JAMA Peddles Rx Payment Propaganda Posing as Research

06/22/2016 05:26 PM | Robert Goldberg

Two recent articles on the alleged negative impact of pharma providing meals to physician practices are first rate examples of how the editors of JAMA and the authors ignore their own questionable data to arrive at a pre-ordained conclusion.  And worse, JAMA peddled these articles to the media as proof positive of this claim.   
 
The articles reinforce the assumption that doctors are influenced to prescribe more expensive brand drugs.  There is has never been any causal evidence of any sort to support this claim, just anecdotes and the torturing of data that is conducted to fit the narrative.  
 
But let’s say for the sake of argument that the more freebies and lunches doctors received is directly associated with more brand prescribing or specific prescribing of brands.  Indeed, that is the hypothesis these two articles seek to test.  Except that now, as opposed to even 10 years ago, the amount spent on pharma freebies like lunches and trips worth about $150 has declined.  And the number of doctors who get them has fallen too for a number of reasons.  At the same time brand prescribing has declined.  So the reduction in payments has led to less brand prescribing right?
 
In Association of Industry Payments to Physicians With the Prescribing of Brand-name Statins in Massachusetts James S. Yeh, MD, MPH; Jessica M. Franklin, PhD; Jerry Avorn, MD; Joan Landon, MPH; Aaron S. Kesselheim, MD, JD, MPH (which I will refer to as Avorn and Co.) claim:
 
“Industry payments to physicians are associated with higher rates of prescribing brand-name statins. As the United States seeks to reign in the costs of prescription drugs and make them less expensive for patients, our findings are concerning.”
 
In Pharmaceutical Industry–Sponsored Meals and Physician Prescribing Patterns for Medicare Beneficiaries  Colette DeJong, BA concluded:
 
“Receipt of industry-sponsored meals was associated with an increased rate of prescribing the brand-name medication that was being promoted. The findings represent an association, not a cause-and-effect relationship.”
 
Dejong and company looked at four specific drugs in different therapeutic class.  They note that Crestor was 8.8% statin prescriptions; Benicar 3.3% beta-blocker prescriptions; Benicar was 1.6% of ACE inhibitor and ARB prescriptions; and Prestiq was 0.6% of SSRI and SNRI prescriptions and like Avorn and Co. conclude that prescribing rate was influenced by drug reps passing out donuts and Chipotle.
 
 Except that in both ‘studies’ the brand prescribing rates were BELOW national averages for Medicare part D
 
The Medicare Payment Advisory Commission reported that
 
“Generic drugs accounted for 81 percent of all prescriptions filled in 2012 compared with 77 percent and 61 percent in 2011 and 2007, respectively. In 2015, generic fill rate increased again but as Express Scripts Drug Trend Report notes, the fill rates differed by plan type, with Medicare Advantage and stand-alone Part D plans with similar generic fill rates (87.5% and 87.2%, respectively), and Employer Group Waiver Plans with the lowest generic fill rate (82.4%).”
 
The Avorn group estimated that doctors who got lunch prescribed brand name statins 23 percent of the time vs 18 percent that were deprived of a free lunch in 2011.  But the 23 percent is the same as prescribing of all brand drugs in Part D, a percentage that began and continued to decline as more medicines went off patent.   We don't know what Avorn and Co.'s data would show in 2012 or 2013 as Lipitor went off patent... We will never know because taking that into account might undermine the conclusion they want to make. 
 
Meanwhile DeJong shows that the undue influence of meals leads to much lower brand utilization in part D than the national market share of each drug. 



And here is the trend in brand vs generic over the past decade.  And neither study took the time to control for this critical variable?













Finally, neither study tested the reverse assumption: that the biggest prescribers of brand drugs were more likely to have drug reps visit their office, provide samples and schmooze than those that prescribe generic.  If they had done that, both groups of authors could have controlled for patent expirations, co-pay effects, etc. that are more highly correlated with prescribing and generic uptake than snacks.  

But that wouldn’t fit JAMA's distorted narrative of unscrupulous drug companies seducing dumb doctors with free lunches.   Read More & Comment...

The CREATES Act: Avoiding Unintended Consequences and Hidden Agendas

06/20/2016 06:49 AM | Peter Pitts

The road to Hell is paved with good intentions -- and often hidden agendas.

Generic drug manufacturers have complained that innovative pharmaceutical manufacturers use FDA-mandated safety-based distribution requirements—called “risk evaluation and mitigation strategies” (REMS)—to prevent or delay generic medicines from coming to market. Some generic and biosimilar manufacturers have argued that innovative manufacturers use REMS to avoid selling samples of their medicines to competitors, which results in some generic and biosimilar manufacturers being unable to complete the testing necessary to obtain FDA approval of their medicines.

To address this issue, generic and biosimilar manufacturers are supporting, the “Creating and Restoring Equal Access to Equivalent Samples Act of 2016” or the “CREATES Act.” The CREATES Act allows a generic or biosimilar manufacturer to bring a civil action in federal court against an innovator to obtain injunctive relief and monetary damages in two instances: (1) where the innovator has failed to provide samples of a drug or biological product within 31 days of a request for samples, and (2) if the companies fail to reach an agreement on the development of a single, shared REMS system. While seeking to address a narrow issue, the bill is drafted in manner that will put patients and medical researchers at risk of serious harm and generate significant and meritless litigation costs for innovative pharmaceutical manufacturers.

Specifically:

The CREATES Act Lacks Adequate Patient Safety Protections

* The CREATES Act fails to adequately protect both patients and medical researchers who participate in clinical trials of REMS drugs conducted by generic or biosimilar manufacturers. This is concerning because REMS drugs are not typical prescription medicines—they are a special class of potentially harmful drugs that may be subject to restrictions called “elements to assure safe use” (ETASU), which FDA deems as necessary to ensure patient safety. In fact, many REMS drugs subject to ETASU may only be distributed with specific safeguards to protect anyone who comes in contact with the medicine.

* The bill fails to ensure sufficient FDA oversight of safety protections for subjects and researchers in studies of drugs having REMS with ETASU. To obtain an authorization, a generic or biosimilar manufacturer may -- but need not -- submit a clinical trial safety protocol outlining its planned testing of the drug in patients. The bill does not require FDA to pre-approve the safety protocol or to even make the determination that it provides equivalent protections for patients and researchers in comparison with the REMS with ETASU. The bill also grants FDA no authority to suspend a generic or biosimilar manufacturer’s access to samples or otherwise modify or revoke an authorization if the generic or biosimilar manufacturer does not implement appropriate safeguards.

* Instead of providing the FDA with authority to address these safety issues, the bill tasks the federal courts with adjudicating the terms. Although the bill contemplates a limited role for FDA in the authorization process for these products, the federal courts will determine what, if any, safety protections imposed on the transfer of samples are reasonable. The federal courts lack the expertise of FDA in evaluating the safety of a medicine and the measures necessary to protect patients and researchers.

The CREATES Act Exposes Innovative Manufacturers to Liability Risks Through No Fault of Their Own

* The CREATES Act will hold innovators responsible for the actions of generic and biosimilar manufacturers because the bill provides innovators liability protection only for claims arising out of failure to follow adequate safeguards during handling or use of product by the generic or biosimilar manufacturer. As a result, innovative manufacturers could still be unfairly liable for others’ negligence, long after the medicine has left their control. The CREATES Act exposes innovators to significant new liability risks based on the actions of their competitors.

The CREATES Act Hurts Patient Access to Life Sustaining Therapies During Drug Shortages

* The CREATES Act could exacerbate drug shortages and further limit the supply of medically necessary drugs. If a medicine has been on the shortage list for more than six months, the medicine is not exempt from the bill. In other words, manufacturers of these products would be forced to divert medicines from their patients—even when the medicines are life sustaining—to ensure supply for their competitors’ clinical trials.

More careful consideration needs to be inserted into the CREATES Act design so that it more clearly addressed its intent and avoids unintended consequences or hidden agendas. Patient safety, public health, and healthcare innovation mustn't become innocent victims.  Read More & Comment...

Besmirching White Oak

06/16/2016 09:11 AM | Peter Pitts

A very upsetting story about an FDA official who sold information to an investor.

This person should go to jail.

For a long stretch.

For shame.  Read More & Comment...

ICER: A Cancer Survivor/Marathon Runner's Perspective

06/15/2016 03:27 PM | Robert Goldberg

This post on ICER from a patient's perspective is by Don Wright.  Don is a lawyer living and working in Minnesota and  has been running marathons since 2002.   In 2003 he was diagnosed with multiple myeloma, a blood cancer with no cure.  He went on an experimental treatment that year and is now on the verge of completing his 97th marathon.  Running is a part of fighting back against myeloma, as well as a celebration of life.  He is a leading advocate for cancer patients around the world.   This post argues that a doctor using ICER guidelines to determine treatment access would at the very least violate the Hippocratic Oath.

Who is ICER?

ICER is the Institute for Clinical and Economic Review.  As far as I can tell, it is funded primarily by insurance companies and by nonprofit organizations who, in turn, are funded by insurance companies.  They claim some funding by the federal government as well.  Other members include pharmaceutical companies who apparently participate in order to have some voice in ICER's proceedings.  A quick Google search shows that the title of many of ICER's documents is "Building Trust through Rationing," which I believe is their mantra and suggests their real purpose.

ICER deals in statistics, not medicine, and a primary goal is to control costs.  I assume that this is why they don't want participation by patients.  They have been working on a report for multiple myeloma, and we myelomiacs have been concerned that they would produce a one-size-fits-all treatment algorithm that doctors might be expected to follow and insurers might try to enforce.

Garbage In, Garbage Out

ICER issued their final report on Myeloma on June 9, 2016, attempting to grade different myeloma treatments to provide comparative medical and cost values.  In my opinion this report is ridiculous on its face, saved only by one of its final recommendations.  ICER claims to have found over a thousand potentially relevant literature references to myeloma treatment, considered 38 worth reading, and exactly six Phase III studies worth analyzing to form their conclusions.

Thus they chose to ignore all Phase I and Phase II studies, which provide by far the largest part (I'd guess 90%?) of the current, up-to-date information that the FDA uses for drug myeloma approval and that doctors actually use in their day-to-day care of myeloma patients.  For this reason, ICER's entire analysis is fatally flawed.  As we say in the computer industry: "Garbage in, garbage out."

Blinders

As just one example of this blinders approach, the report ignores an old but widely-used myeloma treatment called cyclophosphamide (Cytoxan), which is frequently combined with dexamethasone (DEX) and either bortezomib (Velcade) or lenalidomide (Revlimid).  Indeed, many patients will recognize cyclophosphamide with bortezomib and DEX as the CyBorD regimen.  Because cyclophosphamide is relatively low in cost, it certainly should have been included in any economic analysis, but it appears nowhere except peripherally in the addenda. 

ICER's peculiarly superficial analysis also minimizes or omits many other commonly-used and highly-effective regimens.  Worst of all, it gives especially poor grades to the treatments that are newest and possibly the most effective, such as pomalidomide (Pomalyst) and daratumumab (Darzalex).

Saved by the disclaimer:

One recommendation near the bottom of the final report and in the shorter Report-at-a-Glance, saves the report from total disrepute.  This appears under the heading "Insurers:"
Multiple myeloma is a condition in which many patients will cycle through most or all available treatments, and there is substantial variation in drug mechanisms of action and in the personal patient values that guide consideration of the trade-offs between extended survival and different side effect profiles. Given this background, and in the absence of better evidence, payers should not consider step therapy or “fail first” coverage policies for myeloma treatments.Amen.  This statement seems to have two important implications:
ICER recognizes that their report has no value in guiding treatment for any particular patient (i.e. it turns out that we wasted our time producing this report); and
The PATIENT (the payer) is responsible for choosing an insurer or a plan which does not demand step therapy or "fail-first."Let that be a lesson to us patients!  Maybe the best advice I've seen today - if you have a choice of insurers, choose very carefully.

My bottom line opinions:
A doctor attempting to use the results of this ICER report as the primary guide for treating a patient would be committing medical malpractice, and if so
It follows that an insurance company or plan that denied coverage based upon this report would be demonstrating a singular contempt for their own client, the policyholder.    Read More & Comment...

When it comes to Biosimilars -- Knowledge is Safety

06/14/2016 06:15 AM | Peter Pitts

According to a letter to the FDA from the GPhA and its Biosimilars Council:

“… we are concerned about the FDA’s requirement to include a biosimilarity statement on biosimilar labeling. The biosimilarity statement is at best unnecessary. The FDA has never required any similar statement for products found to be therapeutically equivalent, and has not provided sufficient justification for its inclusion in biosimilar labeling. Moreover, the biosimilarity statement will be confusing to patients and providers who are unfamiliar with this type of unprecedented statement. This confusion could put biosimilar utilization, and savings, at risk.”

Not so.

Consider generic drugs and information transparency. According to the FTC’s 1979 report on generic drug substitution, that agency concluded, “increased communication (as well as lower prices) may explain why most pharmacists report that product selection laws have had a positive effect on their relations with patients”

Safety and trust are exactly why transparency-in-labeling is needed. As Sumant Ramachandra, Senior Vice President & Chief Scientific Officer at Pfizer’s Hospira unit, has said, “Communications fosters confidence.”

And Geoffrey Eich (Executive Director, R&D Policy, Amgen) has asked:

Why not transparently label biosimilars to engender patient and physician confidence?

Why not ensure accurate patient medical records that clearly identify specific products?

Indeed, at a time when the FDA is considering a rule for the differential labeling of small molecule generics, why not transparency in biosimilar labeling?

It’s important to mention that the majority of the letter signatories are … payers.

Draw your own conclusions.  Read More & Comment...

TIme to wake up to Antibiotic Resistance

06/10/2016 02:32 PM | Peter Pitts

What Does Future Hold After Woman Infected With Drug-Resistant Superbug?

By  Kathy Ritchie

Updated: Friday, June 10, 2016 -- For the first time, a person in the U.S. has been infected with bacteria resistant to an antibiotic used as a last resort. The woman, who is from Pennsylvania, is recovering after it was discovered she was carrying a strain of E. coli resistant to the antibiotic colistin. But public officials say this should serve as a wake-up call for everyone.

It’s something many of us take for granted— the ability to go to our doctor and get an antibiotic for something as common as a urinary tract infection. But what to do when antibiotics stop being effective?

Colistin is the antibiotic of last resort for particularly dangerous types of superbugs, including a family of bacteria known as CRE. The Centers for Disease Control and Prevention says infections with these germs can be extremely difficult to treat and could be deadly in up to 50 percent of patients who become infected.

Peter Pitts is the president of the Center for Medicine in the Public Interest. He says this one case could turn into thousands unless we do something now. "Shame on us to wait until there are bodies in street to begin to worry about this," said Pitts. "The thing about antibiotics is that we currently have a pretty good supply of various potencies, but you always need new ones. You can’t simply turn on the spigot when you think you need some and it comes out right away."

Pitts says the over-prescribing of antibiotics and the fact that there is no real incentive for companies to develop new antibiotics could lead to dire consequences.

So what does a future filled with drug-resistant superbugs look like?

"The more realistic view of this is that people get sick and have to be on a regimen of very strong antibiotics you know with very nasty side effects for extended periods of time," Pitts said.  Read More & Comment...

Peter Loftus Lead Astray By ICER On Cancer Drug Costs

06/07/2016 03:21 PM | Robert Goldberg

Peter Loftus' article Combination Drug Therapies for Cancer Show Promise at Higher Potential Cost relies on opinion rather than facts. And the opinion he relies on comes from an organization that combines factoids with fiction to demonstrate that new drugs are too expensive. 
            

1.    The assertion of Steve Pearson of the Institute for Clinical and Economic Review (ICER) that the combinations will cost more is inaccurate.  In a recent report I pointed out that Pearson estimates the cost of combining three drugs for multiple myeloma using list prices of all drugs in making the case for ‘group’ discounts.   In fact, the list price of such medicines are already discounted by about 30-50 percent because of rebates drug companies provide.  Pearson never notes that these rebates are pocketed by insurers and pharmacy benefit companies even as they force patients to pay 30 percent of the list price of products. 

2.    Most treatment combinations reduce the cost of other more expensive services and care.  Indeed, I co-authored two abstracts presented at the same ASCO meeting Loftus attended demonstrating that combinations of cancer drugs matched to specific patients cost less or about the same at less effective treatments.  Similarly, Intermountain Health published two studies the came to the same conclusions. 

3.    Pearson and others also ignore the savings and benefits to patients by using combinations that reduce the need for second and third line therapies as well as the end of life care that is more likely required when combinations are not used. 

4.    Finally, cancer costs increase mainly because people are living longer.  ICER notes “…additional progression-free survival leads to higher costs” and recommends restricting use of new medicines to address this ‘problem.’  So letting people die becomes the de facto discount. 

Groups like ICER believe that we need to spend less on cancer drugs they define as not valuable because doing so " often leads to waste that saddles our children and their children with future budget deficits based on our inability to objectively look at the evidence and value of new drugs. We’re siphoning off resources for other things we need like better schools and more resources for local police, roads and bridges."

In fact, spending on cancer drugs – which is less than 1 percent of total health care spending – increases well-being and prosperity by increasing life expectancy.   Reporting based on misinformation could lead to policies that deny patients access to medicines that produce such benefits including more tax revenue for filling potholes.   Loftus and other journalists should look at how ICER leads them astray.  Also, has anybody checked to see if ICER doesn't run a highway construction company?  Read More & Comment...

Serepta and the Final Furlong

06/07/2016 01:30 PM | Peter Pitts

Sarepta Therapeutics said the U.S. Food and Drug Administration has requested for additional data from an ongoing study for its muscle-wasting treatment as the agency decides whether to approve the drug or not.

"We believe there is a good chance these data will demonstrate required dystrophin production and recommend shares ahead of a regulatory decision, which could come in 2016," they said in a Monday note to clients.

The FDA deferred a highly anticipated decision on whether to approve Sarepta's drug, eteplirsen, last month, after an advisory panel determined that the treatment was not effective.

The agency requested that Sarepta provide dystrophin data from biopsies already obtained from the ongoing confirmatory study of eteplirsen, the company said on Monday.

Sarepta's drug has been in the spotlight over the past few months with patient groups and parents arguing passionately in favor of the treatment to pressure the regulator to approve the drug.

Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscular weakness and is caused by a lack of dystrophin, a protein needed to keep muscles healthy. Eteplirsen is designed to increase the production of dystrophin.

Sarepta said on Monday it plans to submit data from thirteen patient biopsy samples to the FDA over the coming weeks to facilitate a prompt decision by the agency.  Read More & Comment...

Expediting Expanded Access

06/03/2016 08:35 AM | 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.  Read More & Comment...

ICER Involved in Unlawful Hep C Drug Rationing Scheme?

06/02/2016 01:47 PM | Robert Goldberg

 
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.   Read More & Comment...

It's the Volume Stupid..and the Rebates

06/02/2016 01:09 PM | 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.  
   Read More & Comment...

ASCO Value Task Force Takes Two Steps Towards Patients, Slaps ICER, Abacus

06/01/2016 12:09 PM | Robert Goldberg

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. 

   Read More & Comment...

ICER's Multiple Myeloma Treatment Evaluation Regards Patients As Side Effects

05/31/2016 06:30 PM | Robert Goldberg

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

Off-Label Intramural

05/31/2016 07:22 AM | 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.”  Read More & Comment...

After the ICER Storm

05/27/2016 03:27 PM | 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?
 
 
 
 
 
 
 
 
 
   Read More & Comment...

Truth in Biosimilar Labeling

05/24/2016 04:22 PM | 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?  Read More & Comment...

Building EvGen

05/16/2016 08:30 AM | 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
   Read More & Comment...

ICER (The Institute for Constantly Extracting Rebates)

05/13/2016 02:30 PM | Robert Goldberg

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.   Read More & Comment...

The Urgency of the TPP

05/12/2016 12:51 PM | Peter Pitts

When it comes to global trade, there is no value to living in the past. We no longer trade in barter or beads. The Trans-Pacific Partnership (TPP) acknowledges that we live in a global village – and innovation is an essential commodity. Why are some people finding this so surprising? 

The evolution of the TPP has been long and arduous – but the basic premise has never changed. In order for global trade to flourish in an equitable manner, there have to be rules. Minus the rule of law, chaos ensues and we find ourselves in a survival of the fittest situation without fairness, predictability, or incentives for innovation. In a world without rules, ambiguity trumps investment and risk outweighs rewards.

One of the more important aspects of the TPP is intellectual property (IP) protection for biopharmaceutical innovation. This is of particular importance to the United States, where the U.S. biopharmaceutical research sector leads the world in the development of new medicines with about 4,000 in development or FDA review in the U.S. and more than 7,000 in development worldwide. This sector generates high-quality jobs and powers economic output and exports for the U.S. economy, serving as the foundation upon which one of the U.S.’ most dynamic innovation and business ecosystems is built. 

Read our new report here.
   Read More & Comment...

BILLIONS: Is ICER Enabling Illegal PBM Rip-off Racket?

05/11/2016 05:44 PM | Robert Goldberg

 If you give someone the tools they use to commit a crime, are you also responsible? 

Consider the following news item from Reuters about pharmacy benefit managers and their rebate contracts with drug companies: 

U.S. probes contracts between drugmakers, pharmacy benefit managers
 
“The U.S. Attorney's Office for the Southern District of New York is investigating contracts between drugmakers and companies that manage prescription benefits, according to regulatory filings… When drugs are knocked off their formularies, patients may have to pay full price for them. PBMs often keep or dump a product depending on whether they can obtain favorable pricing.”
 
As I have noted PBMs get and distribute about $110 BILLION in rebates each year.  That’s a trillion over a decade.  ICER plays, or seeks to play a role in setting prices and determining what products PBMs choose and insurers pay for that is central to enlarging and maximizing rebates.   And the PBMs are working closely with ICER to develop the price and access parameters of the contracts the  U.S. Attorney for the Southern District of New York is investigating:
 
“ICER’s new program will make a huge difference by providing what is sorely needed: an independent, trusted source of information about new drugs,” stated Steve Miller, MD, Chief Medical Officer of Express Scripts, the nation’s largest pharmacy benefits manager. “I believe many payers and policy makers will find this information of critical importance as they evaluate the new drugs, and we look forward to using it to help us improve the ability of patients to get access to new, innovative drugs at a price the system can afford.”
 
PBMs are also looking to ICER’s Steve Pearson to find reasons to pay less for cancer drugs depending on their indication. As a recent article noted: “ Express Scripts is using data from ICER, as well as the DrugAbacus, to inform its indication-based pilot.”
 
As I have noted before, indication pricing, based on average response and on a QALY measure reflecting PBM profit motives, can endanger patients and discourage companies from investing in treatments for the most difficult to treat tumors.  More generally, first in class or first ever drugs to treat the most severe conditions -- particularly rare diseases --  are likely to show modest benefit on average.  Such medicines will never meet ICER's devalued QALY measure. 
 
The most recent example is ICER has already determined that most new multiple myeloma drugs approved to treat patients whose disease has returned are NOT cost effective, even at 90 percent of the list price of the drug.
 
People with myeloma will bear moral, economic and clinical consequences that ICER ignores and the media barely mentions.   So two days before ICER meets to approve the report’s (foregone) conclusions I will be releasing a white paper that measures these essential values.
 
One important point to consider:  ICER recommendations would lead to drug companies forking over billions in rebates in exchange for putting these drugs on their formulary.   It does NOT mean that patients will get access.   It does mean that ICER is enabling the rebate ripoff scheme that the US attorney is investigating.  It may not be cutting the deals but ICER is supplying the PBMs the justification for the rebate enterprise.   


 
 
 
 
 
 
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