The other healthcare hearing

03/29/2012 11:23 AM |

In the midst of all the hoopla surrounding SCOTUS and the Affordable Care Act, let’s not forget today’s Senate HELP hearing on PDUFA.

For those who want to follow this important conversation, follow the twitter feed @modernmeds for the blow-by-blow.

Who said healthcare policy was dull?  Read More & Comment...

My Wish For Obamacare

03/29/2012 10:56 AM |

MARCH 29, 2012 4:00 A.M.
High Court Hopes
(Page 3 of 7)
By NRO Symposium

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JOHN FUND
One of the least-noticed mistakes made by the Obama administration in its arguments for its health-care law this week was the claim by Solicitor General Donald Verrilli that Congress had passed Obamacare to deal with a pressing national problem only “after long study and careful deliberation.” I’m told that even liberal lawyers in the courtroom had pained looks on their faces at that preposterous assertion.

Certainly the general public doesn’t buy that argument, as indicated by the polls. I doubt many lawyers do either. Congressional leaders bullied members into voting for the measure in the dead of night, blatant payoffs such as Nebraska’s “Cornhusker Kickback” and Florida’s “Gatoraid” were handed out, and members were given almost no time to absorb the bill’s 2,700 pages (leading House speaker Nancy Pelosi to say, candidly, “we have to pass the [health-care] bill so that you can find out what is in it.”) At least one Supreme Court justice is said to be astonished that process was so sloppy that Congress left out the traditional “severability” section, which routinely allows that should the courts strike down part of a law the rest would stand.

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Indeed, the third day of oral arguments in the Supreme Court brought a mild surprise: At least five of the justices are considering repealing all of Obamacare, either because the task of figuring out which parts can work absent an individual mandate is too onerous or they don’t believe Congress would have passed the bill without the mandate. In other words, the Court just might take the extraordinary step of sending Congress a stiff rebuke: that its recklessness and sloppiness in lawmaking should have some limits.
Randy Barnett, the Georgetown law professor who has bravely pushed the legal arguments against Obamacare with bulldog tenacity for two years, summed up what has been a good week for the law’s critics: “After these arguments, if the Court strikes down the Affordable Care Act, no one in the country will be surprised.” Indeed, if it happens, a majority of Americans will be relieved. And the battle over putting back some serious curbs on Congress’s ability to run roughshod over the Constitution will then begin in earnest.

— John Fund, a New York writer, is author of Stealing Elections: How Voter Fraud Threatens Our Democracy.


This is my contribution to today's  http://www.nationalreview.com  on-line discussion about how the Court should rule and what should the political response to it's decision (or the decision we hope for) be.  


I hope the Court rules Obamacare unconstitutional. The fact that Congress had many other ways to expand insurance pools has undermined the necessary and proper claim for the mandate. As Roger Vinson noted in his district court ruling, “While the individual mandate was clearly ‘necessary and essential’ to the Act as drafted, it is not ‘necessary and essential’ to health care reform in general.” The mandate is a way to redistribute premium revenue from the young and healthy to other groups. Under Obamacare, it is possible to pay the penalty and use the free emergency care that must still be provided under current law. I think all the judges regard Obamacare as unconstitutional and a failure.

If the Court overturns the law, Obamacare again becomes an issue. Supporters will warn that young people will lose coverage under their parents’ plan and that this will undo community rating. Opponents will be able to say the ruling stops a tax increase, blocks the indirect tax Obamacare imposed on people forced to buy the amount of health-care coverage deemed necessary by government, and pulls the plug on rationing. I hope legislators introduce a market-based reform plan that makes low-cost coverage available. If Obamacare supporters want to reintroduce the law with higher taxes and more regulation, let them.


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Look MAA, no hands

03/28/2012 08:17 AM |

Two important news items courtesy of BioCentury:

The first concerns the necessary and timely updating of how the FDA determines risk/benefit for medical devices.

Final FDA guidance on risk-benefit determinations for devices

FDA released final guidance that seeks to clarify how the agency determines the risk-benefit profile of a medical device during a premarket review. The guidance, which is in line with a draft released last August, says that in addition to safety and efficacy, FDA reviewers will consider criteria such as the novelty of the technology, the characterization of the disease and the availability of alternative treatments or diagnostics.

The guidance includes a worksheet that reviewers will use to make risk-benefit determinations during a review. FDA said it is developing training modules to help industry and sponsors understand how the Center for Devices and Radiological Health (CDRH) will apply the guidance. Reviewers will begin applying it to incoming submissions and to submissions already under review beginning May 1.

Of particular interest to those following the agency’s arduous journey in search of a risk/benefit “grid” is how CDRH will both develop and use its “worksheet” and whether or not that document will be public information after a regulatory decision is reached. Transparency would certainly go a long way towards holding the Center’s feet to the fire vis-à-vis regulatory predictability and consistency.

And, speaking of consistency, some news from our Regulatory Cousins across the pond.

EMA finalizes guidance on MAA transparency

The EMA and the Heads of Medicines Agencies (HMA) adopted a joint guidance for a Europe-wide approach to identify which information included in an MAA can be publicly released after approval, should a request for access to information be submitted. The guidance is part of EMA's policy to increase transparency and access to documents, which came into effect in 2010.

According to the guidance, sections of an MAA dossier that would not be released include detailed information on manufacturing processes and synthesis of the active ingredient, as well as contractual agreements. In general, reports on non-clinical and clinical trials, including efficacy and safety trials, would be made available, although exceptions may be considered when innovative study designs or analytical methods have been used. Identifiable patient information would be redacted. Scientific advice received by a sponsor on an agreed indication would be available, while advice related to new developments and formulations would remain confidential.

(For you Yanks, “MAA” = “Marketing Authorization Application.”)

This is interesting for a number of reasons, let me mention two. First, it points to the ability to harmonize across borders. Need an ocean separate us from this concept? And, second, is this at all predictive of where the FDA might go relative to “going public” with redacted Complete Response Letters?

Inquiring minds want to know.

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CMPI in the Washington Times: Drugs of Choice

03/27/2012 09:29 AM |

Next week, the FDA will be holding a hearing about letting consumers buy commonly used prescription drugs without a prescription, signaling FDA recognition that empowering consumers to make health care choices is the key to better health at a lower cost. The agency’s proposal is a refreshing departure from the usual administration’s practice of expanding government’s role in our daily lives. Yet so-called consumer groups that want the government to tell Americans how to eat, what cars to drive and what medicines to take are opposing even this small step toward medical freedom.

Indeed, the FDA proposal recognizes that individuals, armed with increasingly individualized information on what’s best for them, can make better decisions than a system where what is spent on health care is determined by bureaucrats. More consumers want more personal responsibility and freedom in making health care decisions, not less. They want to spend less time and money on medical care. So why would some groups oppose making more medications that have been widely used for years - for such conditions as high cholesterol, migraines, and hypertension - more accessible?

Groups such as the Center for Science in the Public Interest and Public Citizen claim the FDA proposal will “permit” people to use more medicines without solid information. On the contrary, the explosion of diagnostics, applications and online communities has increased our ability to responsibly take control of our health and well-being. In today’s digital world, we can use our smartphones to obtain test results and access information tailored to our diagnosis, genetics, health goals and lifestyle. Making drugs for osteoporosis prevention, birth control, migraines, cholesterol and erectile dysfunction available without a prescription would, with appropriate safeguards and new health-information tools, empower people to look after themselves. In fact, evidence shows that moving prescription drugs over the counter (OTC) helps people stay on track with their treatment regimen.

Critics often point to what they claim is misuse of cough medicines as an argument for more FDA regulation of OTC products, not less. But a recent survey I conducted on how American families treat coughs and colds underscores the opportunity self-care can offer to improve health. Over the past year, 61 million consumers avoided missing work, school or other scheduled appointments due to illness because they had access to OTC cough medicines to alleviate their symptoms. And nearly 75 percent of all consumers surveyed complemented cough and cold medicine use with rest, fluids (including chicken soup) and other “home” remedies.

The nearly a third of patients who see a physician for a cold wind up spending $7 billion on office visits and another $2 billion on antibiotics a year even though antibiotics don’t work for such ailments. If we were all forced to run to the doctor and get a prescription every time we coughed or were stuffed up, we would be spending a lot more money treating symptoms that can be alleviated effectively by OTC medicines in our pharmacy or supermarket and, more often than not, resolve on their own.

So why do some groups want to keep Americans shackled to our current system of care? As Eric Topol notes in “The Creative Destruction of Medicine,” access to our “own data and information - whether it be DNA sequence or biosensor remote monitoring - will soon be unprecedented, and surely each individual has more at stake about his or her health than the busy physician who is looking after hundreds to thousands of patients … But change cannot take place unless consumers are the driving force.” Without the ability to conveniently access and use more medicines we know and trust in tandem with such valuable personal health information, the paternalism of the present health care system will prevail. Opponents of the FDA proposal are afraid of losing control over our lives.

There is wisdom in the crowd. The benefits of consumer empowerment outweigh the risks that, in fact, can be managed mostly by we the people by educating each other. Turning more prescription medicines into OTC products not only saves time and money. It enhances our ability to sustain health rather than just treat disease.

Illustration: Dangerous drugs by John Camejo for The Washington Times  Read More & Comment...

New Evidence on the Effect of The Individual Mandate On Seniors

03/27/2012 09:22 AM |

Under-treatment is a result of government health systems undervaluing us as we get older and are faced with life threatening illnesses.   And it's a product of 1.  using comparative effectiveness research to guide reimbursement and treatment decisions and
2.   the fact that CER standards and information is not only controlled by government but used in 'academic detailing' of doctors while private companies are banned to discuss new treatments..


UK elderly "being denied cancer treatment"

LYNNE TAYLOR


Under-treatment is one of a number of factors contributing to around 14,000 avoidable cancer deaths in patients aged over 75 in the UK every year, according to a new report.

Cancer mortality rates are getting significantly better for the under-75s, but they are improving at a much slower rate in those aged 74-84 and actually getting worse for people aged 85 and over, says the study, from Macmillan Cancer Support.

PREDICT charter underlines trial bias against older people
The number of people living with a cancer diagnosis is set to rise from two to four million in the next 20 years, and, with half of all new cancer cases in the UK diagnosed in people aged 70 or over, this is an issue which must be addressed as a matter of urgency, says Macmillan.

According to the report, some of the reasons why older patients are less likely to receive treatment than those who are younger include:
- recommendations on treatment are too often being made on the basis of age, regardless of how fit patients may be;
- assessments do not adequately measure an older person's fitness for treatment, and co-existing health problems are often not identified or effectively managed;
- many patients do not take up treatment because they have inadequate practical support to help them at home, with transport, or with care for dependent spouses and other family;
- older people are not represented on enough clinical trials, reducing the amount of evidence available to clinicians on the benefits and risks of cancer treatment and its impact on quality of life: and
- oncologists and cancer surgeons need more support to manage issues specific to older people such as falls, incontinence and multi-drug use. In a survey of 98 oncology trainees, 60% reported that they have never received any training in the particular needs of older people with cancer, despite them making up half of numbers getting cancer each year, the study reports.
"To deny older patients treatment that could cure them based on ill-founded assumptions is an unacceptable act of discrimination," said Ciaran Devane, chief executive of Macmillan Cancer Support.

"We have a moral duty to treat people as individuals and give them the best chance of beating cancer, regardless of their age," he stressed.
"Efforts are begin made to increase early diagnosis and promote healthier lifestyles, but much more needs to be done to tackle under-treatment. The NHS and social care providers must wake up to the specific issues older people face and ensure treatment decisions are based on their overall health, not just their date of birth," said Mr Devane, adding: "writing people off as too old for treatment is utterly shameful."

Added Professor Riccardo Audisio, consultant surgical oncologist at St Helen's Hospital: " it is despicable to neglect, not to offer, not to even go near to the best treatment option only on the simple basis of the patient's age. This has been a horrible mistake that, particularly in the UK, we have suffered from."
Macmillan has, in partnership with Age UK and the Department of Health, set up five pilots to test new models of older people's care. They will report in December this year.

- Meantime, the National Cancer Intelligence Network (NCIN) reported yesterday that almost 10% of bowel cancer patients die within a month of being diagnosed, and of these 56% are over 80% years old, while 60% have been diagnosed following an emergency admission to hospital  Read More & Comment...

Give me Liberty or Give me Teva

03/27/2012 08:08 AM |

A new study by CVS Caremark shows that Massachusetts residents with chronic conditions are more likely than residents of all other states to take generic medication.

Alaska ranks last in terms of patients who obtain the maximum allowable amount of medicine with each refill, New Jersey ranks last with just 57.8 percent of prescriptions filled with generic drugs, and New Mexico ranks last when measuring patients who refill their prescriptions as directed.

According to CVS Caremark, the failure of patients to adhere to their prescriptions layers $290 billion in unnecessary health costs onto a health care system that is crushing federal and state budgets with little sign of abating.

Per CVS Caremark, use of generics is a critical component in determining whether patients are sticking to their prescription regimen because the cost of brand-name drugs can be a deterrent. Patients also tend to stick to mail-order prescriptions more closely than they do to prescriptions they need to pick up at retail outlets.

More than half of Americans suffer from a chronic disease, contributing to 75 percent of the country’s health care costs.

  Read More & Comment...

Apocalyptic Now

03/26/2012 04:15 AM |

On Sunday, the New York Times ran a Sunday Dialogue called, “Equitable Health Care.”

(The complete discussion can be found here.)

The lead commentary was written by Alan B. Cohen, professor of health policy and management at Boston University School of Management and executive director of its Health Policy Institute.

Cohen writes, “The specter of rationing has also been invoked by those seeking to repeal the Independent Payment Advisory Board — a panel that would recommend ways to lower Medicare costs — so that Congress and special interests may retain firm control over Medicare spending cuts. By delegating responsibility to independent experts, such a board would help depoliticize the existing process.”

My response?

“Professor Cohen criticizes those who want to repeal the Independent Payment Advisory Board for invoking “the specter of rationing.” That ignores an immediate and crucial concern.

The board will be composed of 15 presidential appointees, unaccountable to the public. It will be given the task of cutting billions in Medicare expenditures — largely by denying government reimbursement for new and innovative medicines.

In other words, its only viable option will be to further ratchet down reimbursement rates for providers, especially doctors, who are already losing money on Medicare patients. Indeed, according to the American Medical Association, the financial burden of too-low payments under Medicare has driven 17 percent of doctors and 31 percent of primary care doctors out of the Medicare program altogether.

If rates fall any lower, seniors will have an increasingly difficult time securing doctor appointments. Visits will be cut short to squeeze in patients and care compromised. The board is even more insidious because it deflects policy makers’ attention from innovative reform efforts with real cost-saving potential.”

To which Cohen retorts:

“Mr. Pitts’s attack on the Independent Payment Advisory Board, on the other hand, contains an apocalyptic vision of stifled innovation and compromised patient care wrought by “insidious” board decisions. This is what reform opponents and industry groups want the public to believe.”

Apocalyptic?  Just what does that mean?

Here are some synonyms for “apocalyptic” from www.thesaurus.com:

- Important
- Prophetic

If my comments are apocalyptic, what are the antonyms that might represent those of Dr. Cohen?

- Insignificant
- Unimportant

Perhaps a more straightforward way to say it (IMHO) is that I’m right and he’s mistaken.

And a big “thank you” to the Gray Lady for providing the forum.

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Heart is Half a Prophet

03/23/2012 03:38 PM |

Eric Topol and colleagues at Scripps have developed a gene test that quickly and accurately predicts who is at risk for sudden heart attacks. Now here's the question: will we have to wait years for AHRQ and health plans to pay for this diagnostic? How many avoidable deaths will be caused by a delay or underpayment in reimbursement? http://stm.sciencemag.org/content/4/126/126ra33   Read More & Comment...

PDUFA and the Critique of Pure Reason

03/23/2012 07:20 AM |

Immaturity is the incapacity to use one's intelligence without the guidance of another. -- Immanuel Kant

Is "Kant" in the FDA vocabulary?

Here’s the latest House PDUFA discussion draft being circulated by the House Energy and Commerce Committee.

As pointed out in the Pink Sheet, guidance development would take on a much larger and likely more extensive role at FDA if provisions in the draft are ultimately approved. The draft, would require FDA to give at least three months’ notice before it intends to issue a certain type of draft guidance and solicit public comment.

New, more rigid guidelines for public participation prior to issuing some draft guidance documents, as well as a requirement that they be finalized within a year, likely would require an increase in agency resources devoted to those efforts.

The more predictable schedule also would favor industry by ensuring the waiting period for FDA thinking on a subject would not be potentially unending. Industry typically complains about the uncertainty of FDA policy on many subjects.

The rule would apply to a guidance that “sets forth initial interpretations of a statute or regulation, sets forth changes in interpretation or policy that are of more than a minor nature, includes complex scientific issues, or covers highly controversial issues,” according to the draft.

A clause in the proposal allows the agency to skip the advance notice if it is impracticable, unnecessary or contrary to public interest, but it would still be required to meet with stakeholders during a three-month period beginning the day the guidance is issued.

Once a draft guidance is released, FDA would be required to finalize it no more than 12 months later or it would be considered null and void, according to the discussion draft.

Comprehensive reviews of final guidances also would be required every five years under the bill to ensure they are not “outmoded, ineffective, insufficient or exceedingly burdensome.”

The provision would subject FDA to a much more formal schedule of guidance development. While the intention appears to exclude routine guidance updates from the rule, it would be easy to place most, if not all, new agency guidance, into one of those categories, especially “highly controversial” and “more than a minor nature.”

The schedule would create more pressure on the agency to finalize guidances before the one-year deadline, which could leave them incomplete or rapidly outdated. A more structured guidance development process ideally would prevent situations where industry has been penalized for violations of FDA policy, yet still waited for years to receive formal agency thinking on the subject.

Sponsors have complained the agency changed its guidance on clinical trial endpoints in the midst of a development program, making continuation more complicated.

FDA is clearly being forced to battle against congressional efforts to adjust its mission to focus more on issues of innovation … and PDUFA’s first principle of predictability.

A provision would add to FDA’s mission that it would strive to establish a regulatory system that “protects the public health and enables patients to access novel products while promoting economic growth, innovation, competitiveness, and job creation among the industries regulated by this Act,” according to the draft.

The package includes reauthorizations of the prescription drug and medical device user fees, as well as new programs for generic drugs and biosimilars.

The four user fee cycles would run from FY 2013 through FY 2017.

The user fee package is expected to be ready for passage soon. Health Subcommittee Chairman Joe Pitts, R-Pa., already has stated he wants the bill signed by President Obama by June.

He alone is free who lives with free consent under the entire guidance of reason. -- Baruch Spinoza   Read More & Comment...

Downton AbbVie

03/22/2012 08:16 AM |

Abbott Laboratories’ pharmaceutical spinoff will be called AbbVie and will debut by the end of the year.

  Read More & Comment...

Prometheus Bound

03/21/2012 08:45 AM |

Abraham Lincoln wrote that patents, “add the fuel of interest to the passion of genius.”

The Supreme Court of the United States has just doused a lot of that passion – and, along with it, the hopes that personalized medicine (via advanced diagnostics) can become reality sooner rather than later. 

Or, as the Wall Street Journal judiciously puts it, “The ruling sparked uncertainty about the booming field of personalized medicine, in which some of the world's largest drug companies are vying to tailor treatments to patients' unique makeups by using diagnostic tests.”

The Supremes have rules that telling doctors of a new scientific discovery and recommending they use it to treat patients is not patentable. Per Justice Stephen Breyer, that’s all Prometheus Laboratories did when it patented a test to help doctors set drug dosages for patients with Crohn's disease. The inventions claimed in the patents "consist of well-understood, routine, conventional activity already engaged in by the scientific community.”

Justice Breyer said the Prometheus patents recited laws of nature connecting the level of certain chemicals in the blood to a thiopurine dosage that is too high or too low. Then, he said, the patent walked doctors through several obvious steps—such as measuring the chemical levels—to turn that natural law into recommendations for particular patients. He rejected the company's contention that, taken together, the steps represented a patentable method for treating patients.

But isn’t that what “intellectual property” is all about?

Justice Breyer emphasized that patent law should not inhibit future innovations by "tying up the future use" of natural laws. He said Prometheus's patent claims "threaten to inhibit the development of more refined treatment recommendations."

But where there is no patent protection there is no investment.  And where there is no investment there is no development. 

The only people who are going to love this ruling are those who agree with  Jamie Love that separating the markets for innovation from the markets for physical goods will ensure that everyone, everywhere, will have access to new medical technologies at marginal costs.

Baloney.

Prometheus Bound is, after all, a Greek tragedy.
 

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Survival of the Most Personalized

03/20/2012 08:20 AM |

According to Alan Ashworth, head of the Institute of Cancer Research in London, "Today, despite the best efforts of the drug companies, we still use medieval methods to attack most cancers: cutting [surgery], burning [radiotherapy] and poisoning [non-specific chemotherapy]." But, says Ashworth, "Once we understand the Darwinian process by which cancer cells evolve in the body, we should be able to control even advanced disease through combinations of specific drugs."

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Biosimilar Music Theory

03/19/2012 01:18 PM |

Overheard at event bio break:  “EPO is the new Lipitor.”

Last week in Boston I was honored to give a presentation at the 10^th Biosimilars America conference. But, as is often the case at good events, it was more about what I learned than what I had the chance to say.

Or, as my father used to remind me, “Listening is not just waiting for your turn to talk.”

Of the many fine presentations, I refer specifically to the talk given by John Pakulski, head of US Biopharmaceutical Regulatory Affairs at Sandoz, and the current chair of the GPhA Biologics Task Force.

John made many interesting and important points, but the one that resonated the most was that, in an age of technology, we must use technical data more and rely on clinical trials less. In short – when it comes to understanding the complicated concept of biosimilarity (from both regulatory as well as clinical perspectives) it’s a brave new world. But will we have the courage to move beyond “the gold standard.” Perhaps we should say, “the 20^th century gold standard.”

Claude Debussy said, “Music is between the notes.” And the same can be said for biosimilarity. We now have many thoughtful guidance documents but, in many respects, it’s just theory. And just like with music theory, the words on the page are one thing – but when talented performers sit down at different pianos in disparate venues the result is both similarity and uniqueness.

In other words – creativity happens. 

Another difference between biosimilars and small molecule generics is that the developers of biosimilars are very much in the innovation business. 

Innovation happens.
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Second Look At Statin Risk

03/16/2012 02:18 PM |

A couple of weeks ago I posted on the 'statin scare',  and referred to a study that discussed the increase risk of diabetes for people taking cholesterol lowering meds in primary prevention. 

I was skeptical of a single study as the basis for concern (and certainly panic).    Eric Topol's recent videoblog on the issue is underscores the need for careful review of all data, tailoring treatment to patients to maximize benefits and the urgent need for whole genome research on response and metabolism of all statins.

I am on a low dose statin.  I monitor my liver enzymes pretty regularly (normal for someone who works out as regularly as I claim to!).  It never occured to me that the slight elevation in my glucose levels -- something my doctor has always kept an eye on --  was a 'side effect' of statin use.   Since we have a family history of high cholesterol and heart attacks statins are indicated and my risk of a heart attack were probably greater if I didn't take statins compared to taking them and getting diabetes.   But a more precise and individualized measure of risk and benefit would be greatly appreciated!

Dr. Topol raises important questions about the risks and benefits of using statin in primary prevention absent other risk factors.  And he again makes a persuasive case about the value of genomic testing.  As he notes in his excellent book   The creative destruction of medicine: How the digital revolution will create better healthcare   30 percent of people being treated for diabetes are non-responders to metformin, which is pushed as first line therapy.   Why not a test that measures response and reaction to statins and diabetes meds.  Why not make that test available to consumers in the most convenient way possible?

The impact on compliance and health outcomes would be significant in my opinion.  And as for my use of statins,  I will be a bit more careful going forward.

Thanks Dr. Topol. 

http://blogs.theheart.org/topolog
  Read More & Comment...

Progressive Approvals "Over There"

03/16/2012 09:09 AM |

Many tough questions. No easy answers.

From the latest edition of the British Medical Journal ...

Speeding up access to new drugs
Nigel Hawkes
 

The prime minister promised at the end of last year to give some patients quicker access to new drugs. Was this just an empty aspiration?

No. A lot of thought has been given to a faster access scheme since David Cooksey recommended it in his review of UK health research funding in December 2006. A working group from industry and government produced a plan by November 2009. It sat on a shelf until resurrected two years later.

How would it work?

Medicines that have completed phase III trials (or in exceptional circumstances phase II) and that will treat or prevent life threatening, chronic, or seriously debilitating conditions that lack adequate existing treatments would qualify. Manufacturers would have to apply, and a decision would be promised in 75 days.

How would that be any quicker than licensing, if phase III trials have already been completed?

It usually takes a year or more to get licensing approval after a successful phase III trial. The new process is expected to get these medicines to patients a year earlier than otherwise would be the case.

But doesn't it mean that proper risk assessment will be skimped?

There's a danger of that. The burden of risk will be shifted towards the doctor and the patient and away from the manufacturer. Good information will be vital; patients will need to be fully informed and give active consent. Legally, the position will be the same as that for any unlicensed medicine, and the working group believes-but cannot guarantee-that primary care trusts or clinical commissioning groups will not be liable should anything go wrong. And it says that if the decision by a doctor to treat a patient was reasonable in all the circumstances and all relevant information was provided, a successful claim for negligence is unlikely.

If unlicensed medicines can be marketed and sold, what's the point of licensing?

The medicines under this scheme would be exceptional and few in number, perhaps only one or two a year. The NHS already uses unlicensed or off-label medicines in some cases, on the authority of the prescribing doctor (for example, bevacizumab for age related macular degeneration). Patients consulted by the working group were confident that they were competent to make a proper assessment of the risks. Doctors were not so sure.

What happens when the medicine is licensed?

The approval will last a year and can be renewed if necessary. When the medicine gets a licence it will become part of the normal process and the National Institute for Health and Clinical Excellence (NICE) will examine its cost effectiveness. Arrangements for continued funding of early access patients will need to be agreed for each medicine in advance.

So these fast access medicines will bypass NICE?

Yes. NICE deals with licensed medicines; these are unlicensed, so NICE does not have a role.

So how can we tell if manufacturers are overcharging?

We can't. They will set the price. It will be up to commissioning organisations, without NICE input, to decide if it's a price they want to pay.

Where's the money coming from?

An important question, the answer to which could yet put a spanner in the works. Since these medicines will not be approved by NICE, there will be no obligation for primary care trusts or clinical commissioning groups to pay for them. There will be no additional money available, unless the Department of Health or the drug companies provide it. Commissioning bodies will have to fund the drugs out of their normal allocations and in hard times may be reluctant to do so. This could lead to "postcode prescribing," as the working group conceded. But it hoped that if the process were applied only to medicines providing "significant clinical benefit in areas of current unmet need" the variation could be minimised.

Has anybody else attempted a similar scheme?

Yes, the French Temporary Authorisation for Use (ATU) scheme is quite similar and has operated for 15 years. To qualify for this, the medicine must have no satisfactory alternative and patients cannot access the scheme if they could become part of a clinical trial of the same medicine. The scheme is restricted to hospital specialists, and companies must agree to submit an application for a full licence, usually within a year, of the temporary authorisation being granted.

Experience shows that companies usually set high prices, but if the price fixed after market authorisation is lower they can be asked to refund the difference.

What about surveillance of side effects?

Applications for faster access would have to include plans for drug surveillance. "Collecting safety data is essential to protecting patients receiving the medicine," the working group concluded and is also an important way of developing a better understanding of the medicine. But the demands should not be so burdensome as to discourage companies from applying; nor should data gathering be seen as a clinical trial.

What's next?

The Medicines and Healthcare Products Regulatory Agency plans to launch a consultation on the scheme at the end of March. It will allow 12 weeks for responses.

  Read More & Comment...

A Clinical Trial wrapped in a mystery, inside an enigma.

03/15/2012 09:41 AM |

You mean central planning doesn’t work?
 
Russia: Russian Companies Receive Lowest Number of Permits for Clinical Research in Seven Years 
 
Russian companies have received the lowest number of permits for clinical research in seven years, according to a report from Remedium. In 2011, the Ministry of Public Health issued 571 permits for clinical studies, of which 369 were for multicenter studies (an increase of 49% from 2010), and 117 for local studies (a decrease of 27% from 2010).
 
The share of research by Russian sponsors was below average for the last seven years, with domestic producers receiving 14.1% of permissions for drugs research testing—against the seven-year average of 20.6%. According to the report, the number of local studies has fallen following the 1 September 2010 "law on treatment of drugs", which specified that local studies be included in the registration of medicines, meaning that Russian companies must apply for a drug's registration and pay a state fee before testing. There is no such requirement for foreign companies in Russia.
 
The Federal Antimonopoly Service (FAS) is currently preparing an amendment to the law proposing that the requirement to register be cancelled. They are also proposing to cancel the obligation for foreign companies in Russia to conduct local research if not including the country in a multi-center trial.
 
Significance: Russia's healthcare and pharmaceutical industry is currently going through regulatory changes that aim to simplify the registration process and speed market entry for drugs, among other goals. The report will come as a setback to Russia's efforts to expand the pharmaceutical industry and stimulate domestic manufacturers. The changes proposed by the FAS are likely to form a part of the regulatory reforms that are currently being formulated.

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PCORI Spends $9 million and 18 months to define what's in the organization's name

03/14/2012 11:25 AM |

It's the Patient-Centered Outcomes Research Institute.    It will collect $3.5 billion of our insurance premium payments over ten years -- with 20 percent going straight to the Agency for Healthcare Research And Quality -- a federal agency that can now bypass congressional oversight thanks to the pass through. 

PCORI has devoted 9 million taxpayer dollars and nearly two years focusing exclusively on figuring out what patient-centered outcomes research is.   That's like the Department of Homeland Security or Defense Department doing nothing but trying to define what "security" and "defense" is.  

Here's the full defintion of patient-centered outcomes research:

Patient-Centered Outcomes Research Working Definition


Patient-Centered Outcomes Research (PCOR) helps people and their caregivers communicate
and make informed health care decisions, allowing their voices to be heard in assessing the
value of health care options. This research answers patient-centered questions such as:

1. “Given my personal characteristics, conditions and preferences, what should I expect
will happen to me?”
2. “What are my options and what are the potential benefits and harms of those options?”
3. “What can I do to improve the outcomes that are most important to me?”
4. “How can clinicians and the care delivery systems they work in help me make the best
decisions about my health and healthcare?”

(Note that for all the hot air about personalized medicine, there is no specific mention of genetic characteristics)

By way of comparison, it took less time to draft and ratify the Constitution of the United States.  The excuse given for this over-cost and over-due delivery of a definition that leaves everyone more confused than ever is the following: " the difficulty in managing large numbers of healthcare providers with busy schedules and divergent interests."   You see, these board members are much, much busier than Benjamin Franklin, John Adams, and Thomas Jefferson ever were. 
And patient centered outcomes research is supposed to timely and scientifically up to date?  If it took all this time just to agree on a definition,  what can we expect about the effort to develop a research agenda that meets the definition?

As I have noted before, it will take less time to commercialize whole genome sequencing systems that produce same day results for clinical use then it will for PCORI, AHRQ, etc to decide what to study and to pay the groups who control the PCORI and AHRQ apparatus to do the research for the very same organizations.     Did I forget to mention the incredible amount of cronyism involved in the entire CER process?   But more on that in a future post.

A couple oversight hearings on what PCORI is really doing, how it's being done and whether it should be abolished would be beneficial.  I mean, what's a couple more meetings and two days on the Hill when you spend two years defining what your organization should do.   And I bet all those busy providers could clear their calendars pretty quick if called to testify.   Read More & Comment...

PDUFA Predictions Department

03/14/2012 09:03 AM |

Last November, the Center for Medicine in the Public held a confab entitled, “PDUFA without the Politics.” Many of the predictions from the event’s expert panelists seem to be coming true.

AS PREDICTED, the current conversation surrounding the reauthorization of the Prescription Drug User Fee Act (PDUFA) must focus on (among other things) the First Principle of Predictability as well as ensuring that the FDA can fulfill its role as an important ally in advancing healthcare in America.

The draft bill extends FDA's mission to include: advancing medical innovation; promoting economic growth and job creation; promoting predictability and allowing flexibility; identifying and using the most innovative and least burdensome tools for achieving regulatory ends; and incorporating a "patient-focused benefit-risk framework that accounts for varying degrees of risk tolerance, including for people living with a life-impacting chronic disease or disability."

AS PREDICTED, the House Republican discussion draft incorporates the Generating Antibiotic Incentives Now (GAIN) Act, which would extend market exclusivity by five years for drugs that treat antibiotic-resistant pathogens

AS PREDICTED, by Representative Michael Burgess at the CMPI conference, the bill also completely removes limits on issuance of conflict-of-interest waivers for FDA advisory committee participation that were enacted as part of the FDA Amendments Act.

The discussion draft also includes language intended to expand accelerated approval. The accelerated approval provisions are similar to those in the FAST and TREAT acts.

Is there still room for a few more Christmas Tree ornaments?  Perhaps permanent renewal of the Best Pharmaceuticals for Children’s Act and PREA?

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Health Exchange Regs Leave Consumers Short Money, Care and Doctors

03/13/2012 12:54 PM |

Where else but in Washington would the release of 600 pages of regulations be hailed as promoting greater choice, efficiency and reduce cost?

In this regard, the final rule regulating the establishment of "Affordable Health Exchanges" does not disappoint.

Most of the regs deal with how to qualify people living on Indian reservations and setting up systems to determine eligibility and award tax credits to the 9 percent of Americans who might actually get them.   But there are some interesting discussions that crop up which might surprise those who regard exchanges as another step towards utopia:

1.    If your income changes after you buy a health plan with some tax credits,  your costs will go up or down without warning.   And you may have the IRS knocking at your door asking for repayment of the tax credits you planned on having but no longer do as your income changes. 

2.    If your income changes so does your cost sharing.  Even a few bucks a month can make a huge difference.  Hence: A five percent decrease in income
from $47,018 is $44,667 (190 percent of the FPL), would correspond to a total premium, after advance payments, of $217 per month, for a total difference in premium of around $360 per year.  And there's more "... based on the same figures cited above, the difference in cost-sharing between a family at 190
percent FPL and a family at 200 percent FPL is $1,000 per year, due to the change in eligibility for cost-sharing reductions at 200 percent FPL. The difference is $2,000 around 250 percent FPL, which is the upper limit for cost-sharing reductions based solely on household income.

So if your pre-tax income goes up by $2351 you wind up paying an additional $1360.  That's an effective marginal tax rate of 68 percent.

3.   And since such changes do not keep up with tax information, exchanges will be required to create a system to monitor what we make in other ways.  To wit: "the Exchange must use information other than tax data to verify income in cases in which an applicant attests that a change has occurred or is reasonably expected to occur..."   What might that information be?  Bank accounts?  Pay stubs from employers?  Undercover informants?  

4.   If you or I decide to stop paying premiums for three months.  Under Obamacare, we still are able to receive health care services.  Coverage is terminated retroactively after three months and health plans don't have to pay doctors and other professionals for services rendered.   In otherwords, health providers are on the hook for the cost of providing free care if we stop paying premiums.  Or as the regs state: Qualified health plan (QHP)  issuers.. must notify providers who submit claims for services rendered during the second and third months of the grace period that any such claims will be pended, and potentially not reimbursed by the QHP issuer if the individual does not settle outstanding premium payments."

5.  There is other mind-numbing stupidity too.   HHS still hasn't figured out how to get accurate information about income and family size without tax information.  And it will take the word of people that they are poor enough to get tax credits without checking first. 

I am not making any of this up.   I wish I were.
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Mainstream Media Misreads Research on Personalized Medicine

03/12/2012 11:16 AM |

Last week there was doom and gloom about the potential and pace of personalized medicine:  

'Personalized Medicine' Hits a Bump
Wall Street Journal‎ -

    Tumor Evolution More Complex than Imagined
    MedPage Today‎

   Personalized cancer treatment: Genetic differences abound in tumors   Los Angeles Times‎


The source of this pessimism?  A study in the NEJM: Intratumor Heterogeneity and Branched Evolution Revealed by Multiregion Sequencing

http://www.nejm.org/doi/full/10.1056/NEJMoa1113205?query=featured_home

Here's Ron Winslow of the WSJ passing sentence on why the study is a blog to targeting treatments:

"Tumor's genetic makeup can vary significantly even within the same tumor sample, researchers said, a finding that poses new challenges to the personalized-medicine movement in cancer.

One big implication of the new research, being published Thursday in the New England Journal of Medicine, is that analyzing only a single sample of a patient's tumor—the current practice—may miss important genetic mutations that affect the course of the disease.

That, in turn, could hinder emerging efforts to match patients with drugs that target the mutations affecting their tumors, a basic strategy of personalized medicine.

The findings don't diminish enthusiasm for the idea that genetic knowledge about tumors can transform cancer care, the researchers said. But it could make personalized treatment more complex—and more costly."


Winslow and others -- following the bias of the researchers who did the study --  overstate the novelty of finding differences in tumor progression because of differences in mutations identified by exome sequencing and appear to suggest that such subtle changes always mislead.

First,  exome sequencing is becoming commonplace, as is deep sequencing of the type provided by Life Technologies and Oxford Nanopore Technologies.  The costs and speed of such analysis is dropping rapidly.  So cost is not a factor.   Second,  such sequencing will become commonplace precisely because clinicians know that every cancer progresses differently.    Mutations are not always associated with changes in tumor progression.  Sometimes they are, sometimes they are not. 

Winslow and others present personalized medicine as a single gene-single step process of individualizing treatment.  No one who knows anything about the clinical and functional application of sequencing believes that.  Hence, the media -- where many 'thought leaders' still get the primary source material on health and science -- perpetuates both an outdated view of personalized medicine and artificially overstates the costs and barriers of the same. 

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