Advancing Companion Diagnostics

07/07/2016 06:34 AM | Peter Pitts

Important news. FDA Commissioner Robert Califf outlined two draft guidance documents addressing regulatory approval of next-generation sequencing (NGS)-based diagnostics. The guidances were presented as part of an update on the White House's Precision Medicine Initiative, which is recruiting a cohort of 1 million volunteers for a longitudinal research study. 


The first guidance, titled "Use of Standards in FDA's Regulatory Oversight of Next Generation Sequencing (NGS)-based In Vitro Diagnostics (IVDs) Used for Diagnosing Germline Diseases," describes FDA-recognized standards to demonstrate test accuracy and provides recommendations for designing, developing and validating NGS-based tests for hereditary diseases. 


FDA said a second guidance, titled "Use of Public Human Genetic Variant Databases to Support Clinical Validity for Next Generation Sequencing (NGS)-based In Vitro Diagnostics" outlines "an easier path for marketing clearance or approval" of NGS tests that would allow developers to use genomic databases to support clinical claims.


Per a report in BioCentury, “Califf said the draft guidances are meant to strike a balance between encouraging innovation and supporting patient safety.” FDA will accept comments on the guidance for 90 days.  Read More & Comment...

For a more independent FDA

07/05/2016 06:58 AM | Peter Pitts

From the pages of the Boston Globe and reporter Ed Silverman ...

Independence would be good for the FDA and the public

 In a moment remarkable for its symbolism, six former Food and Drug Administration commissioners last month sat together on a stage and argued that their former agency needs more autonomy from Washington bureaucracy. The solution: make the FDA independent and maybe give it a cabinet seat at the White House, too.

The idea has been kicked around a few times over the years, but never gained traction. Yet the panel discussion at the Aspen Ideas Festival has refocused attention on the notion that the FDA — and by extension, the American public — would be better off if the agency’s status was elevated. And the suggestion carried still more weight since the former commissioners worked for both Republican and Democratic administrations.

Right now, the FDA is part of the US Department of Health and Human Services, which adds a big layer of officialdom between the agency and the White House. And in a legislative holdover, the FDA budget is overseen by House and Senate agriculture appropriations committees, which may not always be familiar with matters surrounding cutting-edge medical developments.

“An independent agency could work directly with Congress and the White House on a one-to-one basis and create dialogue that could lead to policy changes,” Andrew von Eschenbach, who was FDA commissioner from 2006 through 2009, and is now president Samaritan Health Initiatives, a think tank, told me. “This would put FDA in a much better position to execute its mission.”

To what extent the notion can become reality is uncertain, but it’s worth considering.

Here’s one reason. In December 2011, former HHS Secretary Kathleen Sebelius overruled the FDA and refused to allow an emergency contraceptive pill to be sold over the counter to young teens. This was the first time the HHS took such a step, but it was politically expedient because it allowed the Obama administration to avoid a contentious battle over birth control during a presidential election season.

The decision smacked of malfeasance, but reflected a long-running battle between politicians and scientists over whether the “morning after” pill should be available without a prescription. The Bush administration initially resisted such a move but later allowed over-the-counter access to women 18 and older. In 2009, the Obama administration lowered the age to 17 in response to a federal court order.

Of course, it’s true that even an independent FDA would still remain beholden to the White House, which suggests the potential for political interference will always exist. This is a fact of life in Washington. But cabinet-level status may confer an added benefit, because whoever heads the FDA would, presumably, have more opportunity for direct contact with administration decision makers.

“It may not entirely change the political dynamic, but I think it would be an improvement, because it could make it more difficult to meddle,” 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. “You want to make it possible for a commissioner to stand firm and do the right thing,” added Pitts who wasn’t at the Aspen conference.

Toward that end, the top FDA job might also be structured so that there is a fixed term of say, six years, that doesn’t directly overlap with a president’s tenure. Yes, the FDA chief would still be a political appointee, but this approach may encourage a midterm president to find the best candidate, rather than use the appointment as a way to pay a political debt following an election.

Another reason to consider a push for independence is the budget process. Since FDA is part of HHS, its budget is vulnerable to cuts and changes — even before Congress gets to decide what to leave in and what to leave out. By elevating the agency to cabinet-level status, the FDA presumably could have more sway over resources.

“In a way, the FDA has always been a stepchild,” said Ira Loss of Washington Analysis, a consulting firm that tracks the pharmaceutical industry and regulatory policy. “And it often gets trapped in the bureaucracy.”

The challenge is to make a good case for independence.

Right now, the odds seem long. A new administration would have to be convinced to make the idea a priority. And persuading Congress, which seems perennially critical of the FDA and is currently crafting legislation to remake some agency functions, would be a challenge. To speed things along, von Eschenbach noted that a few former commissioners may now consider drafting a joint white paper.

To be sure, there will always be bureaucratic hurdles. That’s the nature of government. But any effort that can recalibrate the balance between bureaucracy and resources should be pursued. The FDA deserves a seat at the table where decisions are made.  Read More & Comment...

JAMA and Pharma Freebies: Same Slander. Different Day.

06/29/2016 08:57 AM | Peter Pitts

From the pages of this morning's Morning Consult:

JAMA and Pharma Freebies: Same Slander. Different Day.

The truth is rarely pure and never simple. -- Oscar Wilde

Much ado about pharma freebies to physicians. Much ado about nothing medically and everything politically.

A new study published by JAMA Internal Medicine (Pharmaceutical Industry–Sponsored Meals and Physician Prescribing Patterns for Medicare Beneficiaries) makes it sound (as Meagan McArdle has written for Bloomberg), that your doctor is “willing to sell you out for the price of a sandwich.” It’s not that simple … or true.

The JAMA methodology:

Cross-sectional study of 279,669 physicians that received industry-sponsored meals (retrieved from Open Payments program) and wrote Medicare part D prescriptions in any of four drug classes: statins, cardioselective beta blockers, angiotensin-converting enzyme inhibitors and angiotensin-receptor blockers (ACE inhibitors and ARBs) and selective serotonin reuptake inhibitors (SSRIs)/serotonin norepinephrine reuptake inhibitors (SNRIs). Prescribing rates of promoted medicines were compared with in-class alternatives adjusted for volume, demographic characteristics, specialty and practice setting.

It’s important to note up front the JAMA conclusion stated that, “The findings represent an association and not a cause and effect relationship.” But you won’t find that in the media coverage. Also, the Open Payments data and Medicare Part D prescription data are not temporally linked. As John Adams points out, “Facts are pesky things.”

Mechanism of association cannot be extrapolated from the methodology of the study; systematic confounding variables such as physician self-selection to attend the educational event and the effect of education itself obscure interpretation of the results. The study design is cross-sectional, only 5 months of payment data may not be representative of a full year and beyond. And, importantly, branded medicines that are often newer may represent advances over older generic agents with regard to efficacy and tolerability.

This is not a new debate nor is it new to the pages of the Journal of the American Medical Association. A widely cited 2000 JAMA article in summarized 29 published studies critiquing the interaction between doctors and drug reps. Notable feature of these articles, as quoted in the summary paper: "No study used patient outcome measures." Absent in 2000 and in 2016 was any discussion of how diagnostic and dispensing decisions are often influenced by external cost-control measures. Both JAMA articles allowed politics to trump the public health. The polite term for this is “normative bias.”

Studies and commentary that discuss alternative findings are generally ignored. In the February 7, 2009 edition of The Lancet, Richard Horton points out that the battle lines being drawn and between clinician, medical research and the pharmaceutical industry are artificial at best -- and dangerous at worst.  Dangerous, because all three constituencies are working towards the same goal -- improved patient outcomes. His main point is that we must dismantle the battlements and embrace of philosophy of "symbiosis not schism."  It's what's in the best interest of the patient.

Information is an important lubricant for markets and yields numerous benefits to market participants. Open, honest, and regular communication is critical for alerting both doctors and patients as to what medicines are available, and for what diseases. No single person, especially a general practitioner, can keep up with all of the information available on drugs, let alone health care. By one estimate every year some 1,700 articles are published in each of 325 professional journals on the 25 top medicines. Drug producers use a variety of promotional efforts to stand out in this information flood. One may like or hate the industry’s tactics, but there is nothing illegitimate about them.
 
Per Dennis Ausiello and Thomas P. Stossel (both of Harvard Medical School):

The real intent of these critics goes far beyond food and trinkets, and its true purpose is to curtail strictly or even eliminate all contacts between physicians and private industry. We strongly oppose this agenda. Despite extensive training, physicians cannot know the details of all products, especially new ones. Therefore, company salespersons complement physicians’ information derived from many sources. They tell physicians about a limited range of products about which their employers train them under strict FDA regulations. We believe that the best approach to optimize cost effectiveness of product prescribing is to promote more, not less, interaction among all stakeholders involved in health-care delivery, including company marketing reps.

From a strictly free market perspective, if there were only one drug company, there would be for that entity to speak with physicians. But who marketed anything in the Soviet Union? Imperfect though the process might be, marketing promotes price competition and lowers prices.
 
According to Paul H, Rubin, Professor of Law and Economics at Emory University and former Chief Advertising Economist at the Federal Trade Commission and Chief Economist at the U.S. Consumer Product Safety Commission:

Drug company reps offer overworked doctors useful, lifesaving information in an efficient manner. The drug companies are of course motivated by profit, but economists have known since Adam Smith that the profit motive is the best way to induce someone to do something useful. Marketing and research are both information activities; they work together to get effective drugs to patients. The two activities are not in competition for resources. The denouncers of drug companies don't understand this. One of the senators sponsoring the bill suggests that "the millions of dollars these companies spend on marketing ... could be put into research." In fact, drug companies would not switch money from marketing to research. If they cannot market drugs in the best way, they will reduce spending on research. What's the point of inventing a new drug if doctors and patients don't know about it?
This is crucial -- in all of the medical literature on drug sales, there was no evidence of harm to patients caused by doctors and drug reps sharing a few slices of pizza. Physicians who, by their oaths put patient welfare first wrote these articles. Yet they were critical of the industry based on analyses that totally ignore the only measure that really counts – patient outcomes.

“Good for sales” and “Good for the public health” are not mutually exclusive.

A valuable takeaway from the new JAMA study should be that wide adoption of Open Payments reporting has led to transparent interactions and value exchanges of education, money and meals between the pharmaceutical industry and prescribers. These data are now available to inform and improve educational efforts to meet the treatment needs of patients using the latest advances in medicine and science. However, such data must be cautiously interpreted with full acknowledgement of study limitations and author bias.

In summary, the new JAMA study is devoid of any data regarding patient outcomes; omits all the variables physicians consider when treating their patients; assumes pharmaceutical sponsored meals are purely social gatherings in which no educational information is shared; and reduces complex prescribing decisions to a simple transaction.

“The best interest of the patient is the only interest to be considered.” -- William Mayo, MD


   Read More & Comment...

Platform Prattle

06/28/2016 06:56 AM | Peter Pitts

Draft Democratic platform calls for both drug importation and direct Federeal negotiation for Medicare and Medicaid.

Populist rhetoric isn't good for the public health.

"Facts," as John Adams said, "are pesky things."

   Read More & Comment...

Frank and Honest Disucssion on the Value of Innovation

06/27/2016 10:26 AM | Peter Pitts

Pharmaceutical innovation has not only revolutionized the field of health care and significantly contributed to the fight against cancer, but it also allows Canadian governments to save billions of dollars. This is the general thrust of a Research Paper published today by the MEI, prepared by Frank R. Lichtenberg, Professor at the Columbia University Graduate School of Business and internationally renowned expert in this field of research.

Taking into account just the effects of new cancer drugs, Canadian governments registered savings of $4.7 billion in hospital expenditure in 2012 alone, whereas total spending on cancer drugs, old and new, was an estimated $3.8 billion that same year.

“If no new drugs had been registered from 1980 to 1997, the number of hospital days in 2012 would have been almost twice as high,” says Professor Lichtenberg, who has published numerous articles on the issue in a variety of scientific journals. “This represents in one single year net savings of at least $900 million for the Canadian health care system.”

The costs of new pharmaceuticals are often the subject of critical media coverage, but their benefits are rarely mentioned. Yet pharmaceutical innovation is responsible for a large part of long-term improvements in the health and longevity of patients.

For example, the premature (before age 75) cancer mortality rate declined by 8.4% from 2000 to 2011 in Canada. This rate would instead have increased by 12.3% in the absence of pharmaceutical innovation, implying that 105,366 years of potential life before age 75 would have been lost in 2011 alone.

“Although new drugs are expensive, this cost is small when compared with the benefits they provide for patients,” argues Professor Lichtenberg.

The publication also points out that financial incentives are a prerequisite for the industry to sustain a robust rate of pharmaceutical innovation. “It is important for drugs to be appropriately priced in order for manufacturers to have the proper incentives to invest in the development of new molecules, a very risky process that can be very expensive for the innovating company,” argues Youri Chassin, Research Director at the MEI.

The Research Paper entitled The Benefits of Pharmaceutical Innovation: Health, Longevity, and Savings was prepared by Frank R. Lichtenberg, Courtney C. Brown Professor at Columbia University Graduate School of Business and Associate Researcher at the Montreal Economic Institute. This publication is available here.
   Read More & Comment...

ICER's Moral Vision: Repair Potholes Instead of Rescuing Lung Cancer Patients

06/23/2016 03:34 PM |

After concluding that because myeloma drugs keep people alive longer they cost too much to be cost-effective, ICER through it’s Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) is now looking at new drugs for treating people with non small lung cancer.

And ICER’s Steve Pearson wants you to know that he and his organization care about patients.   ICER’s website has a video of Pearson speaking before the Midwest CEPAC meeting entitled:  “Why we are here today” – Dr. Pearson underscores the moral vision of the Midwest CEPAC”   (Yes, that's the title


In proclaiming this moral vision Pearson said: “we really want to know from patients what outcomes matter most to them.”

He also claims that the quality adjusted life year does not mean a life is “less valuable if they have or disability. a gain is a gain wherever you are starting from.  Sometimes there are conditions where we have a serious condition and the first possible treatment, that’s an important consideration.”

These are outright falsehoods that hide Pearson’s real moral vision.  

Pearson – and ICER – are using a superficially low QALY measure that does NOT take into account what matters to patients, by overstating and misrepresenting the price of new medicines and by clearly using budget caps to cut off and ration care.

And ICER’s metrics and methods are indeed driven by Pearson’s moral vision which he stated clearly in an article entitled: “Which Orphans Will Find a Home: The Rule of Rescue in Resource Allocation for Rare Diseases” where he tells us what he really thinks about lung cancer patients: 

(Spoiler alert:  Pearson thinks lung cancer patients are underserving whiners. )

Before directly attacking lung cancer patients, Pearson argues “There is no apparent obligation to rescue identifiable rare disease patients based on a duty of rescue within personal morality.” 

But what about, as Pearson said, taking into account new treatments for people with the most serious conditions and few options?  

“In practice, however, a sickest-first principle might require allocation of resources even when only minor gains can be achieved and the cost is very high, which is obviously inefficient…coverage decisions must not only incorporate consideration of the benefits gained
but the opportunity costs incurred when covering expensive orphan drugs.

And contrary to Pearson’s claim about not considering cost in recommending what drugs to use, he clearly regards the growing number of expensive therapies that offer benefit only to small populations” to “ensure that an undue burden is not
placed on others for the sake of a few.” 


Then Pearson goes on to show that people with non small cell lung cancer aren’t worth spending money on based on his/ICER’s estimate of opportunity cost. 

First, he claims, contrary to his desire to engage patients, that patient advocacy is a pain in the ass that gets in the way of making cost-based decisions for the good of all:

“Publicity can be a powerful and important tool for advocacy groups, but it is not an appropriate ethical justification for coverage of particular orphan drugs over others.”  

Note that people said the same thing when AIDS activists were demanding faster and broader access to new medicines. 

And he singles out people with lung cancer as a patient group that is unethically using advocacy: the pressure to treat every (lung cancer) patient is a product of lobbying and driven largely by the heightened public consciousness surrounding lung cancer.”

With that bias, Pearson then applies ICER’s benchmark for limiting access to new drugs (in this case Erbitux or cetuximab) based on QALY and budget impact: 

“Lung cancer is often lethal, but the marginal benefits of cetuximab are quite modest. The average survival advantage from adding cetuximab to the standard treatment regimen is approximately five weeks. Cetuximab treatment is also associated with higher frequencies of rash, diarrhea, and febrile neutropenia, a condition that increases the risk of infection. And, lastly, cetuximab is expensive at both the individual and
population level.

Although nonsmall cell lung cancer is technically a rare disease, 60,000 patients are diagnosed with the illness each year in the United States. The treatment costs for each individual patient average approximately $80,000, which translates into an expenditure of $4.8
billion dollars per year. " (My note: In fact, Erbitux total revenues worldwide in 2015 were $2.2 billion)

Pearson concludes: “Considering the sources of identifiability (patient advocacy), the marginal impact on the length and quality of life, and the implicit opportunity costs of this level of expenditure, our framework would suggest that public and private insurers would be justified in refusing to pay for cetuximab.”

Pearson believes that unless someone like ICER decides what a life is worth and how much to spend on it, people with rare and fatal conditions – the sickest first -- will be “siphoning off resources for other things we need like better schools and more resources for local police, roads and bridges.”

That’s Pearson’s true moral vision.  Refuse to pay for lung cancer drugs and spend the dough on pothole repair.  

      Read More & Comment...

CREATing a generic "get out of jail free" card

06/23/2016 03:03 PM | Peter Pitts

From the pages of Drug Industry Daily …

Lawmakers, Generics Companies Praise CREATES Act

Senate Judiciary Committee members and generics makers Tuesday both sung the praises of a bill aimed at preventing branded drugmakers from restricting access to their products. The bill is aimed at companies that restrict access to samples, thereby preventing generics companies from reverse-engineering a product, or requesting a distribution safety protocol and then blocking generic companies from participating.

Innovator drug companies came under fire during the subcommittee hearing, with both senators and generic drugmakers accusing them of using the FDA-mandated REMS process to block generic competition.

Beth Zelnick-Kaufman, assistant general counsel at generics maker Amneal Pharmaceuticals, said the CREATES Act “provides necessary remedies” when innovators refuse to provide samples of their product.

Zelnick-Kaufman cited an example of her company attempting to join a brand REMS with an unidentified drugmaker to launch a product designed to treat drug addiction. She said the brand made $1 billion as a result of delay tactics. The unnamed company’s effort to block generic access ended only after the FDA issued its first waiver of the REMS requirement, she said.

Robin Feldman, a professor at the University of California Hastings College Of Law, also applauded the bill. She cited a study she is currently conducting which has found the increase of REMS abuse to be “abundantly clear.” She said the result of REMS abuse delays has cost billions of dollars in savings in recent years.

Only one of the six panelists demonstrated some degree of opposition to the bill. Peter Safir of the law firm Covington & Burling expressed concerns over the consistency of the CREATES Act with the language of the FD&C Act.

Safir notes that FD&C Act includes several civil and criminal penalties that can be brought against drugmakers for violating a single REMS requirement, saying the CREATES Act fails to amend the FD&C Act to protect innovators. He says this can confuse the brand name drugmakers and expose them to enforcement.

Peter Pitts, president and founder of the Center for Medicine in the Public Interest and a former FDA associate commissioner, also expressed some reservations about the bill. He told DID that the bill significantly overreaches what it wants to accomplish, which could result in unintended consequences to patient safety.

Pitts also described the bill as a “get out of jail free card” for generics makers.

PhRMA spokeswoman Holly Campbell told DID that the drug lobby is currently reviewing the legislation, but that it would be concerned if it jeopardizes patient safety in regards to REMS.

The CREATES Act was introduced last week by members of the Senate Judiciary Committee, including Chairman Sen. Chuck Grassley (R-Iowa), ranking member Patrick Leahy (D-Vt.), Sen. Amy Klobuchar (D-Minn.) and Sen. Mike Lee (R-Utah).

The Senate Judiciary Committee did not return a request for comment by press time as to whether a date for a vote has been set.  Read More & Comment...

JAMA Peddles Rx Payment Propaganda Posing as Research

06/22/2016 05:26 PM |

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 |

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 |

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 |

 
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 |

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 |

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 |

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...