CREATES Act a Tort Torte

03/16/2018 10:45 AM | Peter Pitts

When members of the tort bar start to salivate over a piece of legislation, it’s worthwhile to find out where the red meat resides. In a rush to pass legislation to “lower drug prices” as a budgetary pay-for, lawmakers are pushing two pieces of parallel legislation, the Senate’s Creating and Restoring Equal Access to Equivalent Samples (CREATES) Act and the House’s Fair Access for Safe and Timely (FAST) Generics Act.

 It’s time to take a breath – because neither bill will speed generic drugs to market or lower the cost of medicines for a single American. What they will most certainly provide is a windfall for the trial lawyers.  

Both bills aim to provide a series of new legal provisions that will make it easier for drug companies to introduce generic alternatives, thus spurring competition and bringing down prices. Both are well intentioned. But they are poorly worded and that is a serious matter: Instead of bringing generics to market sooner, these bills could endanger patients’ lives and encourage costly, needless litigation.

To protect consumers, the Food and Drug Administration requires that new drugs undergo a series of clinical trials to prove their safety and effectiveness before entering the market. Generic drugs must also complete clinical trials, but only to prove they’re clinically equivalent to the already-approved brand-name drug. The problem comes when some drugs are so potent, or have such dangerous side effects, that the FDA requires drug companies to develop and abide by specialized safety protocols called “risk evaluation and mitigation strategies,” when selling or dispensing these medicines.

Rather than keep these safety measures, both bills strip the FDA of its watchdog role. Generic manufacturers will be exempt from outlining testing and safety protocols for the FDA to approve. Even if a generic drug maker’s proposed risk evaluation and mitigation strategies are inadequate, the FDA will have no authority to reject or halt the transfer of medicines to the generic company for testing.

The ambiguously worded liability provisions of the two bills further subject innovators to unfair legal risk. The reason is many generic companies, after obtaining brand-name drug samples for testing, ship them to third-party research firms to perform clinical trials. If the third party is negligent with the samples, patients could get hurt. Yet, under the bill’s terms, patients could sue the brand-name drug company, even though it had no control over the testing or safety protocols. The higher legal fees for drug companies will result ultimately in higher costs for everyone else.

Additionally, the bills would allow generic drug manufacturers to sue if brand-name manufacturers fail to hand over their drug samples for testing within 31 days, or if the companies do not reach an agreement on shared risk evaluation and mitigation strategies for risky drugs. Fine concepts, but the actual administrative language is ambiguous and subjective wording is music to trial lawyers’ ears.
 
Over 60 percent of Americans want the government to take action to lower prescription drug prices — and Congress, for once, is listening to voters.

Unfortunately, the legislation before Congress will lead to dangerous unintended consequences. Instead of bringing generics to market sooner, these bills could endanger patients’ lives and encourage costly, needless litigation. Both houses of Congress deserve praise for trying to bring generic medicines to market faster, relieving consumers from high drug prices. Yet good intentions don’t change the fact that the legislation, as currently constructed, is deeply flawed.

Congress could help consumers by reworking the imprecise phraseology to end bad behavior without gutting safeguards for patients or enabling unscrupulous trial lawyers to file costly, pointless suits. Whether it’s the practice of medicine or the development of public healthcare policy two rules apply – first, do no harm and, second, be wary of trial lawyers bearing gifts.  Read More & Comment...

Contract Killers: Inside the Secret World of PBM Practices

03/15/2018 03:02 PM | Peter Pitts

AXIOS: Inside a drug pricing contract
By Bob Herman, March 15, 2018

A contract template used by Express Scripts, the largest pharmacy benefit manager in the U.S., provides a window into how pharmacy benefit managers — middlemen that manage drug coverage for businesses throughout the country — steer negotiations with drug companies to benefit their own financial interests.

Why it matters: These benefit managers have a lot of power over the prescription drug coverage people get through their employers, and they're supposed to negotiate discounts so coverage is cheaper for insurers and employers. If they're not making it cheaper, there's less chance people will get relief from high drug prices.

The details: Axios obtained a 36-page Express Scripts contract template from a source who works in the health care industry. Express Scripts and employers use the document as a starting point to determine how medications are paid for and how pharmacy networks work, but the contract usually is not in the public's view. Since it's a template, there are no hard numbers or terms of any specific agreements.

The big takeaway: There's nothing illegal about these contracts. But the language is clearly written with the PBM's financial interests in mind, and critics say those kinds of provisions can result in lost savings for everyone, especially for small companies and their employees.

Even some of the largest companies think they are protected because they have in-house and outside attorneys vetting contracts, yet that's not necessarily the case.

"That's a little bit like going to Las Vegas and consistently thinking you can beat the house at their own game," said one source who has worked in the industry for many years. "These PBMs have entire departments of lawyers where this is their game."

The other side: Express Scripts, which is in the process of being acquired by Cigna in a $67 billion deal, didn't dispute the contract template was its own. But spokeswoman Jennifer Luddy said in an email the document was "several years old," although some sources said it appeared to be current.
Luddy added that employers are "savvy purchasers of pharmacy benefits" and that these contracts are common: "It is industry standard terminology used by all PBMs, and is well-understood by clients and consultants."

In a follow-up email, Luddy said: "It is clear to us that there are several vocal PBM critics who are eager to provide their biased interpretations of this template contract to serve their own agenda."The details: These are some of the major provisions. The contract was explained in interviews with several people who work in or are familiar with the pharmacy benefit industry, most of whom asked not to be named given the sensitivity of the issue and to speak candidly.

Rebates

A primary function of a PBM is to negotiate rebates from drug companies. Most of those rebate dollars flow back to employers (not workers). But Express Scripts collects other rebate-like fees from drug companies that it doesn't have to pass along to employers.

The Express Scripts contract explicitly says "rebates do not include things" like "administration fees" from drug manufacturers, "inflation payments" and numerous types of "other pharma revenue."

"There are so many carve-outs of what they consider a rebate that it’s very murky of what’s being kept and what’s being passed through (to clients)," an industry source said.

The contract also says Express Scripts negotiates rebates "on its own behalf and for its own benefit, and not on behalf of sponsor."The brand/generic algorithm

Multiple people said the "proprietary" algorithm is one of the most important definitions, as it gives Express Scripts full authority to determine whether a drug is brand or generic without being transparent.

The algorithm allows Express Scripts to pocket the difference between a brand-drug discount and a generic-drug discount — a major tactic to maximize profits.

"This is why they don't miss earnings," said one person familiar with the industry.

Payment schedules

The "MAC list" and "maximum reimbursement amount" also permit Express Scripts to pay for drugs in a way that is "most advantageous to them," according to a source.

For example, using these different lists of drug costs, Express Scripts can charge its employer clients $15 for a particular medication but pay the pharmacy just $1 for the same medication — and keep the extra money for itself.Financial disclosures and auditing

The last two pages rehash some of the initial definitions, but also reiterate how Express Scripts can collect almost any type of revenue it wants and "may realize positive margin" — code for reaping big profits and not having to share with employers.

Employers can choose to have their agreements audited, but they have to get Express Scripts' approval on what auditor is used.

And sometimes they don't get it. Hayes, a pharmacy benefit consultant who agreed to review the document and speak on the record, said Express Scripts has not allowed her firm to conduct audits.  Read More & Comment...

Fighting Illini Fight Non-Medical Switching

03/15/2018 12:08 PM | Peter Pitts

Per this article in the Chicago Tribune, llinois lawmakers are considering a bill that would guard non-medical switching in the middle of a plan year. The proposed bill would prohibit commercial health insurers from modifying coverage of a drug during the plan year if it has previously approved the drug for a medical condition.  Read More & Comment...

Santa Claws

03/14/2018 02:45 PM | Peter Pitts

If drug manufacturers are giving such large discounts for brand name medicines to Pharmacy Benefit Managers (PBMs); while prices of commonly used generics keep going down, why aren’t co-pays going down and why, in some circumstances, are they going up – even for generic medicines?

In short, where’s the money going?

The answer, according to a new study just published in the Journal of the American Medical Association, Frequency and Magnitude of Co-payments Exceeding Prescription Drug Costs, is … from the purses of patients into the pockets of the PBMs.

Per the JAMA article:

Pharmacies collect patients’ co-payments and pass them to PBMs, who reimburse the pharmacy a negotiated rate to cover drug costs, dispensing fees, and any markup. Overpayments occur when the co-payment exceeds the negotiated reimbursement.

The scheme is called “claw-backs.”

Per JAMA:

However, drug co-payments sometimes exceed costs, with the insurer or pharmacy benefit manager (PBM) keeping the difference. Furthermore, some pharmacists are contractually prevented from alerting patients when their co-payment exceeds the drug’s cash price. Although some have argued that the practice is uncommon, a 2016 survey of independent pharmacists indicates otherwise.

No, you read that correctly, PBMs lock-in these claw-backs, going so far as to contractually gag pharmacists who want to help patients lower their drug costs.

Some of the study highlights include:

* Among 9.5 million claims, 2.2 million (22.94%) involved overpayments.

(That means that almost 1 out of 4 prescriptions involved a patient copayment that exceeded the average reimbursement paid by the insurer. The vernacular for this is – stealing.)

* The most commonly prescribed drug, hydrocodone/acetaminophen, involved an overpayment on 36.15% of claims.

(Could this explain why PBMs make time-consuming prior-authorization for abuse-deterrent opioids and non-opioid pain alternatives such common practice?)

* Overpayments were common in this data set, affecting 23% of all prescriptions, and 28% of generic prescriptions.

(Price gouging on generics! Shameful.)

* In 2013, total overpayments by patients amounted to $135 million in the sample studied

The authors conclude:

Cost-related nonadherence is common and associated with increased medical services use and negative health outcomes. By raising patient costs at the point of sale, overpayments may exacerbate these effects. To lower patient expenses, legislation addressing overpayments and gag clauses warrants further investigation.

Amen.  Read More & Comment...

FDA and the Digital Divide: The Battle of Proof & Predicate vs. Errors & Upgrades

03/13/2018 08:34 AM | Peter Pitts

There is a yawning divide between regulatory science and digital development. Digiratti view regulators as stodgy while regulators view digital developers as trigger-happy. There is an unproductive cognitive disconnect.

When we consider the integration of new and exciting digital technologies (ingestible, implantable, portable, app-based, diagnostic, etc.), it's likely that technologists are far more likely to be excited about the possibilities rather than considerate of the risks. The same cannot necessarily be said of regulators/reviewers who reside within a culture of proof and predicate. Technologists inhabit a planet of errors and upgrades. There is no "Beta" approval pathway for the FDA.

For the FDA, risk exists to be minimized while for digital developers risk is an opportunity. Fortunately, there is common ground – and it isn't the technology. It's the public health need for which the technology presents a safe and effective (within the FDA definition of that duality) solution. Interestingly, it's the drug developer who must now play the role of “learned intermediary” between regulator and technologist -- a new and uncomfortable role. But the pay-off is worth the effort for sponsor, regulator and public health advocate -- better patient outcomes through more evolved 21st century technology integration.

Consider Adherence/Compliance, a public health problem of brobdingnagian proportion nowhere more acutely felt than in patients with schizophrenia. That's why products that address new and innovative solutions (such as Abilify MyCite, a pill with a sensor that digitally tracks if patients with schizophrenia  have ingested their medication) are so exciting to developer, regulator and patient alike. It's a real world example that should provide momentum for continued development beyond this one therapeutic area.

As real world data becomes available, the FDA will hopefully feel increasingly comfortable expediting similar programs (specifically) and programs with more innovative uses of digital technologies (more broadly).

Positive signals from the FDA will send potent messages to developers that further investment in such clinical programs is worth the investment risk. And positive signals emanating from “the patient voice” will be crucial.  Read More & Comment...

Cratering CREATES

03/09/2018 09:46 AM | Peter Pitts

The Creating and Restoring Equal Access to Equivalent Samples (CREATES) Act will not be included in the pending omnibus budget bill, according to BioCentury, citing lobbyists. Although the bill has bipartisan support and is aligned with Trump administration policies, a combination of lobbying muscle and political calculation have scuttled efforts to have it included in the omnibus bill. The bill seeks to prevent brand-name drug makers from using Risk Evaluation and Mitigation Strategy programs to avoid selling samples that are needed to develop generic versions and block generic competition.  Read More & Comment...

Gottlieb call out "Kabuki Pricing"

03/08/2018 09:52 AM | Peter Pitts

FDA's Gottlieb blames industry 'Kabuki drug pricing' for high costs

WASHINGTON (Reuters) - U.S. Food and Drug Administration chief, Scott Gottlieb, criticized pharmacy benefit managers, health insurers and drugmakers on Wednesday for “Kabuki drug-pricing constructs” that profit the industry at the expense of consumers.

The comments, made at a conference organized by a leading U.S. health insurer lobbying group, stoked speculation over what steps the administration of U.S. President Donald Trump may take to rein in lofty prescription drug costs.

“Patients shouldn’t face exorbitant out-of-pocket costs, and pay money where the primary purpose is to help subsidize rebates paid to a long list of supply chain intermediaries,” Gottlieb said at the meeting of America’s Health Insurance Plans (AHIP). “Sick people aren’t supposed to be subsidizing the healthy.”

The remarks surprised meeting participants and spurred new accusations between leading members of the drug supply chain. Shares of top pharmacy benefits managers CVS Health Corp and Express Scripts Holding Co fell 1.4 percent and 2.4 percent, respectively.

He criticized the health industry for failing to promote access to so-called biosimilar versions of drugs, and for pricing practices that harm consumers.

Biosimilars are copies of original drugs that are supposed to be as effective but cheaper. Kabuki is a form of Japanese theater characterized by dramatization and elaborate costumes.

Gottlieb said practices in the healthcare industry “obscure profit taking across the supply chain that drives up costs” and discourage competition.

As FDA commissioner, Gottlieb has prioritized approving more generic drugs to help lower prices, allowing more than 1,000 copycat drugs into the market last year, he said.

Still, while the agency has approved nine biosimilar therapies to date, only three have reached the market, Gottlieb said. The rest have been mired in legal challenges brought by drugmakers such as AbbVie Inc to protect its multibillion-dollar rheumatoid arthritis treatment Humira.

Trump has vowed repeatedly that his administration will take more steps to lower drug costs, and included some potential actions in a proposed budget made public last month that Congress is not likely to accept.

Other regulatory actions could come directly from Health and Human Services Secretary Alex Azar, a former drug company executive, and through the department’s Centers for Medicare and Medicaid Services. Azar is scheduled to deliver remarks at the AHIP conference on Thursday.

Gottlieb noted that the top three pharmacy benefit managers - CVS, UnitedHealth Group Inc and Express Scripts - control more than two-thirds of their market. The top three wholesalers - AmerisourceBergen Corp, Cardinal Health Inc and McKesson Corp - control more than 80 percent; and the top five pharmacies more than 50 percent, he said.

AHIP responded by saying drug manufacturers were to blame for the high cost of prescription medicines. The Pharmaceutical Care Management Association, which represents pharmacy benefit managers, also said it was unfair to place blame on payers who cannot control the prices drugmakers set.  Read More & Comment...

Will Price Transparency “Unintentionally Raise Costs”

03/07/2018 10:24 AM | Peter Pitts

Here’s the PBM argument – blame drug companies for high prices and complain when they’re asked to be transparent about their own discounts. Per Express Scripts, transparency will “unintentionally raise costs.” Hm.

Legislators seek to protect consumers from high drug prices

HARTFORD — For six months, Branford resident Robin Comey waited for the price of her son’s prescription asthma medication to drop.
She scoured the internet for coupons and researched generic alternatives, but the cost cut her family needed never came.

Self-employed, Comey has health insurance, she said, but even with deductibles the price of her son’s medications were sometimes out of reach. She was already spending nearly $2,000 a year on epi-pens for her son’s food allergies.

“Our family feels a little bit taken advantage of,” Comey told legislators Tuesday.

Legislators and State Comptroller Kevin Lembo hope to make medications more affordable for Connecticut residents like Comey by increasing transparency around prescription drug pricing.

A bill they unveiled Tuesday would allow consumers to pay post-rebate costs instead of marked-up retail prices for prescription drugs.

In addition, drug manufacturers would be required to justify price increases above 25 percent, and pharmacy benefit managers — middlemen like CVSHealth and Express Scripts — would have to disclose the rebates they receive from manufacturers and how much of the rebate was passed down to consumers.

Lembo said most people pay inexplicable drug price mark-ups without even realizing it through insurance premiums, taxes and buying prescriptions.
“There is only one way to bring any free-market fairness to this realm: by shining a bright light onto a shadowy market,” he said.

Rep. Sean Scanlon, D-Guilford, said he hopes the legislation will uncover why drug prices are rising so health care costs can ultimately be lowered.
“Prescription drug costs are the fastest rising cost in health care and consumers are rarely given an explanation when the costs of their drugs increased,” he said.

Although the proposed bill received support from AARP and many individuals, it faced opposition from pharmacy benefit managers, health organizations and the Connecticut Insurance Department in a public hearing Tuesday.

The Pharmaceutical Care Management Association, a trade association for pharmacy benefit managers, argued that such legislation is preempted by federal benefits law and therefore is unconstitutional. They also said the bill might damage PBMs’ ability to negotiate lower drug costs.

“Any public disclosure of rebate information would allow manufacturers to learn what type of price concessions other manufacturers are giving, thus establishing a disincentive from offering deeper discounts,” said April Alexander, assistant vice president for State Affairs for PCMA. “This transparency will not lead to better health care or lower health care costs.”

Express Scripts, a PBM, also testified that secrecy was a necessary part of obtaining rebates for consumers and disclosing them would  

Connecticut’s largest union of doctors and health care workers District 1199 opposed the bill because they thought it did not go far enough to stop price gouging by drug companies.

The National Physicians Alliance in Connecticut called it “toothless.”

Both groups urged legislators to implement the Connecticut Healthcare Cabinet’s recommendation to establish a Drug Review Board to investigate price abuses and empower the Attorney General to act on uncovered abuses.

The Insurance Department claimed it did not have the authority or personnel to oversee parts of the bill.
   Read More & Comment...

Stem Cell Pioneer Bob Hariri:The Edison of Regenerative Medicine

02/16/2018 10:31 AM |

Today Robert Hariri, one of the pioneers in the development of stem cell therapy, launched Celularity a company with a mission to make regenerative medicine as affordable and as convenient as the polio vaccine. 

To give you an example of what Hariri (full disclosure, who is a good friend) envisions, consider the excitement over immunotherapy medicines that,"harness the power of the body's immune system to fight cancer" and chimeric antigen receptor (CAR) T cell therapy in particular such as those developed by Novartis and Kite. 

CAR-T is not a pill.  Or a shot you can get at Walgreens.  Instead, it is a very complex and expensive process where immune cells are removed from someone with cancer and then armed with new proteins that allow them to recognize cancer.  Then large quantities of these cells are injected into the patient.  These cells persist in the body, becoming “living drugs.”

Put another way, CAR-T is the ultimate hand-crafted therapy.   

They are true miracles.  But the cost, complexity and the need to carefully administer CAR-T at a handful of academic cancer centers means that making them available to everyone who might benefit will not be easy. 
 
Further, the laborious customization characterizing CAR-T is due to its dependence on using one's own cells and then engineering them to avoid an immune response that shuts downs every organ. Donors for bone marrow transplants are hard to come because of the danger that cells from another body will be viewed as invaders by our immune system. 

Democratizing cell therapy requires a reliable source of cells that can be mass produced and used off the shelf in any physician's office.  Only one scientist on the planet has developed a nearly inexhaustible source of stem cells that overcome the immune system's lethal resistance.  

That is Bob Hariri.  And there is only one company on the planet that can broadly manufacture, develop and distribute cell therapies of all kinds - including CAR-T - on an everyday basis. And that's Celularity. 

Many of the media accounts of Celularity's launch note Hariri's distinguished career as a scientist and the fact that he holds more cell therapy patents than any other researcher.  

In an ironic sort of way, this is not surprising.   Hariri, like his hero Thomas Edison, does not consider himself just a discoverer of new gadgets.  As Sir Harold Evans wrote in "They Made America: "Thomas Edison is thought of as America's foremost inventor, with 1,093 patents in his name, but his most important work was translating the insight of invention into the practical reality of innovation through the long process of development and commercial introduction."

"Edison's transcendent innovation was to understand that the lightbulb he invented would be a mere novelty unless he could find a way to integrate it into an economical and safe electrical system. The simple act of flicking a light switch in offices and homes depended on a complex of dynamos, cables, and numerous connections that all had to be devised, costed and manufactured. Edison had also to fulfill the entrepreneurial role of raising the money, arranging the legal rights-of-way and cultivating the market. Edison was a supreme innovator."

So too is Hariri, who in many ways is the Edison of regenerative medicine.  Over 15 years ago, he demonstrated that it was possible to collect 10 times as many stem cells from a single post-birth placenta as have been gathered from any other single source. He then promptly told the rest of the scientific world the recipe for doing so.  His hope was that stem cells would be democratized, and be broadly available to researchers, doctors, and patients instead of being controlled by a handful of well-connected scientists who used their dominance to stifle other forms of stem cell research.   

But incredibly, very few researchers seized the moment.  So, Hariri - while at Celgene - invested nearly a decade and $500 million on democratizing stem cells. 

First, he had to develop a process of procuring placentas under tight quality-control systems and with a high level of predictability. 

Second, he had to create a manufacturing scheme that's necessary for economies of scale and quality control. Both were and are necessary for cell therapy to be a realistic clinical tool.

Third, he had to find the right "light bulb."  (There were other lighting technologies that use electricity before Edison.  And Edison himself develop dozens of light bulbs that worked.  The challenge was developing a product that was not just affordable, but reliable.  And to demonstrate that, Edison, as Sir Harold points out, had to create a whole system of distribution and uses.) 

One of those "light bulbs" were, in fact, CAR-T.  At the time, Hariri build CAR-T to see how they stacked up against placental-derived stem cells is delivering cancer-fighting instructions to the immune system.  The placenta is unique in that it is not rejected by the body.  Rather, a placenta can modify the host immune system and evade any host immune detection. As Hariri told me: "That was the premise behind our searching the placenta for these cells, and it served as the basis for our product development and, ultimately, clinical development, which has shown that allogeneic transplant is accepted without problems."

To demonstrate that capacity, Dr. Hariri has developed and used placental stem cells to treat several diseases at every stage of life. He has shown it is possible to use placental cells to update our biological software, upgrade the cellular 'processors' that create the connections and share the information that regeneration requires. 

Now with Celularity he is poised to manufacture stem cells that as Hariri told me, can be "easily deployed, readily adoptable like a medicine, and could integrate into the existing healthcare system, where practitioners are most comfortable with traditional pharmaceuticals".

That means millions, if not billions, of people, will live longer free from disease and disability.  By anyone's estimation, the lightbulb's impact on humanity would pale in comparison to that accomplishment if Hariri and Celularity succeed in commercializing stem cells.   Read More & Comment...

Express Scripts Prescription For Reducing Drug Spending: Increase Rebates and Limit Access to New Medicines for the Sickest Patients

02/13/2018 02:02 PM |

 
Express Scripts (ESI) reports that drug spending increased at the slowest pace in 25 years.  Or, as the headline from the PBM’s press office touts: “Express Scripts Reduces Employers' Annual Prescription Drug Spending Growth Rate to Historic Low in 2017”
 
That may or may not be a good thing for patients.  A closer look at how the PBM achieved this suggests that it is almost entirely due to a significant reduction in the number of new patients with HIV, Hepatitis C and treatment-resistant high cholesterol for the year before.  From 2016-17 generic drug use in those categories declined as a percent of drugs dispensed while the use of new meds increased.  If the ratio is tilting towards brand drugs it means the total number of peoples being treated for these conditions declined. 
 
It could be a yearly anomaly due to fewer people needing treatment.  Or it could be the result of increased patient cost sharing, the use of drug exclusions, step therapy, etc.  Indeed, Express Scripts credits its SafeguardRx program which uses all these tools for the reduction in the rate of drug cost.   
 
Moreover, the low rate of growth does NOT translate into lower costs for patients.  Indeed, as noted, the increase in cost sharing through separate drug deductibles, higher copays, co-insurance means that the decline is a result of an increased burden on consumers.  The fact that specialty utilization declined in Medicaid and ACA plans, while Medicare specialty spend increased more slowly than in commercial plans suggests that higher cost sharing and restrictive access played a key role in reducing the rate of increase.
 
Further, it appears that Express Scripts is not counting the rebate dollars shared by plans in the estimates.  Yet member cost is what is paid at retail.  ESI backs out rebates from the unit and total costs but includes cost sharing of patients.  Drug mix is another element calculated in coming up with unit cost.  As noted, the mix of generic and specialty spend – as well as the mix of traditional and specialty is moving towards specialty drugs. That means most of the increase in unit cost apart from certain drug categories where utilization dropped, is the result of increased member cost since cost is net of rebates. And as for ESI’s claim they are more than happy to pass rebates to patients instead of plans, it ain’t happening as Drug Channels points out.
 
Bottom line: Fewer people with chronic or complex conditions are getting access to new medicines while others are paying a bigger sharing of the increase in drug costs.
 
Does this translate into better health and lower health care costs overall?  And what or who is SafeguardRx protecting?  My guess is it’s ESI’s failing business model, not patients.
   Read More & Comment...

Fake Patient Group Publishes Fake Pharma Profit Study

02/09/2018 03:18 PM |

 
Patients for Affordable Drugs (P4AD), the wholly-owned group advocating on behalf of the Laura and John Arnold Foundation campaign to reduce the number and price of new drugs and limit their access, has produced what it regards as the real cost of developing Kymriah, “the first gene therapy available in the United States, certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL)”.  The therapy is a cure for most patients.  Kymriah’s costs about $475000 but only if it works.  And Novartis is financing the acquisition cost of the medicine in many cases.
 
But P4AD, run by David Mitchell (whose firm – GMMB -- was responsible for running over a billion dollars’ worth of campaign commercials for the Obama ’12 and Clinton ’16 campaigns, is using the price as a target for the ads its 501c4 (P4ADNow)  will be running (using LJAF money) supporting price controls on prescription drugs and attacking congressional candidates who don’t agree with them.   (Most patient groups help patients with their daily lives and support research.  P4AD simply collected stories and names through the 501c3 and is now using them for their political attack on Novartis.)  
 
Many people, myself included, criticized Mitchell for asserting that NIH invested $200 million in Kymriah and that all Novartis did was manufacture the cells and hand them out.   Now, along with academics like Aaron Kesselheim – another LJAF money recipient, he has come out with a ‘study’ posted in a Health Affairs blog that purports to show that Kymriah’s price should be about $160,000.
 
The simulation is pure fantasy.  It is an exercise in ideological accounting carried out to justify the Arnold supported agenda to cut drug prices, including seizures of patients, step therapy, price controls, etc.     A few months ago, another Arnold funded individual, Vinay Prasad published an article that overstated the profits of cancer biotech firms and understated R&D and concluded that cancer drug development yields a 10-50 fold return on investment.  That study was the source of a lot of deserved derision.
 
Mitchell and Kesselheim apply Prasad’s LJAF funded methodology to Kymriah in the Health Affairs blog.  They presume that the development of Kyrmiah carried no risk. (I wonder if the models John Arnold’s used at Enron used the same assumption about energy exploration and distribution.)  The authors claim that the NIH assumed all the cost at the riskiest part of development, preclinical work.  This is nonsense.   The fact that fewer than 1 in 10000 pre-clinical projects become commercialized products underscores the fact that translating biology into products is the most costly and riskiest of enterprises.  
 
To paraphrase an article in Nature: The authors' calculation ” imply that each clinical trial was a guaranteed success. Instead, clinical drug development should be regarded as a series of high-risk wagers where success in the first wager.”
 
For example, Novartis began clinical trials in 2009.  It did not earn any revenue for almost a decade.  The authors assume away the risks of drug development and the opportunity cost of tying up billions for ten years. 
 
Further using cash flows from operating income only (which include revenue and costs), presents unrealistically high valuations for biotechnologies.  “Risk is mitigated as biotechnologies progress through development. When this increasingly mitigated risk is taken into account, the risk-adjusted cash flow can be discounted to arrive at the risk-adjusted NPV.” 
 
In the real world, the present value of each risk-adjusted cost is subtracted from the present value of the risk-adjusted payoff to arrive at the rNPV.   Only by adding together all of Novartis’s costs and risks and then discounting for time, is the true rNPV is finally revealed.   Mitchell does none of that.
 
Finally, the internal cost of capital (6 percent) is ridiculously low.   The internal cost of capital is based on what the market for investment bears and reflects the fact that over time returns will be quite low or non-existent.  In the real world of biotech, especially projects for small groups of patients, the internal cost of capital is estimated at 20% or higher.  As Ian Coburn notes:“This reflects investors’ expectation of a return sufficient to compensate them for taking on extraordinary risk. Permanently lowering realized returns will lead to lower investment in a critical component of the life sciences industry.”
 
In their fantasy world, the authors claim that expected returns could be 60 percent lower and offer investors a rate of return that is lower than US treasury notes.   The cost of capital increases with risk. The authors assume no risk is being taken by Novartis or any other entity in undertaking clinical development.   Indeed, the authors claim at reducing operating income and profits because it’s not fair and Novartis can afford to make less.
 
Even if we accept the notion that Novartis is not charging a fair price, most companies developing cell therapies are NOT Novartis.  They are smaller firms and their costs of capital will be even higher.  The authors seem to think that it is possible to reduce rates of return without affecting how much a company or VCs need to “pay” for outside capital.  (See Prasad piece for another example of this absurd assumption. and a good laugh.)
 
If they think it possible, then by all means start up a company that can reduce prices.  One of Mitchell’s co-authors, Paul Kleughten, was the CEO of a generic drug company.  Let him enter into a partnership with the NIH that gives the agency control over prices.  Let him try to raise capital or find a partner for a firm that presents financial projections and a research plan consistent with their assumptions.  
 
The fact is, their model will reduce investment and drive up the cost of capital needed to support biotech.   Price controls will steer investment into other sectors.  Voluntarily capping profits means less money for other potential cures and will deprive millions of people in the future of their wellbeing and lives.   That’s the reality P4AD and their LJAF funding compatriots offer.   Read More & Comment...

New Federal Budget Bill Blasts Medicare Part D

02/08/2018 03:10 PM | Peter Pitts

The latest federal funding bill could issue a huge blow to beneficiaries of Medicare Part D, the government program that subsidizes the cost of prescription drugs for 42 million seniors.
 
The bill restructures how costs are divided between beneficiaries, insurers, and drug manufacturers once Medicare recipients' prescription costs hit a predetermined limit -- or what's known as the "donut hole." It's a terrible change, and it stands to raise costs for seniors, particularly those whose drug expenses already are through the roof.
 
Medicare Part D provides seniors with access to affordable prescription drug coverage offered by private insurers. There are more than 780 unique Part D plans available in the country, but each requires patients to follow the same payment plan.
 
First, patients must pay for their own medications until they meet a deductible -- $405 in 2018. Then, insurance kicks in and patients pay about 25 percent of their drug costs.
 
After patient's total drug spending hits $3,750, they enter the "donut hole," where they're responsible for 40 percent of brand-name drug costs.
 
Congress gets that the donut hole burdens patients. So they're trying to phase it out. Under current law, patients' cost-sharing would drop to 25 percent by 2020. Insurers would chip in the same percentage and manufacturers would cover the leftover 50 percent.
 
Now, Congress wants to shift the insurers' costs to drug makers. The new proposal would force manufacturers to front 75 percent of the cost of brand-name prescriptions in 2020, reducing insurers' cost-sharing to zero.
 
That change doesn't explicitly affect the percentage fronted by patients. But it will still affect the amount they pay for medications.
 
With their cost-sharing down to zero, insurers will have no reason to keep patients' drug costs low. Instead, they'll have an incentive to increase it -- and they'll put patients on the fast-track to the donut hole.
 
Proponents of the proposal say that it will save Medicare billions of dollars annually. But Part D already is economical. Its costs are 45 percent lower -- $349 billion less -- than initially projected for its first decade. And seniors like how Part D works. Nine in 10 report that they're satisfied with the program.
 
Part D is one of the highest-functioning branches of healthcare. Its performance and the care it secures for America's seniors should not be jeopardized because insurance companies want to shift some costs to drug makers.  Read More & Comment...

Mail Order Confusion

01/31/2018 10:52 AM | Peter Pitts

The recent announcement by Bezos, Buffett and Dimon that they're teaming up to address health care costs is interesting. They certainly bring a lot to the table. But there seems to be some confusion.

USA Today reports that one of the things that Amazon can bring to the table is "shipping products to consumers." True. But there's a problem. According to the article, "Since drug companies rely heavily on mail orders, Amazon's long-rumored entry in the pharamcy world could introduce price-lowering competition."

Good idea -- except that drug companies don't send a single pill to patients. That's the job of PBMs and insurance companies.

Maybe the Big Three (and and the media covering this story) should get a 100 level course in the pharmaceutival supply chain.
   Read More & Comment...

ICER Joins the Walking Dead

01/25/2018 03:34 PM |

Despite receiving another $14 million from the Laura and John Arnold Foundation, ICER is turning into the walking dead.     

The recent spate of ICER reports all come to the same premature and prejudged conclusion: that every new medicine that does not cure is not worth paying for at almost any price.  Overextended and overexposed, ICER is slowly being destroyed by its ideological rigidity and analytic obsolescence.  At a time when the use of data to match people to the right treatments over time and pay for performance at the patient level, ICER has doubled down on one size fits all reports that focus on saving insurers money by cutting drug prices.   It roams the health care policy terrain in search of new targets to devour, guided by the same research methods and beliefs that shaped the eugenics movement.  Like that movement, ICER is finding itself ridiculed and rejected by the same stakeholders that feared it just a year ago. 

Of course, the unspoken but clear assumption behind ICER reports -- the same assumptions informing those who wanted to use eugenics to save society  -- is another reason that Steve Pearson and company have jumped the shark:  ICER assumes the use of new drugs siphons money from healthy people, wage increases, roads, potholes, etc. and that we need to put a limit on how much we pay and how much we spend for new medicines for people, most of whom, are not receiving many benefits from existing treatments. 

These assumptions are laughable, and everyone knows it.  Better medicines reduce the cost of treatment and staying healthy longer.  Longer and better lives generate happiness and wealth, which in turn makes spending on everything -- including health care -- sustainable.  ICER,  now includes, but does not calculate, those goods, services, and actions we enjoy and product because we live longer and healthier.  By listing those virtues but not measuring them, ICER has exposed how superficial and irrelevant it is. 

Beyond that, ICER is unable to close the gap between a new generation of personalized medicines and finding a way to pay for them in order to "enhance health, prevent disease, track its development, intervene early, and manage disease most effectively if it occurs."  Such treatments are based on a deep understanding of what causes disease as well as the individual differences in disease risk and response to medicine. As result, illnesses such as cancer, heart disease, and multiple sclerosis are being treated with greater effectiveness, while many rare or fatal diseases - such as cystic fibrosis, Hepatitis C, and HIV --now have treatments where none existed. 

Simply put, personalized medicine is a powerful tool for extending life and making the delivery of great health, simple, convenient and more affordable.  Yet ICER, captive of it paymasters and increasingly outdated approach, can't produce information to let consumers and everyone else determine which treatments work best for them to live healthier, longer.  Rather, as the ability to deliver personalized medicine grows, ICER only proposes ways to reduce prices for PBMs and limit access.  Meanwhile, PBMs are expanding step therapy, prior authorization and increasing cost sharing as more personalized or precision medicines become available.  That means they are keeping people sick when they should be healthy and forcing them to spend more time and money on substandard care. 

The rebate driven approach to drug benefits is under siege and intelligent stakeholders are seeking other ways to provide patient-centered coverage.  That does not include ICER.

In addition to its need to carry out the societal rationing agenda of the Arnold Foundation, ICER lacks the bandwidth to help promote personalized medicine. The digitalization of medical data and the rapid increase in computing power now permits identifying what treatments work and measuring outcomes and analyzing such evidence to determine the links between the use of medicines and outcomes.  Traditional analytical approaches employ manual, time-consuming, single hypothesis algorithms.  As a result, ICER is limited in its ability to integrate multiple data types and are often limited to population averaged approaches. 

For example, ICER makes all sorts of assumptions about the condition of patients and treatment patterns based on models it develops from clinical trial data.   Such assumptions - including the selection of the treatment it uses to compare new medicines - are based on correlations that lack any basis in the reality of the life of every patient. 

ICER will become increasingly irrelevant.  Other stakeholders could accelerate that process by ignoring ICER's request for 'input' and invest the millions of dollars into creating models that capture personalized treatment response.  

Such models would be based on the probabilistic and causal relationships between disease progression and treatment response (unbiased by methodological and data choices that characterize much of ICER's work) for each patient.  They are less expensive to produce because the machine learning supporting it is automated.  They are quicker to produce and more useful.  

Indeed, personalized medicine models can be used to demonstrate and qualify an approach for using real-world evidence.  The Food and Drug Administration is required to create a guidance and/or pathway for integrating real-world evidence into their approval processes. If the FDA encourages the use of real-world evidence to measure and predict clinical benefit at the individual level, it will force payors to rely more on such analyses and less on those developed by ICER and other groups.  Speedily in our time.   Read More & Comment...

FDA Transparency? WWPT? (What will Pharma think?)

01/16/2018 01:27 PM | Peter Pitts

Important advance at the FDA (courtesy of the Washington Post). WWPT? (What will Pharma think?)

FDA to release more clinical trial information for newly approved drugs

The Food and Drug Administration is taking steps to make it easier for doctors, patients and researchers to get access to clinical trial data amassed during the process of approving new drugs, Commissioner Scott Gottlieb said Tuesday.

Gottlieb announced the actions just before a speech on FDA transparency at a Washington forum. The meeting, attended by researchers and academics, focused on 18 recommendations for making the agency's decision-making less opaque. The suggestions were part of a report called Blueprint for Transparency.

The FDA has long said it is sharply limited in what information it can release because it often is dealing with drug companies' proprietary material.
Gottlieb, in his statement and in remarks to the forum, said the agency is starting a pilot program this month to release clinical study reports for recently approved drugs. These summaries, which are generated by drug-company sponsors of the treatments, spell out the methods and results of clinical trials. The data don't include patient-identifiable information.

The pilot is expected to ultimately include nine drugs volunteered by their sponsors for the effort, Gottlieb told Joshua Sharfstein, a Johns Hopkins Bloomberg School of Public Health professor, in a question-and-answer session at the forum.

The release of the study reports, which can run hundreds of pages, will allow researchers and others “to do more analysis around our decision-making,” especially on the safety and efficacy of new drugs, Gottlieb said. Some of the information is already released by the agency but in a format that is difficult for lay audiences to use, he said.

The commissioner also said the agency will make it easier to track clinical-research information by adding a study's identifier number from ClinicalTrials.gov to all FDA materials for a specific product. ClinicalTrials.gov is the database of studies maintained by the National Institutes of Health.

On another transparency issue, Gottlieb said the agency is exploring whether there is a “subset” of “complete response letters” that can be released. Such letters to drug companies detail why their drugs were not approved. He said the FDA is looking at possibly releasing information involving safety issues. Critics of the pharmaceutical industry have long complained that the companies don't always give the public accurate and comprehensive explanations of why their products were rejected.  Read More & Comment...

Opioids: Fixing the Problem, Not the Blame

01/11/2018 10:21 AM | Peter Pitts

Speaking of opioids and lawsuits, something very interesting just happened in the MDL (Multi-District Litigation) National Prescription Opiate Litigation case (MDL No. 2804, Case No. 1:17-CV-2804) – Presiding United States District Judge Dan A. Polster  (United States District Court, Northern District of Ohio Eastern Division) made it clear to all parties that he intends to focus on fixing the problem, not the blame.

According to Judge Polster:

I don't think anyone in the country is interested in a whole lot of finger-pointing … People aren't interested in depositions, and discovery, and trials. People aren't interested in figuring out the answer to interesting legal questions like preemption and learned intermediary, or unraveling complicated conspiracy theories.

My objective is to do something meaningful to abate this crisis and to do it in 2018. … We've got all the lawyers. I can get the parties, and I can involve the states. So we'll have everyone who is in a position to do it. And with all of these smart people here and their clients, I'm confident we can do something to dramatically reduce the number of opioids that are being disseminated, manufactured, and distributed … and make sure that the pills that are manufactured and distributed go to the right people and no one else, and that there be an effective system in place to monitor the delivery and distribution, and if there's a problem, to immediately address it and to make sure that those pills are prescribed only when there's an appropriate diagnosis, and that we get some amount of money to the government agencies for treatment.

The full Transcript of Proceedings can be found here.

Stay tuned.  Read More & Comment...

All Budget "Pay Fors" are not CREATE(d) Equal

01/08/2018 07:34 AM | Peter Pitts

In a rush to find “pay fors” to balance the budget package that Congress is working to pass by January 19th, some lawmakers are pushing forward the Creating and Restoring Equal Access to Equivalent Samples (CREATES) Act.

It’s a bad idea with dangerous unintended consequences. It’s time to take a breath – because the CREATES Act won’t speed a single drug to market or lower the cost of medicines for a single American. What it will most certainly provide is a windfall for the trial lawyers, raising legal costs for the pharmaceutical industry and threatening the incentives to invest in development programs for new medicines.

The CREATES Act aims to provide a series of new legal provisions that will make it easier for drug companies to introduce generic alternatives, thus spurring competition and bringing down prices. It’s well intentioned. Unfortunately, it’s worded poorly – and would lead to dangerous unintended consequences. Instead of bringing generics to market sooner, these bills could endanger patients’ lives and encourage costly, needless litigation.

CREATES strips the FDA of its watchdog role. Under its proposals, generic manufacturers won’t be required to outline testing and safety protocols for the FDA to approve. Even if a generic drug maker’s proposed risk evaluation and mitigation strategies are inadequate, the FDA has no authority to reject or halt the transfer of medicines to the generic company for testing. Whatever happened to “safety first?”

CREATES contains ambiguously worded liability provisions that subject innovators to unfair legal risk. Generic drug companies often obtain brand-name drug samples and ship them off to third-party research firms to perform clinical trials. If the third party is negligent with the samples, patients could get hurt. Under the bill’s terms, patients would be able to sue the brand-name drug company, even though it had no control over the testing or safety protocols. Higher legal fees for drug companies ultimately result in higher costs for everyone else.

CREATES would allow generic drug manufacturers to sue brand-name manufacturers if they fail to hand over their drug samples for testing within 31 days, or if the companies do not reach an agreement on shared risk evaluation and mitigation strategies for risky drugs. Such subjective wording is music to trial lawyers’ ears.

Congress deserve praise for trying to find pay-fors that bring generic medicines to market faster, relieving consumers from high drug prices. Yet good intentions don’t change the fact that the CREATES ACT, as currently constructed — is deeply flawed.

Congress could help consumers by reworking the legislative language to end bad behavior without gutting safeguards for patients or enabling unscrupulous trial lawyers to file costly, pointless suits. Whether it’s the practice of medicine or the development of public healthcare policy two rules apply – first, do no harm and, second, be wary of trial lawyers bearing gifts. The CREATES Act as a budget pay-for would be a Pyrrhic victory.  Read More & Comment...

CMPI Awards Billy Madison Pharma Idiocy Award to Yale University Professors

01/03/2018 07:25 PM |

Cary Gross and Abbe Gluck of Yale University get the Billy Madison award for the most incoherent and idiotic article about drug pricing.  The piece: Soaring Cost of Cancer Treatment: Moving Beyond Sticker Shock published in the Journal of Clinical Oncology). There was a lot of competition but the authors managed to synthesize every pedestrian and inchoate assault on drug companies into an editorial that took the genre (if I can use this word without disparaging real scholarship) to a new level. 

The failing heart of the article, entitled can be obtained by reading one paragraph: ( I am sparing you the painful waste of time required to slog through the entire article and endure the smell of decomposing bromides) 
“We know that the cost of cancer drugs has increased dramatically, even though most drugs are brought into the market without compelling evidence that they prolong survival or improve quality of life. We know that these high costs render state-of-the-art cancer treatment unaffordable to patients without insurance and even to some patients with insurance. Furthermore, financial distress associated with paying for cancer treatment is common and is associated with stress, decreased adherence, bankruptcy, and worse outcomes. Finally, we know that the cost of new drugs is not well correlated with their effectiveness, nor with the presence of competing products.”

The authors then conclude that price controls and stricter formularies are the only way to control prices and help patients. 

I won’t take on every citation Gross and Gluck (Gross-Gluck sounds like a Borscht Belt act) use to assert perfect knowledge about the havoc price increases have had on society.  It is enough to say that once again, they all are written by people on the Arnold Foundation payroll.   Or maybe not.  As I and Peter Pitts pointed out, the most prolific of the paid hacks, Vinay Prasad, had an article published that was brazenly misleading and inaccurate.  (Shame on the medical journals that continue to publish any anti-pharma crap if it fits the narrative.)

So here is my fact-based response to the fictional claims to support government regulation of access to new medicines. 

1. As the number (and price) of targeted treatments have increased the percent of health care dollars devoted to cancer spending has remained at 4.6 percent.  How? New cancer drugs reduce spending on more expensive medical services.   In 2001, 64 percent of cancer care went to hospitals and only 3.6 percent to drugs.  By 2016 drug spending ‘skyrocketed’ to about 25 percent of cancer costs but hospitalizations dropped to 38 percent of care.  That’s why a government study concluded, “The net value of (cancer) treatment has grown substantially, consistent with medical technology improving over time and leading to better health outcomes at a lower cost per patient.”   All these benefits have been generated by new medicines that are only .7 percent of health spending.  This relationship – newer, initially expensive medicines (that go generic by the way) reducing the cost of care by letting people live longer and eliminating the need for other medicines -- has held up for nearly half a century.

2. GrossGluck enable the PBM payoff racket by remaining silent on the role PBMs and insurers play in setting cost-sharing levels. 

As scientists find the shut-off switch for specific cancer-causing genes, they can make pills that go after cancer cells and block the specific biological mechanisms that produce them. These pills are not only less toxic than conventional IV chemotherapy; they've turned once-incurable cancers such a myeloma, breast cancer, and even pancreatic cancer into manageable diseases.  They reduce the cost of cancer care over time but PBMs and insurers have responded by making these medicines more expensive and shifting patients to drugs that pay the most rebates. 



Sadly, several studies show, 25 percent of patients don't even fill their initial prescriptions for cancer pills when the co-pays exceed $500. Even more will stop or interrupt treatment.

Neither Medicare nor private health insurers are closing the gap between coverage and innovation. Instead, a survey of plans conducted by the Zitter Group found that insurers "recognize that oral therapy cost-sharing requirements actively encourage patients to use infusible products.

A Milliman study found that shifting to co-insurance would only add about $2 per member per month in private health plans.  GrossGluck never discuss this solution.  Instead, they remain true to the Arnold Foundation edict to give PBMs a pass.  By the way, neither has advocated for co-pay reforms that could relieve the burden on patients.  

3. They ignore the impact of government price controls on access and innovation.  But then again, those with perfect knowledge trust themselves to makes decisions on behalf of everyone else. They don’t mention the National Institute for Health and Clinical Excellence or NICE but they see it as a model for how experts would set prices and determine access for everyone. 

So, it is useful to know that NICE has turned down more new cancer drugs than other countries, even in Europe.  Hence, 5-year survival rates for all cancers are lower in the UK than in any other European country.   It’s mortality rates for many cancers, including breast, prostate, kidney, and others are higher.  A study published in Lancet Oncology on cancer survival rates in Europe found. 
“Cancer survival rates in Britain still lag well behind many other European countries, a study shows. Survival rates for nine out of ten common cancers are lower than the European average, despite improvements in diagnosis and treatment.

And the discrepancy is even worse among elderly sufferers. Patients with nearly all forms of the disease are more likely to die in Britain compared with patients in France, Germany, Spain, and Scandinavia.
For breast cancer, it found that British women have a 79 percent chance of surviving five years, compared with 86 percent in France, 87 percent in Finland, 85 percent in Norway and 82 percent on average.

Only 9 per cent of lung cancer patients in the UK live beyond five years, compared with the 13 per cent average, 17 per cent in Austria and 15 per cent in Sweden.
For prostate cancer, just under 81 percent of patients in Britain live beyond five years.  The rate is 90 percent in Finland and 89 percent in France. Around 68 per cent of over-75s with breast cancer survive beyond five years, compared with 75 percent across Europe. About 45 percent of men over 85 with prostate cancer live for at least five years. The average is 58 percent.”

The only evidence they cite for the effectiveness of their approach is the now old case of Peter Bach claiming he saved patients money by refusing to add Zaltrap to the formulary. 

(Indeed, it is ironic that Bach became crypto famous by getting Sanofi to cut the price of a cancer drug by 50 percent and writing about it in a New York Times op-ed piece.  )

The duo asserted that they wouldn’t prescribe the drug because it cost twice as much as Genentech’s Avastin (bevacizumab), a competing biologic drug with similar expected clinical outcomes for colorectal cancer patients.  In response, Sanofi said they would reduce the price of the drug by 50 percent.

In fact, doctors and prescribing hospitals benefited hugely from Sanofi’s pricing move, while payers and patients did not.   Zaltrap was sold in a dose twice as large as Avastin, thus the price discrepancy.    Further, Sanofi didn’t cut the price of Zaltrap; it gave Memorial Sloan a 50 percent rebate.  The price charged to patients remained the same.  Which meant that MSKCC raked in even more dough.  As an article in Health Affairs noted at the time, “Meanwhile, in the near term, physicians and hospitals will likely enjoy additional revenue opportunities from ziv-aflibercept use. the spread may be considerable: equal to $250 per treatment dose (insurer + patient reimbursement ($750) – discounted acquisition cost ($500)) and for 340B eligible purchases, $450 per treatment dose (insurer + patient reimbursement ($750) – discounted acquisition cost ($300)).  Additional revenues may incentivize physicians and hospitals to favor ziv-aflibercept over bevacizumab to treat colorectal cancer among Medicare-eligible patients, despite the treatments having equivalent expected clinical outcomes.  The strength of the incentive is based on comparing the magnitude of the spread obtained with the use of Zaltrap to that obtained with Avastin."

For this half-baked convoluted diatribe, GrossGluck get the Billy Madison award: 

Principal: Mr. Madison, what you’ve just said is one of the most insanely idiotic things I have ever heard. At no point in your rambling, incoherent response were you even close to anything that could be considered a rational thought. Everyone in this room is now dumber for having listened to it. I award you no points and may G-d have mercy on your soul.

  Read More & Comment...

FDA: Packaging a Punch for Opioids

12/13/2017 07:56 AM | Peter Pitts

Good news! The FDA is interested in using opioid labels to mandate packaging standards, as well as offer claims similar to those available for abuse deterrent products.

Per Commissioner Scott Gottlieb, labeling claims describing the benefits of packaging could be connected to agency efforts to work with medical societies to create better opioid prescribing guidelines.

Package design "opens up certain possibilities about how we drive more appropriate prescribing," Gottlieb said at the beginning of a two-day meeting on opioid packaging, storage and disposal options to enhance safety. "Could we, for example, require that the immediate-release drugs be packaged in units that comport with the majority of these consensus durations?"

Stand by.  Read More & Comment...

Different Pathways for Different Products

11/30/2017 06:47 PM | Peter Pitts

From the page of STAT News …

Gottlieb signals support for both ‘gold standard’ and expedited review for drug approvals

By Meghana Keshavan @megkesh

November 30, 2017

Regulatory standards for some clinical trials may soon slacken, per a new statement from Food and Drug Administration Commissioner Scott Gottlieb. And though his words are vague, they’ve been enough to raise red flags in some corners.

Testifying today before a Congressional committee on the 21st Century Cures Act, Gottlieb sent a mixed message: He wants the agency to “remain steadfast to our gold standard for safety and efficacy,” while making the development of breakthrough products “more scientifically modern and efficient, to meet the urgent needs of patients.”

Gottlieb said that in some cases, “when there’s a clear and outsized treatment effect,” a cancer drug might get expedited approval — and its efficacy will be evaluated only in postmarket studies.

The agency said it intends to release a document soon that will outline how it plans to expand on the Cures Act.  

The term “gold standard” has historically referred to rigorous randomized controlled clinical trials. But this form of drug evaluation is onerous and costly — and many argue that it prevents promising experimental medicines from reaching patients quickly enough. The 21st Century Cures Act was devised, in part, to speed up that process — granting the agency $500 million over the course of a decade to work out the intricacies. And President Trump has spoken of hastening drug approvals.

Still, this gold standard is what keeps ineffective, or even dangerous, drugs away from patients. And there isn’t much evidence that speeding along approvals will bring better results to patients. A recent BMJ study, for instance, found that more than half of all new cancer drugs “show no benefits” for either survival or quality of life.

A big sticking point with Gottlieb’s apparent approach is that small clinical trials simply don’t offer enough meaningful data.

“Empirical evidence suggests that the findings from individual trials may be spurious,” said Joshua Wallach, a research fellow at Yale’s Collaboration for Research Integrity and Transparency. He added, as additional studies are performed, the effects observed in small, early stage trials often attenuate — and can even be invalidated.

“With cancer drugs, the patients in these small trials have been so carefully selected, they will do well whether you give them a good new drug or sugar water,” said Dr. Vinay Prasad, an oncologist at Oregon Health & Sciences University.

Meanwhile, the current system for monitoring postmarket drug efficacy remains highly flawed, according to a 2016 report from the Office of Inspector General. Since it’s harder to track a drug’s efficacy in the real world, which functions rather differently than a controlled trial, FDA already has issues with data management and workflow, the report concluded. As a result, many of these studies aren’t completed on time — or even at all, according to an August study in the New England Journal of Medicine.

The concern is that the agency is not equipped to handle a new influx of postmarket data — and much could slip through the cracks. It’ll take substantial investment to help plug those holes and create a smart system for postmarket analysis, Wallach said.

“FDA understands that different products need different regulatory pathways, from both a science and a patient-need perspective,” said Peter Pitts, president of the nonprofit Center for Medicine in the Public Interest.  Read More & Comment...