Latest Drugwonks' Blog

Expediting Expanded Access

  • 06.03.2016
  • Peter Pitts
Yesterday, the FDA clarified its expanded access policies in a series of documents including a Q&A that outlines the process of obtaining expanded access, guidance that describes how companies may charge for therapies given under expanded access programs, and a new form to request access to investigational drugs.
FDA Commissioner Robert Califf said the agency hoped to simplify and streamline the process to reduce "procedural burdens on physicians and patients."

In one document, the agency outlines requirements for physicians and companies to obtain expanded access for individual patients, or for intermediate or large-scale studies. The document notes that FDA requires IRB review of all expanded access protocols, even in cases of emergency use, as well as reporting of adverse events.
 Addressing concerns from drug sponsors that adverse event reports from expanded access programs could derail development efforts, the agency said that while adverse event reporting from expanded access protocols have been included in drugs' safety assessments in a "small number of cases," FDA reviewers are to interpret safety data in "the context in which the expanded access use was permitted."

In another guidance addressing the process of charging for investigational therapies, FDA said companies must provide evidence that a trial could not be conducted without charging, that the treatment has a potential clinical benefit, and that data obtained from the trial are "essential" to establishing the therapy's safety or efficacy. The company must also submit a calculation of cost recovery. 
In 2013 draft guidance, the agency had said companies can only charge for direct costs involved in making an investigational drug available, and only when the costs of providing the drug are "extraordinary" for the company. The new guidance reiterated both points.

FDA also finalized an Individual Patient Expanded Access Investigational New Drug Application form to speed individual requests for investigational drugs. Califf said the new, "much shorter form" should take physicians about 45 minutes to complete.

 
Ed Silverman at STATnews.com noted that the Washington state Medicaid program has been ordered to lift restrictions on coverage of pricey hepatitis C treatments, according to a preliminary injunction issued Friday by a federal judge in Seattle.
 
In a strongly worded, 12-page opinion, United States District Court Judge John Coughenour agreed with their argument. He wrote that the facts “clearly favor” their contention that state policy violates federal law. In his view, the evidence “establishes that there is a consensus among medical experts and providers that the life-saving [drugs] are medically necessary” for all hepatitis C patients.”
 
The restrictions were developed by ICER.
 
Last week ICER’s boss Steve Pearson told attendees at a meeting to vote on the value of new myeloma drugs that ICER’s recommendations are not used by health systems or insurers to make coverage decisions.
 
In fact, ICER recommended reducing access to Hep C drugs to control spending:
 
"The budget impact and cost offset figures change substantially under our second treatment
scenario in which only patients with advanced liver fibrosis are started on the new treatment
regimens, with other patients treated with existing pre‐DAA regimens."
 
And...
 
"Panel members and outside experts nearly all agreed that for both clinical and cost reasons,
not every patient with hepatitis C needs to be immediately treated with the new drugs.
Informed, shared decision‐making about the timing of treatment should be encouraged.
Given the circumstances, it is reasonable to consider prioritizing treatment with the new
drugs for patients who need urgent treatment and have some evidence of liver fibrosis but
do not have advanced liver disease.
 
The Washington State Healthcare Authority (WHCA) restricted Medicaid access to Hep C drugs by following the ICER recommendation.
 
It was sued and a federal judge ordered the WHA to make Hep C drugs available to everyone.
 
Here’s what the court said about WHA’s hewing to the ICER guidance:
 
“defendant does not come forward with any specific, credible evidence to show that its decision to apply the HCV Treatment Policy’s exclusion of DAA treatment for patients with F0-F2 fibrosis scoring is based upon medical evidence rather than solely budgetary concerns.
 
Or concerns of what ICER refers to as health system value. 
 
ICER claims the provisional health system value is grounded in “the assumption that society would prefer health care costs to grow at a rate that does not exceed growth in the overall national economy. “
 
Here’s what the court said about THAT:
 
The WHCA argues that the injunction would double the State’s Medicaid outpatient Pharmacy budget and cause them to reduce Medicaid enrollments, benefits, or provider rates to compensate for the increased expenditure in HCV treatment. (Dkt. No. 29 at 22.) The Ninth Circuit has also addressed the question of balancing the risk of irreparable harm with the risk of financial hardship for the enjoined institution. Posed with this question, the Ninth Circuit held that when “[f]aced with such a conflict between financial concerns and human suffering, we have little difficulty concluding that the balance of hardships tips decidedly in plaintiffs’ favor.” Lopez v. Heckler, 713 F.2d 1432, 1437 (9th Cir. 1983)
 
ICER’s involvement or influence on WHCA is not just intellectual.  ICER is one of three organizations that WHCA pays to conduct technology assessments:
 
“The mission of the Washington State Health Care Authority’s Health Technology Assessment Program is to ensure that medical treatments and services paid for with state health care dollars are safe and proven to work.  The program contracts for scientific, evidence-based reports about whether certain medical devices, procedures, and tests are safe and work as promoted, and an independent clinical committee of health care practitioners then uses the reports to determine if programs should pay for the medical device, procedure, or test.  The clinical committee has a legislative mandate to consider, in an open and transparent process, evidence regarding the safety, efficacy, and cost-effectiveness of the technology being reviewed.
 
In 2012, ICER was named one of the program’s three Technology Assessment Centers and completes one to three assessments a year for the agency.  To date, ICER has completed projects on cervical spinal fusion for degenerative disc disease, cardiac nuclear imaging, proton beam therapy, appropriate imaging for breast cancer screening in special populations, and bariatric surgery.”
 
ICER has tried to distance itself from the boasts it has made in the past that it serves at the pleasure of PBMs and insurers.  But as my friends at PatientsRising.com recently pointed out: ICER boss Steve Pearson told "ICER’s framework is specifically designed for payers," Dr. Steven D. Pearson, recently admitted in an interview with Evidence-Based Oncology’s managing editor Surabhi Dangi-Garimella. That statement is a direct quote and says everything patients need to know about the independence and value of ICER's tools.”
 
And now we know that ICER is a paid consultant to the government agency that used the ICER value framework to ration drugs.  My guess is ICER will not take responsibility for WHCA’s illegal drug rationing and the harm caused to patients that prompted the court’s decision. That assertion, as well as ICER's claim that it is a trusted third party should be regarded with great suspicion.  

As for patients, it's time to follow suit literally, and take legal action against other insurers and government systems that use ICER to justify illegal rationing. 

It's the Volume Stupid..and the Rebates

  • 06.02.2016
  • Robert Goldberg
An IMS report on Oncology Drug trends was released today. 

I guess it was too much to ask for the media to actually read the full report or report on the full report.  It's easier to feed into the drug spending is unsustainable and creates financial toxicity narrative..  Meg Tirrell came closest to getting it right, noting that most of the increase was due to more people taking new medicines for longer.  

The report breaks it down thusly
  • Over the past five years, the cost of oncology medicines in the U.S. increased by $15.9 billion, or 72% over the 2010 level. Over $9 billion of total growth came from the adoption of new therapies introduced since 2010 and a similar amount is due to increased volume and price of existing branded drugs.
  • Greater use of older brands – due to increasing numbers of patients receiving treatment as well as lengthening treatment durations – led to $9.3Bn in cost growth in the past five years.
Payers are also noticing the impact on drug budgets of longer treatment duration and increased numbers of patients receiving treatment.

What Meg didn't mention and should have because she brought up financial toxicity is that cost sharing for cancer drugs is that insurance imposed cost sharing soared by 30 times more than prices net of rebates..  I just compared the out of pocket data from the IMS report on Developments in Cancer Treatments, Market Dynamics, Patient Access and Value from May 2015 (page 35) with data in the report the media is writing about. 




And rebates remain about 40 percent of the total increase in cancer drug spending.  None of that money directly goes to cancer patients.  At the same time, drug companies are helping patients with out of pocket costs. 

Meg alluded to it.  Most other reporters didn't.

Rebates up.  Cost sharing up.   Someone is making a lot of money and sticking it to patients.  
 
ASCO Revised Framework Takes Slaps At ICER, Abacus

ASCO released Value Framework 2.0 yesterday.  The article “Updating the American Society of Clinical Oncology Value Framework: Revisions and Reflections in Response to Comments Received” published in the Journal of Clinical Oncology makes two changes in how it values medicines.  

First, it uses the hazard ratio – that is the “chance of events occurring in the treatment arm as a ratio of the hazard of the events occurring in the control arm. “ – as a measure of benefit.  In other words, the ASCO framework, replaces absolute increases in benefit with one that measures the probability of benefit as the standard measure

This is a direct slap in the face to Peter Bach’s Abacus and ICER both of which measures increases in overall survival without regard to how bad a prognosis is.  

Also ASCO notes: because an HR expresses a relative difference in risk, a similar HR could be derived for a modest improvement in survival that is measured in weeks or months for a tumor type with a poor prognosis or for a larger absolute gain that is produced by a highly effective therapy in a tumor type more amenable to treatment.

Slap two. 

Third, whereas the first framework devalued progression free survival, in the revision “bonus points are awarded if the test regimen results in at least a 50% relative improvement in percentage of patients alive at a time point that is at twice the median OS or PFS point for the control regimen and if at least 20% of patients receiving the control regimen are alive at this time.” 

This is likely a bar bit too high, but at least the revision framework recognizes that a month of additional average OS or PFS that is a 50 percent increase and affects 20 percent of patients is meaningful to patients.  Again, neither the Abacus or ICER has this feature. 

Slap Three

Further, the framework authors no longer publicly single out cancer drugs as the thing that will destroy the American economy.  And it goes to great lengths to distance themselves from earlier goals of making the framework the tool insurers and PBMs will use to decide which drugs to cover.  

In the 2015 version, the Task Force asserted:

“ASCO recognizes that this work has the potential to influence policymakers and payers as they consider preferred management options and evaluate the relative value of new treatments introduced into the cancer marketplace…As policymakers and payers seek ways to assure the best use of limited resources, they are appropriately turning to physician experts for a better understanding—and definition—of value. ASCO has dedicated significant volunteer time and resources to the issue of cost and has now turned its attention to a formal definition of and strategy for assessing value in cancer care.

…Benefit structures, adjustment of insurance premiums, and implementation of clinical pathways and administrative controls have all been employed as means of controlling cost while emphasizing value. It is in this arena that the ASCO Value in Cancer Care Task Force seeks to contribute to the effort to ensure value for patients while preserving and enhancing quality and sustaining innovation developing policy positions that will help Americans move toward more equal access to the highest-quality care at the lowest cost”.16

Now ASCO states “As currently configured, the framework is not meant to be a policy tool. It is intended for use in the clinical setting between physicians and their patients and is meant to serve as a catalyst and facilitator of individual treatment discussions.”

Slap Four


Ironically, these shifts in values underscores just how subjective any framework will be.   And subjectivity also determines what evidence is used and how it is measured.  The Task Force revisions are welcome.  They don’t go far enough and the adequacy of evidence has nothing to do with it.  

Hence while the Task Force still insists there is no good data to measure patient reported outcomes and drug prices net of rebates, that begs the question: why the framework accepts the lack of data to measure of Net Health Benefit?  

Further, the framework still undervalues incremental benefits to specific groups of patients with limited clinical options.   The tool is still biased against significant changes in outcomes that average measures of clinical benefit ignore.  It is still a highly static tool that cannot take into account the impact of matching patients to combinations of treatments. 

As structured the framework still ignores role of cost shifting, the pocketing of rebates and focuses obsessively on drug prices.  

The Task Force had no problem asserting, “Health care costs in the United States present a major challenge to the national economic well being” and then blaming the danger on “the introduction of costly new drugs and techniques in radiation therapy and surgery.”

The Task Force still fails to acknowledge that PBM and health plan cost shifting and rebate driven formulary decisions are the principal reason patients will face high costs.  ASCO supports oral parity and opposes reimbursement experiments that endanger patients. 

Most important, this still NOT a patient-developed framework. The focus should be on developing one instead of tinkering with and fighting about the patient hostile approaches taken by ICER.   

In sum, the ASCO value framework is an improvement over it’s first version.  It is more patient-centered and less a tool for policy making and political attacks. As a recent comparison of the ICER and ASCO frameworks concluded: “ Substantial drug price reductions may be necessary in order to meet ICER thresholds even when maximum NHBs are present as assessed within the ASCO framework. “

 In that important regard, it is a better value framework than those developed by ICER and Peter Bach. 

 
Here's a link to the report CMPI released entitled: Not At Any Price: How ICER Robs Myeloma Patients of Life and Hope

Off-Label Intramural

  • 05.31.2016
  • Peter Pitts

Lawmakers accuse HHS of delaying FDA guidelines for off-label marketing
By Ed Silverman @Pharmalot

Two high-ranking House Republicans have accused the US Department of Health and Human Services of delaying eagerly awaited guidelines for off-label marketing of drugs and devices. And the charge comes amid growing frustration among companies that the US Food and Drug Administration is squelching their free speech-rights.

In a letter sent Thursday to HHS Secretary Sylvia Burwell, the lawmakers wrote they are “perplexed” the FDA has not yet issued new guidelines covering off-label marketing or held a promised meeting. And they worry that court rulings will, instead, become the basis for determining policy. Then they claimed the delay is the result of disagreement between HHS and FDA leadership.

“It is our understanding that HHS has not allowed FDA to issue its completed draft guidance addressing the scope of permissible ‘scientific exchange,’” of useful information about drugs and devices, wrote Fred Upton, a Republican from Michigan and Joe Pitts, a Republican from Pennsylvania. They also attached a draft bill that would allow companies to market products for unapproved uses.

Drug and device makers have increasingly argued that current regulations prevent them from distributing important data to physicians about unapproved, off-label uses of their products. Doctors can prescribe medicines for any purpose, which is called off-label use, but companies can only promote medicines for uses approved by the FDA.

For its part, the FDA maintains public health can be compromised if marketing claims are not backed up by solid evidence. The agency, however, promised to issue new guidelines after a federal appeals court overturned a criminal conviction of a sales rep for off-label promotion. The court ruled his speech was protected, since the information was truthful and not misleading. The FDA also agreed to holding a meeting on the topic, but that has also been delayed.

The issue accelerated last year when Amarin filed a lawsuit to argue its right to distribute information about unapproved uses of a drug is protected by the First Amendment. Amarin sought to give doctors clinical trial data not directly pertaining to approved uses of a pill. In March, the FDA settled the case by allowing Amarin to proceed if information given doctors is truthful and not misleading.

Only a month before that, a federal court jury in Texas decided that the medical device maker Vascular Solutions and its president were not guilty of off-label promotion since the information the company gave to doctors about a laser therapy device was deemed truthful and not misleading. These developments underscored industry anxiety over the delay in issuing a guidance.

We asked the HHS for comment and will update you accordingly. In a speech earlier this month, FDA Commissioner Dr. Robert Califf said the agency is “reviewing our policies,” but did not provide a timeline for releasing the guidance. An FDA spokeswoman said the agency will review the letter, but did not have any further information on the release of the guidance.

One source, who speaks regularly with both agencies, told us this: “HHS leadership doesn’t trust industry to do the right thing … HHS leadership believes off-label speech will lead to more aggressive marketing of new products that will raise costs to [Medicare and Medicaid]. They are allowing both their prejudices [industry as the bad guy] and priorities [keeping spending down] to get in the way … The White House shares these fears, and as a result the FDA’s desire to issue guidance is stymied.”

One Washington insider cautioned that the may lose the initiative if the agency does not release a guidance.

“After the recent court decisions, the FDA realizes that it must either lead the effort to disseminate off-label information that is truthful, accurate, and non-misleading, or lose its ability to direct the speed, direction, and quality of these communications,” said Peter Pitts, a former FDA associate commissioner, who heads the Center for Medicine in the Public Interest, a think tank that is funded, in part, by industry. “Things are moving fast and unless the FDA acts, we’ll have federal judges making these decisions for us. That’s not good for the FDA or the public health.”

As for the draft legislation, a spokeswoman for the House Committee on Energy and Commerce wrote us that the bill may not be introduced. She noted that the language was discussed with the FDA during conversations related to the 21st Century Cures Act, but was ultimately not included in the bill.
One consumer advocate, meanwhile, criticized the draft.

“The threat to patient health posed by the draft bill attached to their letter is tremendous,” said Michael Carome, who heads Public Citizen Health Research Group. “FDA approval of a drug for one use tells us nothing about whether the drug is safe and effective for another use. Yet this bill would allow drug manufacturers to advertise and promote drugs and devices, both to doctors and directly to consumers, for uses never approved by the FDA.”

After the ICER Storm

  • 05.27.2016
  • Robert Goldberg
Here are the takeaways from the ICER meeting on” Treatment Options for Relapsed or Refractory Multiple Myeloma”.
 
First, it is obvious that the Midwest Comparative Effectiveness Advisory Council is nothing but a cheap coat of paint used to poorly cover-up the fact that that, from start to finish, ICER and all the so-called advisory councils (whose participants were added a week before the meeting) are run directly by Steve Pearson, the President of ICER who wants to reduce drug spending to pay for roads and bridges.  Either he dreams of being Rationer in Chief or public works czar, maybe both.
 
The voting members of the panel had as much experience in treating myeloma as they did in translating Beowulf into Klingon.   Yet they voted.  They tried to do so with care.  But it became evident early that the panel knew it was clueless and simply deferred to the one panel member who was an expert on myeloma.  They followed, almost blindly, because informed discussion was impossible.
 
Indeed, after sitting through the meeting (in the comfort of my bedroom via live stream) I came away surprised at how unsophisticated and sloppy the ICER presentations were.  Then again, I don’t think ICER was quite ready for how many patient groups and companies were ready to challenge them.   You could sense the discomfort and the confusion of being held accountable.
 
Second, Steve Pearson, made a strategic retreat by NOT voting on the ‘value’ of new myeloma drugs to the health care system.  Pearson knew ICER would be pummeled for forcing a vote that essentially validated his long held view that most new drugs for seriously ill people with rare diseases will ‘siphon’ away resources that could be used to pay for roads and bridges.
 
Rather Pearson insisted that such meetings were just discusson groups and not intended to guide policy.  That is untrue.  ICER studies and votes have been adopted in making coverage decisions for Hep C, cholesterol and heart failure medicines.   Perhaps Pearson forgot about this statement from Express Scripts:
 
“The Institute for Clinical and Economic Review (ICER) has announced that, through a public and transparent review process, it will establish a value-based price benchmark for the PCSK9 inhibitors later this year. We plan to reference the findings from this independent, trusted organization in our subsequent negotiations with manufacturers to ensure that patients get to access this innovative class of drugs at a price the healthcare system can afford. “
 
 
Third, perhaps it was the setting but no one – from the patient community or the panel --- pointed out that drugs for cancer are very small part of health care spending or that more people living longer means more taxes and premium dollars.  It is crucial at the outset of every pricing discussion to establish a context based on facts. 
 
 It would have been useful if that point was made when Ryan Barker of the Missouri Foundation on Health said that he couldn’t vote for using new myeloma drugs because it would take money away from other patients.  Apparently, letting people die is a better way to generate money than reducing hospitalizations and increasing productivity.  
 
Barker told the group that he teachers medical ethics.  Note to Barker: the litmus test of an ethic is whether you apply it to yourself.  So if it’s your kids dying of myeloma, I expect you to reject any myeloma drug that doesn’t have the evidence you needed to vote yes.  That’s my gut reaction to Barker. 
 
Then again, after sitting through the ICER meeting, it became clear that Pearson aside, there is little malice, just a lack of understanding and opportunities to discuss tangible solutions.  As it stands now, ICER is just the blindly ambitious leading the blind.
 
I believe that it’s time to spend less time picking apart ICER and more time working on ways to create and apply the kind of knowledge needed to reward innovation and patient-defined outcomes. 
 
Is anybody else interested in joining me?
 
 
 
 
 
 
 
 
 
 

Truth in Biosimilar Labeling

  • 05.24.2016
  • Peter Pitts
From today’s edition of The Hill  …

Truth in biosimilar labeling
By Peter J. Pitts

Recently the FDA issued draft guidance on labeling for biosimilar products. In two words, no surprises – which for some is better news than for others.

The top of Page 8 will get a lot of attention:

FDA recommends that biosimilar product labeling incorporate relevant data and information from the reference product labeling, with appropriate product-specific modifications. The relevant data and information from the reference product labeling that should be incorporated into the biosimilar product labeling will depend on whether the applicant is seeking approval for all conditions of use (e.g., indication(s), dosing regimen(s)) or fewer than all conditions of use of the reference product for the biosimilar product.

And further:

In sections of the biosimilar product labeling that are based on the reference product labeling, it is anticipated that the text will be similar. Text based on the reference product labeling need not be identical and should reflect currently available information necessary for the safe and effective use of the biosimilar product. Certain differences between the biosimilar and reference product labeling may be appropriate. For example, biosimilar product labeling conforming to PLR and/or PLLR may differ from reference product labeling because the reference product labeling may not be required to conform to those requirements at the time of licensure of the biosimilar product. In addition, biosimilar product labeling may include information specific to the biosimilar product necessary to inform safe and effective use of the product, which could include differences such as administration, preparation, storage, or safety information that do not otherwise preclude a demonstration of biosimilarity.

To “inform safe and effective use.” Sounds good on paper – but what does it mean? Always? Sometimes? What are the parameters? If there’s information available, why not share it all the time? Or not at all? Who’s to judge? Can we rely on regulatory predictability – and this early in the biosimilar experience? Isn't more information better?

Of course, in order to maintain maximum regulatory, um, flexibility --

FDA acknowledges that there will be variations on the general concepts outlined in this section because the approach to product identification will depend on the specific statements.

(Note – author highlights, not FDA’s.)
 
An update article in Modern Healthcare makes an interesting point, “Federal regulators are likely trying to simplify physicians' understanding of the products' efficacy and safety. By definition, a biosimilar product has no clinically meaningful difference in terms of safety, purity and potency.”

If what practicing physicians understand about biosimilars is anything akin to the knowledge scale of the FDA’s Arthritis Advisory Committee -- as demonstrated during the meeting that considered the biologics license application for a proposed biosimilar to Remicade (infliximab) – then the agency’s attempt at “simplification” may result in some very serious unintended consequences. From the very beginning of the adcomm, it was clear the expert members of the committee didn’t understand what biosimilars really are, nor the pathway the agency uses to review them. Not good.

Per being upfront that the product is a biosimilar, the agency is unambiguous:

FDA recommends inclusion of a statement, on the line immediately beneath the initial U.S. approval date in Highlights, that the product is biosimilar to the reference product.

Although the FDA notes that the labeling does not need to be identical to information based on the reference product, it’s a pretty safe bet that manufacturers of biologics will likely take issue with competitors using their data for a product that is not exactly the same.
But from a straightforward public health standpoint, why omit truthful and accurate information from the information available to physicians and patients?

Shouldn’t labeling transparency be a key tenet in the Era of Biosimilars?

Building EvGen

  • 05.16.2016
  • Peter Pitts
What We Mean When We Talk About EvGen Part II: Building Out a National System for Evidence Generation

By: Rachel E. Sherman, M.D., M.P.H., and Robert M. Califf, M.D.

In an earlier FDA Voice blog post, we discussed a pair of concepts – interoperability and connectivity – that are essential prerequisites for the creation of a successful national system for evidence generation (or “EvGen”). In this post, we take a look at how we would apply these constructs as we go about building such a system.

Building EvGen

Creating knowledge requires the application of proven analytical methods and techniques to biomedical data in order to produce reliable conclusions. Until recently, such analysis was done by experts operating in centers that typically restricted access to data. This “walled garden” approach evolved for several reasons: the imperative to protect the privacy and confidentiality of sensitive medical data; concern about the negative consequences that could arise from inappropriate, biased, or incompetent analysis; and, the tendency to see data as a competitive asset. Regardless of the specific reason, the result has been the same: widespread and systemic barriers to data sharing.

If we are to reverse these tendencies and foster a new approach to creating evidence of the kind envisioned for EvGen, we must bear in mind several critical principles:

There must be a common approach to how data is presented, reported and analyzed and strict methods for ensuring patient privacy and data security.
Rules of engagement must be transparent and developed through a process that builds consensus across the relevant ecosystem and its stakeholders.
To ensure support across a diverse ecosystem that often includes competing priorities and incentives, the system’s output must be intended for the public good and be readily accessible to all stakeholders.What Would EvGen Look Like in Practice?

What would a robust national platform for evidence generation look like? It may be helpful to envision EvGen as an umbrella for all activities that help inform all stakeholders about making treatment decisions.

The task of evaluating drugs, biologics, or devices encompasses different data needs and methods. However, all share a common attribute: the characterization of individuals and populations and their associated clinical outcomes after they have undergone diagnostic or prognostic testing or been exposed to a therapeutic intervention.

Moreover, when medical practice itself is part of the evaluation, characterization of the organization and function of delivery systems is critical. In other words, the kinds of evidence needed to evaluate medical products for safety and effectiveness and the kinds of evidence needed to guide medical practice overlap substantially.

Over the last decade, there has been enormous progress in the area of “secondary use,” in which data collected for one purpose (for instance, as part of routine clinical care) can be reused for another (such as research, safety monitoring, or quality improvement).

The Sentinel Initiative, launched in response to a Congressional mandate to develop an active postmarket risk identification and analysis system, is one example. Modeled after successful programs such as the Centers for Disease Control and Prevention’s Vaccine Safety Datalink, Sentinel allows FDA to conduct safety surveillance by actively querying diverse data sources, primarily administrative and insurance claims databases but also data from electronic health record (EHR) systems, to evaluate possible medical product safety issues quickly and securely.

Another example, the National Patient-Centered Clinical Research Network (PCORnet), is a national system that includes many of the attributes needed for EvGen. PCORnet includes participation from government, industry, academia, and patients and their advocates. Whereas FDA’s Sentinel system is built primarily on claims data repurposed for safety surveillance, PCORnet is designed to leverage EHR data in support of pragmatic clinical research.

The NIH’s Health Care Systems Research Collaboratory has demonstrated through its Distributed Research Network that the concept of secondary data use can be extended into the realm of prospective pragmatic interventional trials. The NIH Collaboratory program, which includes many of the same health care systems involved in Sentinel and PCORnet, has 10 active trials underway.

In addition, the Reagan-Udall Foundation Innovation in Medical Evidence Development and Surveillance (IMEDS) Evaluation Program is exploring governance mechanisms to ensure that private-sector entities, notably regulated industry, can collaborate with Sentinel data partners to sponsor safety queries about marketed medical products. Such measures have the potential to expand the involvement of private-sector partners beyond the arena of methodology, further helping to ensure that Sentinel continues its expansion into a national resource.

Similarly, efforts are underway to establish a National Device Evaluation System (NDES). As currently envisioned, the NDES would be established through strategic alliances and shared governance. The system would build upon and leverage information from electronic real-world data sources, such as data gathered through routine clinical practice in device registries, claims data, and EHRs, with linkages activated among specific data sources as appropriate to address specific questions.

As substantial work already is being done in all of these areas, valuable experience is being gained. The next step is to ensure that these pioneering efforts coalesce into a true national resource. More on that in future postings.

Rachel E. Sherman, M.D., M.P.H., is FDA’s Associate Deputy Commissioner for Medical Products and Tobacco
Robert M. Califf, M.D., is Commissioner of the U.S. Food and Drug Administration
 
Yesterday Sara Edmond, the chief operating officer of the Institute for Clinical and Economic Review and I danced around whether ICER is really patient-centered as she claimed, or rebate-centered as I did. 

Ms. Edmond was a panelist at an  excellent Financial Times sponsored  US Healthcare Life Sciences Summit yesterday.     https://live.ft.com/Events/2016/FT-US-Healthcare-Life-Sciences-Summit  She was joined by Peter Bach, Director, Center for Health Policy and Outcomes, Memorial Sloan Kettering Cancer Center Jonathan Emms, Senior Vice President, Global Health and Value, Pfizer Jeff Myers, President and CEO, Medicaid Health Plans of America and Leonard Schleifer, President and CEO, Regeneron.

Praluent, the drug targeting people with untreatable high cholesterol was (along with Amgen’s Repatha) was considered low value by ICER and claimed that it should only be given to 15 percent of all patients who could benefit and at 80 percent of the list price.  

As a Reuters article reports, Dr. Schleifer blasted ICER as unscientific:

"They did all the calculations and they said it's X, which is ok. I could have lived with that. But ... they said society can't afford X, so we are going to say it's one-third X," Schleifer said during a panel discussion.
"They had value-based pricing, but they just decided that well we can't afford it. That wasn't scientific. There was no intellectual honesty there."

ICER Chief Operating Officer Sarah Emond, on a panel with Schleifer, countered that budget impact is part of the equation.
"You're attacking the science of an independent non-profit whose entire mission is tied to opening the black box of pricing," she said.

Which lead me to challenge Edmond from my seat in the audience about ICER’s so called independence. 

I said:  “ICER is not independent, it is largely funded and overseen by PBMs and insurers.”

Edmond responded that most the money came from the Arnold Foundation but I pointed out that the Arnold foundation grant is a one time thing.  

I then said that ICER’s value metric reflects the PBM and insurer interest and ignores patient value.  Further, the low QALY by default becomes a price control or price cap that doesn’t make drugs affordable or available.  Instead, the lower price only maximizes rebates that don't go to patients. More important, for all her talk about opening a black box I noted that ICER never mentions that PBMs run the $115 billion rebate racket.  

She said that since ICER doesn't have rebate data they have to use list price.

People laughed and Schleifer asked:  You estimate everything else, why not rebates? 

Finally I reiterated that under the ICER framework, not only would drugs for HIV, cancer, etc. be rejected but that next generation medicines would not be developed.    She responded, if we pay for Hep C drugs then we will have to lay off teachers and close schools." 

That drew another laugh.  

Edmond claims that ICER "uses science, we use math."  But she was unable to rebut the claim that the math supports the kind of creative accounting that reduces drug prices and maintain PBM and insurer profit margins. 

ICER's self anointed claim of being a trusted and independent organization was debunked.  Indeed, the other FT summit panelists talked about plans, providers and drug companies sustaining innovation and generating value while realizing that in doing so some ‘stakeholders’ – hospitals and PBMs – would go the way of Blockbuster video.   

Maximizing PBM and insurer rebate revenue is a self-serving enterprise that is sustained by increasing the spread between list prices and acquisition cost.   ICER was created and is funded by groups that profit from this enterprise to validate a QALY for that financial goal.  

In its current form ICER stands for the Institute for Constantly Extracting Rebates.

Now the black box is opened. 
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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