Paging Jenny McCarthy and Andrew Wakefield...

07/01/2011 02:47 AM |

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505(2)b or not to be?

06/30/2011 08:31 AM |

I’ve just returned from the big BIO bash, where I was honored to moderate the panel discussion, "Lessons From In-Licensing Partnership: Biotech Company Partners with Global Pharma to Deliver Cutting-Edge Follow-On Biologics.” I was joined on the podium by joined by Arun Chandavarkar, Chief Operating Officer of Biocon Ltd.; Stephen Hoge, Principal at McKinsey & Co.; and Diem Nguyen, General Manager, Biosimilars, Pfizer.

Here’s how I opened the discussion:

Woody Allen said that, “Change should always be expected – except from vending machines.”  But management guru W. Edwards Deming hits closer to the mark, “Change is not required.  Survival is not mandatory.”

Many believe that the age of the blockbuster is over. Cost concerns are more challenging than ever. And we are struggling with what “personalized medicine” really means.

We are now in the era of post-patent medicine where advances in manufacturing, incremental innovation, and molecular diagnostics are as important as new molecular entities, and safety is as important (and as improvable) as efficacy.

The era of post-patent medicine is also the epoch of biosimilars.  But will biosimilars really be as important an element of change as many believe.  Will it be a game changer?

I believe the answer is “yes,” but I am not sure whether or not all the changes will positively affect the advancement of the public health.  I fear the expectations that biosimilars will radically reduce costs are overstated. I fear that safety concerns are being understated and that the risks to innovation are real.

As Eli Lilly & Co. CEO John Leichleiter said, "Creating and maintaining the conditions for innovation to flourish is challenging and complicated work - work that is never finished.”

Case in point -- there seems to be general consensus that, with a clear FDA pathway still off in the future, BLAs are the way to go.  Hence a redefinition of BLA as “Beat Legislative Ambiguity.” No aBLA biosimilar.

EMA and FDA recently announced that they are setting up a "cluster" on biosimilars to increase communications between the two agencies on the topic and the two agencies will discuss harmonization of regulatory requirements. Yet fundamental differences in the laws they administer make harmonization difficult.

Beyond a standardized regulatory pathway, there are many issues on the table:

* The role of “next generation biologics” (or, if you prefer, “biobetters”)

* Bioequivalence, interchangability, and therapeutic substitution

* The debate over distinguishable names and robust post-marketing surveillance

* GMP standards

* Reference products and clinical trial requirements and design

* And, last but not least, Patent Life vs. Data Exclusivity

And that’s not even considering PDUFA V. Nobody said it was going to be easy.

This morning’s panel won’t answer every question – and it is very likely to raise a few more. Our hope is that the panel will provide useful and timely insight on how global pharma and biotech companies can collaborate to develop, gain authorization, and globally commercialize cutting-edge follow-on biologics.

(And I believe we succeeded  in delivering a thoughtful and thought-provoking discussion.)

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The Avastin Vote -- Sadly, So 20th Century

06/29/2011 09:17 PM |

Avastin Fight Highlights Limits Of Big, Random Trials

By DAVID HOGBERG, INVESTOR'S BUSINESS DAILY Posted 07:08 PM ET

A Food and Drug Administration special advisory panel voted 6-0 on Wednesday to withdraw approval of the drug Avastin for treating late-stage breast cancer, sticking to its usual scientific standards despite some patients' pleas to disregard them.

The recommendation was based on the best scientific evi dence currently available, but the controversy highlights the limits of the science the FDA uses to determine drug effectiveness.

"Large, randomized-controlled trials are the 20th century," said Peter Pitts, president of the conservative Center for Medicine in the Public Interest. "The 21st century is the age of personalized medicine, where we need better molecular diagnostics to determine which subpopulation reacts to which treatments."

Avastin blocks the growth of new blood vessels to tumors, and is already FDA-approved for late-stage colon and lung cancer.

In 2008 the FDA granted accelerated approval of Avastin for use in late-stage breast cancer based on one randomized trial that found it slowed the progression of late-stage breast cancer.

In late 2010, the FDA withdrew approval based on two trials that found Avastin did little to slow progression, did not improve overall survival from late-stage breast cancer, and that it posed serious health risks, including death.

Randomized-controlled trials often involve large samples of patients. Such studies determine if a drug has a widespread benefit. But this is also a limitation. A treatment may work for a subpopulation in the sample, but since it doesn't work for most people in the sample, the average shows that the treatment is ineffective.

"The concept that you're looking for one magic bullet that cures a ton of patients at once is the wrong idea," said Terry Kalley, who founded Freedom of Access to Medicines in response to the FDA's 2010 decision on Avastin. "Cancer is likely to be cured by several therapies that target different subpopulations."

Kalley says his wife Arlene has been taking Avastin, along with chemotherapy, for 27 months.

Roche asked the FDA to extend its approval of Avastin until it conducts a study that "would include a biomarker component to identify patients who may be more likely to derive a more substantial benefit from Avastin."

But that is in the future.

"Unfortunately, the data we have right now (don't) allow us to know if there is such a subpopulation," said Karuna Jaggar, executive director of Breast Cancer Action.

Jaggar is concerned that Roche is using this as a stalling tactic to keep Avastin on the market. The Swiss drug giant could lose nearly $1 billion in sales, analysts say.

"We must insist that breast-cancer patients are receiving drugs that have been demonstrated to work," Jaggar added.

Others say regulators should keep Avastin approved for now.

"The FDA should say the evidence isn't there right now, but we will work with Genentech and patient groups to develop the appropriate diagnostics," said Pitts. "But the FDA needs to leave the indication on the label so people can still use it and be reimbursed for that."

If the FDA rescinds Avastin for late-stage breast cancer, at least until subpopulations are studied, then Genentech/Roche cannot market the drug for that purpose.

Doctors could still prescribe Avastin for the off-label use. But it's unlikely that government programs like Medicare and Medicaid or private insurance will cover Avastin — which costs $80,000 or more per year — for breast cancer.

An estimated 17,500 women suffer from late-stage breast cancer.

Many people like Terry testified at the hearings about Avastin. But without better data, such stories are little more than anecdotal.

The flip side is that if it turns out that Avastin does help some subpopulations, such women today will suffer as long as the FDA does not approve the drug for late-stage breast-cancer treatment.

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FDA Loses Battle and The War

06/29/2011 08:52 PM |

Media accounts and medical progress haters like Merrill Goozner are claiming that the FDA said Avastin in advanced breast cancer is "unsafe." 

What the FDA advisory panel (stacked by Richard Padzur, who heads up the Office of Cancer Drugs, with 6 advisors who wanted to yank Avastin last year) said was that based on the FDA's read of the data, Avastin despite causing a doubling in median progression free survival and objective response in tumors as well as an overall medial survival of two additional months in a subgroup of patients who were the most likely to die without Avastin (that's my take) and were more likely to die of cancer should not be used for breast cancer because it caused too much hypertension, bleeding and swelling.

Hal Barron,  Executive Vice President, Head of Global Development: Oncology, Inflammation, and Virology at Genentech -- Avastin's creator --  summed up the FDA's (Padzur) deadly decision:  The FDA (Padzur) would have allowed the Avastin indication to stay if it was really treating an  unmet medical need.  In otherwords,  the FDA (Padzur) would have not stacked the deck against Avastin if the FDA (Padzur) thought that giving thousands of women longer life and better health.

The FDA switched the endpoints and oncologists, health plans and patients know it even as The Gooz oozes glee over the Avastin decision and derides the additional benefit to thousands of patients as just in their minds.  Here's what Padzur said in his summary of evidence against Avastin.

"AVADO showed a statistically significant improvement in PFS for the bevacizumab-containing arms, with a HR of 0.70 (95% CI 0.55, 0.90) for 7.5 mg/kg
bevacizumab arm and HR of 0.62 (95% CI 0.48, 0.79) for the 15 mg/kg bevacizumab arm. The magnitude of treatment effect, as commonly assessed by clinicians based on
differences in median PFS, was marginal."

Genentech had called out this shifting of evidentiary standards in a pre-hearing summary of evidence:

"Although it was clear that AVADO and RIBBON1 (the two studies FDA, I  mean Padzur, wanted to confirm the benefit of Avastin) were not powered to show an OS benefit, the agency now cites the studies for failing to show a statistically significant OS effect. Then, CDER stated only after its decision to withdraw Avastin’s MBC indication that any PFS effect “must confirm the magnitude of treatment effect of E2100." 

Also: " CDER also has not articulated a clear rationale for its view that a 5.5-month improvement in median PFS is clinically meaningful but lesser improvements are not."
www.gene.com/gene/news/news-events/avastin/documents/051311.pdf

FDA might believe it sent cancer drug developers a message, but I believe it's get tough approach will backfire:

1.   Medicare and health plans will still continue to pay for Avastin.

2.  Congress will hold oversight hearings and will rake Padzur over the coals (repeatedly) en route to amending FDA statute through one of many bills calling for faster access to new treatments.

3.  The Obama administration will toss the FDA under the bus as the attacks target the President for presiding over the rationing of new drugs.

4.  Cancer patients who moblilized to keep Avastin's label will become enraged and engaged long term.

5.  The FDA has unleashed public sentiment that will overwhelm the medical progress haters --  Avorn, Angell, Wolfe, Goozner, Mahar, Soros, Furberg -- who will find their Congressional patrons have abandoned them. 




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True NORD

06/29/2011 06:26 AM |

The Avastin situation raises a lot of issues – not the least of which is the future of reimbursement for expensive treatments (both on and off label) for not only cancers, but also rare diseases.

First some facts, figures, and definitions:

• A “rare disease” (according to the IOM) is one that affects fewer than 200,000 people (in the United States)
• An estimated 25-30 million Americans suffer from a rare disease
• According to the NIH Office of Rare Diseases, there are more than 6800 “rare diseases”
• 80% of rare diseases are of genetic origin
• 50% of rare diseases affect children
• 85-90% of rare diseases are life threatening

And, most germane to this conversation …

• Only 10% of rare diseases have available treatments

So, what are we doing about it?

Well, of course there’s the Orphan Drug Act of 1983 which:

• Modified the Food Drug and Cosmetics Act to increase market incentives and reduce regulatory barriers for orphan drugs
• Provided the following incentives for industry to research, develop, and manufacture drugs for rare diseases:
• Seven years market exclusivity from the date of marketing approval of a drug with an orphan designation
• Tax credit of up to 50% for clinical research costs of a designated orphan drug
• Grants to support clinical development of products for use in rare diseases
• Waiver of PDUFA fees normally charged to sponsors
• Assistance with trial design for sponsors by FDA staff about nonclinical and clinical studies that could support approval of a rare disease drug

And the FDA Modernization Act of 1997 which:

• Provided additional incentives for industry to develop drugs for serious or life threatening conditions and which potentially address unmet medical needs (not exclusive to orphan drugs) including fast track, accelerated approval, and priority review designed to facilitate the development and expedite the review of new drugs that fall in the above category.

And what have these well-meaning pieces of legislation delivered?

Well, as of January 2011:

• There are 460 rare disease drugs in development in late stage clinical trials or awaiting FDA approval
• More than 350 medicines have been approved to treat rare diseases since 1983 (compared to 10 in the 1970s
• 2,313 medicines have been designated orphan drugs by the FDA
• Between 2000 and 2008:
  • Orphan drugs comprised 22% of all new molecular entities (NMEs) and 31% of all significant biologics (SBs) receiving market approval
  • Orphan drugs receiving priority review status rose from 35% of all orphan NMEs in 2000 to 50% in 2008; orphan SBs receiving priority review status rose from 17% to 67%
  • Big Pharma’s share of orphan drug approvals grew from 35% to 56%
• Average total development time for orphan products dropped by 2.3 months for NMEs and 37.5 months for SBs

Mazel tov. All good things. Solid and important achievements – as intended.

Why the success? Many reasons – not the least is which is that investment in innovation was rewarded.

But now comes the unintended part – reimbursement problems.

Scuttlebutt across the industry is that payers are beginning to, increasingly, question Tier One formulary status for many new drugs designed (and designated on label) to treat rare diseases. And these conversations are, increasingly, taking place well in advance of FDA review. The result is that Big Pharma is (increasingly and not surprisingly) recalibrating go/no-go decisions on rare disease development programs. It’s yet another unintended (and dire) consequence of short term cost-centric care trumping patient-centric medicine.

Stifling for innovation. Worse for patients.

Widows and Orphans is not an option.

Attention must be paid.  Read More & Comment...

Peter Pitts on Avastin

06/28/2011 09:29 PM |

  Read More & Comment...

Avastin in the Balance

06/28/2011 05:30 PM |

It seems that the deck  and the Oncology Drugs Advisory Committee is stacked against Avastin despite the outpouring of patient support for restoring the FDA indication for it's use in advanced breast cancer.   The FDA staffers who are pushing to keep the Avastin ban in place are spinning the issue as one of evidence vs. patient anecdotes.  But the patient anecdotes are based on measurable and measured improvements in survival:

tinyurl.com/3cthlwf

"There seems to be this perception that there are all these kinder, gentler treatments for metastatic breast cancer, but I'm not aware of those treatments, said Dr. Kimberly Blackwell of Duke Cancer Institute.

Blackwell helped conduct the trials of Avastin in breast cancer and believes the FDA is "moving the goal posts" on the drug's effectiveness, which could discourage drugmakers from pursuing new drugs.

"If the label is withdrawn, we will not see a new drug for metastatic breast cancer for another decade," said Blackwell, who directs Duke's breast cancer program.


Watch these discussions of Avastin's use in HER-2 positive metastatic breast cancer underscores just how misleading is the claim that there is no scientific evidence to support what patients are saying."

www.ecancermedicalscience.com/tv/video-by-category.asp

tinyurl.com/6b5o8pz

 

For the patient view I commend you to this article by Terry Kelley on how the FDA is deciding whether his wife will live or die..

www.foxnews.com/opinion/2011/06/28/fda-panel-is-deciding-life-or-death-for-my-wife/
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Me and Mitch

06/28/2011 03:33 PM |

I went out to talk to consumers, doctors, health plan executives and Governor Mitch Daniels about the state's Healthy Indiana Plan.    Here's a photo of me with the Governor looking like I had struggled with his security detail en route to my appointment.   If you have suggestions for captions, pass them along. 





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BIO Beyond PDUFA

06/28/2011 08:35 AM |

According to a report in BioCentury, “A few weeks after finishing negotiations with FDA over a PDUFA V deal that seeks to tweak drug oversight with changes to the review process, BIO now plans to propose sweeping changes to the U.S. regulatory system.”
For example:

A progressive approval pathway to get new therapies for unmet conditions to patients rapidly; with intensive surveillance and limits on off-label prescribing if necessary

A revamping of the advisory committee process to ensure that conflict-of-interest rules do not prevent FDA from gaining access to needed expertise

A fixed term for the FDA Commissioner to insulate the agency from political pressure. BIO takes political independence a step further, calling for FDA to be removed from HHS and turned into an independent agency with a head who reports directly to the president, like the Environmental Protection Agency.

A refining of FDA’s mission to emphasize its role in biomedical innovation. To bolster FDA’s capacity to advance scientific innovation, BIO wants Congress to lift restrictions on funding the Reagan-Udall Foundation.

Further, BIO calls for the FDA to have a “chief innovation officer with “authority to pilot and develop strategies for implementation of promising scientific and regulatory approaches in review divisions.”

Underscoring the connection between regulatory innovation and PDUFA, a member of BIO’s board of directors will present the organization’s recommendations at a July 7 House Energy and Commerce Committee hearing about PDUFA.

Bravo BIO.

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IPAB Jab

06/27/2011 01:03 PM |

From the pages of the Des Moines Resister.

(Why Iowa?  You know why.)

Our shortsighted plan for dealing with Medicare
By:  Peter J. Pitts

On June 7, the Department of Health and Human Services launched two important health care cost-saving initiatives.

First, HHS announced it would be making $42 million available to enhance coordination efforts between primary care physicians and other health care providers treating Medicare patients. An Emory University study concluded that better coordination in Medicare could save $125 billion over the next 10 years.

Second, HHS launched a $40 million effort to help states combat chronic disease.

Chronic diseases are responsible for 75 percent of our health care costs. Diabetes, heart disease and strokes alone account for nearly a trillion dollars in medical spending annually.

Both those initiatives have the promise to save money and lives. Unfortunately, they represent exactly the opposite approach to Medicare cost control set forth in last year's health care reform law.

Deep in the heart of the 2,700-page law is the creation of something called the Independent Payment Advisory Board (IPAB), a panel tasked with reining in soaring Medicare costs.

Here's how IPAB will work. The president will appoint 15 "experts" to serve on the board. Starting in 2014, they will be charged with keeping Medicare annual spending in line with designated targets. Any time spending looks to exceed its target, IPAB must draw up cuts to bring it in line.

Incredibly, IPAB's Medicare spending recommendations automatically become law whether a majority of Congress approves of them or not. Overriding IPAB requires a three-fifths majority vote in the Senate, a very high legislative hurdle. There's also no way for doctors or patients to legally or administratively challenge IPAB's decisions.

There are numerous legal problems with IPAB. A lawsuit is challenging the board's constitutionality, arguing that it has been improperly delegated authority over the federal budget (about 13 percent of the total) that constitutionally belongs to Congress.

The policy repercussions of IPAB are even worse. In meeting its spending targets, IPAB is ostensibly not permitted to ration care or adjust Medicare premiums and fees. Its main tool for cost-cutting is to lower Medicare reimbursement rates to doctors and other health care providers.

Unfortunately for Medicare patients, reimbursement rates are already well below market rates. Because of this, the number of doctors limiting or refusing Medicare patients is at an all-time high. IPAB threatens to make doctor access a major problem for Medicare.

Worse still, the scramble IPAB will produce each year to meet spending targets will deflect policymakers' attention from innovative new programs such as the two announced by HHS.

Even though the HHS primary care coordination and chronic disease initiatives are comparatively inexpensive and could eventually yield major savings, it will likely take years to measure their impact, let alone demonstrate that they're reducing costs. Not only that, they increase health care costs in the short-term.

On one hand, these two new HHS initiatives show that the Obama administration is making credible efforts to target the areas of the health care system that could produce the most savings. On the other hand, the Affordable Care Act through IPAB seeks to devalue efforts such as these in favor of squeezing doctors and other providers.

The right way forward is to get rid of IPAB and substitute for it a Medicare cost-savings plan that encourages long-term strategic thinking along the lines of these HHS studies.

The partisan acrimony over health care reform in Washington would seem to make all-new Medicare legislation unlikely. Fortuitously, though, bipartisan concern about IPAB's obvious shortcomings might prove to be the catalyst for a new and improved reform initiative.

House Budget Chairman Paul Ryan, R-Wis., has made repealing IPAB a major part of his high-profile budget proposal. Politico reports that several House Democrats have signed on to repeal IPAB. The repeal legislation's House sponsor, Phil Roe, R-Tenn., has floated the possibility of attaching repeal to the must-pass increase in the debt ceiling.

Support for IPAB is rapidly and rightly collapsing as citizens become better informed about the danger this all-powerful panel of unelected bureaucrats poses to their health care. It's time to urge Congress to get rid of IPAB and stand up for real Medicare reform.

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How can you regulate prescription drugs in a country with 246 varieties of cheese?

06/27/2011 12:17 PM |

In France the Ministry of Health has outlined a proposal to overhaul the country’s regulations on reimbursement, marketing, and safety of prescription drugs. 

Details are incomplete, but Xavier Bertrand, the Minister of Health, highlighted these provisions: 

• A new law on transparency and conflict of interest, to be called the “French Sunshine Act”;
• Pharma companies will be “held responsible for off-label use management with financial consequences”;
• For reimbursement, new drugs will be required to have an active comparator in Phase 3 studies,
• Products that have been on the market for some time will need to have a benefit/risk valuation prepared;
• A new tax will be created on pharma to finance physician medical education;
• A limit on sales reps beginning in hospitals (no personal detailing - only in group meetings) that may be extended to physician offices.

We’ll see.  As General De Gaulle said, “How can you govern a country which has 246 varieties of cheese?”

Or, perhaps more to the point, “I have come to the conclusion that politics are too serious to be left to the politicians.”

Bien sûr -- and especially when it comes to healthcare.

Please pass the cheese.

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Early Fireworks

06/27/2011 08:41 AM |

As we count down to the launch of the Avastin hearing, BioCentury reports that seven advisors will participate at the June 28-29 hearing on FDA's proposed withdrawal of bevacizumab’s metastatic breast cancer indication -- including five who voted at a panel last July to remove the indication and one who doesn't have a vote.

Four of the five who previously voted against the Genentech Inc. drug are ODAC members: Ralph Freedman from the M.D. Anderson Cancer Center; Brent Logan from the Medical College of Wisconsin; Mikkael Sekeres from the Cleveland Clinic Taussig Cancer Institute; and Wyndham Wilson from the Center for Cancer Research at the National Cancer Institute. The fifth is patient representative and temporary voting member Natalie Compagni-Portis.

The non-voting member is industry representative Gregory Curt from AstraZeneca’s oncology division. Curt was a non-voting member on the July panel. The final advisor participating next week is ODAC member Frank Balis, who was not on the committee last July. He is from the University of Pennsylvania School of Medicine.

Director Karen Midthun (who is the presiding officer for the hearing) said some committee members who had participated in previous meetings on the breast cancer indication had rotated off the committee and will be replaced before the June 28-29 hearing.

Caution – fireworks can be dangerous.

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Patent News, Joint Reviews, Preemption Coups

06/24/2011 09:27 AM |

A robust policy triptych courtesy of BioCentury.

Good News: House passes patent reform bill

The U.S. House of Representatives voted 304-117 to pass the Leahy-Smith America Invents Act (H.R. 1249). The lawmakers also passed by a vote of 283-140 a manager's amendment from Judiciary Committee Chair Lamar Smith (R-Texas) that would end diversion of fees from the U.S. Patent and Trademark Office but would require the PTO to wait for subsequent congressional appropriations acts to access excess funds. Much of the money the PTO collects from fees is currently diverted to fund other parts of the government.

 

In March, the U.S. Senate voted 95-5 to pass S. 23, its version of the bill. That legislation would allow the PTO to use all the money it collects from fees without fiscal year limitations.

 

Both bills include elements supported by the biotech industry, such as eliminating the ability of an alleged infringer to invalidate a patent based on the description of the invention's best use and creating a new post-grant review procedure. A joint congressional committee will next be appointed by the House and the Senate to iron out the differences between the two bills.

Joint Reviews: FDA, EMA to accept first application for joint review

EMA and FDA agreed to accept the first application submitted under a pilot program for parallel assessment of Quality by Design components of NDAs and MAAs. The application was submitted by Pfizer for an undisclosed product.

 

FDA and EMA are collaborating on reviews of seven applications from April 1, 2011 to March 31, 2014 under the program, which is intended to prevent regulatory agencies from applying Quality by Design standards inconsistently.

Preemption Coups: Supreme Court rules for preemption on generics

The U.S. Supreme Court ruled 5-4 on Thursday in Pliva Inc., et al. v. Mensing that FDA-approved labeling for generic drugs preempts claims under state tort law because federal regulations prevent generic drug manufacturers from independently changing a drug's label.

 

In the court's opinion, Justice Clarence Thomas wrote that today's ruling does not conflict with a 2009 decision in Wyeth v. Levine because branded drug manufacturers can add to or strengthen a drug's label without preapproval from FDA. In that case, the court held that an FDA-approved label for Phenergan promethazine from Wyeth did not preempt state tort law in a product liability suit.

  Read More & Comment...

Even Stevens

06/24/2011 09:10 AM |

Update from London-based CMPI Senior Fellow Philip Stevens:

After years of focusing the majority of its attention on HIV/AIDS (and to a lesser extent malaria), the global health community is finally waking up to the fact that the biggest health problems now facing developing countries are actually ‘non communicable diseases’ such as diabetes, cancer and cardiovascular disease.  And not before time: 80% of global deaths from these diseases occur in developing countries, and already cause double the number of deaths that result from things we more normally associate with these parts of the world, such as infectious diseases, maternal and perinatal conditions, and malnutrition.[1]

Although the UN and its specialised health agency, the WHO, have already started discussing how to respond to this issue, real flesh should be put on the policy bones at a special UN Summit on Non-Communicable Diseases, to be held in New York in September 2011. We can expect whatever global agreement emerges from this gathering to focus on prevention (watch out Big Food and Alcohol), as well as making treatments and diagnostics more readily available in developing countries.

None of this is going to be easy, no matter what is agreed and what funds are committed in New York.  The international response to the AIDS crisis in the late 1990s and 2000s highlighted the difficulties of delivering modern medicines and treatments to patients in developing countries, the vast majority of which have dilapidated health infrastructures and demoralised and underfunded health workforces, a large proportion of which has emigrated to better opportunities in countries like the US.

In order to bring some perspective to this issue, CMPI will be discussing this very issue ahead of the NY Summit in Geneva -- the administrative and policy heart of the UN.  Our inaugural “Geneva Round Table” event (13^th July) will focus on the challenges faced by clinics and hospitals in responding to this epidemiological shift, and we are lucky enough that Dr Eric Roodenbeke, the head of the International Hospitals Federation, has agreed to give his perspective based on his extensive knowledge of hospital policies and health systems approaches.

It should be a great event, and the guest list includes ambassadors, health attaches and other members of the UN health community.  If you happen to be in Geneva on 13^th July, please do drop by for what should be a most illuminating discussion!

  Read More & Comment...

Where there's smoke there's fire

06/23/2011 11:11 AM |

From the pages of Advertising Age:

ANA Weighs Lawsuit With FDA Over Graphic Cigarette Labels
Trade Group: Government 'Can't Put Words in the Mouths of Advertisers'

By: E.J. Schultz

The Association of National Advertisers says it will fight the federal government's move to slap graphic new warning labels on cigarette packs.

"We are still discussing whether we go directly with a lawsuit or whether we will enter of friend-of-the-court filing," Dan Jaffe, ANA's exec VP for government relations, told Ad Age. But "we will certainly join with others in opposing this proposal." Mr. Jaffe added that the ANA thinks the proposal is unconstitutional because "the government on its own ... can't put words in the mouths of advertisers."

The nine warning labels, unveiled Tuesday by the Food and Drug Administration, are the biggest change to cigarette packaging in more than 25 years. By September 2012, all cigarettes made or sold in the U.S. must display the images, which include graphic depictions of diseased lungs, decaying lips and teeth, a man wearing an oxygen mask and a morgue scene with a message: "Smoking can kill you."

As defenders of advertising, it's no surprise that the ANA is fighting back, as fears swirl that the government might use the momentum to more aggressively target other categories, such as alcohol. Indeed, the ANA is already on record opposing the cigarette labels, filing comments in January after the initial proposal was first unveiled. The association has also sided with six tobacco companies that are challenging in federal court the underlying Tobacco Control Act, passed with bipartisan support in Congress and signed by President Barack Obama in 2009.

But while the ANA is compelled to defend what is sees as free-speech rights, the association has a tricky task of defending Big Tobacco, hardly a sympathetic industry. Former FDA Associate Commissioner Peter Pitts, who served during the Bush administration, said: "It would be very tough for me to defend anybody who believes an anti-tobacco campaign is somehow deleterious to the national good."

He added: "Any image that scares the bejesus out of people away from cigarettes is a step in the right direction. It's been clearly shown by research that visuals are more powerful than words."

The FDA says it chose the images from a list of 36 potential warnings after reviewing scientific literature and reviewing more than 1,700 comments from various interest groups. The warning will be accompanied by a smoking-cessation-help phone number.

"The Tobacco Control Act requires FDA to provide current and potential smokers with clear and truthful information about the risks of smoking -- these warnings do that," FDA Commissioner Margaret Hamburg said in a statement.

But the ANA, in a blog post, said "this is precisely the kind of paternalism that the First Amendment does not permit."

  Read More & Comment...

Data Mining in the Land of Cherry Garcia

06/23/2011 11:07 AM |

As expected, overturned 6-3.

The ruling can be found here.

Next!

  Read More & Comment...

Say it ain't so Jo, Value-Based Pricing on the Down Low, and FOB GPS at BIO

06/22/2011 08:24 AM |

Today’s update on interfering with Non-Interference, paying the value-based piper, and biosimilars at BIO.

Say it ain’t so Jo

Repeating her mistake from last tme around, Representative Jo Ann Emerson (R-Mo.) is co-sponsoring legislation requiring the Medicare to negotiate prescription drug prices.

 

(The same provision was in the House-passed healthcare reform bill but didn't make it into the final bill as part of “the deal.”)

 

"The ability to negotiate the cost to taxpayers of prescription drugs purchased through the Medicare program could be a substantial savings at a critical moment," Emerson said in a statement.

 

Except that’s not true. Politics aside, consider the facts:

"It is not obvious that allowing the government to negotiate with pharmaceutical companies will lead to lower prices than those achieved by private drug plans. Private plans like Kaiser or United are able to negotiate deep discounts with pharmaceutical companies precisely because of the plans' ability to say no – the ability to include some drugs and to exclude others, allowing the market to judge the resulting formulary. On the other hand, when the government negotiates, its hands are tied because there are few drugs it can exclude without facing political backlash from doctors and the Medicare population, a very influential group of voters. Neither economic theory nor historical experience suggests government price negotiation will achieve lower drug prices. Congressional Democrats need to be careful in making the logical leap from market share to bargaining power. Empowering the government to negotiate with pharmaceutical companies is not necessarily equivalent to achieving lower drug prices. In fact, neither economic theory nor historical experience suggests that will be the outcome. Members should think carefully before jumping on the bandwagon – this promise may bring just the opposite of what was ordered."

Stanford Business School's Alain Enthoven and Kyna Fong

And in the words of the American vox populi (aka, USA Today):

"Both the non-partisan Congressional Budget Office and Medicare actuaries have said they doubt the government could negotiate lower costs than the private sector. The theory behind Part D is that market forces and competition among drug plans, overseen by government, can achieve better results than a government-run program. The multitude of plans allows seniors to pick one that best meets their needs. Government price negotiation could leave people without drugs that manufacturers decide aren't sufficiently profitable under the plan. Medicare recipients account for half of all drug prescriptions. 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."

Many of the President’s men and women are ready with the following talking point, “Look at how successful direct Federal negotiation works for the Veteran’s Administration,” suggesting that allowing the feds to directly negotiate for Part D is no different from the current VA scenario. But suggesting that the Veteran’s Administration “negotiates” prices for prescription drugs is a false premise. 

Under rules set by Congress, to sell drugs to the VA, companies must offer each drug at a price that “represents the same discount off a drug’s list price that the manufacturer offers its most-favored nonfederal customer under comparable terms and conditions.” The medication must be offered “at a discount of at least 24 percent off [the] nonfederal average manufacturer price (NFAMP). An excess inflation rebate is also required, equal to the percentage by which the price increase for [the] drug has exceeded the consumer price index (CPI) in the prior period.” The manufacturer must make all of its drugs available through the Federal Service Schedule for any of its drugs to be eligible for reimbursement under the VA and Defense Department health systems, the Public Health Service (including the Indian Health Service), the Coast Guard, and the various state Medicaid programs.

A study by Professor Frank Lichtenberg of Columbia University found that the majority of the VA formulary’s drugs are more than eight years old and more than 40 percent are 16 years old or more. Just 19 percent of all prescription drugs approved by the FDA since 2000 are available to veterans; only 38 percent approved during the 1990s are.

There’s a big difference between negotiating and mandating – and it’s not a thin line. My fear is that a government negotiated Part D plan is but the first step towards a more strident program of government price controls.

 

But putting politics aside to advance the public health is, alas, easier said than done. "There's savings to be had by negotiating those drug prices and addressing how Medicare reimbursement takes place for drug benefits," House Minority Leader Nancy Pelosi (D-Calif.) said last month. "This is a rip-off of the taxpayer, to have the pharmaceutical companies make as much money as they do."

 

Facts?  We don’t need no stinkin’ facts.

 

U.K. provides more color on planned pricing scheme

BioCentury reports that the U.K. Department of Health said that under a planned value-based pricing (VBP) system, the NHS would be mandated to "consistently fund medicines with a value-based price." The government's previously published consultation on VBP had been silent on whether or not the NHS would be required to pay for drugs for which value-based prices had been negotiated. Under the current system, a positive recommendation from the National Institute for Health and Clinical Excellence (NICE) requires the NHS to pay for a drug within three months of publication of the final guidance. The VBP system is slated to be in place by Jan. 1, 2014.

 

The DoH's statement came in its response to a report by the NHS Future Forum, a group of government and NHS leaders who collected input from professional groups, patients and the general public on the future of the NHS. The forum's report, published last week, recommended changes to the NHS.

FOB GPS

Will you be at BIO?  If so, please join me on Wednesday, June 29 (from 10:00 to 11:30)  to learn how follow-on biologics are influencing collaborations between global pharma and biotech’s. I’m moderating a session titled, "Lessons From In-Licensing Partnership: Biotech Company Partners with Global Pharma to Deliver Cutting-Edge Follow-On Biologics.” The panel will provide useful and timely insight on how global pharma and biotech companies can collaborate to develop, gain authorization, and globally commercialize cutting-edge follow-on biologics. Discussion topics will include the critical success factors needed to navigate new technology platforms, tech transfers, and global positioning, and how to build strong alliances to advance the development of novel therapies.  The panel comprises Arun Chandavarkar, Chief Operating Officer of Biocon Ltd.; Stephen Hoge, Principal at McKinsey & Co.; and Diem Nguyen, General Manager, Biosimilars, Pfizer – EM/EPBU Unit. 

Hope to see you there.

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My Response To Dr. Avorn

06/21/2011 12:43 PM |

Dr. Avorn:

I appreciate you taking the time to respond to my post and email.  I will publish your response on my blog without editorializing on my part.

Your analysis did nothing to help patients.. you only raised alarms about conducting smaller studies not randomized to your standards.  That's an old saw used by you and others -- along with your assertion that unless you understand the  mechanism of action endpoints, even if met, really don't matter.   Your assertion that cancer patients might suffer more is a counterfactual argument that frankly is a function of individual response to medicines that, absent biomarkers or algorithms derived from patient level data, cannot be tested.  What we do know is that life expectancy is higher from orphan drugs so the fact that you want longer and more expensive clinical trials for orphan drugs translates into you being willing to let sick kids die.   And your agenda is not secret:  you believe most drugs should not be marketed unless they meet your standards.  

Regarding CER,  you claim RCT is the gold standard for demonstrating effectiveness.  Yet when ALLHAT was all but eviscerated by ACCOMPLISH and other studies, I didn't see you call for a change in clinical practice. 

The evidence gathered from smaller trials is not "less valid."  Such data does not comport with your cultural and ideological bias against the commercialization of medical information.   I have a different bias.  And I am not on the Right or the Left when it comes to science. 

Finally, regarding BiDil, you should read your own work and what FDA officials have written in response. (See Annals in Internal Medicine, Jan, 2007 vol146 pages 57-62)  You did object to it's approval on grounds of political correctness and the mechanism of action argument.  In a perfect world we would know the MOA for drugs and diseases.  But your view of how to evaluate drugs,  RCTs above everything else, will not bring that about. 

Best wishes,

Bob Goldberg
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Avorn Responds

06/21/2011 12:33 PM |

Dr. Goldberg –

Our JAMA piece was not even about comparative effectiveness research, or about paying for treatments.  We agreed in it that orphan drugs for cancer may sometimes need to be approved with less valid data than is usually required, and called for better understanding how well these drugs work by studying their outcomes once they are on the market. But cancer patients do not benefit – and may well suffer more -- if a drug that does not work is approved and then given to them. We undertook this  analysis to help understand how patients with rare diseases could be provided with drugs that actually are going to help them; we certainly did not publish it because of some secret agenda to delay or deny treatment to people.  Your making such a wild charge just makes you look silly.

I also do not recall ever having said that BiDil should not be approved because we don’t know its mechanism of action. In any case, since it was just a combination of two widely available generic drugs, the treatment would be readily available whatever FDA did with the application for its approval as a patentable combination.

Reasonable people can (and do) differ about the best way to evaluate new medications, but this statement is so wild that it just looks weird. A flaming, inaccurate diatribe like this reflects badly on its author. Most others on the right have reassessed the wisdom of depicting those with whom they disagree as “malignant presence[s]” who want sick children to “drop dead,” and calling  for someone to “pull the plug” on them. Such hate speech just makes those who use it look like they are flailing around in ill-founded rage.

                 
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Targeting IPAB

06/21/2011 09:00 AM |

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