PDUFA Cheat Sheet

06/12/2012 07:32 AM |

To support congressional work on user fee authorizing legislation and other FDA-related drug, device, and biological product provisions, Congressional Research Service analysts have prepared tables comparing provisions in the Senate passed bill that originated in the Senate Committee on Health, Education, Labor, and Pensions and the House suspension document that originated in the House Committee on Energy and Commerce.

• S. 3187, the Food and Drug Administration Safety and Innovation Act, passed by the Senate on May 24, 2012; and

• H.R. 5651, the Food and Drug Administration Reform Act of 2012, revised and dated May 24, 2012, as posted on the websites of the House Committee on Energy and Commerce and the House Committee on Rules.1

CRS has also provided brief summaries of relevant provisions in current law, mostly the Federal Food, Drug, and Cosmetic Act (FFDCA), but also the Public Health Service Act (PHSA), the Controlled Substances Act (CSA), and several others as noted.

The complete report can be found here.

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Dal Pan-acea?

06/12/2012 07:30 AM |

CDER Staff:

It gives me great pleasure to announce the appointment of Gerald Dal Pan, M.D., M.H.S., as the permanent director of the newly reorganized Office of Surveillance and Epidemiology (OSE). CDER conducted a nationwide search for this position, and I am happy to say that the best candidate was right here in CDER.

OSE plays a critical role in monitoring and safeguarding drugs once they are on the market. As OSE director since 2005 and then, more recently, as acting director of the reorganized OSE (super office) since June 2011, Gerald has been instrumental in conceptualizing, standing up, and managing CDER’s postmarket drug safety and risk assessment programs. Throughout this time, he has provided strong leadership and direction to his staff and to the Center on a broad range of drug safety, epidemiological, risk management, and information technology activities.

Gerald has extensive knowledge of the scientific basis, regulatory laws, and best practice guidelines used to evaluate the essential components of postmarket drug safety: pharmacovigilance, pharmacoepidemiology, risk management, and medication error prevention. He has implemented numerous projects that have proven critical to advancing our postmarket drug safety programs. Under Gerald’s leadership, OSE has grown from a staff of approximately 116 to 250 over the last five years.

He has been a dedicated leader on CDER’s Safety First Initiative--working effectively across lines within the Center to build collegial and team-based work groups. He has also forged new relationships with other federal health agencies and the international community—all of which are helping to advance our oversight of marketed drugs. Such efforts strengthen our credibility and transparency, and the public’s trust in our ability to protect them from harm.

Gerald first joined FDA in July 2000 as a medical officer in the Division of Anesthetic, Critical Care, and Addiction Drug Products. He became director of OSE (then known as the Office of Drug Safety) in November 2005. He holds a medical degree from Columbia University and a Master’s degree in clinical epidemiology from Johns Hopkins University. He is board certified in internal medicine and neurology.

Gerald has been a trusted and valued advisor to me due to his medical and scientific background, his ability to tackle complex regulatory issues, and his grasp of the expectations of the public and policymakers. He has done an excellent job in his role as acting director of OSE, especially as the depth and volume of work in OSE have continued to increase and the pace has quickened. I would like to extend my sincere appreciation for all that Gerald has accomplished, and for his dedication to our public health mission.

Please join me in congratulating Gerald on his permanent position and wishing him continued success in this role.

Janet Woodcock

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The Pill with the Dragon Tattoo

06/11/2012 07:55 AM |

China has overhauled its intellectual property laws to allow Beijing to issue compulsory licenses to eligible companies to produce generic versions of patented drugs during state emergencies, or unusual circumstances, or in the interests of the public.

For "reasons of public health", eligible drug makers can also ask to export these medicines to other countries, including members of the WTO.

According to Reuters, “All eyes are now trained on how China battles it out with big foreign drug exporters, especially from 2013 when the Geneva-based Global Fund to Fight AIDS, Tuberculosis and Malaria will no longer give grants to China to fight HIV.”

It seems that IP doesn't permeate the Great Wall.  It's time for the Middle Kingdom to seek some middle ground.
 

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BIO Breakout: Drug Shortages

06/11/2012 07:39 AM |

If you’re attending the upcoming BIO convention, please join an important and timely conversation on the past, present and future of drug shortages.

Drug Shortages:  How will the United States Ensure Patient Care is not Compromised will take place on June 18^th from 2-3:30pm in Room 258B.

I will be joined by Jouhayna Saliba of the FDA, Nancy Davenport Ennis, CEO of the Patient Advocate Foundation, Louis DeGennaro of the Leukemia & Lymphoma Society, as well as representatives from industry to discuss and debate the regulatory, legislative, economic and provider aspects of this troubling issue.

Hope to see you there.

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Cheaper is Better 'Study' Of Abraxane Attacked At ASCO

06/08/2012 10:26 AM |

As I noted in an earlier blog,  a study claiming paclixtel was cheaper and better than Abraxane  -  a study paid for by The National Cancer Institute -- was biased and flawed.    Here's an article reporting on the pushback.  Nothing new for comparative effectiveness research where the gold standard is torturing data to make it cry cheaper is better..   

Eyebrows raised at ASCO over Abraxane vs paclitaxel study

Many observers have been puzzled by the presentation at the American Society of Clinical Oncology meeting in Chicago of a study which claimed that two new breast cancer drugs - Celgene Corp’s Abraxane and Bristol-Myers Squibb’s Ixempra - were no better than paclitaxel.

The Phase III study enrolled 799 patients with locally advanced or metastatic breast cancer who were randomised to receive one of the three therapies - paclitaxel (the standard of care), Abraxane (nanoparticle albumin bound -'nab' - paclitaxel) or Ixempra (ixabepilone) - on a weekly basis with each cycle consisting of three weeks of treatment followed by a one-week break. Some 98% of patients also received Roche's Avastin (bevacizumab), which had its approval for breast cancer revoked by the US Food and Drug Administration in November 2011.

The data from the study, presented at ASCO by lead investigator Hope Rugo at the University of California, San Francisco, stated that median progression-free survival was 10.6 months for those receiving paclitaxel, 9.2 months for nab-paclitaxel, and 7.6 months for ixabepilone.

Grade 3 or 4 non-haematologic toxicities were also lowest in the paclitaxel arm, including sensory neuropathy (16% versus 25% for both experimental arms), while haematologic toxicities were lowest in the ixabepilone arm, and highest for Abraxane (12% versus 51%), compared to paclitaxel (21%). Dr Rugo said that the study "demonstrates that we should not simply assume that newer drugs are always better than the standard therapies".

150mg dose used in study never used in practice

However the findings, particularly in the Abraxane arm, caused raised eyebrows among oncologists at the meeting. The dose of the Celgene drug used was 150mg, well above the 100mg for which Abraxane is approved in over 40 or so countries, and when asked by PharmaTimes World News as to why the higher dose was selected, Dr Rugo noted that it had been used in a Phase II trial by the company.

However, Brian Gill, head of global corporate communications at Celgene, noted that there is only one trial to date "that is a true head-to-head study between Abraxane and paclitaxel". Those results showed statistical significance in time to disease-progression, PFS and overall survival. He added that "it was very curious to see why the decision was made to use the 150mg rather than the successful dose on the label" and expressed his surprise over the use of Avastin.

While Avastin is "a wonderful drug," lots of peer-reviewed publications note that the Roche drug, used in combination with other therapies, exacerbates toxicities, he told PharmaTimes World News. Between using the higher dose with Avastin, it appears that about 50% of patients actually discontinued the therapy in the latest study.

William Sikov of the Brown University School of Medicine, said he had no problems about the conduct of the study but questioned its design in terms of the 150mg dose, which would have led to missed and delayed doses in that arm. He was surprised by the broad statement that patients can do equally well on paclitaxel than Abraxane, a stance he felt was "contradictory" given that the latter is highly effective when used at the right dose.

When asked by PharmaTimes World News whether the 150mg dose was ever used in clinical practice, Dr Sikov gave a clear 'no', although occasionally 125mg is used. He noted that the 150mg used in the Phase II study had appeared to be the most active, although toxic as well, "but it is very different going from a 75-patient Phase II study to a 200-plus patient trial".

Other breast cancer specialists that spoke to this publication were also surprised that ASCO had chosen to highlight this study, with one suggesting that while making a case for the use of cheap generics is a valid one, in the case of Abraxane versus pacitaxel, the argument falls down.

Celgene has recently stated that peak sales of Abraxane, which is also being studied in lung and pancreatic cancer, as well as melanoma, could be in the region of $1.5 billion, with as much as a quarter of that coming from pancreatic cancer.




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No Incremental Innovation Please. We’re British.

06/08/2012 02:09 AM |

BioCentury reports that Stephen Whitehead, CEO of the Association of the British Pharmaceutical Industry, said Wednesday that the U.K. government is too focused on encouraging "breakthrough drugs" at the expense of incremental innovation. In a statement accompanying a report on examples of incremental innovation, Whitehead said the government "wants to target resources at big breakthroughs, but the science shows us that developments in medicine are made in small steps."
 
The report and Whitehead's comments come ahead of discussions about implementing the U.K. government's proposed value-based pricing system, which is slated to come into effect at the start of 2014.

He’s right. 

When it comes to innovation in health-care technologies, there are some tough but important basic principles:
 
Innovation is slow. As any medical scientist will tell you, there are few "Eureka!" moments in health research. Progress comes step by step, one incremental innovation at a time. Biopharmaceutical companies more often profit by improving existing molecules and making processes more efficient than by revolutionizing the whole field with new "miracle" products.
 
Innovation is difficult. Today, it takes about 10,000 new molecules to produce one FDA-approved medicine. And if that's not frightening enough, only three in 10 new medicines earn back their research and development costs. And here's the kicker: Unlike other R&D-intensive industries, biopharmaceutical investments generally must be sustained for more than two decades before the few that make it can generate a profit.
 
Innovation is expensive. In 2003, researchers at the Tufts Center for the Study of Drug Development estimated the costs to bring a new medicine to market at $802 million. Others suggest that the total cost is closer to $1.7 billion. And that number is on the rise.
 
Innovation is under attack. From accusations of the "me-too" variety, to crackpot schemes to replace pharmaceutical patents with a "prize" system, life for innovator pharmaceutical companies is rough and tough. The late Israel Makov, founder of the generics giant Teva, once said that he wasn't really in the pharmaceutical business, but rather "the litigation business."
 
But innovation is important -- and not just for biopharmaceutical industry profits. Increases in life expectancy resulting from better treatment of cardiovascular disease from 1970 to 1990 have been conservatively estimated at bringing benefits worth more than $500 billion annually. In 1974, cardiovascular disease was the cause of 39 percent of all deaths. Today, it's responsible for about 25 percent.
 
Cerebrovascular diseases were responsible for 11 percent of deaths back then. In 2004, they caused 6.3 percent of deaths. Kidney diseases were linked to 10.4 percent of deaths then, and now they're associated with 1.8 percent. And that's just in the United States.
 
And, with all due respect to the pharmaceutical industry, innovation mustn't be only about medicines. We have to embrace innovative technologies for medical records and prescribing medications. We need innovative clinical trial designs and molecular diagnostics so that we can develop better, more personalized medicines faster and for far less than the current $1 billion-plus delivery charge.
 
So we'd better start taking innovation -- of both the incremental and discontinuous varieties -- seriously. And that means spending more on more complex developmental R&D (with concomitant higher investment risks).
 
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Isn't Knowledge Power?

06/07/2012 07:01 AM |

Shame on the National Coalition on Health Care.  The NCHC is foolishly calling on the FDA not to insist that biosimilars carry different names than their innovator precursors. Joining the NCHC are the AARP, AFL-CIO, the American Federation of Federal, State, County and Municipal Employees, Blue Cross and Blue Shield Association, California Public Employees Retirement System, and Service Employees International Union.

Why do these well-intentioned groups (including the Blues and the AARP – two of our nation’s biggest payer organizations) feel the urgency to remove an important level of safety and … knowledge for physicians (who write the prescription), pharmacists (who fill them), and patients (who ingest them)? Could it be a question of transitory cost savings over patient care? Just asking.

(In Europe, many healthcare systems are developing HTA standards and practices to ensure that product quality and therapeutic value are not inappropriately influenced by short-term cost considerations. We should learn from their experience.)

And, after all, regardless of your position on narrow therapeutic index, biosimilarity and interchangability (not to mention pharmacovigilance) – isn’t knowledge power?

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When Less is not More

06/06/2012 07:04 AM |

Today, a House appropriations subcommittee will mark up a bill that would give FDA $44 million less to spend in Fiscal 2013 than a measure produced by the Senate Appropriations Committee. Look to a conference committee to be the final arbiter of agency funding -- again.

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Health Innovation Solyndra Style

06/05/2012 05:51 PM |

From the Wall Street Journal 
Inside ObamaCare's Grant-Making

George Washington University is getting $1.9 million for a project to reduce health costs by $1.7 million.

By STEVEN E. GREER

Early this year, I was briefly involved with one of the Affordable Care Act's bureaucracies called the Center for Medicare and Medicaid Innovation, or CMMI. Despite its lofty ideals, it is one more pork program and venue for political cronyism, as I learned firsthand.

The innovation center is supposed to test better ways to deliver and pay for health care than the current fee-for-service system, and the outfit will spend $10 billion over the next decade on awards, grants and contracts. In a recent letter to Congress defending its work, the center touted its "structured clearing process" and "rigorous evaluation of models." But I found there are few safeguards and little transparency in practice.

This January, I was invited to review grant applications for something called the Health Care Innovation Challenge. Local health systems, state Medicaid programs and the like could apply for awards ranging from $1 million to $30 million, with priority for "projects that rapidly hire, train and deploy new types of health care workers." I was selected to become one of the chairmen overseeing the volunteer panels of outside experts. Our grant application reviews were supposed to help the CMMI in making the final award decisions. There were more than 3,000 grant applications in total.

Having written numerous other federal grant applications as a medical researcher, I was surprised by the very short time allotted to review 12 applications, each of which ran more than 100 pages. We had only two weeks to assemble a team and grade the applications on such criteria as the promise of the project design and its workforce goals.

Applications to the government's National Institutes of Health or the Patient-Centered Outcomes Research Institute, by contrast, undergo months of thoughtful review by scientists who are well-regarded in their fields. I began to wonder how much CMMI was interested in high-quality input from the grant reviewers.

To make matters more challenging, the computer system that handled our grant-scoring input was malfunctioning and deleting the painstaking comments and scores that our reviewers were inputting. When I reported the problems to the outside IT contractor, a support technician accused me and my team of never writing the reviews in the first place. All but one of my reviewers quit due to the digital hassle and the time-consuming workload.

That is when I sent a memo to CMMI Director Richard Gilfillan and Health and Human Services Secretary Kathleen Sebelius. I said that the rushed process was simply collecting "junk in, junk out" reviews. When I received no response, I left the CMMI program as well. Based on my experience, it seems unlikely that the 3,000 applications were closely vetted.

In May, the innovation center announced the first batch of grant recipients, 26 in all. George Washington University earned $1,939,127 because it expected to reduce health costs by a mere $1.7 million. Similarly, the Center for Health Care Services in San Antonio received $4,557,969 to save $5 million. At least CMMI didn't pick one of the thinly veiled handout requests that I personally oversaw, such as a for-profit company that claimed to offer holistic healing through human touch.

Of particular note was a $5,862,027 grant to the University of Chicago medical center to "train and create new jobs for an estimated 90 individuals from this high-poverty, diverse community." The program is part of the school's Urban Health Initiative. Perhaps creating this is a noble use of federal funds, but job creation seems far afield from the CMMI mission to innovate.

 

Dr. Donald Berwick was Administrator of the Centers for Medicare and Medicaid Services from July 2010 to December 2011. CMMI, which was established during his tenure, started another program called the Partnership for Patients. That $500 million initiative is supposed to reduce hospital-acquired conditions and hospital readmissions.

In December 2011, the Partnership for Patients awarded a contract to the Health Research and Education Trust, which in turn awarded a subcontract to the Boston-based Institute for Healthcare Improvement—which Dr. Berwick ran for 19 years before he moved to Medicare. A source involved with Partnership for Patients told me about the relationship.

I emailed Dr. Berwick in May to confirm the subcontracts between the institute and the trust. "I don't think there are contracts between them, but they're good friends," he replied. He was careful to note that he is no longer the institute's CEO, though he now works out of the institute's Boston offices as an adviser. The Health Research and Education Trust and the Institute for Healthcare Improvement have not responded to requests for information about the subcontract.

Even if the innovation center's grants were chosen via a squeaky-clean peer-review system and awarded solely on merit, they would still be a waste of taxpayer money because Medicare should not be straying from its core competencies into areas like job creation. Congress ought to dismantle and defund the program if CMMI survives the Supreme Court ruling on the Affordable Care Act.

Dr. Greer, a surgeon, is CEO of the Healthcare Channel (thehcc.tv) and a financial analyst.

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PCORI's Subtle Rationing Bias

06/05/2012 02:02 PM |

I have read the PCORI call for grant proposals.  On the surface it all sounds so...patient-centered.  PCORI encourages the social scientists who will receive billions in grant money (assuming Obamacare is not overturned) to ask the following questions:

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?

How can you argue with that?  The problem is, PCORI in investing heavily in the dissemination of research and the use of methods that are NOT patient-centered.   PCORI relies upon the US Preventive Services Task Force as the foundational source of CER to determine what screening tools to use. 

USPSTF decisions are based on one-size fits all data and demands long term studies that show a screening tool directly reduces all-cause mortality   As I have noted before, that means nothing can be proven to be preventive.

Moreover,  there is no mention in any PCORI publications or grant propsoals about genomics or  personalized medicine.  Zero.  Check that, one mention in the context of questioning the value of personalized medicine. 

Third, (and this my pet peeve) the bias toward chiropractors is rife in research proposals.  PCORI implies that chiropractors -- who as a profession oppose vaccinations -- are a cheaper approach to orthopedic problems even though once you go to chiropractor you are expected to keep going and buy all their herbs, vitamins, etc.  Could it be that the sop to an industry that combines auto mechanics, biology, New Age babble and anti-vaccination bias is a result of having a chiropractor on the PCORI board has anything to do with it.  And by the way, will PCORI issue a statement defending vaccines? 

"One of her neighbors is a chiropractor who treated two of her coworkers successfully for on-the-job back injuries. The chiropractor recommended that
she avoid narcotic medications, stay active and try a course of spinal manipulation for a few weeks before considering an MRI or surgery.  An acupuncturist whom the worker has consulted for temporary relief of neck pain in the past also recommended a series of treatments. "

And this evidence based statement:  

I have a patient who has a knee problem
and, by chance, went to a chiropractor
and was there for four sessions; and
now she is normal. She was in need of
knee surgery, and now she isn’t.
—Cardiologist

Fourth,  the questions posed cannot be answered by PCORI or any one else.  

Fifth, PCORI cannot keep up with the flood of individualized treatments for diseases such as cancer, multiple scleroris, HIV,  hypertension and admits it:

"The rapid pace of introduction of new diagnostics and the diversity of new technologies raise questions about the role and added benefit of these new options for guiding clinical decisions and changing patient outcomes include the role of molecular diagnostics in managing the care of patients with cancer. Some new diagnostic technologies have potential uses for a wide range of conditions, but the evidence base demonstrating benefit for these new indications fails to keep pace with use. For example, there continues to be a desire for evidence to support the use of PET imaging, MRI, and CT for a number of conditions, including oncology and lower back pain. Finally, questions sometimes remain about the long-term safety of well-established modalities, even some modalities perceived traditionally as having relatively little risk, such as dental x-rays. "

Does PCORI really think it or any entity develop an 'evidence base' a la USPTF that will "keep pace with use?"   By design and definition then, CER will delay and discourage innovation, no matter how patient-centered PCORI claims to be.   
t will by definition and design not only hold up access to these treatments by requiring CER before they are adopted, the studies -- which cannot capture the diversity of experiences and responses it claims it will measure -- will be outdated.  In any even PCORI presumes that CER must precede use of new technologies before they are used or paid for.

Finally,  the so-called patient-centered processes it claims to be developing are designed to discourage use of new treatments.  "Shared decisionmaking" has been show to be biased against intervention by  presenting patients the bad news about a treatment -- rare and dramatic risks -- before 'sharing' information about the benefits.     

Here's an example of how PCORI frames patient-centered decisionmaking:

It used to be accepted that we screen everyone
for prostate cancer who is a candidate. Now we
don't know what to do.
—Primary care physician

As far as exercise goes, is there something nonmedical like yoga or breathing exercise, or
should [my son] be doing anything? Is there
anything he can do other than take medicine, or
will he be on medicine for the rest of his life?
—Parent of pediatric patient

The theme is less should be more, cheaper is better in most cases.  Or at least innovations should be considered guilty before being found -- after years of CER research -- proven effective.   Of course PCORI is not measuring the impact of CER on the pace of innovation and how it will affect life expectancy and well-being compared to accelerating personalized medicine.

We have and will continue to do so even though it seems 'stakeholders' have been frightened into not challenging PCORI on that score.  Instead of investing in research on the value of their innovations, they are not only playing ball with PCORI,  they are buying the equipment and ball field to sustain the stupidity. 


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Dear Doctor

06/05/2012 08:01 AM |

U.S. drug recalls common, not well publicized: study

By Andrew M. Seaman

NEW YORK (Reuters Health) - The U.S. Food and Drug Administration (FDA) recalls potentially harmful drugs about once every month, but they could be doing a better job of letting doctors and patients know about them, says a new study.

Over an eight-year span, researchers found that the FDA failed to send notifications for one in five of the most serious recalls through its two electronic systems used to alert doctors and the public.

The so called Class I recalls, according to the FDA, are issued for drugs that, if taken, have the potential to cause "serious adverse health consequences or death."

The 2008 contamination of the blood thinner heparin, one of the most notable and recent examples of a Class I recall, resulted in serious reactions and some deaths in dialysis patients. That recall, according to one of the study's authors, was reported.

"I think a good system would indicate all of the Class I recalls, and it wouldn't necessarily communicate recalls the FDA deems less important, such as Class II and Class III," said Joshua Gagne, from Brigham and Women's Hospital in Boston.

Between 2004 and 2011, Gagne and his fellow researchers counted more than 1,700 drug recalls listed in the FDA's enforcement reports. Of those, 91 were serious Class I recalls.

During that time, the FDA issued about 2,900 announcements through the Recall Alert System, which sends notifications to subscribers about recalled drugs and products. The system, however, only sent alerts for 55 of the 91 Class I recalls.

MedWatch, another system used by the FDA to report drug recall information, sent alerts for 18 of the remaining recalls. Another 18 -- or one-fifth -- of the Class I recalls were never reported through either system.

"Despite recent efforts by the FDA to address the drug recall burden, health care providers may be inadequately informed about clinically important recalls that threaten patient safety," wrote the authors in a letter to the Archives of Internal Medicine on Monday.

An FDA spokesperson told Reuters Health there are a number of ways the agency communicates with doctors, but she wasn't able to say why notifications weren't sent in these 18 cases.

ACTIVE TRACKING

There's no way, according to Gagne, to know if the lack of notifications were linked to any patient harm.

He added that the FDA has moved to address communication problems, including recall notifications.

"It's certainly on their radar and they're constantly starting initiatives to address it, but there is still room for improvement," he said.

Dr. Lisa Schwartz, co-director of the Center for Medicine and Media at the Dartmouth Institute in Lebanon, New Hampshire, told Reuters Health that the FDA should try hard to let everyone know about Class I drug recalls until there is a system that allows the agency to track the recalled drugs.

"It's unclear why it shouldn't be 100 percent of the time they're notifying people on both systems," said Schwartz, who was not involved in the new study.

Gagne and his fellow researchers echoed Schwartz's call for an active tracking system.

"The tracking could all be computerized so pharmacies can know which bottles the recalled products came from," said Gagne.

Until then, he told Reuters Health that there are other ways doctors can find out about recalls. One way is for the drug companies to send letters directly to doctors. His team, however, was not able to look into those methods of notification.

But ultimately, Gagne said, the FDA needs "a more specific system that only communicates all of the Class I information."

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Да Bomb

06/05/2012 07:29 AM |

Da bomb

Last week I testified in front of the Russian Duma’s committee on non-communicable diseases (NCDs). That committee is preparing (at President Putin’s direction) a series of recommendations to address the many significant health issues that NCDs pose to the Russian Federation (with smoking at the top of the list).

Here are my remarks (as presented):

NCDs (such cancer, diabetes, Alzheimer’s Disease, chronic lung and heart disease) are the leading causes of death and disease worldwide, killing more than 36 million people in 2008, and are projected to increase by 15% globally between 2010 and 2020 due in large part to progress made in combating infectious diseases through economic growth, development, and better treatment options.

But this frightening increase it is also largely linked to lifestyle choices.

For most infectious diseases, rapid access to diagnosis and treatment is an imperative for patient survival.

But for NCDs the “lifecycle” is different, and there may be many actions that can take place, such as prevention, before medicines or other treatments need to be prescribed. A major epidemiological shift is underway.

The bad news is that NCDs sit at the center of a complex series of social and economic factors that it is hard to influence from the top down.

The good news is that there are many inexpensive and proven preventative medical interventions that can significantly reduce the burden of NCDs.

A neglected area of discussion within the NCD debate is medical research and development, which has the very real promise of introducing cures for currently incurable chronic conditions, as well as treatments for diseases where there is currently a lack of pharmaceutical options.

Just as innovation in HIV/AIDS treatment has turned it from a terminal disease into a manageable chronic condition, it is not entirely unfeasible that future innovation will throw up a cure or prophylactic vaccine for many diseases currently considered as “chronic.”

The current trend of increase in Alzheimer’s, for example, threatens to overwhelm health systems in OECD countries because not much can be done beyond labor-intensive care. If a cure were invented, this huge economic burden would be significantly reduced as would much pain and suffering.

Remember that tuberculosis used to be an incurable major chronic disease until the invention of treatments. Innovation is crucial – but it is not the only tool available to us in the war against NCDs.

Fortunately for NCDs, there are plenty of off-patent and widely available preventative medicines (such as statins and medicines for diabetes and hypertension) that can dramatically reduce the overall disease burden

The good news is that half of deaths caused each year by NCDs are preventable and lifestyle choices, such as unhealthy diet and tobacco use, are part of the explanation. Reducing disability or deaths through increased investment in prevention programs will contribute to higher economic growth and allow limited resources to be focused efficiently on patients most in need.

Money is tight everywhere. Before governments agree to increased spending on top-down schemes, it is worth taking a look back at the last major global effort to fight disease: HIV/AIDS.

Some lessons are clear, like the need to focus on prevention. The UN’s big mistake with HIV/AIDS was to prioritize treatment. This neglect of prevention led to new infections piling up more quickly than they could be treated, creating unnecessary suffering and costs. Actions on access and affordability of NCD treatments include promoting the right policy, along with smart regulatory and supply chain environments that secure optimal quality of care for patients.

But in order to accomplish this, there must be a confluence of interests.” And the good news is that there is.

There is no substitute for the power of partnership. It’s not about having pharmaceutical companies write checks, but rather combining the resources of private industry with the expertise of leading global health organizations, to identify new models of patient care that increases access and speed to treatment – and not just medicines.

Part of the role of government is to facilitate partnerships that propel progress. In the United States we’re learning that, in the battle against NCDs our health systems needs to get smarter, specifically: 

·      Our reimbursement systems need to be less focused on outputs and more on individual patient outcomes;

·      Health professional education should be more holistic addressing all issues concerning both acute and chronic conditions;

·      Our view of drug safety relative to what is Rx vs. OTC is going to have to change (such as OTC statins and HIV/AIDS preventative medicines);

·      Our delivery systems will have to dramatically reform to take advantage of new e-health technologies; 

·      The partnership between social services and health services will have to become closer (including smoking cessation and weight reduction strategies), and 

·      Many aspects of our healthcare system need to be decentralized as far as possible to the local level – thus making it easier to deal with complexity of multi-chronic conditions.

Most importantly, we must place personal responsibility at the center of the NCD debate – because it is the most effective way to ensure that early detection and prevention is maximized whenever and wherever possible.

I look forward to working with you in your efforts to create a US/Russian Healthcare Foundation that will help both of our countries and all of its citizens live longer, healthier and more productive lives.

Thank you.

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Obamacare To Healthy Indiana:Drop Dead

06/04/2012 11:21 PM |

The Healthy Indiana Program was established by Governor Mitch Daniels to provide affordable and comprehensive health insurance to lower income Hoosiers.  Using a combination of tobacco taxes and Medicaid dollars, it serves more than 42,000 Hoosiers.  The plan requires enrollees to contribute up to 5% of their gross income to a POWER account, which is used to pay for medical expenses. The state covers the balance needed to raise the account to $1,100.  People who use preventive services get a reduced premium and get to carry over a portion of the POWER account from one year to the next. The average premium is $130

Unfortunately, Governor Daniels had to freeze HIP enrollment in March 2010 because under Obamacare HIP enrollees will have to go into Medicaid.

Health and Human Services Secretary Kathleen Sebelius told an audience at the Indiana University School of Medicine on May 14, 2010 that the health reform law didn't outlaw Healthy Indiana. "There's no change that [health reform] made that would make this waiver program difficult to pursue in the future."

PS   The federal waiver authorizing Healthy Indiana expires at the end of 2012.

PPS  In November of 2011, Sebelius rejected Indiana’s request to keep Healthy Indiana  and exempt it from Obamacare.

About 25,000 adults without children have been on a waiting list for the program, while adults with kids are still being enrolled.

Unless Obamacare is overturned or dramatically reformed, they will still be waiting.  Their only choice will be Medicaid.  As the video on Healthy Indiana shows, for those who rely on HIP for coverage, that’s not a choice at all. 

http://www.youtube.com/watch?v=qoWothG4b2Y
 

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In Cancer 'Cheaper' Is Not Better, Just Less Available

06/04/2012 05:42 PM |

Too often media coverage of scientific studies are rife with what behavioral economist Daniel Kahenman calls "the availability heuristic."   The availability heuristic is a mental shortcut that uses the ease with which examples come to mind to make judgements about the probability of events. 

In reporting terms that translates into taking top line results and fitting into a pre-conception or narrative.

Such is the case with reporting on a study comparing the effectiveness of paclitaxel and bevacizumab with a nano-sized form of paclitaxel called Abraxane & Avastin as well as Xyempra and Avastin.

Here's the MedPage account of the study: 

"Patients with metastatic breast cancer did just as well -- and maybe better -- with paclitaxel-based chemotherapy compared with two newer, more expensive agents, results of a randomized trial showed.

First-line treatment with weekly paclitaxel and bevacizumab (Avastin) resulted in a median progression-free survival (PFS) of 10.6 months compared with 9.2 months with nanoparticle albumin-bound paclitaxel (nab-paclitaxel or Abraxane) and 7.6 months with ixabepilone (Ixempra), each paired with bevacizumab.

The ixabepilone arm had significantly worse PFS at an interim analysis and was dropped from the trial.

Patients who received paclitaxel also had less peripheral neuropathy than with either of the other drugs, and less hematologic toxicity than those who received nab-paclitaxel, Hope Rugo, MD, reported here at the American Society of Clinical Oncology meeting.

"Neither weekly nab-paclitaxel nor ixabepilone is superior to weekly paclitaxel," Rugo, of the University of California San Francisco, said during an ASCO press briefing. "Weekly paclitaxel appears to offer better progression-free survival than ixabepilone."

"These data suggest that similar patients could be appropriately treated with weekly paclitaxel," she added.

During the discussion that followed her presentation, Rugo described cost issues surrounding cancer therapies as "enormously complicated," but said that generic drugs such as paclitaxel will always be less expensive than newer drugs that are not available as generics.

Funded by the National Cancer Institute (NCI), the trial came about as a result of recent developments in the treatment of metastatic breast cancer."

Of course, the funding had nothing to do with comparative effectiveness research efforts to claim that cheaper is better.   But I digress

The study and the Medpage article is yet more evidence --  pouring in at the ASCO meeting as I write -- that targeted therapies or targeted use of therapies based on biomarkers and mechanisms of action implicated in both toxicity and tumor response -- is replacing the one size fits all approaches and comparisions that NCI supports. 

The fact that you have to get into the weeds about the study and have to put it into context of other studies looking at Abraxane and Xyempra underscores this point.

For instance:  Abraxane was designed to be longer lasting and administered at lower doses and less often.  

The study used 150 mg/m2 weekly and has a 30% higher weekly dose intensity than the approved dose of 260 mg/m2 every 3 weeks.  

Still Abraxane has been used in weekly fashion, to great effect: "in phase 2, median progression-free survival (PFS) was 18.9 months for 150 mg/m2 weekly nab-paclitaxel vs 8.5-13.8 months for all other regimens. In phase 3, median PFS for q3w nab-paclitaxel and solvent-based paclitaxel were 5.6 months and 3.5 months, respectively. Weekly nab-paclitaxel resulted in less serious adverse events compared with all other regimens.  
Breast. 2011 Oct;20(5):468-74.

The outlier in all Abraxane studies is the current one.  Why?  Abraxane is formulated as a monotherapy for metastatic breast cancer, not in combination with Avastin.  It's not just a stronger or smaller form of paclixtel.. it also targets a previously unrecognized tumor upregulated path through the blood vessel wall.

Nearly half of the people enrolled in the study dropped out due to toxicity issues.  So in effect, the investigators forced cancer patients to stay on a regimen that was toxic.. 

Yet, even using Abraxane in an off-label manner, patients who took the combo lived an average (again, a suspect number in cancer trials these days) of 9.6 more months compared to 10.4 months for paclixtel and Avastin.

Did the higher toxicity result from the combination of a higher than prescribed dose and using it with Avastin?  They researchers and the media account don't even ask that question.  Instead they hew to the availability heuristic:

 Rugo described cost issues surrounding cancer therapies as "enormously complicated," but said that generic drugs such as paclitaxel will always be less expensive than newer drugs that are not available as generics.

Well, yes they are.  For instance:

1.   One smaller dose of Abraxane costs slightly more than the same as four weeks of paclitaxel.  
2.   Several studies have shown that Abraxane use in women with MBC cuts down on the amount of time and services required to treat toxicity, to administer the drug and enhances quality of life.   For isntance: " Nab-paclitaxel had the lowest incidence of grade III/IV toxicity. This translated to lower overall costs for managing the grade III/IV events relative to both docetaxel and paclitaxel ($597 vs. $2626 vs. $1227). Using the median number of cycles administered and the cost impact of grade III/ IV toxicity, the overall cost for nab-paclitaxel would be $15,105 compared to $15,268 for docetaxel and $3557 for paclitaxel. When treatment preferences were assessed, 20 of 24 (83.3%) respondents selected nab-paclitaxel as their preferred choice compared to only 4 who selected docetaxel."    J Oncol Pharm Pract. 2009 Jun;15(2):67-78.
3.  Then there's the fact that there are severe shortages of  paclitaxel:

Chemo Drug Taxol Shortage Puts Cancer Patients at Risk


So is it worth switching breast cancer patients to a drug that incurs more costs and reduces quality of life, a drug that people die waiting for?  

No one asked or answered that question.  







 

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The "Pay-As-You-Go" PDUFA

06/04/2012 06:51 AM |

CBO Estimate of the Statutory Pay-As-You-Go Effects for an Amendment in the Nature of a Substitute to H.R. 5651, the Food and Drug Administration Reform Act of 2012, as posted on the Web site of the Committee on Energy and Commerce on May 25, 2012

Source: Congressional Budget Office.

The bill would modify how the Food and Drug Administration regulates drugs and devices in a broad range of areas. Several provisions of the bill would affect when lower-priced drugs enter the market. Changing the timing of availability of lower-priced drugs affects spending in federal health programs that pay for prescription drugs and biological products. It would also affect the costs of health insurance plans and thus federal subsidies for health insurance purchased through an exchange. On net, the bill would result in a reduction in the deficit of $114 million over the 2012-2017 period, which is the sum of a reduction in direct spending of $113 million and an increase in revenues of $1 million. Similarly, the 10-year net reduction in deficits of $370 million is the sum of a reduction in direct spending of $365 million and an increase in revenues of $5 million.

The CBO chart can be found here.

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Bloomberg: Junk Food Fan

06/01/2012 09:50 AM |

City Marks Nat'l Donut Day On Heels Of Proposed Sugary Drink Ban
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Living in the Post IPAB World

05/31/2012 04:17 PM |

Living in a Post-IPAB World

The Riverdale Press

May 30, 2012

By Peter J. Pitts

The second anniversary of President Obama’s Affordable Care Act (ACA) just passed. And two years after its enactment, the debate over health care reform is every bit as contentious. The recently released forecast from the Congressional Budget Office (CBO) offers fodder to both sides.

On one hand, the law may cost $50 billion less over the next ten years than projected in January — but the national budget deficit is now projected to be $93 billion higher. Medicare and Medicaid expenditures are projected to more than double over the next decade, despite 4 million fewer Americans getting health insurance.

For anyone in favor of improving our healthcare system, these numbers make clear just how desperately our county’s entitlement programs need real reform. 

Unfortunately, the president’s chosen cost-cutting strategy for Medicare — the establishment of the Independent Payment Advisory Board (IPAB), an all-powerful panel of budget enforcers — is one of the most harmful aspects of ACA. Lawmakers must cast this flawed approach to reform aside and focus instead on innovative initiatives that address the program’s real cost-drivers while protecting seniors’ access to care.

IPAB is a board of 15 presidential appointees. Unelected and unaccountable, these bureaucrats are tasked with drawing up budget cuts if Medicare expenditures exceed preset targets. 

Those targets will almost certainly be surpassed. Medicare spending is expected to be $575.7 billion this year, jumping to over $1 trillion by 2022, as the country ages. Over the next 75 years, the program is projected to accumulate a $38 trillion budget shortfall.

Much of this enormous price tag goes towards financing Medicare Parts A and B. And yet, IPAB has no authority over either. In fact, the board can’t make any substantive structural changes. Neither the fee-for-service structure nor enrollee premiums and fees can be altered.

The board’s only viable option is to further ratchet down reimbursement rates for Medicare providers, especially doctors, who are already losing money on Medicare patients. Indeed, the financial burden of too-low payments under Medicare has driven 17 percent of doctors and 31 percent of primary care physicians across the country out of the Medicare program altogether, according to a study from the American Medical Association.  

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. 

IPAB is even more insidious because it deflects policymakers’ attention from innovative reform efforts with real cost-saving potential.
Last June, the Department of Health and Human Services (HHS) launched two such initiatives. 

The first would make $42 million available to improve coordination of care for Medicare enrollees. Consider that just about half of Medicare beneficiaries suffering from congestive heart failure are readmitted to a hospital within six months of discharge. Each readmission costs an average of $7,000. The health and monetary benefits of improved coordination to make such readmissions unnecessary would be huge. Evidence shows that well designed transitional care between providers can dramatically reduce costs and improve health care outcomes. 

Limiting preventable Medicare hospital readmissions could save nearly $245 billion over the current ten-year budget window, according to MedPAC. Even a 40 percent reduction could save $100 billion over that same period.

The second HHS initiative combats chronic illness. This has enormous cost-cutting potential, as 75 percent of health care expenditures go towards the treatment of chronic diseases. Diabetes, heart disease and strokes alone cost $1 trillion annually.

Patient coaching and health education could both improve enrollee health and reduce the costs of treating such illnesses. A clinical trial of the Diabetes Prevention Program found that enrolling overweight adults aged 60-64 in a community-based weight loss program could save Medicare between $1.8 and $2.3 billion over ten years.

Both HHS initiatives hold the promise of saving money and lives. The same cannot be said for IPAB. Instead of generating savings by improving the health of beneficiaries, IPAB devalues these efforts, squeezing doctors and providers to the detriment of seniors.

It’s time to support a Medicare cost-saving strategy that encourages long-term strategic thinking. Congress must repeal IPAB and stand up for real Medicare reform.

Peter Pitts is President of the Center for Medicine in the Public Interest and a former FDA Associate Commissioner.
 
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Bloomberg Bans Big Gulps

05/31/2012 12:29 AM |

According to stories that will run in the NY Times and NY Post:

“The health department/City Hall will tomorrow propose that any location that gets a restaurant letter grade will be barred from selling sugar-sweetened beverages over 16 ounces. This would also apply to food trucks.”

And here’s something from the NYC Fox affiliate:

“Get ready to sell smaller containers of soda and other sweetened drinks. The obesity task force with the New York City Department of Health is ready to recommend that containers for sweetened beverages be limited to 16 ounces. The rule would affect drinks sold at delis, fast-food franchises, sports arenas, and sidewalk carts. Anyone who violates the regulation would be fined $200 for every sale.”

I can’t wait for the first 7-11 sting to stop the sale of Big Gulps, the undercover operation to the illegal sale of Mountain Dew refills at movie theaters and the Gatorade bodega/salad bar busts designed to break up multiple bottle sales. 

It would also apply to those souvenir soft drink cups at Yankee Stadium, CitiField and Madison Square Garden.   But not the 24 ounce beer cups.  (As we all know, beer does NOT add weight.)

Or jumbo sno-cones.  (I think.)  Or coffee with lots of sugar and cream.   Then again, will we be allowed to add sugar to our beverages?  

I am sure some people actually care if such a regulation will – as Bloomberg claims – reduce obesity.  It really doesn’t matter.    If obesity rates  (using the unreliable measure of Body Mass Index) remain essentially unchanged in The Big Apple(and the have not really budged since 2005 ) since the Mayor started his crack down on sugar and soft drinks  ( the consumption of the latter also remains essentially flat) then it will be argued that the assault on serving size is critical.   

If Bloomberg claims (incorrectly) – as he has– that the subway ads linking soft drinks to morbid obesity and a ban on soda sales in schools have reduced obesity (and they have not) then a container size crackdown will lead to even greater gains, I mean losses.    In fact, sweetened beverage consumption has declined nationwide over the past few years.   Do we really know why?  No.    Does it have any affect on health?  No one knows.   Are NYC statistics on sweetend-beverage consumption reliable.   The data is based on self-reported consumption.   For all we know, all the kvetching about soft drinks is just making people lie.. as in telling those conducting surveys what they think they want to know.   But who cares?  And who in the media has ever looked at the data apart from using it to restate the narrative of evil corporations poisoning our kids.   So much for the media being more careful reporting statistics and science after perpetuating the Wakefield vaccine fakery.

We have arrived at this silly place in public policy because the operators of the Nanny State and the media willingly accept the spurious correlation between soda consumption and obesity because it reaffirms pre-existing biases.  These biases are reinforced by sloppy “studies” that associate watching TV with drinking soft drinks and both with obesity.    Next thing you know NYC is running ads linking soda to leg amputation.

Nagging –  by our parents or a billionaire baby sitter with too much time – and one term too many – on his hands has never produced changes in habits, especially eating, drinking and exercise.   People – especially in NYC – will find a way to chow and slurp down what they want, when they want. 

Which they will.  After they stop laughing and ridiculing the latest Bloomberg effort.

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Fourth of July Fireworks?

05/30/2012 10:27 PM |

The U.S. House of Representatives voted 387-5 on Wednesday to pass its version of PDUFA reauthorization legislation. The bill will now need to be reconciled with a version that passed the Senate last Thursday. Majority Leader Eric Cantor said he hopes to reach an agreement with the Senate by July 4 on a final bill to send to President Obama.

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Will USPSTF Say No to Breast Cancer Detection Innovations?

05/30/2012 05:20 PM |

The US Preventive Services Task Force standard for approving a preventive screening technology is as follows:

You need to show a decline in rates of death in a randomized clinical trial conducted over ten years in mostly white Europeans

The decline in death rates has to be from all causes, not the disease you were screening for

The determination of what a sufficient decline in death rates is  at the sole discretion of USPSTF.

In otherwords,  no new screenings such as this new breast cancer detection tool being developed in Israel.

A game-changer in breast cancer detection

Early detection is the key to improving breast cancer survival rates, but mammography is not the ideal method to accomplish this goal. On this point, medical experts across the globe agree.

Not as clear is what could do the job without the disadvantages of mammography — which often causes pain or discomfort; emits radiation; cannot properly image dense breast tissue; relies on a radiologist’s interpretation of the image; and is not recommended for routine screening of women under age 40 or 50.

Of several approaches being developed worldwide, an Israeli solution pioneered by electro-optical engineer Boaz Arnon holds particular promise in providing a game-changing device for early detection of breast cancer.

Arnon’s mother, Ruth, succumbed to the disease in 2004. Through Real Imaging, the company he founded in 2006, he was determined to offer an accurate alternative that would address all issues of concern and still be cost-effective.

Appropriately named RUTH, the device he invented uses a new trademarked platform he calls MIRA (functional Multidimensional Infra-Red Analysis). Built on principles from existing technologies and mathematics, MIRA enables functional quantitative analysis of 3D and infrared signals emitted from cancerous and benign breast tissue.

“Our solution is not sensitive to age or breast density, and works without radiation,” Arnon tells ISRAEL21c. “We image the patient from a distance of 70 centimeters (25.5 inches), with no physical contact or radiation, and we have developed an automatic method that aims to detect breast cancer early, easily and as cheaply as possible.”

No more guesswork

“Physicians should be highly praised for their success rate in diagnosing breast cancer with the tools available today,” says Arnon, “but still, the death rate from breast cancer is unacceptable.”

Breast cancer is by far the most frequent cancer among women, with an estimated 1.38 million new cancer cases diagnosed in 2008 (accounting for 23 percent of all cancers), and is now the most common cancer both in developed and developing regions.

Though a medical doctor will oversee screenings with RUTH, “automatic” is one of its most key features. Results will not have to be interpreted by human eyes, thanks to the device’s unique process of calibration using mathematical algorithms formulated from three-dimensional models of hundreds of women with and without malignancies. The algorithms provide unprecedented accuracy, as Real Imaging has demonstrated in blind studies.  Read More & Comment...