Latest Drugwonks' Blog
Are Politics Driving the CDC’s Pending Decision on an Infant Meningitis Vaccine?
For decades, the Centers for Disease Control and Prevention has championed immunization against a host of deadly diseases. But, now, in a sudden reversal, CDC is signaling it may not recommend the routine administration of a proven new vaccine to prevent infant meningitis, an often fatal disease.
This briefing will feature the release of brand new research evaluating the disparities in how government programs place economic value on human life, as well as testimonies from health care and FDA experts.
Please join us as we examine how and why the CDC may be restricting access to a new meningitis vaccine for infants, and the impact this may have on patient care.
Special Remarks By
The Honorable Michael C. Burgess, M.D.
US House of Representatives (TX-26)
Panelists:
Christopher Stomberg, Ph.D., Partner, Bates White Economic Consulting
Gretchen Moen, RN, MS, CPNP-Pediatric Nurse Practitioner, Eagan Child and Family Care
Richard G. Judelsohn, MD, FAAP-Medical Director, Erie County Department of Health
Peter Pitts, President & Co-Founder, Center for Medicine in the Public Interest
Moderated by Merrill Matthews, Ph.D., Institute for Policy Innovation
Event Details:
Friday, October 14, 2011
10:00 am to Noon
Rayburn 2325
Rayburn House Office Building
Capitol Hill, Washington DC
Complimentary refreshments will be served
*This event is widely attended
RSVP to Erin Humiston
erin@ipi.org or (972) 874-5139
Are Politics Driving the CDC’s Pending Decision on an Infant Meningitis Vaccine?
For decades, the Centers for Disease Control and Prevention has championed immunization against a host of deadly diseases. But, now, in a sudden reversal, CDC is signaling it may not recommend the routine administration of a proven new vaccine to prevent infant meningitis, an often fatal disease.
This briefing will feature the release of brand new research evaluating the disparities in how government programs place economic value on human life, as well as testimonies from health care and FDA experts.
Please join us as we examine how and why the CDC may be restricting access to a new meningitis vaccine for infants, and the impact this may have on patient care.
Special Remarks By
The Honorable Michael C. Burgess, M.D.
US House of Representatives (TX-26)
Panelists:
Christopher Stomberg, Ph.D., Partner, Bates White Economic Consulting
Gretchen Moen, RN, MS, CPNP-Pediatric Nurse Practitioner, Eagan Child and Family Care
Richard G. Judelsohn, MD, FAAP-Medical Director, Erie County Department of Health
Peter Pitts, President & Co-Founder, Center for Medicine in the Public Interest
Moderated by Merrill Matthews, Ph.D., Institute for Policy Innovation
Event Details:
Friday, October 14, 2011
10:00 am to Noon
Rayburn 2325
Rayburn House Office Building
Capitol Hill, Washington DC
Complimentary refreshments will be served
*This event is widely attended
RSVP to Erin Humiston
erin@ipi.org or (972) 874-5139
It is no use to blame the looking glass if your face is awry. -- Nikolai Gogol
The survey found that FDA regulatory challenges were most frequently cited as having a significant effect on investment trends.
Blame the FDA? It’s a convenient excuse when sponsors aren’t transparent (I hesitate to say “honest”) about their interactions and communications with the agency.
For example, upon receipt of a Complete Response Letter, many companies issue public statements to the effect of, “We were shocked! The FDA never said there were any problems. We were most definitely expecting product approval.”
Some companies will blame the FDA with excuses like, “The FDA won’t allow us to discuss the letter” or “The FDA will be upset if we release the letter.” Hogwash.
And when you read the CR, all of a sudden it seems there is often blame to be shared. But, since CRs (and all FDA-sponsor communications) are commercial confidential, sponsors rarely (if ever) offer them with the media or, for that matter (and germane to the NVCA survey), the investment community. Why is that?
Might it be that sponsors make mistakes and that there is (de minimus) blame to be shared? Might it be that companies sometimes try to game the system (accelerated approval issues come to mind) – and fail? Blaming the FDA when corporate strategy flounders is convenient when the agency cannot release (in its own defense) any information.
Might Complete Response Letters be made public? Consider this statement by FDA Commissioner Hamburg when asked whether FDA is discussing unilateral disclosure of information about drug development programs even if a sponsor objects, “It’s a discussion that we’re having, and I think it’s a partnership working with industry. We need to move in directions that make sense, that will have value added, and where everyone understands the expectations and the opportunities.”
Here it is straight from the Bob Temple:
“If I were a reporter, I would say, ‘if you don’t show me the letter I don’t even want to talk to you.’ That’s what I would say.”
Wisdom of Temple or Temple of Wisdom, no matter how you slice it, it equals the FDA’s strong support for transparency.
Blame the FDA? Perhaps. But the fault, dear Brutus …
Take your life in your own hand and what happens? A terrible thing: No one to blame. -- Erica Jong
The NVCA also noted that, after FDA issues, the most potent challenge to investors are reimbursement issues.
And that’s another story.
But frankly it reads like a publication edited, rewritten and vetted by the FDA's political minders and not the work of scientists. The glossy document spends half it's pages extolling the virtues of activities that have nothing to do with accelerating the development of new products through cheap and easy to use tools and metrics. Rather, we get a roll call of Obama administration initiatives such as Startup! America, the FDA Small Business Liaison Program, Young Entrepreneurs, Partnering with the Small Business Administration and programs with one time funding from the stimulus bill. Taken together they are a Potemkin Village to divert attention to the increasing regulatory uncertainty flowing from comparative effectiveness research requirements, projected cuts in reimbursements and other features of the new health care law that undermine innovation.
When the report discusses activity that the FDA will undertake to 'drive innovation' it largely restates work already underway. To be fair, efforts to integrate the use of biomarkers, adaptive trial designs, etc., that could accelerate development of drugs and devices takes time. Often companies, fearing it could actually doom a product by using new approaches, simply load up on old ones. But by the same token when you have the FDA demanding more safety data on the back and front end of the regulatory process and certain divisions change endpoints, revoke approvals and raise the bar so high as to make approval unlikely -- think drugs for obesity and diabetes and Avastin -- a 37 page brochure celebrating 'regulatory science' and 'partnerships' should make anyone who follows the FDA highly skeptical of real progress. Which means that specific initiatives to accelerate targeted treatments and combination products -- initiatives that were launched in 2004 under the Critical Path Initiative -- will need it's own targeted effort to be adopted.
And even here driving innovation is undermined by the stealth effort to turn the FDA into an accomplice of those seeking to use comparative effectiveness research as rationing tool:
"FDA has launched the Partnerships in Comparative Effectiveness Science (PACES) program to support the development of new mathematical methods to support patient-centered outcomes research. PACES provides funds to pilot out the technical, infrastructure, scientific and legal constructs that will be used as foundations for science computing communities involving FDA scientists and data. These activities will support scientifically sound assessments of medical interventions consistent with FDA’s public health responsibilities."
The PACES program is nothing but the mathematical manipulation of claims data -- data that tells you nothing about differences in patients -- to create the illusion that you are truly measuring differences in patients. The FDA's solicitation for the PACES program begins:
"CER could extend beyond the application of specific intervention methods to controlled clinical trials or within specific health care settings. For example CER could be used to better understand what interventions work best for individuals and subgroups within populations."
Let it be noted that there is already a lot of this sort of analysis included in clinical trials already. So why do we need another initiative of this nature? The solicitation explains:
"Both pre- and post-approval data collected and housed at the FDA can be combined with other datasets on long term health outcomes that reside in other agencies or the private sector. As personalized medicine develops, FDA expects an increase in the need for comparisons across products (and associated delivery mechanisms) to define how these products should be used in combination for individualized health care before and after FDA approval."
Does this sound like an requirement that will speed up product development. Why should clinical trials to compare response across products (CER) be a requirement for FDA approval? Why should it be a requirement after FDA approval? The additional cost and time of such trials will be hugely expensive. At least $100-$200 million per product. And by requiring the use of algorithms derived from claims data, the FDA guarantees that detection of no differences when in fact they exist in the real world.
Worse, the FDA's Janus project, an effort to standardized the submission of patient-level data to expedite biomarker development and promote smaller trial designs is being hijacked for CER purposes (from the PACES solicitation):
• Component 1- The Contractor shall plan and organize the CER project with FDA stakeholders, including participation in annual FDA-hosted planning workshops (three workshops, two days each) to define candidate CER questions and priorities, analyses strategies and datasets to be utilized.
• Component 2- The Contractor shall provide training on methodologies developed to FDA staff during two of the FDA-hosted planning workshops.
• Component 3 - The Contractor shall identify or develop appropriate analytic methodologies for CER and apply them to FDA and/or other data; develop new scientific computing strategies and hone existing computational strategies to perform these CER analyses. The Contractor shall prepare reports and manuscripts of the results suitable for publication in scientific journals.
• Component 4- The Contractor shall develop innovative clinical trial design strategies for prospective CER clinical trials and analyses of healthcare data including providing formal recommendations for best practices for submission of studies to the FDA when they involve product comparisons. These strategies and recommendations shall be documented in reports and manuscripts suitable for publication in scientific journals.
Even if all the happy talk about driving innovation was real, it could all be undermined by this effort to use FDA's most significant data program into a tool for rationing. It will drive innovation all right, off a cliff.
I believe that senior leadership at the FDA from Commissioner Hamburg on down have the best of intentions. And I know for a fact that Janus and other activities undertaken in response to the Critical Path were to be used to bring new and better products to market with greater speed and at less expense. It's those who want to slow down the development and adoption of innovation and who have politicized the FDA with CER claptrap that are at fault. They think they know better and their monstrous certainty will kill innovation for decades to come.
The FDA has released a new “blueprint” document, “Driving Biomedical Innovation: Initiatives for Improving Products for Patients,” containing immediate steps that can be taken to drive biomedical innovation.
Addressing the sustainability of the medical product development pipeline, which is slowing down despite record investments in research and development, FDA Commissioner Peggy Hamburg comments, “America is at an important crossroads, where the science before us presents unprecedented opportunities to create new and better medical products and to promote better health for the public. Our innovation blueprint highlights some of the initiatives FDA will be implementing to ensure that these opportunities are translated into safe and effective treatments that can help keep both American patients and American industry healthy and strong.”
The report’s proposals stem from a review of FDA’s current policies and practices, as well as months of meetings with major stakeholders nationwide, including key industry leaders, small biotech, pharmaceutical and medical device company owners, members of the academic community, and patient groups.
The blueprint focuses on implementing the following major actions:
• rebuilding FDA’s small business outreach services
• building the infrastructure to drive and support personalized medicine
• creating a rapid drug development pathway for important targeted therapies
• harnessing the potential of data mining and information sharing while protecting patient privacy
• improving consistency and clarity in the medical device review process
• training the next generation of innovators
• streamlining and reforming FDA regulations.
The complete report can be found here.
An important read. But it’s got to be more than rhetoric.But then again, The Shink probably has insights we cannot share. He was the ace reporter of Marijuana Business Reporter, a job he was recruited for. Actually he found the job on Craigslist. Which is fitting in so many ways. Nothing like finding a job while checking out garage sales and massage parlors. But I digress. In addition, The Shink broke new journalist ground with his Irony Supplement blog. Get it? Irony (as in iron) Supplement? If Twain were alive he would be jealous. Here is a sample of The Shink's writing skills and prophetic reporting gifts:
Why Bernard Madoff Should Face The Death Penalty
A rational discussion as to why disgraced financier Bernard Madoff should face capital punishment.
Father Limbaugh?
A comparison of Rush Limbaugh and Charles Coughlin's political tactics.
And my favorite..
Hurry Up And...@*&%# WAIT?!?!?
An examination of the media's dementia in covering the stimulus package. (This is where The Shink attacks the media for doubting the benefit of the stimuls and not believing the recession was over in 2009 when The Shink said it was.)
Why do I bother with such a bottom-feeding paranoid? Because he writes for what once was and should be a respectable online publication that cares about the mindset and journalistic stability of it's editors.
I hope The Shink has to hit Craigslist for work real soon. He shouldn't have a problem getting another job. After all, the recession is over, isn't it? Or maybe BIG PHARMA is cooking up that crisis too.
“I don’t want any yes-men around me. I want everybody to tell me the truth even if it costs them their jobs.” -- Samuel Goldwyn
Important article in the October edition of Southern California Physician, “The Devil in the (Academic) Details.”
Some verbatims:
Since they are not employed by a pharmaceutical company nor do they receive incentives from them, then they must be completely unbiased, right?
Not so fast.
“Of course more information is always better,” says Peter Pitts, President of the Center For Medicine in the Public Interest, a nonprofit, nonpartisan research and educational organization that seeks to advance the discussion and development of patient-centered health care. “But to argue that academic detailing is “pure” is absurd. The information being shared is only as good as those sharing.”
Pitts says that the Patient-Centered Outcomes Research Institute—a research organization dedicated to the support and promotion of comparative clinical effectiveness research, which was created through the Patient Protection and Affordable Care Act—plans to offer Continuing Medical Education Credits to physicians who meet with academic detailers. The practice is potentially dangerous if the academic detailers are acting in the interest of government’s push to drive down health care costs, for example by promoting generics over other new types of drugs.
The Real Bottom Line
Whether you are a fan of academic detailing or not, the bottom line is that federal and state governments are driving and funding the effort. And it is clear that they are tasked with reducing the cost of health care, even if it is not necessarily in the best interest of the patients.
If you get a visit by an academic detailer, keep in mind that they may not be any less biased than a pharmaceutical representative and they definitely do not have to follow the same rules.
The complete article can be found here.
Last week the wsj editorial page artfully described how comparative effectiveness research is being used to justify government deciding what medical technologies will be developed and what treatments we will receive:
What the editorial implied but not discuss is that CER methodology is used to 1) establish that a drug will work but that 2) the cost of the treatment is "not good value for money". Which means 'society', meaning government-paid CER munckins say it's not worth paying for the treatment. In 1994 NICE decided that the cost of Cerezyme, the first treatment for Gaucher's for the ‘average’ Gaucher’s disease patient exceeds the normal upper threshold for cost-effectiveness seen in NHS policy decisions by over ten-fold."
In a similar vein, Alan Garber suggests that individual response to new treatments should not be considered in determing what benefits health plans should cover under Obamacare because if we add every new techology that "is proven effective" would result in a "costly bundle of services." Cue the CER experts to decide what services and treatments are valuable and which are not, even if they help people live longer healthier lives. Garber also urges the US to adopt the CER tools used around the world.
WIth that in mind, here's how NICE, the Nirvana of the CER crowd handles Benlysta, the first new drug in 50 years to treat lupus:
"Lupus drug Benlysta from GlaxoSmithKline PLC (GSK, GSK.LN) and U.S.-based partner Human Genome Sciences Inc. (HGSI) doesn't represent value for money and should therefore not be made available on the publicly-funded National Health Service, Britain's healthcare watchdog NICE said on Friday.
Benlysta, or belimumab, is the first new lupus treatment in 50 years. It won U.S. marketing approval in March and European approval in July. Lupus is a chronic autoimmune disorder, with symptoms ranging widely in type and severity. An estimated five million people worldwide have the disease.
In draft guidance, the U.K.'s National Institute for Health and Clinical Excellence said, "NICE's independent appraisal committee has looked very carefully at the evidence provided on the use of belimumab for treating SLE [Systemic lupus erythematosus], including the views of people with the condition, those who represent them, and clinical specialists.
"The evidence considered did not persuade the committee that belimumab was good value for money compared to standard care, as the cost per year of improved health is very high."
NICE also said Benlysta should be compared with rituximab, sold by Roche Holding AG (ROG.VX), because some people with severe lupus currently receive rituximab, although it isn't licensed for this use. However, NICE noted there were no reliable data to show the relative efficacy of the two drugs.
"Whilst recognizing the severity of the disease, the committee concluded that based on this evidence, belimumab could not be considered a good use of NHS resources. We welcome comments on this draft recommendation as part of the consultation," the agency said. The draft guidance is now open for consultation before further decisions on the medicine are made.
Glaxo said it was disappointed by the agency's stance.
"This initial decision is very disappointing for patients living with lupus who currently have very limited treatment options and we will do everything we can to change NICE's mind in an effort to ensure they get access to Benlysta on the NHS," said Simon Jose, General Manager, GlaxoSmithKline UK.
"NICE's current methodology means that it is difficult to meet their cost-effectiveness threshold given the nature of the disease and the comparison with unlicensed or cheap generic medicines. We had hoped that our offer of a patient access scheme would help overcome these challenges," Jose added.
Navid Malik, an analyst with Merchant Securities, echoed that surprise, saying, "It seems incredulous for NICE to say that just because patients are on Rituxan for Lupus, they should not be reimbursed and given Benlysta, (as) Rituxan doesn't work in Lupus and yet Benlysta has been shown in two phase III trials to work."
Proposed Swedish Pharma Pricing Model Suggests VBP Is Not Enough To Control Costs
The Swedish government published on Sept. 22 a framework directive designed to revolutionize the pricing, reimbursement of and access to patent-protected pharmaceuticals.
This will alarm pharma manufacturers that the VBP systems now being implemented by countries such as Germany, France and the U.K., simply may not go far enough in terms of controlling costs and that harsher measures may follow.
The directive examines the possibility of introducing a mechanism that would ensure that drug prices in Sweden are below or the same as prices in other comparable countries such as Norway, Great Britain and Denmark - known as a reference pricing system, the government suggests it could lead to annual savings of between SEK 500 million ($74 million) to SEK 2 billion ($294 million).
Sweden also is examining the possibility of introducing reimbursement limits for certain products - which should generate savings of SEK 900 million over a three-year period. The Rapporteur's report will be presented to government on Sept. 1, 2012.
"This implies that some of the proposals could be implemented as soon as in the beginning/middle of 2013," said Anders Blanck, Director General of the Swedish pharmaceutical industry association, Lif.
tinyurl.com/4y67ndh
Today's news that Blue Shield of California will no longer pay for Avastin to treat breast cancer, though "exceptions may be considered on a case-by-case basis," makes a new nationwide survey by the Coalition of State Rheumatology Organizations (CSRO) big news. The survey shows broad dissatisfaction with the insidious practices of preauthorization and step therapy – specifically the ways in which it impacts the ability of physicians to treat patients.
(Prior authorization, also known as pre-authorization, pre-certification or prior notification, is an extra set of steps some insurance carriers require before determining whether they will pay for a medical service or prescription medication. The physician, or other medical provider, is required to obtain approval from the insurance carrier before the carrier will agree to cover the cost of the medical service or prescription medication. Step therapy, also referred to as “fail-first,” requires patients to “fail’ on one or more less costly medications before the health insurance carrier will agree to cover a more expensive medication, even if a physician thinks it is a better option for the patient.)
“Rheumatologists around the country have increasingly voiced their concerns about the impact of health insurance protocols such as prior authorization and step therapy on patient care,” said Reuben Allen, CSRO Executive Director. “These practices are stripping rheumatologists of the ability to direct the most appropriate and effective courses of treatment, which causes patients to suffer delays or outright denials of proper medical care. Individualized treatment plans that can restore, enhance, and preserve quality-of-life over time are essential to rheumatology patients and their struggle against autoimmune and destructive arthritic disorders.”
Specific findings of the CSRO survey include:
Nearly 99% of rheumatologists surveyed say they have had to alter treatment plans including changing prescription medications to accommodate restrictions imposed by patient health insurance carriers;
91.5% of survey respondents say prior authorization has a “negative” to “very negative” effect on their ability to treat patients;
Nearly 97% of rheumatologists surveyed agree, “There should be enforceable legislation to regulate restrictions that insurance companies place on health care providers in regards to treatment modalities they prescribe for their patients;”
Nearly 98% of survey respondents agree that decisions about what medications are best for a patient should be made by the patient’s own health care provider and not by the health plan or insurance company;
Nearly 73% of respondents say they are only “sometimes” or “rarely” able to easily determine what procedures will be covered by a patient’s health plan at point-of-service;
52.2% of rheumatologists surveyed say they have considered re-establishing their practices as fee-for-service only because of prior authorization constraints.
Currently, prior authorization and fail-first protocols are primarily paper-based, and non-standardized. Each insurance carrier has its own set of requirements, which can vary among plans, even within the same carrier’s portfolio of coverage options. To meet prior authorization requirements physicians must complete a time-consuming series of faxes, phone calls, emails, input of data into insurance carrier web sites and, in some cases, letters.
Prior authorization should be standardized and improvements in the current process can be made by the adoption of a universal prior authorization form;
Electronic prescribing platforms are provided on neutral and open platforms that do not advance the commercial interests of any particular participant (e.g., health insurers, hospitals, pharmacy benefits managers, pharmaceutical companies, etc.) to the potential detriment of the patient;
Adjudication of prior authorization requests occurs within a reasonable time frame (hours as opposed to days or weeks); and communication between physicians and payers should be on a peer to peer basis;
Electronic prescribing platforms include access to information about all FDA-approved medications and medical services without restrictions;
Complete, up-to-date information about prior authorization and fail-first criteria is available through electronic prescribing platforms at point-of-service;
Prior authorizations should not be required on a repeated basis. It should only be necessary with a change in medication dictated by a change in clinical status;
Prior authorization should not be necessary for low cost medications; for example, prednisone and methotrexate.
“Physicians are responsible for the administrative costs associated with meeting prior authorization and fail first requirements.
“Prompt diagnosis and specially tailored treatment can improve the long-term outcomes of patients with rheumatologic diseases," said CSRO's Allen. "State legislatures and insurance commissioners should take appropriate steps to ensure that patients suffering from chronic rheumatic diseases and chronic pain do not have to needlessly suffer.”
The complete CSRO survey can be found at http://www.csro.info/
In April, the Food and Drug Administration approved a first-of-its kind study to test whether marijuana can ease the nightmares, insomnia, anxiety and flashbacks common in combat veterans with post-traumatic stress disorder.
But now another branch of the federal government has stymied the study. The Health and Human Services Department is refusing to sell government-grown marijuana to the nonprofit group proposing the research, the Multidisciplinary Association for Psychedelic Studies.
The HHS official in charge of the review, Sarah A. Wattenberg, declined to answer questions when reached by phone.
Can you imagine the hue and cry if this had happened under the previous administration?
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.
