Latest Drugwonks' Blog

Democrats are blaming Ebola on budget cuts to NIH made by Republicans.  Francis Collins agrees.  They may not be in cahoots but they are both way wrong. 

The facts:

NIH budgets have increased since 2001 except for 2011.  

The NIH - through the National Institutes for Allergies and Infectious Diseases -- has received $11 billion for research and development to find diagnostics, drug and vaccines for Ebola and other lethal pathogens.  In fact in 2010,  NIAID received an additional $304 million when Congress diverted money for buying vaccines from the Bioshield procurement fund.  And each year the budget was never cut, except for 2011.

If you go to clinicaltrials.gov and type in Ebola, Marburg or other viral hemorrhagic fevers and limit your search to NIH sponsored trials here's what you get.  

The only study completed that actually reported results was a test of yellow fever vaccine on people with excema 

The NIH developed 7 Ebola vaccines.. None of which worked.   $11 billion.  7 vaccines.  No results. 

There are several drugs that do work.  TKM-Ebola and three drugs from a company called Sarepta.  AVI-7537 has already shown effectiveness in non-human primates against the Zaire Ebolavirus, the species implicated in the current outbreak. Moreover, Sarepta had been conducting Phase 1 safety trials with the drug alone and together another Ebola-directed PMOplus molecule (AVI-7539, with the combination called AVI-6002).   Sarepta's work was being funded by the Department of Defense, NOT NIH.   The administration and Congress could have transferred the money from Bioshield to the DOD work under a cooperative research agreement but it did not.   The dough went to NIH. 

According to a recent article in Forbes, Sarepta " does indeed have clinical trial-quality drug on hand. The Marburg program that has continued “uses the same backbone chemistry,” so the safety studies that are continuing with that drug at higher human doses could be applied to AVI-7537.

The company's CEO Chris Garabedian told Forbes: “We’re here to raise awareness that we do have a technology that might be helpful, and that we do have drug substance on hand if we received a request from a government agency,” said Garabedian. “We, of course, would have to get the appropriate waivers and approvals from the Department of Defense who supported the development of this compound as well as the FDA in terms of an emergency use authorization.”


In his article in The Hill, entitled "FDA Should Play Key Role in Lower Drug Costs", former Congressman Ed Towns gets it wrong.  He claims drug companies want the FDA to make it harder for doctors to prescribe biosimilars."  He wants the FDA to hand over clinical data developed by innovator firms to generic companies well before the full patent life of the product expires.  

Actually it's the small biotech startups and medical innovation that would be screwed if Towns has his way 

My late colleague John Vernon Jr. explains why cutting IP protections to biologics will hurt Americans (from is article EXPLORATION OF POTENTIAL ECONOMICS OF FOLLOW-ON BIOLOGICS AND IMPLICATIONS FOR DATA EXCLUSIVITY PERIODS FOR BIOLOGICS. )

"A minimum of 17 years of data exclusivity or data protection is required to provide the necessary incentives for continued biotech R&D investments...evidence suggests that these incentives are in fact inadequate for pharmaceuticals in light of the social rate
of return to both pharmaceutical and medical R&D."

Vernon concluded that at present, the U.S. is under-investing in new medicines. "One study suggests that for every $1,345 invested in pharmaceutical R&D, a U.S. life year is gained, on the average. "

This return could be even higher if FDA regulation did not ratchet up the cost and complexity of drug approvals. We can find common ground on this important point!

Spears Speaks

  • 10.10.2014

In 1992, Congress created a program -- known as "340B" -- to help caregivers serving disproportionately large numbers of low-income beneficiaries and uninsured patients. Under 340B, drug manufacturers are required to sell their products at a discount to such institutions. The discounted prescriptions are dispensed either through the caregiver's in-house pharmacy or through a contractual arrangement with an outside pharmacy.

340B has a noble cause. And many of the medications discounted through 340B do in fact go to clinics, hospitals, and medical facilities providing care almost exclusively to uninsured and poor patients.

However, some 340B participants are exploiting the program and the vulnerable patient populations 340B was intended to help are often still stuck struggling to gain access to affordable pharmaceuticals.

To wit, Pharmaceutical Research and Manufacturers of America (PhRMA) Executive Vice President and General Counsel, Mit Spears has issued the following statement:

“PhRMA strongly supports the 340B program, which is intended to help vulnerable and uninsured patients obtain access to life-saving medicines. We are committed to working with all stakeholders to improve the program to ensure it benefits those it was intended to help. To achieve this important objective, it is critical the program operate in a manner consistent with the clear direction of Congress. 

“At issue is the Health Resources and Services Administration’s (HRSA) interpretation of the 340B orphan drug exemption, enacted as part of the Affordable Care Act (ACA). The ACA significantly expanded the type of entities that can access 340B discounts for prescription drugs. To preserve incentives to invest in research and development of new treatments for rare diseases, the ACA expressly exempts manufacturers from having to provide these discounts on orphan drugs to newly eligible providers.

“After the Federal District Court of the District of Columbia vacated the HRSA July 23, 2013 rulemaking regarding the 340B orphan drug exemption, in July 2014, the agency issued the exact same rule, but labeled it ‘interpretive.’  HRSA’s action in this regard is unlawful.

 “While we value the hard work and efforts of all agencies, it is important federal agencies recognize and work within the bounds set by Congress. PhRMA is therefore filing suit against the U.S. Department of Health and Human Services to challenge its second attempt to issue a rule conflicting with the plain language of the statute.

Go get’em, Mit!

In case you missed the FDA’s Viewpoint commentary in JAMA, here it is:

Addressing Prescription Opioid Overdose: Data Support a Comprehensive Policy Approach

And it’s not about banning Zohydro:

“The risk in singling out a single drug like Zohydro ER in this complex, multidrug epidemic is that resulting policy is unlikely to have an effect on the underlying causes—the abuse of multiple opioids and other drugs and inappropriate prescribing.”

The first sentence of the concluding paragraph says it all, “The problem of opioid overdose demands well-informed policies.”

Not knee-jerk. Not political. Not bowing to threats. Well-informed policies.

Attention must be paid.

Center for Medicine in the Public Interest Challenges Accuracy of “60 Minutes” Cancer Story
Leading medical innovation think tank claims CBS is ‘dead wrong’

New York, NY (PRWEB) October 08, 2014

The Center for Medicine in the Public Interest (CMPI.org– a non-profit research organization specializing in promoting medical innovation) criticized CBS’ “60 Minutes” segment on cancer treatment prices as not just distorted but ‘dead wrong.’ “‘60 Minutes’ ignores the contribution new medicines make in reducing health care costs, improving wellbeing and saving lives,” said Robert Goldberg, Vice President for Research of CMPI. “Featuring physicians that argue that treatment prices are too high does not change the fact that the cost of cancer drugs are about 12 percent of what health insurers spend on cancer or that new cancer drugs actually save health insurers money. Yet insurers are increasing oral therapy cost-sharing requirements to actively encourage patients to use infusible products.

While a Milliman study found that shifting to co-insurance would only add about $2 per member per month in private health plans, 60 Minutes never discusses this solution. Instead, 60 Minutes remains silent about the cost shifting and has never advocated for co-pay reforms that could relieve the burden on patients.
Spending on cancer treatments has climbed from $24 billion in 2004 to about $40 billion today. But such treatments represent only 0.6 percent of U.S. healthcare spending, and that proportion has been fairly consistent for the last several decades.

Such medical innovations were largely responsible for a 40 percent increase in cancer survivors (from 9.8 million to 13.6 million) since 2004. Since 2004 the use of new therapies saved $188 billion on hospitalizations, and avoided nearly $100 billion in lost productivity each year. Since 1990, new cancer drugs have helped generate 51 additional years of life, worth nearly $5 trillion.

A doubling in the use of new-targeted therapies will raise the amount spent on medicines as a percentage of total health care spending. But that increase must always be compared to what would be spent on healthcare, disability, unemployment and rehabilitation in the absence of medical innovation.
Peter Pitts, the President and Co-Founder of CMPI noted, “New medicines almost always reduce the cost of living longer and healthier lives and increase the value of such improvements. It is disappointing that “60 Minutes” failed to investigate why some health plans have deliberately made these innovations unaffordable by requiring patients to pay up to half their costs. And it is disheartening to know that doctors are more interested in attacking innovators than in defending patients from such cost shifting.”

Pitts also remarked, “We are happy to talk about the prices of cancer therapies, but only in the context of value and the rising cost and prices of other healthcare services that constitute a substantially bigger portion of total spending. In addition, the discussion should only be in the context of what can and should be done to cut the cost of developing new therapies through smarter, faster regulation.”

CMPI has a website—valueofmedicalinnovation.org—that provides a balanced view of the role new medicines play in our lives.

The Center for Medicine in the Public Interest is honored to join the US Pain Foundation and other patient-centered organizations in offering our support to the FDA in general and Commissioner Hamburg specifically for savvy and strategic management of pain medicine regulation.

Why now? Well for starters because of the unfair, unbalanced, ad hominem and just plain erroneous attacks on the agency’s efforts.

It’s all spelled out in this letter of support just sent to HHS Secretary  Burwell. It’s designed for impact.

Have a look and please share. This issue is too important to allow the lunatic fringe to drive the agenda.

BioCentury reports that the EMA's management board unanimously adopted its clinical data transparency policy, to be introduced in two phases starting Jan. 1, 2015. 
In the first phase, EMA will publish clinical reports supporting MAA submissions while excluding individual patient level data. 
The policy will apply to any MAA submitted after Jan. 1, 2015.The agency noted that as a practical matter, this would mean the first data for newly approved medicines would become available in 18 months, allowing for review time and approval by the European Commission. For line extensions and extended indications of approved drugs, EMA will publish clinical data for applications submitted as of July 1, 2015. 
EMA said the second phase, disclosure of individual patient level data, will follow a consultation with stakeholders to determine how such data can be made available in compliance with privacy and data protection laws. 


The agency said commercially confidential information will be redacted at its discretion with input from sponsors, noting "the starting point of the redaction principles is that clinical reports do not, in general, contain CCI."
After reviewing stakeholder concerns that clinical data would only be accessible by viewing it on a screen, EMA amended the policy to allow identified users to download, save and print trial data for academic and non-commercial research purposes. The policy provides two permission routes for access, one for "general information purposes" by individuals and another for "academic and non-commercial research purposes."
 EMA also published a document addressing frequently asked questions relating to the policy.

Speaking of opioids, here are three interesting items from today’s edition of DIA Daily. One thing, however, that remains absent in the debate over how to address the issue is the Hamburg Manifesto – enhanced physician education. Now it's time for the DEA to step up to the plate and mandate specific education as a must-have for prescribing rights.

So far, alas, only silence from the DEA on this topic. This must change.

Pharma Association CEO Offers Possible Reforms To Curb Prescription Drug Abuse.

Mark Merritt, president and CEO of the Pharmaceutical Care Management Association, writes in the The Hill (10/2, Merritt) about options in trying to abate prescription drug abuse issue in the US. Merritt offers “two basic reforms”: first, a “safe pharmacy” or “lock-in” policy in Medicare in which “the small segment of patients who are at risk of abusing opioids would choose – along with their health plan – a single, convenient pharmacy to fill their prescriptions for controlled substances.” This eliminates the possibility of “drugstore shopping,” or “the practice of filling prescriptions for controlled substances...at multiple drugstores to avoid detection.” A recent HHS OIG report called for such an approach. A second option would be to “close the loophole in Medicare that prevents Part D plans from suspending payments to pharmacies suspected of fraud or diversion.” A second HHS OIG report outlined the benefits of this potential reform.

Neurologists Urge Caution With Opioids For Non-Cancer Use.

The Los Angeles Times (10/2, Healy) reports that patients on opioid painkillers for chronic pain unrelated to cancer, such as headaches, may face a situation where the risks of taking the medications outweigh the benefits. The paper notes that patients “are more likely to risk overdose, addiction and a range of debilitating side effects than they are to improve their ability to function,” citing the American Academy of Neurologists. In a new position statement released Wednesday, the group warned that even for patients “who do appear to benefit from opioid narcotics,” doctors “who prescribe these drugs should be diligent in tracking a patient’s dose increases” and should insist as a condition of continued use “that opioids are improving a patient’s function.”

Abuse-Resistant Version Of Controversial Painkiller Unveiled.

The Wall Street Journal (10/2, Burton, Subscription Publication) reports, Zogenix Inc., the maker of the controversial opioid painkiller Zohydro ER (hydrocodone bitartrate), disclosed Wednesday it filed an application with the FDA for a modified version of the medication that it says would be harder to abuse. According to the firm, the new version will be a capsule containing a gel, making it more difficult to snort or inject. Still, some doctors noted that it could be abused by addicts, who could choose to orally ingest the contents.

My letter to the NY Times ...

To the editor:

Per  Detailing Financial Links of Doctors and Drug Makers (NYT, October 1, 2014), collaborations between physicians and industry are fundamental to advancing medicine. Without in any way diminishing the governmental and non-profit agencies that support research, it is the partnership between physicians and industry that has created many, if not most, of the major medical breakthroughs that have reduced the rates of death and other serious outcomes in recent years – as any literature search of major medical journals will quickly confirm. 

Conducting clinical research has become so rigorous and sophisticated that those who serve as consultants and investigators to industry recognize it as a major commitment or even a primary career path.  In addition, as studies are completed, physician researchers add to their professional commitment by becoming the teachers of this new information.

Critics of these collaborations see compensation of physicians as evidence of undue influence on medical practice.  But doctors, like all professionals, should be paid fairly for their time and work. The truth is that physicians who are busy with these activities are not as well rewarded as their fellow specialists in full-time clinical practice.  Adding to this remunerative divergence is that a key part of research, writing articles for publication, takes weeks of work -- often unpaid so as to avoid any suggestion of bias.

The Sunshine Act will create troubling misconceptions for and about physicians.  Payments reported for physicians by industry will likely include funding they didn’t personally receive nor will they take into account costs incurred by these physicians in paying their own staff and covering overhead expenses.  Doctors involved in industry-supported research and education may easily get discouraged and frustrated explaining these complexities to an audience already biased and sated by sensationalistic media reports of physicians “on the take.”  

And yet the Sunshine Act, paradoxically, could have a positive effect.  Despite the near impossibility of reliably interpreting all the reported data, this information might well serve as a yardstick of cooperative achievement --identifying those physicians at the forefront of medical innovation.

Peter J. Pitts

Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest

CMPI

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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