Good news! The UK’s Code of Practice regulator has issued guidance on social media and other digital communications tools.

Bad news!  There’s precious little relevance for American marketers.

Nevertheless …

The PMCPA has put together a Q&A document to explain how digital communication fits into its existing regulations. It took this step, rather than amend UK pharma’s self-regulatory Code, because “there have been few complaints about digital communication.”

Ah, our transatlantic cousins doth protest too little!

The Q&A document explains the place of digital communication tools within the existing rules, which ultimately comes down to companies can use digital media if they stick to the Code. It also adds particular emphasis on the Code’s existing ban on promoting prescription-only medicines to the public. “Therefore, pharmaceutical companies need to identify ways of utilising digital communications whilst complying with this restriction."

The PMCPA’s Q&A can be found here.

Obama Panel to Curb Medicare Finds Foes in Both Parties

By ROBERT PEAR

WASHINGTON — Democrats and Republicans are joining to oppose one of the most important features of President Obama’s new deficit reduction plan, a powerful independent board that could make sweeping cuts in the growth of Medicare spending.

Mr. Obama wants to expand the power of the 15-member panel, which was created by the new health care law, to rein in Medicare costs.

But not only do Republicans and some Democrats oppose increasing the power of the board, they also want to eliminate it altogether. Opponents fear that the panel, known as the Independent Payment Advisory Board, would usurp Congressional spending power over one of the government’s most important and expensive social programs.

Under the law, spending cuts recommended by the presidentially appointed panel would take effect automatically unless Congress voted to block or change them. In general, federal courts could not review actions to carry out the board’s recommendations. The impact of the board’s decisions could be magnified because private insurers often use Medicare rates as a guide or a benchmark in paying doctors, hospitals and other providers.

Last week, in his speech on deficit reduction, Mr. Obama said he wanted to beef up the board’s cost-cutting powers in unspecified ways should the growth of Medicare spending exceed certain goals. Supporters say the board will be able to make tough decisions because it will be largely insulated from legislative politics.

Lawmakers do not agree. Representative Paul D. Ryan, Republican of Wisconsin and chairman of the House Budget Committee, called it “a rationing board” and said Congress should not “delegate Medicare decision-making to 15 people appointed by the president.” He said Mr. Obama’s proposal would allow the board to “impose more price controls and more limitations on providers, which will end up cutting services to seniors.”

Senator John Cornyn, a Texas Republican who introduced a bill last month to repeal the Medicare board, said the president’s proposal “punts difficult decisions on health spending to an unelected, unaccountable board of bureaucrats.”

Representative Allyson Y. Schwartz, a Pennsylvania Democrat prominent on health care issues, said: “It’s our constitutional duty, as members of Congress, to take responsibility for Medicare and not turn decisions over to a board. Abdicating this responsibility, whether to insurance companies or to an unelected commission, undermines our ability to represent our constituents, including seniors and the disabled.”

Ms. Schwartz signed up on Friday as co-sponsor of a bill to repeal the board.

The purpose of the panel, according to the health care law, is to reduce the rate of growth in Medicare spending per beneficiary. The law sets annual goals — “target growth rates” — for Medicare spending below the average of the last 15 years.

Board members will be subject to Senate confirmation — no easy feat in the current political climate. Terms are six years. Members can serve no more than two full consecutive terms. The White House has yet to submit any nominations for the board.

“Why have legislators?” asked Representative Pete Stark of California, the senior Democrat on the Ways and Means Subcommittee on Health.

In some ways, Mr. Stark said, expanding the power of the board could be as bad as giving vouchers to Medicare beneficiaries to buy private insurance. “In theory at least, you could set the vouchers at an adequate level,” he said. “But, in its effort to limit the growth of Medicare spending, the board is likely to set inadequate payment rates for health care providers, which could endanger patient care.”

Representative Shelley Berkley, Democrat of Nevada, said she wanted to repeal the Medicare board. “I have great faith that this administration can put together a strong, independent and knowledgeable board,” Ms. Berkley said, but she said she had less confidence in future administrations.

Mark Parkinson, president of the American Health Care Association, which represents nursing homes, said his members disliked the board because it would allow Congress and the president to “subcontract out difficult decisions.”

Still, the idea of a more potent Medicare board could be a live option if the White House insisted on it in budget negotiations with Congress.

Mr. Obama said last week that he would “reduce wasteful subsidies and erroneous payments,” cut spending on prescription drugs and take other steps to save $500 billion in Medicare and Medicaid by 2023. “But if we’re wrong and Medicare costs rise faster than we expect,” he said, the Medicare board would have “the authority to make additional savings by further improving Medicare.”

The president’s proposal would set stricter goals for Medicare spending and establish some type of automatic cost-cutting device as an “enforcement mechanism,” but Mr. Obama did not say exactly how it would work.

Kathleen Sebelius, the secretary of health and human services, described the board as a backstop to “ensure that health costs are reduced.” The board might not have to take action if the president’s other proposals slow the growth of Medicare spending, she said.

The board grew out of proposals by Mr. Obama and Senator John D. Rockefeller IV, Democrat of West Virginia.

“Medicare payment policy should be determined by experts, using evidence, not by the undue influence of special interests,” Mr. Rockefeller said.

AARP, the American Medical Association and the American Hospital Association voiced concern about the president’s latest proposal.

“Relying on arbitrary spending targets is not a good way to make health policy, especially when decisions may be left to the unelected and unaccountable,” said A. Barry Rand, chief executive of AARP, the lobby for older Americans.

Oodles of fascinating and important data on the use of medicines in the United States hot off the presses from the IMS Institute for Healthcare Informatics.

And the policy implications are enormous.

Some tidbits to jump start your cogitation process:

* Medicare Part D beneficiaries filled 871Mn prescriptions in 2010, up 6.4% and accounting for nearly 22% of all prescriptions.

* Spending on branded drugs totaled $229Bn, but declined by 0.7%, while spending on unbranded generics increased 21.7% and branded generics by 4.5%.

* Spending on medicines mainly dispensed by primary care physicians grew by 0.5%, while those medicines primarily used by specialists grew by 4.8%.

* Small molecule spending totaled $240Bn, an increase of 0.5% as biologics grew by 6.6%, amounting to $67Bn.

* Spending on drugs through retail channels increased by 2.0%, while institutional channels rose by 3.0%.

* Oral forms of medicines declined by 0.1%, but spending on injectables increased by 5.7%.

* New therapy starts for 17 chronic conditions declined by 3.4Mn patients in 2010.

* 3.2Mn more patients started their therapy with a generic while 6.6Mn fewer patients started therapy with a brand.

* Continuations and refills within 17 chronic therapy areas increased by 6.7Mn in 2010.

* Generic continuations increased by 11%, or 67.8Mn in 2010, and now represent about two-thirds of all continuations.

* The number of brand continuations declined by 12% or 61Mn prescriptions in 2010.

* The top 5 classes in 2010 based on spending were oncologics ($22.3Bn), respiratory agents ($19.3Bn), lipid regulators ($18.7Bn), antidiabetes (16.9Bn) and antipsychotics ($16.1Bn).

* Absolute spending growth gains were highest for antidiabetes, antipsychotics, respiratory agents, HIV antivirals and autoimmune disease.

The complete report can be found here.

Roche has managed to reverse the European Medicines Agency's rejection of use of Avastin in combination with capecitabine for treatment of first-line metastatic breast cancer.

While this is a different indication than the one under appeal with FDA, the success of overcoming the regulator's concerns with a re-presentation of the data and an oral defense can only be confidence boosting as Genentech prepares for a similar interaction with FDA on combination use with paclitaxel, which is supported by better data.

The CHMP issued a positive opinion on April 14 on the indication for use with capecitabine, though with a limitation. The summary of product characteristics will restrict use to patients in whom other chemotherapy options, including taxanes or anthracyclines, is not considered appropriate. Further, patients who have received taxane or anthracycline-containing regimens in the adjuvant setting within the last 12 months should also be excluded, CHMP decided.

CHMP's original decision not to allow the indication was issued Dec. 16, 2010 - the same day that FDA similarly decided against a label expansion for use in combination with capecitabine, but more importantly, also announced it was moving to withdraw the metastatic breast cancer indication for Avastin in the U.S.

The initial breast cancer approvals were based on a strong progression-free survival advantage when used with paclitaxel chemotherapy in the E2100 study. Roche subsequently submitted other studies with different chemotherapy combinations - AVADO with docetaxel and RIBBON1 with capecitabine (Roche's Xeloda).

But those other studies failed to live up to the magnitude of the benefit in E2100. EMA had granted a full approval for Avastin use in combination with paclitaxel and decided the new findings did not impact that determination. FDA, however, had issued an accelerated approval on the condition that the other studies confirmed the benefit. When that didn't happen, it decided to rescind the approval - an action Genentech is now appealing.

The proposed label expansions based on the AVADO and RIBBON1 studies were denied by both EMA and FDA. While Genentech's efforts in the U.S. are currently aimed at keeping the paclitaxel indication, in Europe Roche requested a re-examination of the capecitabine indication.

The closing speaker at least week’s PhRMA annual meeting (aka, the “Pharmitzvah”) was Renaissance Man Fareed Zakaria. He gave a wonderful view of the current global economic and political landscape and was in equal parts equally euphoric about the potential and brutally honest about the challenges.  Most interesting were his views on the changing nature and role of the nation state.

His was the only presentation that permitted audience participation and I was fortunate enough to ask the first question, “What is the role of the 21^st century nation state when it comes to the future of healthcare?”

His answer was as dynamic as it was simple, “The x factor is innovation.”

Specifically, he believes that for a nation to be a global healthcare leader, said nation must invest its national treasure in innovation. He chose as exemplars Singapore, China, India and South Korea.  The percentage of public wealth invested by the United States doesn’t even begin to match up with that fearsome foursome.  Zakaria also pointed out that the difference between research facilities in Beijing and Cambridge isn’t quality … it’s cost.

In other words, not only are other nations investing more – they’re getting more bang for the investment yuan. All this at the same moment that President Obama disrespects pharmaceutical innovation by suggesting that we reduce the patent life of biologics from twelve years to seven.

In his closing remarks, PhRMA Chairman Chris Viehbacher summed it up nicely, “The question isn’t will our companies be successful? It’s will they continue to be successful in the United States?”

To be or not to be, that is the question. 

It certainly puts the slings and arrows into perspective.

Greetings from the 53^rd annual PhRMA Annual Meeting in jaunty Jersey City. No beach. No golf. No swag.  Just a lot of content.

Some samplers from the Pharmitzvah bima:

PhRMA Chairman (and sanofi-aventis chief) Chris Viehbacher, “As I was walking up to the podium, I was asked if I needed anything.  I said, how about a President and Congress that understands the need for a research-based pharmaceutical industry.”

Quite germane and timely considering President Obama’s speech on Wednesday. (For more on that speech see here).

Viehbacher teed-up the meeting by calling on all present to embrace “the three t’s” of truth, trust and transparency.  He also mentioned that, alas, there is not (at least as of yet) “a billing code for prevention.” Amen Brother Chris.

Later on we heard a pretty dynamic speech from Governor Chris Christie who insisted that he is (and we should all be) about “results over politics.”  Amen Brother Chris.

Governor Christie was followed by our 42^nd President, William Jefferson Clinton, who came on stage saying that he wanted to say something nice about the Governor, “but didn’t want to ruin his (Christie’s) reputation.” He then proceeded to give a rambling (by which I mean, charitably, eclectic) speech. Whatever he was paid it was too much -- unless he was paid by the word.

The luncheon speaker was Mike Krzyzewski (“Coach K”) who gave a very stirring and emotional talk, largely about his experience coaching the Olympic and World Championships basketball teams.  His philosophy of “standards not rules” resonated. (And his jibes about Kobe Bryant – who reneged on a promise to attend Duke after high school – were priceless.)

John Castellani, now eight months into his job as PhRMA President, captured the frustration of the crowd when, in commenting on the current political environment, quoted Oscar Wilde -- “A cynic knows the cost of everything and the value of nothing.” Nuff said. 

Amen Brother John.

Why did President Obama declare war on healthcare innovation on Wednesday?

It’s hugely disappointing that the same man who (as a United States Senator) once said that …

“Realizing the promise of personalized medicine will require continued federal leadership and agency collaboration; expansion and acceleration of genomics research; a capable genomics workforce; incentives to encourage development of genomic tests and therapies; and greater attention to the quality of genetic tests, direct-to-consumer advertising and use of personal genomic information."

… is now advocating a series of policies that would result in precisely the opposite.

That’s not class warfare – it’s no-class warfare. And it’s deleterious to the public health.

Exhibit A:  The Non-Interference Clause

The first question to ask is, what did Senators Tom Daschle and Ted Kennedy know that President Obama does not?  The answer is that allowing the Federal government to directly negotiate for Medicare drug prices is a bad idea.  That’s why they (Daschle and Kennedy) drafted the original language for the Non-Interference Clause.

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.

The bottom line here is that Part D is a tremendous success – due in no small part to the Non-Interference Clause. 

Consider:

* The projected cost for Medicare Part D is $117 billion lower over the next decade than experts estimated just last summer. This means that over the 10-year period from 2008 to 2017, the estimated $915 billion cost of Part D fell to $798 billion.

Why?  Marketplace competition.

* And, according to a study published in the Annals of Internal Medicine, the Medicare drug benefit led to a 17 percent decrease in out-of-pocket expenses, or $9 a month, for seniors who enrolled in the new Medicare Part D benefit in 2006, the first full year prescription coverage became available in the federal health insurance program for the elderly and disabled.

* And the savings amounted to an extra 14 days of medicine for those who signed up, or a 19 percent increase in prescription usage.

Can Part D be made even better? Absolutely. But this is good news worth sharing -- and not because it helps any particular partisan political agenda but because it means that more Americans -- tens of millions of more Americans -- are getting access to the medicines (largely chronic medicines) that will help them live healthier lives. And this, in no small measure, significantly reduces more drastic medical interventions -- which in turn reduces our overall national health care spending.

We shouldn’t interfere with success.

By revoking the Non-Interference clause, Uncle Sam will be able to "negotiate" prices for Part D drugs.  That's kind of like negotiating with your hands tied behind your back and a gun pointed at your head.  There's also the potential for Uncle to dictate that Part D prices be tied to prices in other countries -- a kind of Medicare reference price.

“Direct negotiations” means price controls.  And price controls = choice controls.

Exhibit B:  Biosimilars

The President wants to reduce the number of years of patent exclusivity for biologics. After speaking (during the State of the Union and a widely quoted op-ed in the Wall Street Journal) about the need for America to embrace innovation – President Obama is trying to make it more difficult, specifically when it comes to the desire to invest in pharmaceutical innovation – a sure bet under no circumstances.

The President’s now seeks to hasten availability of biosimilars by cutting the market exclusivity of innovators from 12 years to seven.

Bad idea since a longer period of exclusivity funds an innovator company’s research and development efforts. If the President’s proposal becomes law, the US would provide less data protection for innovative biologics than Europe.

12 years of exclusivity also gives hope to those suffering from rare diseases or conditions. If innovator companies think they will have a short time before a follow-on versions of their products hit on the market, they will likely only focus on drugs for major diseases and conditions -- potentially ignoring ailments that are less common, but equally as serious, to those suffering.

What’s next – an executive order instructing the FDA to approve biosimilars without clinical trials?  Alas – this is unfortunately not a far-fetched idea considering the tone and substance of President’s speech on Wednesday. 

If innovation is one of the key answers to our national economic recovery, then the President should abide by what he said, “Our economy is not a zero-sum game. Regulations do have costs; often, as a country, we have to make tough decisions about whether those costs are necessary. But what is clear is that we can strike the right balance. We can make our economy stronger and more competitive, while meeting our fundamental responsibilities to one another.”

Exhibit C:  IPAB

And then there’s the President’s idea that Congress should lower the threshold for the Independent Payment Advisory Board (IPAB) to make Medicare cost-cutting proposals, which could include containing drug costs. As BioCentury correctly explains, “Under PPACA, the board's recommendations would automatically be adopted unless Congress enacted alternative cuts of the same size. Under the law, the board will only act if Medicare growth per beneficiary exceeded GDP per capita by at least 1%, a rate of increase that the Congressional Budget Office recently said is not likely to occur. The White House wants to lower the threshold to GDP plus 0.5%”

There’s already the very real risk that IPAB will be insensitive to the needs of Medicare patients. After all, board members are unelected appointees with an incredible amount of power. The IPAB is liable to enact cost-cutting measures that might sound good in the boardroom, but actually lead to worse health outcomes for Medicare patients and strap them with unbearable costs. The President’s proposal makes this twice as bad.

Well – at least the President didn’t trot out drug importation. Well – at least not yet.

For the record, the solution is innovation.

We have to embrace innovative technologies for medical records and prescribing.  We need innovative clinical trial designs and molecular diagnostics so that we can develop better, more personalized medicines faster and for far less then the current $1 billion plus delivery charge.  We need innovation in access and reimbursement policies that rewards speed-to-best-treatment rather than more lower-cost patients per hour.

So we’d all better start taking innovation – of both the incremental and discontinuous varieties – seriously.  And that means both spending more on harder developmental R&D (with concomitant higher investment risks).

There’s lip service to the need for more robust comparative effectiveness – although this is a battle yet to be either defined (comparative effectiveness or cost effectiveness or clinical effectiveness?) or fought (do we need a U.S. version of NICE?).

L’audace, l’audace, toujours l’audace. This isn’t even the end of the beginning. Let’s keep our eye on the prize.  No, not the 2012 elections – the real prize: better access to healthcare for all Americans.  Innovation that focuses on creating a chronic healthcare culture that embraces prevention and prophylactic care.  Rather than wasting time on spin, let’s redouble our efforts on innovation.  Then, when we succeed through brainpower and teamwork (and, hopefully some civil bipartisanship), the circus surrounding the President’s budget proposal will be but a footnote in the history of American healthcare.

From the Pink Sheet ...

FDA is making the case for increased data-sharing and disclosure about drug development failures in the name of bolstering regulatory science.

In a speech to the Food and Drug Law Institute’s annual meeting on April 5, FDA Commissioner Margaret Hamburg said agency disclosure of reasons why a drug failed to win approval would strengthen the scientific underpinnings of drug development and regulatory review.

In addition, the agency is examining how it can mine its databases of submitted applications and approved products to help address scientific uncertainties in the development process.

 Hamburg used the FDLI speech to link two of her key initiatives since joining the agency in May 2009 – strengthening regulatory science and increasing transparency of agency actions.

Despite the agency’s stance that increased data-sharing and disclosure would speed the drug development process, such concepts are sure to see pushback from pharmaceutical companies concerned about protecting proprietary information.

Spurring Innovation …

Hamburg’s speech to FDLI focused on the theme of innovation and what she said was FDA’s responsibility “to take advantage of our unique position” to help deliver new, ground-breaking medical products.

“Innovative products that are truly transformative create unique scientific and regulatory challenges, so we’re rolling up our sleeves, developing FDA’s regulatory science and innovation initiative strategy and doing everything that we can to rise to the challenges and to ensure that FDA is a consistently powerful catalyst for innovation,” Hamburg said.

“Moving forward, we’ll focus our efforts on several distinct yet inter-related areas of activity, each of which must be addressed if we are to make a meaningful difference in supporting innovation.”

The first area she touched on is regulatory reform, saying, “We’ll continue to work hard to streamline and modernize regulatory pathways and make them more predictable and transparent.” She cited several examples of FDA action in this regard, including its ongoing implementation of the “21^st Century Review Process,” also known as good review management practices, and encouraging a quality-by-design approach to drug manufacturing.

“In each of these cases regulatory reform involved changing practices but also developing and applying better scientific understanding and tools to the review process, which brings me to the second area, strengthening science and reducing scientific uncertainty,” Hamburg said.

Hamburg unveiled her regulatory science platform in October. Among the initiative’s goals are modernizing product development; improving the speed, efficiency and predictability of the development, application review and manufacturing process; and using informatics to enhance health and drug safety

FDA’s fiscal 2012 budget request seeks $48.7 million in new funding for the program

Regulatory uncertainty is rooted both in scientific uncertainty and the failure to capitalize on existing knowledge, she said.

“At FDA, we have all of the data for every medical product ever submitted and approved in the U.S. There are huge opportunities to mine this information for the benefit of everybody to learn how to more effectively design drugs, to learn more about why products fail and how to better predict failure, and perhaps how to repurpose certain … drugs as we learn more about how to better assess subpopulations of responders.”

Developing new ways to share data and enhance communications with companies, without compromising trade secrets and other important commercial confidential information, is an important priority for FDA and should be for industry as well, Hamburg said. “This is part of what I mean by changing systems. We need to replace what is outmoded or not working with stronger, more effective patterns of practices.”

…Instead Of Waiting For Failure

During the question-and-answer session following her speech, Hamburg was asked how the concept of data-sharing would work “on a nuts and bolts basis” given industry concerns with such a proposal.

“This is an area that we’re obviously looking at carefully and cautiously and any decisions going forward would be done in partnership … with our key stakeholders,” she said.

She noted that agency scientists already have begun looking at data-mining opportunities with external partners, and the agency’s Transparency Initiative is examining some of the issues pertaining to disclosure.

FDA’s Transparency Task Force issued draft recommendations in May aimed at increasing public disclosure about agency actions. These included disclosing the existence and contents of “complete response” and “refuse to file” letters for NDAs and BLAs, as well as summary safety and effectiveness information for investigational applications or pending marketing applications if doing so would benefit public health

Many of the disclosure proposals drew strong objections from the Pharmaceutical Research and Manufacturers of America and the Biotechnology Industry Organization, which raised concerns about protecting trade secrets and the recommendations’ legality

However, Hamburg pointed to the benefits of disclosing information about why a drug was not approved.

“That can contain very, very important information to the R&D field more broadly,” she said, noting that there might be “multiple sponsors developing drugs following scientific pathways that, based on earlier work done, we know will fail. It’s not in the interest of those companies or the broader public good to just sit and watch them fail.”

Hamburg suggested that industry and individual companies would have to be on board with the disclosure concept. “A given company would have to be willing to say, ‘I understand there is a common good here … even though it might be demonstrating my dirty laundry just a little bit.’”

FDA is not alone among regulators in seeking to encourage such data-sharing and disclosure on drugs in development and clinical failures, she said.

“This is something that other regulatory authorities are also thinking about and working on as well because it’s an issue of really helping to build out the underlying science as we think about how can we make both the medical product development process as targeted and as effective and as efficient as possible, and how can we utilize the information that we have as regulators to support that process.”

Hamburg was asked whether FDA is discussing unilateral disclosure of information about drug development programs even if a sponsor objects. “It’s a discussion that we’re having, and I think it’s a partnership working with industry,” she said. “We need to move in directions that make sense, that will have value added, and where everyone understands the expectations and the opportunities.”

By Sue Sutter

Good feature in the April edition of Nature Biotechnology.  Penned by Jeff Fox, the atrticle is titled, "Mixed Messages from Washington."

How unusual!

Good parts about FOBs and FDA funding as well as other excellent bits.

One snippet, "PDUFA negotiations provide an excellent opportunity to lay it on the line honestly and explain to Congress why FDA needs to be funded robustly.  PDUFA, "gave industry better predictability and FDA more money, but it swerved off that path and promises were not kept.  Industry now wants to get back to those promises."

Who said that?  For the answer to that question and many others, see the complete article here.

A BILLCommentsClose CommentsPermalink

To protect all patients by prohibiting the use of data obtained from comparative effectiveness research to deny or delay coverage of items or services under Federal health care programs and to ensure that comparative effectiveness research accounts for advancements in personalized medicine and differences in patient treatment response.CommentsClose CommentsPermalink

Be it enacted by the Senate and House of Representatives of the United States of America in Congress assembled,CommentsClose CommentsPermalink

SECTION 1. SHORT TITLE.

This Act may be cited as the ‘Preserving Access to Targeted, Individualized, and Effective New Treatments and Services (PATIENTS) Act of 2011’ or the ‘PATIENTS Act of 2011’.CommentsClose CommentsPermalink

SEC. 2. PROHIBITION ON CERTAIN USES OF DATA OBTAINED FROM COMPARATIVE EFFECTIVENESS RESEARCH; ACCOUNTING FOR PERSONALIZED MEDICINE AND DIFFERENCES IN PATIENT TREATMENT RESPONSE.

(a) In General- Notwithstanding any other provision of law, the Secretary of Health and Human Services--CommentsClose CommentsPermalink

(1) shall not use data obtained from the conduct of comparative effectiveness research, including such research that is conducted or supported using funds appropriated under the American Recovery and Reinvestment Act of 2009 (Public Law 111-5) or authorized or appropriated under the Patient Protection and Affordable Care Act (Public Law 111-148), to deny or delay coverage of an item or service under a Federal health care program (as defined in section 1128B(f) of the Social Security Act (42 U.S.C. 1320a-7b(f))); andCommentsClose CommentsPermalink




(2) shall ensure that comparative effectiveness research conducted or supported by the Federal Government accounts for factors contributing to differences in the treatment response and treatment preferences of patients, including patient-reported outcomes, genomics and personalized medicine, the unique needs of health disparity populations, and indirect patient benefits.CommentsClose CommentsPermalink

sun-sentinel.com/news/opinion/fl-nbcol-avastin-brochu-0407-20110407,0,5456672.column

South Florida Sun-Sentinel.com

Put Avastin, and the FDA, back on the fast track

Nicole Brochu

Sun Sentinel Columnist

April 7, 2011

Copyright © 2011, South Florida Sun-Sentinel

According to the Daily Telegraph:

Cash-strapped health authorities are doubling the effective cost of medicines for some patients with long-term conditions. They are urging GPs to reduce the number of pills on a given prescription, which now cost £7.40 a time in England. In some cases the number of pills per prescription has halved.

While health authorities say the guidance is to help reduce the NHS bill for wasted medicines - estimated at up to £300 million a year - there is suspicion that health authorities are increasingly resorting to the measure for financial reasons.

Health care trusts have been asked to changed their guidance to GPs in order to get them to issue shorter prescriptions for some patients.

Our friend and colleague David Taylor, professor of pharmaceutical policy at the University of London, warned that shorter prescriptions for those who were "well established" on medications could actually increase costs because of higher dispensing fees.

He said: "You need a flexible approach and not a rigid rule."

David Stout, chief executive of the PCT Network (one of the largest health care trusts), emphasized the prescription rationing idea was to save money through cutting waste, rather than increasing prescription charge revenue.

Further, study after study demonstrates that the more frequently a patient has to refill a prescription, the more likely that patient is not to refill that prescription.

Non-compliance is a bad strategy for cost-containment.

Biocentury reports ...

Genentech, CDER submit joint statement on Avastin

Genentech Inc. and FDA's Center for Drug Evaluation and Research said in a joint statement they have been unable to agree on "the central questions that must be answered" for Commissioner Margaret Hamburg to render a decision about whether to withdraw metastatic breast cancer from the label of Avastin bevacizumab.

Instead of submitting a single joint list of facts in dispute, the parties submitted a partial list that includes whether actions by other regulatory authorities and data from studies outside the first-line setting are relevant. Each party now intends to submit a separate document summarizing what it considers to be the central questions to be presented and resolved. Genentech and CDER have until May 5 to submit their summaries.

Karen Midthun, who is the presiding officer for a hearing on the matter scheduled for June 28-29, had asked the Roche (SIX:ROG; OTCQX:RHHBY) unit and CDER to submit a joint statement outlining facts that are not in dispute and issues that are disputed.

The Association of the British Pharmaceutical Industry has selected a comms man, Stephen Whitehead, to be its next chief executive. Whitehead has spent the past few years working in corporate affairs at a U.K. financial services group. He was group communications director at Prudential PLC, between 2007 and 2010; before that he was group corporate affairs director at the U.K. bank, Barclays PLC. But Whitehead also has pharmaceutical industry experience - he spent 10 years at Glaxo PLC and Eli Lilly & Co. in corporate affairs, becoming European director of corporate affairs at Lilly before leaving the sector in 2003.

As the Pink Sheet reports, “The association's choice of a leader with public communications and lobbying experience is timely. Whitehead comes on board at a time of policy transition - perhaps more accurately, turmoil - for U.K. pharmaceutical companies. They are facing the replacement of a 50-year-old method of controlling their pricing and profits, the Pharmaceutical Pricing Regulation Scheme (PPRS), with a so far vaguely defined value-based pricing scheme.”

We are also pleased to report that our friend and colleague Richard Bergstrom has begun his new job as director general of the European Federation of the Pharmaceutical Industries and Associations EFPIA. (Richard was formerly director general of LIF, the trade association for the Swedish pharmaceutical industry.)

Is there are rhyme or reason to the FDA’s approach to safety?

On food dyes and ADHD there was no reason to revisit an issue that channels the same approach to causality the Andrew Wakefield, Jenny McCarthy and anti-vax crazies used.   (And shame on the media for not referring to the European Food Safety Agency report trashing the Lancet article most reporters cited – in tandem with a real life story of a kid being cured of ADHD by going natural.  As in:  “In 2009, EFSA re-evaluated the safety of the six color additives used in the Southampton study and concluded that the available scientific evidence does not substantiate a link between the color additives and behavioral effects.)

Are reporters that biased or lazy that they can’t get beyond the he-said/she-said approach to reporting. 

In any event,  why, with this knowledge in hand did the FDA grant Center for Science in the Public Interest’s ( the same group that sued McDonald’s for offering Happy Meals to kids) citizen petition which was submitted in 2008?

Now the FDA has decided to allow pharmacists to compound a drug (Makema) developed by KV Pharmaceutical  that significantly reduced the risk of certain preterm births in women who have had at least 1 prior preterm birth. Previously, the agent had only been available from pharmacies that had compounded the drug.”  The FDA and ob-gyns had wanted a version of the injectable  product that meet specific safety and efficacy standards.  Since “Makena is a sterile injectable with a risk of contamination, greater assurance of safety is provided by an approved product. “

http://www.modernmedicine.com/modernmedicine/Clinical+News/FDA-wont-take-action-against-pharmacies-that-compo/ArticleStandard/Article/detail/714333?contextCategoryId=40157

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 for each 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 for free for a Medicaid price to a wide range of organizations.

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.  In case anyone didn’t know, 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 then there were the pre-filled syringes of heparin and contaminiated compounded CF drugs.There are many more such examples.

To be sure the FDA does have 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.

But that was a well-considered and organized action.  This is a reaction to the media hype.  Indeed,  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 reason to allow the compounding they sought to eliminate.   Give that logic, maybe every drug developed in cooperation with the NIH or based on NIH research should be compounded.   How about a nice home made batch of Herceptin or HIV drugs such as Prestiva and Norvir?

All of this is preceded by the FDA’s children’s cough medicine obsession, you know that deadly drug that is linked to 39 deaths over 20 years, most of them do to overdosing by parents?

The FDA is in a shambles because of it’s pursuit of a politically correct position instead of a science-based regulatory policy.  It's already discourage the makers of cancer drugs from pursuing follow studies by shifting the Avastin endpoint to overall survival.  Now, its piling on KV and permiting the type of compounding that KV's drug was supposed to eliminate in an effort to protect expectant mothers. This is the Precautionary Principle run amok.

“In the multitude of counsellors there is safety.”

--  Proverbs xi.14.

CDER’s  Office of Surveillance and Epidemiology has been raised to the level of a a “super-office” to provide (at least in theory) greater management support to the unit’s growing staff and responsibilities.

As outlined by Super CDER Director Janet Woodcock, OSE will become a super-office that houses two new subordinate offices. According to Janet, “As OSE continues to build capacity and take on new responsibilities, and as the breadth, depth and volume of its work continue to increase, we need an organizational structure that provides adequate management support for these vital program areas.”

The changes will result in a new layer of management, in the form of the two new offices under OSE, which will have their own directors and deputy directors. The creation of a second epidemiology division, with its own director and deputy director, also will provide additional management support.

The Pink Sheet opines that, “The reorganization opens the door to a leadership change atop OSE.” Although OSE’s current director, Gerald Dal Pan, will serve as acting director of the new super-office, FDA will conduct a nationwide search for a permanent director.

“There is no safety in numbers – or in anything else.”

-- James Thurber
 

When it comes to the patient voice at the June 28-29 ODAC meeting on Avastin, silence most certainly does not imply consent

FDA's Karen Midthun, who will serve as presiding officer for the second ODAC hearing on the future of bevacizumab's (Avastin) breast cancer indication, has rejected Genentech’s request to allow members of the public to speak at the meeting.

This seems to be in direct contradiction to statements from CDER Office of New Drugs Director John Jenkins, who has gone on the public record that a crucial piece of risk/benefit assessment is the patient perspective.

"I think it's very important to understand the patients' perspective about how they value the benefits and how they are willing to accept the risk … A lot of us are basing these decisions in the abstract. We don't have the disease, we haven't achieved the benefit, and we do not actually have to weigh, personally, that benefit against the risk."

And further, "Regulators and others may not consider those benefits to be very important, but to the patients, they are extremely important and allowed them to go on about their lives.”

Dr. Midthun’s decision to ban patient comment is also contrary to the spirit of the 11/22/10 PDUFA negotiating session, where the FDA said that information about patients’ understanding of existing treatment tools is considered valuable, but is not consistently available during the review process.

The upcoming “Avastin Meeting” is, in all particulars, the  perfect opportunity to take the “patient involvement” hypothesis out for a spin. Why is the agency afraid of what patient groups might say? Or does ODAC simply consider the patient voice not relevant?  Advice mustn’t only be sought and considered when it supports your position.

After all, in the words of Dr. Jenkins, “We have to be aware of the societal expectations of how we interpret our standard and how we make our decisions.”

Bad Medicine

Organized ignorance endangers health.

Tevi Troy