Clinical Trial by Jury

11/11/2014 06:48 AM |

Curiouser and curiouser.

From the Economic Times (of India):

Government panel moots clinical trial waiver for two cancer drugs

The (Indian) government's top advisory panel on medicines has recommended waiving off of clinical trials for two new cancer drugs, allowing them to be sold without testing on Indian patients. This, according to the panel, is permitted to cater to unmet medical needs.

The move is significant as it comes despite a recent directive from the Supreme Court asking the government to be careful while approving clinical trials as well as new medicines.

The two medicines - Aflibercept and Trastuzumab emtansine - are used in treatment of metastatic colorectal cancer and metastatic breast cancer respectively.

The Drug Technical Advisory Committee, headed by director general of health services Jagdish Prasad, considered that since both the drugs have been tested in various other countries and found to be effective, these can be allowed for sale in India in "public interest".

The law allows waiver of clinical trial in Indian population, only for drugs approved outside India, if there is national emergency, extreme urgency, epidemic, orphan drug or a disease for which there is no therapy.

However, many health experts feel the proposed clinical trial waiver to the two cancer drugs is in violation of rules and can have serious implications for patients.

According to CM Gulati, editor of the Monthly Index of Medical Specialities and an expert on the rational use of medicines, Aflibercept and Trastuzumab emtansine do not even qualify for exemption as there are alternative therapies available.

"This is a false and fabricated claim because there are other therapies available for metastatic breast cancer, notably Lapatinib plus Capecitabine. As a matter of fact Trastuzumab emtansine was compared with Lapatinib Plus Capecitabine for efficacy and safety," Gulati said.

Colorectal cancer, one of the lifestyle-related cancers, is still not much prevalent in India. Experts say the incidence of colorectral cancer in India is around 40,000 to 50,000 every year. Doctors say it is on rise as people are pursuing a western lifestyle and diet, high in protein and fat, low in fibre and vegetables.

On the other hand, breast cancer is developing into epidemic proportions in India with almost 1.5 lakh new cases being diagnosed every year and close to 70,000 women dying of breast cancer, according to Globocan (WHO) Data 2012.

As per the expert committee's suggestions, Aflibercept can provide for a second line therapy for metastatic colorectal cancer. However, experts contest that by this logic even third-line therapy and fourth-line therapy can be approved without conducting clinical trials on Indian patients.

However, there are also some who feel clinical trials can be waived for drugs that are available outside India for a specific period and which have therapeutic benefits.

"Generally clinical trials conducted in India do not come up with new data. On the contrary, trials cost a lot of money which consumers have to pay later. Moreover, it unnecessarily causes a delay in entry of crucial medicines," says Amit Sengupta, co-convenor of Jan Swasthya Abhiyan, a public health advocacy movement.

  Read More & Comment...

Jonathan Gruber: Obamacare Passed Because True Costs Hidden From 'Stupid' American Voter

11/10/2014 05:10 PM |

Jonathan Gruber was caught on a just released Youtube video of the 24th Annual Health Economics Conference hosted by the University of Penn Leonard Davis Institute claiming Obamacare was passed by hiding it’s worst features and thanks to the stupidity of the American voter.   That is generating headlines in the conservative blogosphere.  But they missed the most breathtakingly honest  statements made by Gruber that underscore that Obamacare is filled with hidden taxes on everyone, especially the middle class Americans with chronic illnesses.   The GOP should use Gruber’s observations as the starting point for whatever actions they take on Obamacare.  Here's what Gruber said: 

Called it a system of draconian taxes

Called employer mandate a political fiction to raise taxes.   But it is NOT central to law.   Shouldn’t care where people get insurance.

Cadillac tax is really a 100 percent tax on cost of insurance (better than nothing)

Transparent spending is a fiction

Healthy people subsidize sick people except that percent of income ramps up over time..Points out that out of pocket costs will increase by 100 percent by 2018.  It has already increased by 100 percent for chronically ill patients

150-200 pct of poverty will have deductibles of $3000. 

Small business have limit on deductibles of $2000.

Limited networks:  Not a good thing for patients.   Gruber notes that people went to specialists less, hospital less.  But not a long run solution. 

Concluded by saying "We don’t have the right answer.   Force politicians to be humble and patient.”

Well now you tell us.  Better late than never..   Read More & Comment...

Doctor Uses Back of the Napkin Math To Make Life and Death Decisions

11/10/2014 11:21 AM |

Dr. Cary Presant thinks calculating the value of a new treatment by measuring cost over overall survival is a neat idea.  I mean, it's so easy you can use the back of a napkin to make life and death decisions!!!  

He ignores other aspects of the cost and value of medical innovation that take into account patient needs and long term impact.

True, spending on cancer treatments has climbed from $24 billion in 2004 to about $37 billion today. But that’s less than a half a percent of total US health-care spending.
More important: While expensive, since 2004 such innovations were largely responsible for a 40 percent increase in living cancer survivors, from 9.8 million to 13.6 million. The new therapies also saved $188 billion on hospitalizations.
In fact, a new study by Dr. Lee Newcomer confirms this result: United Healthcare’s cancer costs dropped as spending on new cancer drugs increased.
Finally, new drugs help people go back to work. The value of the increase in ability to work is 2.5 times what we spend on new therapies.

Presant's biggest problem, though, is that his cost diagnosis is one-size-fits-all: It treats all patients as the same, ignoring the genetic variation in patient response that a new class of “targeted” cancer drugs will soon address.

Dig a bit deeper, and it’s clear that Presant may have a more ideological motivation:  By ignoring the role of insurers in jacking up the out of pocket cost of cancer drugs to discourage use,  he sides with payors, not patients.  It's the health plans that want docs to use 'pathways' -- back of the napkin calculations -- to make life and death decisions.  Docs who stick to the pathways get bonuses and if they prescribe drugs outside the pathway they don't. 

And this line of thinking does away with the Hippocratic Oath. No longer is the doctor’s first obligation “to apply for the benefit of the sick, all measures that are required.” Instead, Presant seems to believes three months of added life isn't worth it.

In fact, three months or less of survival can lead to a lifetime free from disease because average survival masks greater gains in many groups. Back in the 1980s, experts predicted AZT, the first anti-AIDS drug, would add less than three months of life. Yet nearly 90 percent of people taking AZT lived for two years. That allowed them to survive long enough to get the next-generation anti-retroviral combination that now keeps HIV in check.
Even when innovations don’t work miracles, refusing to die has a value not measured by the ASCO app.
When my friend Lynne Jacoby was diagnosed with advanced pancreatic cancer in April 2012, she was told she’d die in weeks. She accepted the diagnosis, but not the prognosis. She entered a clinical trial and received an innovative treatment tailored to her tumors. She was able to travel, work and spend time with her wife and family.

Lynne died last Oct. 6, less than three weeks before her genome was to be sequenced for the next innovation. Her last written words measure the value of innovation Presant ignores:
“For someone like me, who is . . . told that my life would be measured in weeks, I guess I would just want everyone to realize that all of our lives are just measured in weeks, and we have to do whatever it takes to make that as many weeks as possible for everyone.”  Read More & Comment...

Deterrence Strategy

11/10/2014 07:22 AM |

According to Dr. Janet Woodcock (Director of FDA’s Center for Drug Evaluation and Research), opioid abuse-deterrence technology is in its “infancy” and FDA is unlikely to remove opioids that lack abuse-deterrence features from the market soon.

Speaking on the BioCentury This Week television program,Woodcock said approved abuse-deterrent opioids are “version 1.0.” FDA has approved three abuse-deterrent formulations, including Embeda morphine sulfate extended-release capsules with sequestered naltrexone from Pfizer; and reformulations of OxyContin oxycodone and Targiniq ER oxycodone/naloxone from Purdue Pharma. Woodcock added that there will be a “long path” to travel before FDA would require the incorporation of abuse-deterrence features as a condition for marketing opioids. Rather than forcing conventional opioids off the market, FDA is focused on getting more abuse-deterrent products approved, she said. “We are seeking input on how to get to a point where many, at least, of the formulations on the market have deterrent properties.”

Woodcock’s remarks come after the FDA’s public meeting on the future of abuse deterrent opioids. While the two-day session failed to produce any earth-shattering revelations, it did provide some additional insight into the agency’s strategic path forward. For example, the potential for swifter approval of generic AD products via truncated/bridge studies. One item that wasn’t on the agenda, but that was a topic of conversation during the breaks and after-hours was the increased pressure payers are putting on manufacturers to provide abuse-deterrent products.

That which gets measured gets done – and that which gets reimbursed gets manufactured.

  Read More & Comment...

Don't make patients pay for insurers' mistakes

11/06/2014 08:40 AM |

From today's edition of the Morning Consult ...

Don’t Make Patients Pay for Insurers’ Mistakes

The health insurance industry continues to warn of financial ruin unless America institutes pharmaceutical price controls of the sort mainly found in Europe and Canada. Or, in the absence of regulatory action, insurers are simply sticking their customers with the tab through increased cost-sharing.

It would be highly unfortunate if the insurance industry campaign sparked bad policy decisions that hinder pharmaceutical innovators’ ability to respond to the next epidemic, such as Ebola. Or to illnesses such as hepatitis C that afflict some three million individuals and can lead to cirrhosis or liver cancer – and costs that can reach nearly $600,000 for a liver transplant.

Yet here we are debating miracle drugs that cost one-sixth of such pricey surgical procedures. Take Sovaldi, Gilead Sciences’ breakthrough hepatitis C drug, typically administered with ribavirin plus an interferon injection for a total cost of $94,726 for a full course of treatment (or around $150,000 if taken off-label with Johnson & Johnson’s Olysio, which eliminates the need for injections).

Gilead last week secured regulatory approval for an updated regimen, Harvoni, that combines the active ingredient in Sovaldi with protein inhibitor ledipasvir into a single pill with fewer side effects and a higher estimated cure rate. At $94,500, the price is slightly lower for a more effective, all-in-one oral treatment. Moreover, as many as 40 percent of hepatitis C patients can be cured with eight weeks of Harvoni treatment versus the typical 12-week course, at a significantly reduced $63,000 cost.

Insurers claim such prices will bust budgets and hurt patients (never mind that insurers are making patients pay more out of pocket), despite the fact that pre-Sovaldi hepatitis C treatments typically cost $65,000 to over $100,000. But these prior treatments were less effective and had greater side effects, so either had fewer takers or more patients prematurely ending their treatment. As the IMS Institute for Health Informatics noted in a recent report, “a key issue around the launch of [Sovaldi] is that payers did not accurately predict the demand from patients for the treatment or the price at which it would launch.”

The evidence suggests the industry had at least an inkling, however. Consider that pharmacy benefits manager Express Scripts’s 2012 Drug Trend Report discussed the “increasing challenge of specialty prescription drug spending” and the fact that 22 new specialty drugs were approved in 2012, “many of which will cost more than $10,000 per month of treatment.” In August 2013, UnitedHealth Group’s pharmacy benefits management (PBM) unit published an article citing projected costs of as much as $100,000 for a full course of Sovaldi treatment.

What’s really happening is insurers want someone else to pay for their failure to adequately price demand for highly effective, potentially lifesaving drugs. If the critics had their way and new regulations required price slashing, inevitably patients would lose access to lifesaving therapies, both directly and as a result of reduced research and development expenditures on what could be the next Sovaldi, or Ebola-fighting ZMapp.

Insurers also are hardly powerless, which is evident in their ability to shift drug costs to patients. While critics lambaste the American health system as free enterprise run amok, in reality the U.S. health insurance sector is more like a regulated monopoly – with a mandated customer base that will keep growing as Obamacare expands its reach and as America continues to age. This gives insurers enormous power to bargain with providers and pharmaceutical manufacturers.

Express Scripts, a vocal critic of specialty drug pricing, is a good example. As the largest PBM in the U.S. – with nearly $105 billion in 2013 revenue – Express Scripts enjoys enormous leverage in the marketplace. The company recently told its customers it planned to save $1 billion in 2015 by excluding 66 medicines from its list of covered drugs.

However, noticeably absent from the list was Sovaldi, for two reasons: one, they can’t afford not to cover a miracle drug with a 90 percent cure rate for a deadly disease that claims the lives of 15,000 Americans each year. And two, there is an explicit promise to drop Sovaldi once lower-priced competitors come online that demonstrate comparable effectiveness.

Meanwhile, insurers to date are hardly seeing major dents in their bottom lines. UnitedHealth, the first of the commercial payers to report earnings and an industry bellwether, released Q3 earnings that beat the Street’s expectations. At five percent, United’s overall medical cost increases were far below what they were a year ago at this time when they hit 13 percent, well before Sovaldi came to market. We’ll see what the other major commercial payers have to say, but thus far the concerns raised by the insurers’ Washington, D.C., lobbyists sounds like a case of tail-wags-dog.

Prescription drugs currently make up just over 11 percent of the nation’s nearly $3 trillion health care tab; simple math indicates pharmaceuticals are not the major driver of runaway U.S. health expenditures. America needs a national conversation on healthcare costs, not European-style price controls that will do nothing but deprive patients of potentially life-saving medicines. Insurers suffering through temporary blips in their stock prices should remember what’s really at stake, rather than waging expensive lobbying campaigns and engaging in scare tactics.

Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest.  Read More & Comment...

Saving Money through Innovation

11/04/2014 08:52 AM |

Improved Access to Medical Innovation Slows Medicare Spending

By: Peter J. Pitts

Recently the outlook for spending on federal health programs has been improving. While many have been quick to attribute the slowdown in the growth of spending to the Affordable Care Act, the data suggest improved access to prescription medications is the real hero.

The trustees of Social Security and Medicare recently reported that Medicare should have enough money in its trust through 2030, which is 13 years longer than they projected in 2009. Meanwhile the Congressional Budget Office (CBO) projected last month that federal spending on health programs in 2039 would equal 8.0 percent of gross domestic products (GDP), which is about 15 percent less than was projected in 2010.

While some policymakers and journalists might conclude that the Affordable Care Act (ACA) of 2010 is responsible, evidence of the decline actually predates the bill’s implementation. From 2000 to 2005, the annual growth in spending per Medicare beneficiary was 7.1 percent. But from 2007 to 2010, that rate dropped to 3.8 percent.

What changed between 2005 and 2007 was the introduction of Medicare Part D, which increased access and reduced the cost of prescription medications for millions of eligible Americans. As a result, prescriptions filled by Medicare beneficiaries jumped 14 percent in 2007.

In 2012, the CBO found that this increased use of medications was lowering Medicare’s spending on hospital and physician services. For every 1 percent increase in prescriptions filled by Medicare beneficiaries, spending on medical services dropped by about 0.2 percent, according to the agency.

The main driver of the decline in health care spending is increased access to prescription medications

A more recent analysis found that Medicare Part D reduced hospitalizations by 8 percent and saved the government about $1.5 billion during the program’s first four years. And Medicare Part D is costing less than expected, which is also contributing to the slowdown in health spending.

The main driver of the decline in health care spending is increased access to prescription medications. The health care system is using medication to control illnesses, and it’s replacing expensive hospitalizations, nursing homes and other medical services.

Medicare Part D provides prescription medication coverage for 35 million seniors through private insurers. Seniors pay low monthly premiums of $30 on average for their coverage and 90 percent said they felt satisfied with their prescription medication coverage in a recent survey. Not only is the program providing a valuable service, it is also costing 45 percent—or $348 billion—less than the original estimates — and with a more than 90 percent customer satisfaction.

The program works well because Part D medication prices are determined through negations between private insurers and manufacturers. The savings are realized because market competition effectively drives down the costs, and these savings are passed on to beneficiaries in the form of lower premiums.

Some government officials have proposed that the government should interfere with the program’s success by imposing new rebates on Part D medications. Such rebates, however, would reduce the savings from the program, increasing prices for Medicare Part D beneficiaries by up to 40 percent. The CBO has stated that government interference with the Part D negotiations between insurers and manufacturers would have a “negligible effect.” Precious few programs in Washington deliver both savings to the taxpayer and results to beneficiaries. On both counts, Medicare Part D succeeds. There isn’t a better model to recommend market-driven approaches to health care.

In contrast, the U.S. Department of Veteran Affairs (VA) negotiates its own medication prices with manufacturers but excludes many newer therapies as a result. About 38 percent of drugs approved in the 1990s and 19 percent of the drugs approved since 2000 are not covered under the VA formulary, impacting the overall life expectancy of veterans. As a result, 40 percent of veterans with VA benefits choose to enroll in Medicare Part D instead.

While Medicare beneficiaries have affordable access to life-saving medicines, those enrolled in health insurance plans through the exchanges established by the ACA might not, thanks to specialty tier drug pricing.

Based on the CBO findings, it is abundantly clear, that medical innovation plays a critical role in the solution to curve health spending in the United States. Affordable access to life-saving therapies and continued support through pro-patient and pro-innovation policies are essential to improving patient lives, health care and the economy.

  Read More & Comment...

Mistruths and Half-Truths about Oncology Meds

11/03/2014 06:58 AM |

From the pages of Medical Marketing & Media ...

Washington Insider: Mistruths and half-truths about oncology meds

Demonizing new treatments distracts from the real problem: policies that focus on the near-term

There's been much legitimate consternation over the October 5th 60 Minutes segment on the cost of oncology meds. Hopefully the anger and indignation I've heard will drive some hard thinking toward smart and forceful actions to address the mistruths, half-truths, and straight-out lies presented during the program.

But why is anyone surprised? Did anyone expect a “fair and balanced” story from the same media that was complicit in helping to legitimize vaccine denial?

The 60 Minutes broadcast is only the most recent example of “value denial”—and it's important to understand the program not as a unique and unfortunate incident, but as a set-up for ASCO's pending announcement on it's new methodology for determining the cost-effectiveness of new cancer medicines.

But it's not cost effectiveness and it's not clinical effectiveness. It's a denial of personalized medicine. It is value denial.

Drugs aren't the cause of rising healthcare costs—they're the solution. Demonizing new treatments distracts from the real problem: top-down cost-centric policies that focus on the near-term, short-changing long-term patient outcomes, and so endangering “sustainable innovation” by denying fair reimbursement for high-risk investment in R&D.

New treatments are a bargain. Disease is always much more costly.

Until we counter the Orwellian newspeak that worships at the altar of the “high cost of drugs” with a fact-based and firm explanation of value, the minions of 60 Minutes will own the hearts and minds of the American public. And innovation loses.

And that is not acceptable.

Peter Pitts is a former FDA associate commissioner and president of the Center for Medicine in the Public Interest.

  Read More & Comment...

Trumpeting Trumenda

10/31/2014 01:05 PM |

If you have any doubts that vaccines are on the cutting edge of innovation consider this, Pfizer has just received FDA-approval (via the agency’s Breakthrough Therapy Designation and Priority Review program) for Trumenda, a vaccine for the Prevention of Invasive Meningococcal B Disease in adolescents and young adults -- the first and only approved vaccine in the U.S. for the Prevention of Meningococcal Meningitis B.

As part of the accelerated approval process, Pfizer will complete its ongoing studies to confirm the effectiveness of Trumenda against diverse serogroup B strains.

The next time someone challenges the importance of innovation – trumpet Trumenda.

  Read More & Comment...

Random Acts of Cruelty

10/31/2014 11:44 AM |

From the WSJ:

A consortium including European universities and medical groups plans to give experimental drugs to West African Ebola patients without assigning some to a placebo group, touching off an intense trans-Atlantic quarrel over what is ethical and effective in treating the virus.

Academics and medical groups in the U.K. and France, such as Oxford University, the Wellcome Trust, Doctors Without Borders and Institut Pasteur of France, have decided to give the drugs to sick African patients without randomly assigning other patients to a control group not getting the medicines. They say that in a ghastly epidemic, it is unethical to hold back treatment from anyone.

That has put them at odds with senior U.S. officials at the Food and Drug Administration and the National Institutes of Health. Dr. Luciana L. Borio, FDA assistant commissioner for counterterrorism and emerging threats, told public-health officials at the World Health Organization in Geneva this month she was extremely concerned by the plans to give the medicines to patients without better evidence they work and aren’t highly toxic. “This is too urgent an issue for us not to start out with what we know is scientifically best,” said Dr. Borio. “The fastest and most definitive way to get answers about what are the best products is a randomized clinical trial.”

What crap.   Randomized trials are not scientifically best, they are just tools no better -- probably worse -- than others to establish what works in who.  Dr. Borio, spare us your holier than thou invocation of the randomize trial catechism...


Then there is this:  The FDA yet again asked for more data on a drug for  Duchenne muscular dystrophy, a fatal disease that kills people before they are 30.  The agency doesn't think the measure of response is statistically precise enough...

After receiving criticism the agency assured the public:  "The FDA also plans to hold an expert advisory committee “to gain advice from outside experts and interested stakeholders on the adequacy of the data to support approval, including the possibility of ‘accelerated approval’—a mechanism to approve drugs in particular situations prior to the availability of definitive evidence of effectiveness.”

More crap.

 The FDA had already received expert advice from Parent Project Muscular Dystrophy, an advocacy group founded by family members frustrated by a lack of research on Duchenne muscular dystrophy, (who) initiated and wrote a draft guidance for pharmaceutical companies trying to develop drugs to treat the fatal condition.



...  Read More & Comment...

Courting Biosimilars

10/31/2014 08:46 AM |

Per reporting in BioCentury, in the first lawsuit filed by an originator company over a biosimilars application, Amgen alleged that Sandoz has unlawfully refused to follow the patent resolution protocol laid out by the Biologics Price Competition and Innovation Act of 2009 (BPCIA).


The suit, filed in the U.S. District Court for the Northern District of California, concerns Sandoz's application to market a biosimilar version of Amgen's Neupogen filgrastim G-CSF, a recombinant methionyl human granulocyte colony-stimulating factor.
Under BPCIA, Sandoz was required to provide Amgen with its BLA and manufacturing information for the biosimilar within 20 days of FDA’s decision to accept the application for review.

According to Amgen, Sandoz failed to disclose this information, thus depriving Amgen of the opportunity to assess potential patent infringement claims and potentially seek an injunction to prevent Sandoz from commercializing its biosimilar candidate.
Amgen is seeking restitution for unfair competition and an injunction to prevent Sandoz from commercializing the biosimilar filgrastim until Amgen is "restored to the position it would have been [in] had Defendents met their obligations under the BPCIA."


Amgen is also seeking an injunction to prevent Sandoz from advancing through FDA's approval process until the company has obtained permission from Amgen to use the filgrastim license.
Additionally, Amgen is seeking a judgment of patent infringement for U.S. patent No. 6,162,427. The suit also demands a jury trial.

  Read More & Comment...

Vouching for Ebola

10/29/2014 07:39 PM |

Senator Tom Harkin (D-IA), Chairman of the Senate Health, Education, Labor, and Pensions (HELP) Committee, and Ranking Member Lamar Alexander (R-TN) announced their intention to introduce a bill to speed the development of treatments and vaccines for Ebola by adding Ebola to FDA’s priority review “voucher” program, a program at the Food and Drug Administration (FDA) designed to incentivize the development of new drugs for neglected tropical diseases. Currently Ebola is not listed as a qualifying tropical disease. The Harkin-Alexander bill, which will be introduced when Congress reconvenes, will seek to add Ebola as a qualifying disease under the program.   
 
The bill would add Ebola to FDA’s priority review voucher program, which Congress first authorized in 2007 to promote the development of new treatments and vaccines for neglected tropical diseases. Under the program, a developer of a treatment for a qualifying tropical disease receives a voucher for FDA priority review to be used with a second product of its choice, or this voucher can be sold. 
 
It's an interesting idea worthy of  immediate discussion and debate.  Read More & Comment...

Milwaukee Journal Sentinel and MedPage Win Pulitzer For Getting the Facts Wrong

10/29/2014 01:08 PM |

The Milwaukee Sentinel Journal ran an article entitled,  FDA approves cancer drugs without proof they're extending lives

I don't think I have ever seen or read a more misleading, deceptive and outright false description of the drug development process.   The gist of the article is that surrogate endpoints are not only useless, they are false markers of effectiveness that drug companies use to get worthless but profitable meds approved.   Not suprisingly,  a lot of so-called health care reporters have lauded MSJ piece as extraordinary journalism.   Which just goes to show you that facts need not get in the way of a good story.

But for those who still care about facts, here they are: 

Just this weekend at an educational forum about lymphoma, a well- respected hematologist-oncologist from a major medical center – not a drug company – supported the new FDA Breakthrough designation to approve drugs with significant data after phase 2 clinical trials. He says drugs approved on PH 2 data are now helping people with lymphoma. Conversely he notes that despite powerful PH 2 data for Gleevec, the FDA required an additional three years for Ph 3 data. He says, "A significant number of patients died during that period."

Let me put it this way: In short, the article is based on a fundamental misunderstanding of the clinical trial process, confuses overall survival with the impact on life expectancy and concludes that what we need is to expose more people with incurable diseases to interminable waits that are a prolonged death sentence.



1.   We are using surrogate endpoints to expedite Ebola treatments.   We used them for HIV, Hepatitis C, heart disease, dozens of orphan drugs.  The results:  faster access, more lives saved.  Would the authors oppose using surrogate endpoints — measures of disease progression used to estimate treatment response — for dying Ebola patients.     Surrogate is not code word for fake or ineffective.  Quite the contrary. 

2.   They imply that cancer trials take shortcuts.  In fact, cancer drugs take longer to approve than many other types of medicines.  And only 7 percent of cancer drugs get approved.   If companies were controlling the drug development process you would think they would have better success!

3.   The authors should have asked why haven’t we made even more progress?  They blame surrogate endpoints when in fact they are looking relying upon randomized clinical trials that ignore subgroups of patients, surrogate endpoints that establish linkages between genotype and phenotype and genetic variations.   They ignore the rapid growth in targeted therapies and the significant gains in quality of life and life expectancy they yield

4.  As a result, The authors claim the solution is more randomized controlled trials (RCTs) and (we assume) the number of people enrolled in each trial.  Setting aside the obvious result: an increase in the cost and time required to develop and use new products, this approach is based on wishful thinking and a willful ignorance of the genetic, molecular and clinical diversity that, when ignored, turn RCTs into an tool that harms patients.

Specifically, RCTs are totally inadequate in evaluating treatments in cancer therapy where genomic analysis is uncovering the tremendous heterogeneity of what was previously considered one disease, eg, colon cancer.  Genomic analysis of individual patient’s cancers is disclosing the large number of mutations, and thus targets, within one person.  Developing RCTs for targeted therapies would likely be impossible and heartless for those waiting for effective treatments.

For example, Bruce Chabner gives the example of PLX4032 for the treatment of metastatic melanoma.  That compound had an 81 percent response rate in patients with BRAF mutations in a phase 1 trial. Yet in a phase III study, patients were randomized to PLX4032 or dacarbazine, which has a 15% response rate. Dr. Chabner and others ask whether it is ethical to randomize patients to a drug that everyone knows won’t work if you know in advance which treatment will work for whom.

"If patients with incurable disease who have the right biomarker for response are informed of these impressive early results they will want and perhaps deserve access to the new drug and may not accept random assignment to a modestly effective and toxic standard agent."  (N Engl J Med. 2011 Mar 24;364(12):1087-9. doi: 10.1056/NEJMp1100548. Early accelerated approval for highly targeted cancer drugs.
Chabner BA)

Actual clinical RCTs are often statistically underpowered leading to inconclusive or incorrect results precisely because such studies are biased against prior knowledge. This is due in part to the effect of diminishing returns, because larger sample sizes are needed to achieve sufficient power capable of detecting the necessarily smaller positive effect sizes available as fitness improves.

5. Do potent cancer drugs carry risks? Of course. People also die surging surgery. We see the advertisements on TV - certain drugs for arthritis carry a risk of leukemia. But patients still want and need these potent drugs that change their quality of life! Drug approval is not always about survival, and it’s never risk free.
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The Salk Mythology

10/28/2014 07:58 PM |

A Google doodle marks the 100th birthday of Jonas Salk, who as everyone knows, discovered the vaccine that cure polio without regard to making money.

That's a nice narrative but it is history rewritten by those who want to claim that every company in the drug or vaccine business should cut their prices or give away their products.   

It is true that when Edward R. Murrow asked Jonas Salk who owned the patent to the polio vaccine Salk said:  “Well, the people, I would say. There is no patent. Could you patent the sun?"

Since then, Salk's statement has been use as trump card by individuals who claim that protecting the intellectual property of medicines is immoral or greedy. As an article in Slate notes:  One critic of the big pharma called Salk “the foster parent of children around the world with no thought of the money he could make by withholding the vaccine from the children of the poor.”

Let's set aside the fact that solar energy companies have, in effect, patented the sun.   There was a reason the Salk and the Sabin vaccines (more on that later) were not patented.   The National Foundation for Infantile Paralysis was the beneficiary of a massive campaign to eradicate polio that makes the ALS Foundation Ice Bucket Challenge and Standup2Cancer efforts seem anemic (which they are NOT.)   

"In the single year that the polio vaccine was unveiled, 80 million people donated money to the National Foundation for Infantile Paralysis, which spearheaded the vaccine effort.... Schools, communities, and companies joined in a remarkable display of unity against the disease. Even Walt Disney’s cartoon characters contributed their talents, appearing in a film that adapted the Snow White and the Seven Dwarfs’ song “Heigh Ho” to an anti-polio ditty. In the 13 years leading up to the vaccine’s roll out, the budget of the National Foundation for Infantile Paralysis swelled from $3 million to $50 million.  

In today's dollars that's about $500 million.  The need for IP was removed because the passion capital had already been raised. 

Further, it is true that a vaccine (an inactivated virus) does occur in nature as does the sun.  But we patent both.  How  many solar energy companies have repurposed the sun's power with patented technology.  A lot.  And a lot more than vaccine companies have.  

Then there is the myth that the Salk vaccine eliminated polio.  In fact as Angela Matysiak noted in a review of a Salk biography:

"In 1959, epidemiologists reported findings on the pattern of the disease. These suggested a shift in incidence according to age, geography, and race. By 1960, less than one-third of the population under 40 years of age had received the full course of three doses of the Salk vaccine plus a booster. Most of those who had were white and from the middle and upper economic classes. The disease raged on in urban areas among African Americans and Puerto Ricans and in certain rural locales among Native Americans and members of isolated religious groups."

There were several reasons for the lag.  Most important was the limited effectiveness of the Salk vaccine which was killed version of the polio virus required several shots.  Matysiak oberves that Sabin put the point most succinctly: “The need for inoculating large amounts and the need for repetition are bad.” In contrast, an oral vaccine with a small dose of the attenuated versions of each of the three strains, administered once, would give lifelong immunity.

The Sabin vaccine was cheaper and easier to administer.  But it became clear that both vaccines were needed because each protected certain subgroups that the other could not protect.   The Salk vaccine could be used in people with compromised immune systems while the Sabin vaccine could not.

Both were required to eradicate polio.   Ironically, the Sabin vaccine could be considered a me-too vaccine by the same ilk who claim patents don't matter.  

And the same anti-patent crowd ignore another inconvenient truth:  That Alexander Fleming, who discovered penicillin, regretted that he had not secure a patent because the lack of funding and private investment postponed it's development and commercialization by a decade and cost the lives of millions around the world. 


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There ain't nothin' like a name

10/28/2014 01:15 AM |

Here’s the headline -- The GPhA now acknowledges the importance of differential nomenclature for biosimilars.

And now here’s the spin

The Generic Pharmaceutical Association (GPhA), out-flanked, out-thought, out of its league, and on the wrong side of history, has proposed a bizarre naming scheme for biosimilars – and shared it with the FDA last January. The agency agreed to the meeting on the condition that it was a ‘listening session’, meaning that the FDA would be unable to answer questions or expand on any issues beyond what is in the public domain and what they have stated in the published draft guidance documents.

Wink and a nod.

Officially, the GPhA requested the meeting to discuss the dialogue held at a recent World Health Organization (WHO) meeting regarding non-proprietary names of biosimilars and the possible addition of identifiers (specifically a three-letter random alphanumeric suffix) to the INNs of originator biologicals in order to name biosimilars.

The GPhA’s brainstorm is, rather than the WHO concept (also supported by the US The Pharmacopeia), attaching the company name as a suffix to the INN without changing the INN as a way to distinguish between products. They also proposed that this then also apply to the originator product, e.g. filgrastim Amgen.

Really? What happens when a biosimilar manufacturer merges with another firm or sells a product line to another company? And since when are drugs named “for a company.” That’s why FDA regulations stress names that are designed to avoid confusion. Using a “company suffix” makes things more confusing, not less. (For example, what happens when one company has more than one drug is any given therapeutic category?)

The GPhA stated that it does not disagree with ‘distinguishability’, but believes that this can be done without changing the INN.

That’s a start.

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Sunshine on my shoulders makes me happy?

10/26/2014 04:09 PM |

From the pages of the Birmingham News ...

Without context, Sunshine Act's health care 'transparency' is useless

The biopharmaceutical industry is buying off America's doctors.

That's the bogus conclusion from some who have examined new data from the Centers for Medicare and Medicaid Services. The data set shows that from August to December of last year, drug and device manufacturers paid $3.5 billion to physicians and teaching hospitals.

Those payments were real, but there were no payoffs. The truth behind the numbers is that industry-physician collaboration is one of the main drivers of medical innovation today.

The purpose of the publication of this data is at least partly well-intentioned. As part of the "Physician Payments Sunshine" provision of the Affordable Care Act, it aims to increase transparency in the healthcare industry and help patients make better informed decisions.

The provision requires CMS to provide information on the financial relationship between doctors and the pharmaceutical industry. The data covers all payments and gifts provided to doctors and teaching hospitals from drug and medical device manufacturers. This includes meals, travel expenses, and speaking fees. Disclosure here is all to the good.

But for some, publication of the data was designed to produce exactly a "gotcha" moment: It was intended to embarrass the pharmaceutical industry and healthcare professionals, hopefully leading to a diminution of the supposedly undue influence the industry has on medical practice.

If that's the main impact of the release of the data, it will be doing more harm than good. First, the data reported by CMS is incomplete. Nearly 40 percent of the records aren't actually connected to a specific doctor or teaching hospital. Considering that the information is supposed to help empower patients, this gap is critical.

The data also come with little context. Industry-physician collaborations drive research and development in medicine. Pharmaceutical companies work together with doctors in clinical trials to create novel, lifesaving drugs. In fact, $1.5 billion of the payments reported by CMS were for research. These companies are merely reimbursing doctors for their professional services.

Biopharmaceutical companies and doctors bring separate skill sets and knowledge bases to the research table. Doctors working in the field have a better sense of what types of treatments patients need. And those in the biopharmaceutical industry are better versed in the drug development process. According to a recent survey, 94 percent of physicians say that the role of pharmaceutical and biotech firms in sponsoring clinical trials for new treatments is useful.

Thanks to these partnerships, there are currently 3,400 drugs being developed in the United States. These are drugs that will combat diseases like diabetes, heart disease and Parkinson's.

Currently, 93 medicines are undergoing clinical trials or awaiting FDA approval for Alzheimer's and dementia alone. For the more than 5 million Americans with Alzheimer's, one of these treatments could be the key to higher quality of life.

Physician-industry partnerships are also on the forefront of medical research. Take the collaboration between the University of Alabama at Birmingham and drug manufacturer rEVO Biologics. The two are conducting Phase III clinical trials for a therapy that could prevent dangerous pregnancy complications.

These collaborations are especially critical considering that federal research dollars are quickly drying up. In fiscal year 2013, burdened with the sequestration and budget cuts, the National Institutes of Health (NIH) - the country's main source of biomedical research funding - awarded 722 fewer grants than the previous year.

If the data released under the Sunshine Act continues to present the financial relationship between physicians and industry without this crucial background information, these collaborations could be in jeopardy. Doctors may end up resisting them in order to avoid sensationalist claims that their medical judgment is for sale to the highest bidder. Patients will be the real losers.

The Sunshine Act aims for transparency in healthcare, but fails to be transparent itself. CMS must present a more detailed view of the relationship between doctors and the biopharmaceutical industry. Doing so could even help patients choose doctors who are leading innovators in medicine.

Peter J. Pitts, a former Food and Drug Administration associate commissioner, is president of the Center for Medicine in the Public Interest.  Read More & Comment...

NYC Doctor Flunks Ebola Vaccine Test

10/24/2014 12:47 PM |

In a guest post on Matt Herper's Forbes.com blog  Peter Bach -- the latest doc seeking celebrity status by demonizing drug prices -- asks the loaded question:   "Just how high do prices need to be? When does the potential to profit exceed that which is needed to drive innovation and pervert the market?"

I have previously asked Peter Bach to answer his own question in a different way:  What if his idea of how high prices have to be winds up killing off drugs and vaccines that save peoples lives?   What if the price he thinks is too high induces lots of companies to develop an Ebola vaccine. What is morally more reprehensible,  drug prices higher than what Bach prefers or drug prices that he likes but kills off Ebola vaccine development?  t I have asked Bach to discuss or debate this issue to no avail. . Perhaps Matt Herper could arrange for this to happen since Bach has been ducking me for months. 

In this recent post Bach actually argues that prices that he regards as too high are bad for innovation.  He never explains why.  Instead he complains that there is too much innovation designed to treat rare diseases and specific cancer mutations.   You tell me if he makes a strong case.  Maybe I am missing something. 

1. Bach – an oncologist – misrepresents the research activity of companies pursuing drugs to “target lung or and/or other cancers caused by an acquired genetic abnormality called the ALK rearrangement.” He claims that seven drugs targeting the ‘same cancer causing mechanism’ is too much. 
 
In fact, there are at least seven ALK gene rearrangement variants that are involved in NSCLC alone. And he wants us to believe that one drug targeting ALK will do the job. In fact, most patients who have used the first 2-3 ALK inhibitors (let’s call them Bach’s Enough Bunch or BEBs) undergo a relapse…In particular, the central nervous system (CNS) is one of the most common sites of relapse in patients with ALK-positive NSCLC. 

For this reason, most cancer doctors who understand the limits of the first generationof drugs welcome the development of new ALK inhibitors that have greater potency and different kinase selectivity compared with the BEBs. 

Bach alludes to that fact that other cancers are caused by ALK mutations. Indeed, there are several. Genomic aberrations in ALK are involved in lymphoma, renal cell carcinoma, rhabdomyosarcoma, thyroid
carcinoma, colorectal cancer, and some rare melanomas. 

2. His economic reasoning is circular when it’s not silly. He claims that there is a price high enough to induce companies for 2-3 companies to develop products, but not 7 or 8. Bach presumes that if the prices were at levels he deemed appropriate companies that would otherwise invest in such a small group of patients would invest in yet another small subgroup of patients, presumably at a price not too high, which would in turn lead to companies investing in other small groups of patients where the prices are lower than the ‘right ‘ number of drugs. 

I defy Bach to could come up with one example – any example – of a pricing system that generates just the right amount of innovation while inducing and moving capital to other important medical condition. Can he show, for instance, that lower prices for drugs for Pompey’s disease (a rare pediatric condition) stimulated investment in Huntington’s disease or Ebola? Indeed, most orphan diseases are priced well above $100K a year. According to Bach, a lower price would only discourage companies that are developing medicines that are superfluous AND cause them to tackle another small disease group where prices are capped. 

Failing logic and empirical evidence, Bach resorts to the use of a red herring: that drug prices are too high because they are lower elsewhere. He asserts, “When the FDA approved a drug this month” (called Ebriet for a previously incurable disease, idiopath pulmonary fibrosis or IPF) “Roche announced a $94K per year price tag, more than twice what the drug costs in Europe. Simple math – if they ever could achieve full market penetration Roche would make back their entire investment in a single year. “

Except it won’t. Because IPF also has several mutations involved. Only 10 percent of patients with the disease have the mutation Ebriet targets. According to Bach then, charging $46K per year for the drug is a sure fire way to induce the development of treatments for the 90 percent of patients that have one or more other mutations. 

Let’s set aside the real value of a drug that delays the progression of a previously fatal and untreatable disease for a small and neglected group of patients as Bach does. Or that new cancer drugs and treatments for other rare diseases have saved more money and generated more life expectancy than the half-way or hopeless treatment they replaced. Those are two very good reasons why a new medicine should have a high price. 

And let’s set aside the facts that lay waste to his unstated conclusion: the high drug prices are driving American health care spending into bankruptcy. In fact, apart from the value they generate and money they save, drug costs for such illnesses are less than 2 percent of total health care spending and have been that level for decades. Let’s ignore all that, as does Bach. How about applying his Euro-pricing to what he makes or Memorial Sloan Kettering where Bach works. MSKCC generates about $3 billion a year in revenue. It pays 6 executives in excess of $1 million a year. Doctors and hospitals also cost more than twice that they do in Europe. MSKCC is probably even more. 

When Bach proposes the same cut in price for MSKCC I can take his conviction about drug prices seriously. He will still be misguided or misleading. And I bet he will still be afraid to debate me.  Read More & Comment...

Tap Dancing to Therapeuic Failure

10/21/2014 09:20 PM |

From the pages of the Morning Consult.

Stepping Towards Failure

By Robert Popovian

As healthcare professionals, we should always do what is best for the patient. This includes practicing evidence-based medicine. Unfortunately a growing practice of the healthcare ecosystem ignores this principle: the institution of payer policies that try and curb pharmaceutical utilization. One such policy is “Fail First” or “Step Therapy”. This “utilization management” strategy is when a payer, often a Pharmacy Benefit Manager, decides that a patient must first try and fail on one or more medicines that their physician did not select. This can mean forcing the patient to take a drug that had been previously tried – and failed to work – or even mean a patient is forced by the payer to take a medicine that does not have an FDA indication for their disease. The patient suffers through weeks, if not months, of inappropriate therapies all in the name of cost-effectiveness. There are instances when this type of policy is truly cost saving such as when an AB rated generic is required first, and greater use of real world evidence including the patient’s treatment history could target the right patient for this type of intervention, but applying the policy broadly means that patients needlessly experience substandard treatment.

Today, 70 percent of employer-sponsored insurance plans use this approach to control medication costs. Payers assert that by mandating physicians to follow a certain prescribing algorithm that the “per member per month” pharmacy costs will be lowered. However, payers don’t consider whether it will also result in overall reductions in healthcare costs or an improvement in patient outcomes or even long term savings in pharmacy expenditures. In addition, no consideration is given to the indirect costs incurred by physicians who now have to shuffle patients in and out of their offices for additional visits to ensure that they will eventually get to use the appropriate drug the physician had selected for them in the first place.

More importantly, payers don’t define what “failure” means for a patient. Is “failure” simply a lack of efficacy of the payer’s drug choice? Is “failure” a hospitalization incurred because of the payer’s pre-determined algorithm? Or worse, is “failure” a patient who is now seriously ill because they had to endure an ineffective therapy? As Dr. Brenda Motheral pointed out in her findings published in The American Journal of Managed Care, 17 percent of patients on step therapy algorithms end up without any treatments as they get left behind in the administrative maze.

Another interesting point is that when patients reach the zenith of their fail first/step therapy regimen and have the opportunity to utilize the drug that was chosen initially by their provider, they are hit with significant out-of-pocket costs because such medications are usually in higher copay or co-insurance tiers. So not only did the patients have to endure months of ineffective or at times hazardous therapy, they now have the burden of paying higher out-of-pocket costs.

Remarkably, there is absolutely no evidence that fail first/step therapy provides a reduction in overall healthcare costs and improvements in patient outcomes short or long term. There is no empirical data published in peer reviewed journals that demonstrates definitively that there are both health benefits and cost savings from these policies. In fact, despite evidence of potential harm (i.e., patients ending up on no therapy), payers have not been required, nor have they taken the initiative, to demonstrate that fail first/step therapy policies are safe for patients. Payers quite often and at times rightfully ask biopharmaceutical companies to provide safety and efficacy data or ever-expanding pharmacoeconomic product dossiers for their products. Payers also demand that physicians justify every single intervention they utilize through mounds of paperwork. Isn’t it time to make payers provide a similar level of evidence for their policies?

As Dr. Motheral pointed out, “Adoption of step therapy is quickly outpacing decision makers’ understanding of the clinical, humanistic, and economic value of these programs.” In other words, we have no idea what the outcomes are but payers continue to demand patients should trust their decisions rather than those of their own doctors.

Utilization management is an important part of ensuring that this country’s significant investment in healthcare is used efficiently. However, policies without clear clinical rationale that achieve short term cost savings at the expense of long term health isn’t efficient. Let’s use the growing wealth of real world patient experience data to ensure the right people get the right treatment as quickly as possible, and that money isn’t wasted in the wrong places. This should apply not just to medicines but other parts of the healthcare system.

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Did The FDA Delay Ebola Cures?

10/20/2014 11:15 AM |

How the feds block Ebola cures


By Robert GoldbergOctober 16, 2014 | 8:30pm

How the feds block Ebola cures

We have technology to potentially control Ebola and other viral outbreaks today. But the federal bureaucracy refuses to catch up with 21st-century science.
For example, diagnostic startup Nanobiosym has an iPhone-sized device that can accurately detect Ebola and other infectious diseases in less than an hour.
Two other companies, Synthetic Genomics and Novartis, have the capacity to create synthetic vaccine viruses for influenza and other infectious diseases in only four days. Both firms can also share data about outbreaks instantaneously and make real-time, geographically specific diagnosis and vaccine production possible.
These companies could start producing Ebola vaccine/treatments tomorrow — except that the Food and Drug Administration’s insistence on randomized studies and endless demands for more data means firms have to spend millions on paperwork instead of producing medicines.
And for every small company drained by such tactics, many others conclude it’s not even worth trying.
These advances aren’t available because the FDA is using 19th-century science to decide which medical technologies should be used in the 21st century.
Two years after 9/11, Congress created Project Bioshield to speed up the commercialization of vaccines, drugs and diagnostics. A key part of the plan: Get the FDA to evaluate innovations quickly by using the same scientific advances that were used to discover them.
The agency balked.
Pandemic vaccines and drugs don’t move through the FDA approval process faster. Instead, drug- and device-development times actually increased more than 70 percent over the past decade because the FDA keeps demanding more studies and more data using outdated techniques.
And, no, the FDA is not using the best science to ensure safety. Time and again, it has waived regulations when politically expedient.
Back in 1984, at the start of the AIDS epidemic, the FDA claimed that reviewing HIV treatments would take at least six to eight years.
Only after loud, large and sustained demonstrations did it state that new AIDS drugs could be OK’d in two years or less and that most people who wanted to try them could. Millions of lives were saved as a result.
In 2008, it took Synthetic Genomics scientists a month to sequence the genes of every strain of the meningitis virus and engineer a vaccine that protects against them all. In Europe, Canada and Australia, the vaccine was approved for use in children (the group most likely to get meningitis and die from it) in 2010.
But the FDA demanded another study in the United States. Only after meningitis hit Princeton University and UC-Santa Cruz this year did the agency allow the vaccine to be imported and given to students on both campuses.
And the agency still hasn’t approved its general use.
Ebola is the same story. Take ZMapp, a combination of antibodies designed to block the virus from replicating.
Citing safety concerns, the FDA ordered the drug’s maker, Mapp Biopharmaceutical, to stop testing in July — just days before the Ebola outbreak. Now, of course, the FDA is letting people use it.
The same goes for an anti-viral drug (TMK-Ebola) made by Tekmira Pharmaceuticals. The FDA suspended research in January because of safety concerns. It changed course only after Ebola killed thousands.
Part of the problem: FDA scientists receive no reward for approving breakthroughs, but suffer public anger if but one person dies because a drug is misused. The price we pay for this culture of caution rises every day.
Africa will have to spend billions to treat those infected, rebuild health systems and bury the dead. Here at home, public officials find themselves a step behind Ebola.
As they lose our confidence in their ability to respond to biological threats, they blame the Ebola crisis on — what else — budget cuts.
Asked why there is no Ebola vaccine, National Institutes of Health Director Francis Collins claimed we’d have one if not for NIH budget cuts. Nonsense: NIH funding for infectious diseases has doubled since 2001. But in 13 years, it developed three (ineffective) vaccines.
Nobel Laureate microbiologist Joshua Lederberg noted, “The single biggest threat to man’s continued dominance on the planet is a virus.” The second-biggest threat: a federal culture that rewards the delay of medical progress.
Ultimately, Congress must change the FDA’s mission and bureaucratic culture. Reviewers shouldn’t be allowed to use science to keep new technologies from doctors and patients.
We must force the FDA to focus on accelerating innovation and stop “protecting” us to death.  Read More & Comment...

Opioid Abuse Deterrence and a Step in the Right Direction

10/19/2014 07:11 PM |

Abuse deterrence will take many forms.

In advance of the FDA’s upcoming two-day meeting on opioid pain medicines and abuse-deterrent technologies, some good news. The agency has approved an updated label for the opioid pain medicine Embeda to include abuse-deterrence studies.

Embeda’s updated label states that it has properties that are expected to reduce abuse via the oral and intranasal (i.e., snorting) routes when crushed. However, abuse of Embeda by these routes is still possible. The updated label also includes data from a human abuse potential study of intravenous (IV) morphine and naltrexone to simulate crushed Embeda. (It is unknown whether the results with simulated crushed Embeda predict a reduction in abuse by the IV route until additional post-marketing data are available.)

Per Bob Twillman, Director of Policy and Advocacy, at the American Academy of Pain Management. “Prescription opioids are an important treatment option for people with chronic pain. However, misuse and abuse of opioids in the U.S. is a serious societal concern, which is why the development of abuse-deterrent formulations of these medicines is a high priority,” said “All opioid medications, including morphine products, have the potential for abuse. We believe that anything that can be done to reduce this risk is a significant development for healthcare providers and their patients.”

Embeda capsules consist of extended-release morphine sulfate and sequestered naltrexone hydrochloride, an opioid antagonist. Naltrexone is intended to remain sequestered when the product is taken as directed. The in vitro and pharmacokinetic data demonstrate that crushing Embeda pellets results in the simultaneous release and rapid absorption of morphine sulfate and naltrexone hydrochloride.

Per Pfizer, “More than one-third of extended-release opioids prescribed are morphine, and Embeda is the first extended-release morphine with the potential to reduce abuse via the oral and intranasal routes when crushed,” said Dr. Steven Romano, senior vice president and head, Medicines Development Group, Pfizer Global Innovative Pharmaceutical Business. “Pfizer believes that abuse-deterrent products, like Embeda, are important to help address the growing public health problem of opioid abuse in the U.S.”

It’s an important step in the right direction.

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The Dangerous Myth of Sci-Fi Innovation

10/17/2014 07:10 AM |

I’ve just returned from Dubai (no, I didn’t do any shopping) where I was honored to speak at the Economist’s conference on Health Care in the Middle East: Evolution and Reform. My talk was on “Enhancing Public Health Through Innovation.”

One of my main points is that innovation doesn’t happen in a vacuum. People make it happen – and it’s an ecosystem: academics, manufacturers, physicians, patients, pharmacists, hospitals, caregivers, and government. If we allow ourselves to believe that innovation happens in bursts of “eureka” moments, we do a disservice to those who advance progress through a lifetime of work and through important failures. Innovation happens incrementally – and it’s expensive. The myth of “eureka innovation” is dangerous science fiction.

In the majority, the role that government plays in advancing innovation isn’t, as many think, the science that comes from institutions like the National Institutes of Health (although the NIH makes important contributions), it’s in being a public health partner across the board – and that includes medicine regulatory policy.

So, what is the role of regulators in advancing healthcare innovation? Regulators can be partners in innovation three ways: Through robust oversight. Through active collaboration. And, most importantly, by being an innovation enabler

And regulatory predictability is Step 1 in being an innovation enabler. And one aspect of predictability is an even playing field when it comes to quality. There can be only one level of quality for medicines – whether they be of the innovator or generic variety.

There must also be a dedication across the healthcare ecosystem to safety and quality in the post-market environment – more robust and actionable pharmacovigilance.

There must be the recognition that new medicines enhance, extend, and save lives and, as such, should be reviewed and licensed with all due speed.

There should be a recognition that medicines regulation must never be an arm of domestic industrial policy.

And, finally, that government’s role as an innovation enabler must go beyond words to deeds.

In the words of the American poet, John Andrew Holmes, “Speech is conveniently located midway between thought and action, where it often substitutes for both.”

The most important role government can play in supporting and advancing innovation is to enable action.

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