Science Writing Online 2012 will change the way... Read More » By Amy Maxmen of Nature magazine People who live with clinical depression must also suffer the ‘trial and error’ approach that psychiatrists take when prescribing antidepressants. Now, a study published this week signifies the beginning of the end of guesswork. In it, a blood test predicts who will respond well to a novel treatment for depression, and who might even fare worse.“We haven’t had a test like this in psychiatry before,” says Andy Miller, a professor of psychiatry at Emory University and an author on the study in Archives of General Psychiatry. “There is no brain scan, no physiological measure that tells you whether a patient will respond to one drug more than another.” The test identifies an inflammatory protein in blood, C-reactive protein or CRP, that indicates internal inflammation. Whereas 62% of depressed participants with high CRP levels responded well to the new treatment, only 33% of participants with low CRP levels did. The correlation was not entirely unexpected, because the drug suppresses inflammation, and Miller thinks that inflammation underlies depression in some people. To test whether a potent anti-inflammatory could soothe the malady, his team recruited 60 people who had lived with major depression for more than a decade and had received no relief from antidepressants. Half of the participants received monthly treatments of the rheumatoid arthritis drug, Janssen’s Infliximab, and half received a placebo. Overall, Infliximab did not seem to work. However, when Miller’s team analyzed how the subset of participants with high CRP faired, it turns out they responded well to the drug, with a relief from sadness, suicidal thoughts, anxiety and other symptoms. Since the late 1980s, researchers have sporadically hypothesized that inflammation can lead to depression. The theory is that depressed behavior might be beneficial in the short term because it reserves an injured animal’s energy for healing rather than romping around in the sunlight. Although the hypothesis has never received widespread support, researchers have found that some depressed patients indeed bear elevated levels of inflammatory proteins. On the basis of the results from this relatively small study, a biologic drug such as Infliximab might be a better option in the anti-inflammatory realm than Cox-2 inhibitors such as aspirin, which come with unwanted side effects, says Miller. Although he knows of no Infliximab-like drug in development for depression, he says that companies might be encouraged by his team’s results. What’s more, with a biomarker to predict a response, companies will have a better chance of success. Robert Dantzer, a neuroimmunologist at MD Anderson Cancer Center in Houston, Texas, notes that some of the participants in the low-CRP group fared worse on Infliximab than on placebo. Thus, the CRP test could be as important a tool for excluding depressed patients from taking anti-inflammatory therapies as for predicting responders. This article is reproduced with permission from the magazine Nature. The article was first published on September 5, 2012.   Read More & Comment...

Precautionary Principle Pablum

09/05/2012 08:10 AM |

Here we go again. Thomas J. Moore and Curt D. Furberg (in a new JAMA article) accuse the FDA of compromising safety for speed.

As usual, Janet Woodcock places the matter in the appropriate perspective. "I'd like to stress that where there are unmet medical needs, the public has told us they are willing to accept greater risks," Dr. Woodcock said. "The cancer community in particular says we haven't used accelerated approvals enough."

Here’s the truth -- there is no such thing as a "safe" drug. It's the patient who must understand the risks required to achieve the benefit. That’s why the patient voice must be heard during all phases of the regulatory review process.

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