Contracting Biosimilars

06/24/2019 11:19 AM | Peter Pitts

Per Denny Lanfear ‘s op-ed, “How Big Pharma Suppresses Biosimilars” (WSJ, June 24, 2019), it’s nice to see that the group Patients for Affordable Drugs has finally recognized that Prescription Benefit Managers (PBMs) are  a major roadblock to affordability and access to medicines. It just goes to show you that even David Mitchell can't be wrong 100% of the time. Shenanigans such as exclusionary contracting that prioritize PBMs profits over patient access to FDA-approved, safe and effective biosimilars must end. Just because such actions aren’t illegal doesn’t make them right. It’s past time to prioritize what’s best for patients.
   Read More & Comment...

Drug Importation: Stupidity Redux

06/03/2019 08:10 AM | Peter Pitts

From the pages of Politico ...

Vermont considers insulin, HIV drugs for importation
 
By Rachana Pradhan
05/31/2019
 
Vermont is eyeing birth control, insulin and pricey medications for HIV and multiple sclerosis patients as possible candidates for the state’s landmark program to import cheaper drugs from Canada, according to documents obtained by POLITICO.
 
The drugs are among the 17 most expensive that two private insurers identified for state officials and are potentially eligible for importation, show the documents, which were obtained through a Vermont public records request. Vermont officials determined the importation program could save insurers up to $5 million annually, based on that list of drugs.
 
The medications include Gilead’s Truvada, a $20,000-a-year HIV prevention pill whose price tag drew scorn from congressional Democrats during a hearing this month; Harvoni, among the recent class of drugs effectively curing hepatitis C; Merck’s Nuvaring, a contraceptive costing up to $200 per month without health insurance; and Zytiga, a blockbuster prostate cancer drug manufactured by Johnson & Johnson.
 
The list, which is from last year, isn’t a final catalogue of drugs Vermont would seek to obtain from the United States’ northern neighbor, but it provides a glimpse into the state’s deliberations more than a year after state lawmakers authorized the nation's first wholesale drug importation program.
 
“The 17 is our first cut, or our first time analyzing what would be candidates for importation that would generate savings,” said Ena Backus, the state's director of health care reform. “I think you do it a different quarter of the year you’re going to get a different list.”

Florida and Colorado approved similar importation programs this year. The idea has support from President Donald Trump but has encountered resistance from his top health officials.

Vermont still has several hurdles to overcome before its program could get off the ground. It still must finalize a plan and submit it for the Trump administration’s approval. Vermont is also delaying a statutory deadline to send its plan to HHS by a year — giving itself until July 2020 — because it wants to huddle with other larger states trying to build importation programs.

Because the process is breaking new ground, Backus said state officials were essentially "flying blind" on how to best approach federal officials.
 
“That’s why we felt like it was a great opportunity to slow down on submitting our application and to understand how more people are thinking about this," she said, adding that the state has not yet discussed its plan with HHS.
 
Vermont and other states seeking HHS approval must prove the importation program wouldn’t pose additional risks to patient safety and that consumers will pay less for drugs under the new model.

HHS set up a working group last summer to study importation, but its work — and even who’s in the group — has been kept secret.
 
The pharmaceutical industry and other importation opponents, including many Republicans, have argued that it will open up a dangerous pipeline into the United States for drugs whose safety cannot be accounted for. Further, they say it won’t produce much savings because importation could drain Canadian drug supplies, creating shortages and driving up prices, resulting in minimal savings for U.S. customers.
 
“We simply can’t fathom a duration or a scheme or a structure in which this plan will drive significant savings,” said Tom Rutkowski with CBPartners, a consulting firm focused on pharmaceutical and device issues which has analyzed the Vermont program.
 
The company said Vermont was unlikely to save much because discounts Canadian provinces negotiate with drugmakers would not be passed down to U.S. customers, and some especially expensive drugs that could drive greater savings don't qualify for importation.
 
“It’s a dumb idea now and a dumb idea in 2020,” Peter Pitts, president of the Center for Medicine in the Public Interest and former associate FDA commissioner, said of importation. “It’s a political ploy.”
 
The idea of importing drugs from Canada is gaining steam in states as officials grapple with rising costs and see a steady stream of new drug approvals with eye-popping prices. Maine and Connecticut are among the other states also considering creating their own programs, although legislation is unlikely to pass this year.
 
Trump has recently heaped praise on the idea, and he’s frequently complained that U.S. patients often pay much higher prices for drugs. He’s asked HHS Secretary Alex Azar — who has called importation a “gimmick” — to work with Republican Florida Gov. Ron DeSantis, a close ally, on his state’s importation plan.
 
“We may allow states to buy drugs in other countries if we can buy them for a lesser price — substantially less price,” Trump said at a White House event on health care earlier this month.

A plan won’t come easy, though. Vermont officials are still figuring out how much operating an importation program would cost and how it would pass cost-savings on to consumers. Backus, the health reform director, said the list of import candidates is a moving target because of changing prices and drug usage.
 
Further, the state doesn’t have the full picture of which drugs cost health insurers the most. Blue Cross Blue Shield of Vermont and MVP Health Care provided information for the state’s analysis but Cigna declined, Backus said.
 
Other regulatory issues could alter Vermont’s plans. For example, the FDA next year will regulate insulin as a biologic rather than a drug, which could make it ineligible for importation.

Backus and others said should Vermont manage to get its program off the ground, the state would likely update its list of importation candidates every quarter.
 
“This is all new ground,” said Trish Riley, executive director of the National Academy for State Health Policy, which is working with Vermont, Florida and Colorado on their importation plans.

“They want to be consistent and they want to think it through together,” she said.
 
Sarah Owermohle contributed to this report.  Read More & Comment...

Vinay Prasad: Precision oncology is not about the patient but all about the Benjamin

05/08/2019 01:22 PM |

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Vinay Prasad is an associate professor of medicine at the Oregon Health & Science University School of Medicine. As his Wikipedia page states: “Prasad is a noted critic on the direction of cancer research. He has developed a following using Twitter as his platform.”  More to the point, Prasad has is adored by a group of journalists, policy types and cranks who believe that drug companies use shoddy clinical studies to market expensive medicines that are marginally effective.  
 
In particular, Prasad has singled out targeted cancer drugs as a waste of time and money, a position for which he has been praised and quoted by health care journalists who, like Prasad, publish this perspective with funding from Arnold Ventures.
 
In a recent episode (1.57) of his podcast called Plenary Session (which deserves all the neglect and inattention it receives) Prasad personally attacked University of California at San Diego cancer researchers Razelle Kurzrock and Jason Sicklick, the authors of a study published in Nature showing that attacking multiple genetic and non-genetic drives of tumor growth with combinations of targeted cancer drugs improve survival and reduces toxic side effects.
 
Before explaining why he believes precision oncology -- and the Nature study -- is largely a fraud, Prasad accused Kurzrock and Sicklick of grabbing $200 million for producing what he calls negative results about targeted cancer treatment.  (Around 3:28 into the podcast)
 
He then goes on to claim that Kurzrock and Sicklick effectively silence critics of their methods with “retribution” in the form of denying them funding for their own studies or the opportunity to publish responses.  He implies that he has experienced such retribution from Kurzrock in particular. (Around 4 minutes into the podcast.)
 
Prasad concludes that the researchers can only demonstrate clinical benefit by treating only people they know they can respond and denying others that don’t access to standard of care or by delaying the process of sequencing tumors that people die waiting. (21 minutes into the podcast)
 
Both Kurzrock and Sicklick are Jewish and both live near the Chabad synagogue in California recently targeted by an anti-Semitic animal who killed and maimed congregants during Passover services. Prasad’s personal attack intentionally or not invoked the trope about Jewish control over banking, media, manufacturing, etc. Indeed, by asserting that the researchers let people die in order to make more money through the expansion of personalized combination cancer therapy, I believe Prasad implicitly invokes the blood libel.  
 
Prasad has crossed two red lines. He has alleged, without any evidence, that the principal investigators have grabbed hundreds of millions of dollars for negative results, threaten those who dare to challenge them, covered up the deficiencies of targeted oncology and designed trials that harmed the sickest in order to enrich themselves. And he tells whoever is listening or following him on Twitter that Kurzrock and Sicklick that they did so to make as much money as possible by escalating the use of their junk science.  In other words, Vinay Prasad claims that precision oncology is not about the patient but all about the Benjamins.
 
 
   Read More & Comment...

Restoring the Balance Between Innovation and Generic Manufacturing of Medicines

05/07/2019 03:22 PM |

Restoring the Balance Between Medical Innovation and Generic Manufacturing of Medicines.    Read More & Comment...

Op-Ed from Real Clear Health: FDA Attack on Underage Vaping Could Backfire

05/06/2019 06:05 PM |

FDA Attack on Underage Vaping Could Backfire  
By Robert Goldberg
May 06, 2019
REAL CLEAR HEALTH

Scott Gottlieb’s extraordinarily productive tenure as Commissioner of the Food and Drug Administration (FDA) will be remembered for his crusading against underage use of E-cigarettes and the companies that produce them.  But his legacy is not to be found in his jawboning against the retail distribution of vaping products but in the guidance the FDA issued on developing a nicotine replacement therapy (NRT) product that reduces tobacco use.

Gottlieb announced the guidance in February, but it received little, if any, media coverage. That’s too bad because the document lays out a clear path to fully realize the contribution of vaping to the public health.  Specifically, he noted:


“Novel products with different characteristics or routes of nicotine delivery have the potential to offer additional opportunities for health-concerned smokers interested in quitting. This could also include products such as electronic nicotine delivery systems like electronic cigarettes, but which would need to be proven safe and effective for smoking cessation and regulated as a drug product. This would allow them to be marketed as a prescription or over-the-counter drug products with medical claims for smoking cessation or related indications – ultimately reducing the likelihood of someone continuing to suffer the clinical consequences of smoking. This is different from our regulation of e-cigarettes as tobacco products.”

And it is different from the public statements Gottlieb was making about e-cigarettes as tobacco products as well.  Last September Gottlieb announced the FDA was taking "historic action" by threatening e-cigarette manufacturers with criminal prosecution and sales bans unless they developed plans to reduce the "epidemic" of teen use. 

Juul, the leading manufacturer of vaping devices had anticipated the FDA move and had already begun implementation a "Youth Prevention Plan" that eliminated pod flavors, such as cucumber and mango, from the 90,000 or so retail outlets across the country. Flavored pods are still available online, but Juul also had stopped using social media, instituted age verification tools to ensure that purchasers are 21 years of age or older introduced new purchase limits and product serialization.

These actions did not mollify Dr. Gottlieb.  Instead, he stepped up his attack.  He told Vox: “The dramatic spike of youth [vaping] — that was driven in part at the very least if not largely by Juul.  I hope they recognize the problem that’s been created has been created largely by their product.”

While Gottlieb “acknowledged there’s, no definitive data showing the teens now experimenting with vaping are using Juul” he still holds the company responsible for triggering what the regards as a spike in teen addiction to nicotine that could lead to cigarette use.

Meanwhile, the guidance discusses how companies should test the effectiveness of E-cigarettes in reducing tobacco use in the pediatric population: “Given that use of tobacco products frequently starts in early adolescence, drug products approved for smoking cessation have the potential to benefit and be used in the pediatric population. Based on the current prevalence of smoking in younger children, the Agency has waived…requirements for clinical studies of NRT drug products in patients younger than 12 years of age because clinical studies would be highly impracticable in that age group.”

So, the opportunity is there to turn e-cigarettes into medical products that could be available to anyone who needs to quit smoking at any age.  Conceivably, vaping products could be available behind the counter along with other heath products that have taken the reverse course from by prescription only to OTC.  

Hence, Gottlieb was trying to prod e-cigarette companies to re-introduce themselves as an OTC medical device. The guidance notes to receive OTC approval an NRT product must demonstrate effectiveness in reducing smoking and evidence that the marketing of e-cigarettes increases the safe and effective use of products.  This is something companies such as JUUL have shown they can do.

The question is whether his efforts have made it impossible to get an e-cigarette approved as an NRT.  In stressing the risk of underage vaping the FDA has contributed to the fact that two-thirds of American adults mistakenly believe that e-cigarettes are just as harmful as smoking.  The media’s fearmongering fueled by the FDA attack and spread by so-called consumer groups has incited a movement to ban e-cigarettes altogether. Rather than being able to use vaping to reduce the use of nicotine, people will pick up a pack of cigarettes instead. 

Robert Goldberg is Vice President at the Center for Medicine in the Public Interest.  Read More & Comment...

ICER Cites Date Rape Drug as Being Cost Effective Treatment for Depression

04/23/2019 06:02 PM |

 
My colleague Peter Pitts has written an article on a Democrat proposal to submit the price and use of a new drug for binding arbitration before Medicare or another government program pays for it.   Arbitration is supposed to be a fair way to impose drug prices, but it is price controls all the same. 
 
As with everything, proof in practice is more instructive than proof in principle.  So, let me provide you with a real-world example of how such arbitration would proceed.  Proponents use the German approach to pricing arbitration as a wonderful example of how spectacular arbitration is.  There, the process comes after a company and the committee set up to determine prices can’t agree on reimbursement levels for the medicine in question. 
 
Throughout, the goal is to set a price for a drug based upon the additional value it generates and converting that into a price premium compared to the average price of an existing drug for a condition selected by the committee.
 
The selection of comparator (as well as the reference price) is one critical element.  The other is whose value is being measured.  Interestingly, the German approach does not use quality-adjusted life years to make value determinations.  The Germany process defers to an independent group to determine what comparator to use and whose value should be preferred.
 
In the US, proponents of pricing arbitration envision the Arnold Ventures funded group ICER serving as the arbitration organization, or something else that John Arnold would pay for by sponsoring its formation or operation.   The Center for American Progress, in particular, loves the idea of the ICER driven pricing deals.
 
ICER puts limits on how many people could use a drug using a price that is sensitive to insurer and PBM profits.  And that price is also based on a comparator that ICER also selects.  The cheaper the comparator, the greater the hurdle for a new drug regardless of benefits, particularly since the goal is to save insurers money and maintain the PBM spread between net and list pricing.
 
Here’s an example of how the binding arbitration proposal in the US would work using a real-world example and ICER’s most recent analysis of a new drug:
 
Spravato, a new drug for major depression was recently approved, a form of ketamine that quickly improves mood without resort to ketamine’s hallucinogenic effects.
 
ICER’s initial review of the drug determined Spravato was effective but said that at the list price it was not cost effective and could only be used by a small percentage of people. 
 
ICER’s comparator?  Injectable Ketamine which is now used to knock out animals and some patients.  The ketamine that, in the ICER preferred formulation, has the same mechanism of action at PCP.  What’s more, ICER’s comparator is not even approved for treating depression.  Untested, off-label use of ketamine.  You might know it as Cat Tranquilizer, Cat Valium, Jet K, Kit Kat, Purple, Special La Coke, Super Acid, Super K, and of course, Special K, the medicine preferred by Bill Cosby.
 
You read that right.  ICER used a form of a date rape drug as the comparator for determining price and is recommending the use of a date rate drug as a more cost-effective treatment. 
 
That might sound extreme, but in fact, the capricious and one size fits all selection of comparators is a hallmark of every price negotiation.  Standard of care is chosen to make price decisions.  Benchmarks are adjusted, goal posts are moved to maximize rebates, not well-being.  Drugs that require price cuts based on comparators are often not sold in a particular market because they lose money.   Advocates of arbitration claim that is just an example of value-based care.  Yet in Germany, 82 percent of drugs that were withdrawn because they didn’t meet the comparator price-value threshold were recommended by at least one guideline.  Hence, drugs recommended because of their clinical benefit were no longer available.   
 
Early assessment of new medicines is fraught with danger because prediction is not just political, it is unscientific and uncertain.   ICER’s involvement assures that such arbitration related assessments and comparisons will be downright immoral.   Read More & Comment...

Virginia's Northam Leaves PBMs Red-Faced

03/28/2019 07:38 AM | Peter Pitts

From the pages of the Roanoke Times …
 
Med Beat: Drug makers' coupons to count toward deductibles

Gov. Ralph Northam signed a bill that will require insurers to count toward deductibles and co-pays any payments made on behalf of a patient, including assistance by pharmaceutical makers.
 
The practice known as copay accumulator adjustments excluded from patients’ deductibles and maximum out-of-pocket costs the coupons and assistance programs that lower the price of medication.
 
Fair Health Care VA Coalition lauded passage of the bill.
 
“As out-of-pocket costs continue to rise, Virginia patients already face enough barriers to accessing the health care coverage that they need. Copay assistance programs are a critical resource, particularly for patients whose health care costs could bankrupt their families or force them to live without the care they need,” Dr. Bruce A. Silverman, a Richmond nephrologist and advocate for the collation, said in a news release. “Patients should not be denied one of the key benefits of copay assistance programs, particularly since insurers are already getting the value of negotiated drug price discounts while withholding these benefits from patients.”
 
When the change goes into effect July 1, all payments made by patients or on their behalf will count toward maximum out-of-patient payments and deductibles.  Read More & Comment...

Drug Rebate Head Fake

03/12/2019 03:30 PM |

Today United HealthCare announced that they will be passing rebates directly to patients.  But the impact is not as impactful as may seem. 

United noted that “all new employer-sponsored health plan customers that use UnitedHealthcare must give discounts they get for including certain drugs in their lists of covered medications directly to consumers at the point of sale… UnitedHealthcare said Tuesday that its expanded requirement does not apply to existing employer customers that do not already give rebates directly to the consumer.”

As Drug Channels notes: “PBMI found that only 4% of employers reported that rebates were used to reduce member out-of-pocket costs at the point of sale.”

Indeed, despite the announcement of these offering, Drug Channels demonstrates (see chart below) that employers really don’t want to use rebates to cut out of pocket drug costs.

 

So much for sharing the savings.  

But even passing rebates on to patients is not enough.  In fact, it could make things worse in a perverse fashion.  For example, a pass-through model would reward the most rebated drugs but lead to even more step therapy or greater formulary restrictions in an effort by PBMs to make money on contracts that still reward them for lowest net cost of medicines.   Even drugs that offer zero copay but do little to affect overall net cost of medicines could be excluded.  

Another question:  Will insurers and PBMs increase the use of (illegal) co-pay accumulators – programs that intercept money designated by drug companies to reduce out of pocket costs is confiscated by insurers and NOT used to reduce cost sharing -- and then pass that cash to employers.  Can we say racketeering?  (You can read my colleague Peter Pitts article on the wholesale thievery called copay accumulators here. 

To be sure, many business health groups claim they want to optimize the use of medicines.  However, they lack the data and bandwidth to do so.  Some individual companies do make an effort to support value-based design, but most of these offerings are designed to drive people to more generic drug use.  

Absent these insights, the well-intentioned effort to reduce out of pocket costs by shifting rebates will help some but likely hurt others. Which is why at some point, large employers will be asked to explain why they aren’t doing more through smarter changes to benefit design to help their employees stay health – and alive. 

   Read More & Comment...

Is Everyday Oncology Day at the FDA?

03/12/2019 01:55 PM | Peter Pitts

Two exciting pieces of news from White Oak. The first is that the Director of the National Cancer Institute (NCI), Dr. Ned Sharpless, has been tapped to be the interim Commissioner of the FDA. Whether or not this translates into his getting the official nod for the nomination is anyone’s guess. But he’s clearly the odds on favorite – and that’s a good thing.
 
The second item of interest is the release of new recommendations for broadening cancer clinical trial eligibility criteria. Per the FDA, these new guidelines will facilitate the design of clinical trials that are more representative of the patient population and maximize the generalizability of the trial results and the ability to understand the therapy's benefit-risk profile across the patient population likely to receive the drug in clinical practice.  
 
This is Big News across a spectrum of issues – including expanded access. As Janet Woodcock mentioned earlier this year, “Expanded access programs are only an iterative step towards more regular and robust use of platform trials.” And, per Bob Temple, “Expanded access protocols can produce data that demonstrate effectiveness in populations outside those studied in registration trials, potentially leading to broader indications.”
 
Hopefully, Dr. Sharpless will continue to urge divisions – beyond Oncology – to embrace new clinical trial protocols for both orphan diseases and other areas of urgent public health concerns.  Read More & Comment...

Gottliebensraum

03/10/2019 03:36 PM | Peter Pitts

Gottliebensraum: Will the FDA need more (or less) living space in a post-Gottlieb world?
 
Since Scott Gottlieb’s decision to step down as FDA Commissioner the tributes have been coming in fast and furious. And they're all, save for Sid Wolfe of course, well deserved. But here’s something you haven’t thought about – the exceptional role and excellence of the FDA career staff.
 
One of the reasons that Scott was so successful in so many was because he acknowledged the importance of the FDA's professional staff. Their participation and enthusiasm to share his agenda has helped create a more elastic and angular FDA.
 
So, now what happens? Can the many initiatives begun at say, CDER, gain and maintain velocity? Or do they kind of just fade away? How sharp will FDA elbows be as we slowly march our way towards another glorious round of PDUFA-Palooza? And e-cigarettes? Will planned agency actions back a half-step back to stay quiet and avoid attention? Or will we get a surprise?
 
Swap out NCI for FDA? It’s been done before and wasn’t a disaster, but why disrupt two agencies that are functioning well when you don’t have to. Alex Azar has seen this before as well and his suggestions will carry a lot of weight. As will the Vice President’s.
 
Is the President a wildcard? Are you kidding me? So, there’s that.
 
Momentum comes from without and within. A Scott-Free FDA presents a lot of opportunity space. (But, then again, I’m a cockeyed optimist.)

Stay tuned.  Read More & Comment...

Great Scott!

03/07/2019 08:56 AM |

Those of us who have worked with Scott Gottlieb were not surprised at the extraordinary energy and focus he brought to the job as FDA Commissioner.  Similarly, it was unsurprising that Scott announced his resignation effective next month.   As dedicated as he was to the role at the FDA, he is even more devoted to his family.  

His tenure was short but his impact on the agency and the public health will be enduring.  Scott spent most of time and energy implementing the 21st Century Cures act and building the infrastructure to move product development away from randomized controlled trials and encouraging the use of predictive biomarkers and real-world evidence.  His public pronouncements about drug prices, PBMs and vaping were not mere rhetoric.  Though all the initiatives in these areas are not fully implemented, it is to be noted that Scott never said or tweeted anything he was not prepared to back up with action. 

Most important, Scott raised the bar for the next FDA commissioner.   The agency – and the country – has been blessed with a series of solid FDA commissioners over the past decade or more.  Mark McClellan, Andy von Eschenbach, Peggy Hamburg, Rob Califf all made important contributions to the FDA’s modernization and its movement away from what Scott called the hunger for statistical certainty.  It can be truly said that the FDA is now leading the way in personalized medicine, regenerative medicine and applying regulatory policy to promote competition.  

There are some who still wish to turn the clock back on a decade of FDA reform.   Organizations funded by John Arnold, in particular, want to use FDA regulation to slow down drug development, limit investment in orphan drugs and eliminate patient-centered drug development.  But Scott has, by showing how smart regulation is done, demonstrated that much would be lost if we turn back now.   He built, enlarged and secured a broad consensus about FDA’s mission. In doing so, Scott did something nearly impossible in public policy today: he turned a government agency into a source of health, hope, and possibility. 

Well done. 


   Read More & Comment...

Dispatches from the Inquisition

02/26/2019 09:41 AM | Peter Pitts

What the best advice for a bevy of Big Pharma execs as they prepare for a Congressional crucifixion? Don’t bend over.
 
Just the other day, the New York Times ran an editorial titled, “It’s Time for Pharmaceutical Companies to Have Their Tobacco Moment.” Do the CEOs need a roadmap to know where this is going? Medicines save lives. Tobacco kills. While the headline is off-base and objectionable, the prose makes a number of important points that can be summed up in one statement: Pharma has a lot of explaining to do.
 
So, here’s the good news – it’s a terrific opportunity to speak truth to power. But, will that happen? Here’s what Senator Charles Grassley had to say on the matter:
 
“I hope that the drug CEOs testifying tomorrow don’t try to blame everyone but themselves/take no responsibility for their role in fixing the problem. We already understand there are other factors to consider. Tomorrow is about the part drug companies can do to lower costs for patients and taxpayers.”
 
Wise words from a wise man. But will those posing the questions be seeking sage advice or their very own "I am Torquemada" moment?
 
It’s time we all took to heart the Japanese proverb, “Don’t fix the blame. Fix the problem.”
 
And to those CEOs preparing their remarks – be honest, forceful and helpful. Call it as you see it. Have solutions. That’s why you earn the big bucks.

As the great Frank Douglas reminds us, "It's not what you control, it's what you contribute."

Do the right thing.  Read More & Comment...

Purdue Punches Back

02/26/2019 08:51 AM | Peter Pitts

How bad is it getting for Purdue Pharma? Bad enough that they're actually fighting back – and not pulling any punches.
 
They’ve responded forcefully to the recent 60 Minutes episode with a tick-tock response and a very unambiguous lawyers letter.
 
Some snippets:
 
Contrary to assertions that FDA’s changes to the OxyContin indication in 2001 broadened the medicine’s use to chronic pain, the opposite was actually the case. In July 2001, to address extensive abuse of OxyContin, FDA added a black box warning and narrowed the indication to “the management of moderate to severe pain when a continuous, around the clock analgesic is needed for an extended period of time.”
 
Purdue Pharma expressed concerns in the following letter to Mr. Bill Owens, Executive Producer of 60 Minutes …
 
Despite multiple meetings, phone calls, and email exchanges between representatives of Purdue and 60 Minutes Associate Producer Sam Hornblower (during which detailed information was exchanged), we are still concerned that Mr. Hornblower and 60 Minutes intend to air a biased and one-sided segment rife with significant errors and inaccuracies, and that 60 Minutes will refuse to disclose to its viewers critical information about the sources it intends to rely on (and even put on the air), including their personal biases toward Purdue and their financial incentives in making false and misleading statements about the company and OxyContin.
 
Dr. Kolodny has admitted, in the form of a court-filed expert disclosure report made under the penalty of perjury, to the fact that he is a paid consultant and advisor, earning $725/hour for his services.2He also lists on his CV that he submitted to the U.S. House of Representatives Committee on Energy and Commerce on February 27, 2018 as part of his required disclosures prior to his testimony under oath, that he served in a “CONSULTING AND ADVISING” role for “CBS 60 Minutes” in 2017 as part of 60 Minutes’ “The Whistleblower” segment. A copy of this expert disclosure report and Dr. Kolodny’s CV he submitted for his congressional testimony is enclosed for your reference. The facts demonstrate that Dr. Kolodny admits that he worked for 60 Minutes as a consultant and advisor within the last two years (and on a segment that touched on substantially similar subject matter is this segment) and that he is also a paid consultant on behalf of plaintiffs currently involved in active litigation with Purdue.
 
In that same vein and upon information and belief, we understand that Dr. Kessler has also performed extensive work for and has long consulted with and on behalf of plaintiff-side law firms who are or have been engaged in litigation against Purdue regarding the very same issues he purports 6to comment on for 60 Minutes’ planned segment. These types of consulting and advising roles for law firms involved in active or potential litigation are rarely if ever performed for free. It is incumbent upon 60 Minutes, as the ultimate publisher and airer of segments featuring biased and conflicted individuals like Dr. Kolodny and Dr. Kessler, to thoroughly investigate and vet its own sources and intended on-screen interviewees to confirm any such biases and financial incentives for promoting certain viewpoints and commentary. And where such biases and financial or otherwise personal motives to provide one-sided, incomplete, and even false commentary on a subject matter such as opioid use in the United States exists(as it clearly does with Dr. Kolodny and Dr. Kessler who are, at a minimum, paid-for consultants by individuals and parties who are currently suing Purdue), 60 Minutes has a duty to its viewers to clearly and unambiguously disclose such bias and the facts supporting their incentive and motivation for their viewpoints and commentary during any aired segment in which Dr. Kolodny and Dr. Kessler participate in, and to confront such bias head-on.
 
Transparency for thee but not for me?
 
Powerful stuff.
 
As Pharma CEOs prepare to step up to the plate and face a Congressional grilling, it’s time for Pharma to forcefully defend itself with context and … the truth.  Read More & Comment...

"Universal Healthcare" and the Realities of Rationing

02/17/2019 10:00 AM | Peter Pitts

Per the New York Times editorial, “How Much Will Americans Sacrifice for Good Health Care?, one urgent issue relative to a broader government role in providing healthcare is rationing. No nation (and certainly not those in Europe or Canada) provide universal access to everything. Government bureaucrats make decisions about what medical treatments (new cancer drugs, surgeries, new genetic interventions, etc.) are paid for.

Today, of the 74 cancer drugs launched between 2011 and 2018, 95% are available in the United States, 74% in the U.K., 49% in Japan, and 8% in Greece. Access to these cutting-edge drugs means that patients can use the latest treatments to help cure their conditions; it’s why the United States has the highest five-year survival rate for cancers in the world.

Do we want Uncle Sam, MD to replace the considered opinions of our own physicians? Government-paid healthcare presents significant hurdles as well as interesting opportunities and healthcare rationing is an important part of the discussion about any kind of government-run healthcare proposal.  Read More & Comment...

Why the Trump PBM Reform Plan Has Promise and Why Democrats Oppose IT

02/13/2019 12:33 PM |

I wrote two op-eds that just happened to be published the same day (today)

The first one explains whyTrump's Prescription Drug Price Reform is Promising.

Soaking the Sick to Make the Rich Even Richer discusses how Democrat opposition to the Trump proposal has led legislators from that party propose giving PBMs more control over access to medicines.  


   Read More & Comment...

Frank Douglas: The Definition of a Free Man

02/12/2019 05:19 PM |

Not many people are told, at gunpoint no less, by a dictator to take the next flight out of his country and never come back.   Fewer still have replied by asking the dictator to pay for the trip, let alone live to do so.  

But such quiet acts of defiance connect the "Defining Moments of a Free Man From a Black Stream" by Frank Douglas.  Douglas, one of the most highly honored and respected leaders in the pharmaceutical industry, could have written an entire book about his many careers ranging from a physician, a drug development executive, venture capital advisor, biotech CEO and director of two initiatives to accelerate the commercialization of academic life science research and that alone would have been interesting, informative and inspiring.   But the formative experiences, the events that truly defined the life of Frank Douglas, were forged not in corporate boardrooms or high-level meetings but instead in how he chose to act when faced with beatings, homelessness, outright racism, and political intimidation. 

The autobiography opens with a 12 year Frank Douglas being whipped by his mother after being told that he deliberately dumped a week's worth of groceries from his bicycle basket by her emotionally unstable and sadistic sister, Edith.  It wasn't the first time.  Edith took a sick delight in blaming Frank for things he did not do, knowing that it would lead to a beating.  After this incident, Frank decided he had no choice but to kill himself by plunging into the deep waters off the coast of his native Guyana.  

He asked himself how much "hope is there for a boy of twelve when in his own home he cannot defend himself or defend against injustice?"  Ever the rational being, Frank realized that the possibility of jumping and living in pain wasn't worth the effort to commit suicide.  Instead, he "concluded that there had to be a different solution to my dilemma."

He ran to the home of a woman he called "Moms" who is the mother would take to visit every Sunday after church and declared he wanted to live there.  It was then that Moms told Frank that her son was his real father.  The revelation did not devastate him, it made him stronger.  He went home, no longer a victim, and told his mother and aunt Edith he would not be beaten or manipulated again. 

Such defiance was forged from faith and fearlessness in confronting events and forces that appeared to be beyond his control.  Much like Jacob wrestled God from darkness into dawn before receiving the name Israel, Frank Douglas truly earned his name (which literally means a free man from a black stream) by virtue of his willingness to confront malevolent, even violent forces throughout his life.  

Throughout his academic career which took him from a small private school in Guyana to Lehigh University and then to Cornell for a Ph.D. in Chemistry and a medical degree, Douglas faced outright racism.  Others would have endured it or, especially today sought to triumph by being defined as a victim.  Instead, Frank Douglas took on the threats, often without regard to short term consequences, by confronting those who wanted to squash him because he was black. 

If he had acted otherwise, the world would be a lesser place.  His novel approaches to conducting drug discovery and development spread from the companies he worked for and transformed practice throughout the industry.  The hundreds of students and entrepreneurs that sustain medical innovation would be fewer in number and less effective.  And those fortunate enough to have read the book would not have the privilege of being taught an ageless lesson about the human condition:  that our character is not just our destiny, it is the legacy we leave behind.   And as Defining Moments demonstrates, that heritage is shaped by the work of our own hearts and hands and not the faceless, inexorable traverse from past to future.   Read More & Comment...

Supplementing DSHEA

02/11/2019 10:25 AM | Peter Pitts

Very important initiative from the FDA to update DSHEA – without even once mentioning CBD oil. If you don't think the agency takes the twin issues of quality and claims seriously -- think again. Some excerpts:

Statement from FDA Commissioner Scott Gottlieb, M.D., on the agency’s new efforts to strengthen regulation of dietary supplements by modernizing and reforming FDA’s oversight

The opportunity to strengthen the framework that governs dietary supplements couldn’t come at a more pivotal time. On the one hand, advances in science and the growth and development in the dietary supplement industry carries with it many new opportunities for consumers to improve their health. At the same time, the growth in the number of adulterated and misbranded products – including those spiked with drug ingredients not declared on their labels, misleading claims, and other risks – creates new potential dangers.
 
Legitimate industry benefits from a framework that inspires the confidence of consumers and providers. Patients benefit from products that meet high standards for quality.
 
Dietary supplements can, when substantiated, claim a number of potential benefits to consumer health, but they cannot claim to prevent, treat or cure diseases like Alzheimer’s. Such claims can harm patients by discouraging them from seeking FDA-approved medical products that have been demonstrated to be safe and effective for these medical conditions. In recent years, we’ve also taken action against companies and dietary supplements making similar claims regarding treatment of serious conditions such as cancer and opioid addiction. These enforcement actions are just one part of our overall efforts to update our policy framework governing dietary supplements. 
 
Our first priority for dietary supplements is ensuring safety. Above all else, the FDA’s duty is to protect consumers from harmful products. Our second priority is maintaining product integrity: we want to ensure that dietary supplements contain the ingredients that they’re labeled to contain, and nothing else, and that those products are consistently manufactured according to quality standards. Our third priority is informed decision-making. We want to foster an environment where consumers and health care professionals are able to make informed decisions before recommending, purchasing or using dietary supplements.
 
I’m pleased to announce that we’ve recently created the Botanical Safety Consortium, a public-private partnership that will gather leading scientific minds from industry, academia and government to promote scientific advances in evaluating the safety of botanical ingredients and mixtures in dietary supplements. This group will look at novel ways to use cutting-edge toxicology tools, including alternatives to animal testing, to promote the goals of safety and effectiveness we share with consumers and other stakeholders
   Read More & Comment...

Greedy Tort Bar Tarts Up the CREATES Act

02/07/2019 07:40 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,” lawmakers are pushing forward H.R. 965 (Cicilline and Sensenbrenner) and S.340 (Leahy). The worthwhile goal of this new version of the CREATES Act is to prohibit pharmaceutical and biologic companies from engaging in anti-competitive conduct that blocks lower-cost generic drugs from entering the market.

Both bills address an important problem – but create an even bigger one.

This proposed legislation establishes a private right of action for “eligible product developers” to sue “license holders” for failure to comply with the process set forth in CREATES for providing access to covered product.  Good! But …

As written, the CREATES Act is ripe for abuse by entities that have no intent to actually develop a generic or biosimilar version of the covered product.  This potential for abuse is exacerbated by the significant monetary damages available under CREATES—up to the amount of revenue generated on the covered product during the period of violation.  Indeed, in certain instances it may be more profitable to litigate and obtain damages under CREATES than it would be to actually market a generic/biosimilar product. 

Can you hear the tort bar drooling? Can you hear the tort bar … drafting?

As currently drafted, the CREATES Act could have significant unintended consequences:

The damage provisions of the current draft create the potential for windfall damages, which will distort incentives. Specifically, the bill allows for damages up to the entire gross profit of the brand medicine during the period of negotiations.  This creates a powerful incentive for generic companies to prolong negotiations (increasing their damage award), which will actually delay generic entry and competition in the marketplace.

Indeed, generic developers would be able to earn more from a lawsuit than from actually selling the proposed generic drug.  An "opportunistic" company (Can you say "Shkreli?") could develop a business model of demanding samples and engaging in litigation for damages without ever submitting an abbreviated application to FDA—undermining the bill’s stated goal to speed availability of lower-cost drugs for patients.

Smart changes will fix these problems, save the government money and achieve the desired policy goals of facilitating generic access to samples and increasing competition; maintaining safeguards for products subject to a REMS; and ensuring generic developers actually develop generic products. 
Here’s a savvy path forward:

Establish an affirmative defense for license holders where the license holder has made a timely offer to provide sufficient quantities of samples at commercially reasonable, market-based terms.

Such an affirmative defense is intended to prevent frivolous litigation—where samples are offered on commercially reasonable terms, the eligible product developer should not be able to decline the offer and continue litigation.  The term “commercially reasonable, market-based terms” is defined to provide further clarity to all parties and avoid unnecessary litigation.  Remedies would not be available if the license holder has established an affirmative defense by a “preponderance of the evidence.”This affirmative defense also protects good acting companies from protracted litigation and stops generic sponsors from unnecessarily prolonging negotiations to increase damages.

Revision to the definition of “eligible product developer” to clarify that the eligible product developer must be a person that seeks to develop “and submit” an application for a generic or biosimilar product. 

These change will help ensure that only legitimate manufacturers are considered eligible product developers for purposes of CREATES; entities that seek only to engage in frivolous litigation and do not seek to submit a generic/biosimilar product application would not be eligible for the remedies under CREATES. The tort bar won’t like it – but this important legislation must be about lowering drug prices – not raising their income.  Read More & Comment...

Separating the Healthcare Wheat from the Political Chaff

02/05/2019 10:09 PM | Peter Pitts

Last night during his State of the Union, the President addressed the issue of drug costs. Some of the ideas are sound while others deserve, shall we say, more careful consideration. Let’s look at the record.
 
BAD IDEA: Adopting an international pricing index (IPI) payment model for Medicare Part B in which a “reference price” for a prescription drug will be generated by taking the average price of that drug from 16 countries.

The president claims that proposal will lower the cost of drugs, makes America first again by stopping foreign freeloaders and limits “out-of-control” prices.
 
In reality these proposals reduce access for patients:
 
* These countries utilize government controlled and single-payer healthcare systems where governments leverage their purchasing power to dictate prices, meaning that these “reference prices” have nothing to do with actual market values. 

* In these systems, governments can only contain healthcare costs by restricting or refusing care, meaning that if a drug costs more they are willing to pay, they can refuse to cover it for patients or limit the number of patients who can receive the treatment. 

* This is why “of 74 cancer drugs launched between 2011 and 2018, 70 (95%) are available in the United States. Compare that with 74% in the U.K., 49% in Japan, and 8% in Greece.”

Access to these cutting-edge drugs means that patients can use the latest treatments to help cure their conditions; it’s why the United States has the highest five-year survival rate for cancers in the world.
 
Price controls reduce innovation:
 
* By leveraging CMS’ purchasing power to impose these price controls, the proposal creates a dramatic market imbalance which would force innovators to adjust business models and reduce investment in R&D – reducing high-risk research into complex conditions such as Alzheimer’s Disease. For example, there were numerous, high-profile failures in late stage clinical trials for Alzheimer’s treatments just in 2018. These failures represent years and billions of dollars in research which will yield no return – all part of the risk of scientific research. 

* The U.S. “funds about 44% of world medical R&D, invests 75%of global medical venture capital, and holds the intellectual property rights for most new medicines,” due to our market forces which incentivize the kind of risk that companies routinely take despite potential failures. 

GOOD IDEA: Increasing transparency around rebates in Medicare will bring down costs for patients.
 
The administration has proposed increasing restrictions around how pharmacy benefit managers (PBMs) and insurers process rebates for pharmaceutical drugs with the intent of ensuring such rebates and savings go to the patient at the point of sale.
 
This proposed rule also reduces incentives for PBMs to opt for higher-cost treatments:
 
* As higher-cost treatments often have some of the largest rebates, PBMs are currently incentivized to choose more expensive treatments and pocket the rebate savings, increasing costs for insurance companies, keeping list prices high without lowering costs for patients. 

* By mandating rebates and savings are provided to patients at point of sale, PBMs are no longer incentivized to keep list prices high or to choose the most expensive treatments. 

BAD IDEA: A rule weakening protections for six-protected classes in Medicare Part D will hurt patients.
 
The administration has proposed loosening regulations in Medicare Part D which ensure access to “all or substantially all” medications that treat diseases in six-protected classes. These medicines are protected because the treat serious, chronic and often life-threatening illnesses like clinical depression, HIV and cancer.
 
The president believes this proposal Is another example of reigning in out-of-control healthcare costs and that the new rule will force providers to make better drug choices and help fix Medicare’s cost issues
 
The reality is that this proposed rule will restrict access for patients.
 
The rule would implement what’s known as “step therapy,” commonly referred to as “fail first.” 

This means that even if a patient’s doctor has recommended a treatment, insurance companies are empowered to force a patient to first try a different treatment, waiting to see if it fails before a patient can “progress” to other options or eventually the doctor’s original prescription. 

For patients, this means that they will need to endure long periods of treatment before finally arriving at one that works, lengthening the period of illness and causing increased discomfort. 

GOOD IDEA: Transparency on the hospital pricing front.
 
The President rightly noted that hospitals are largely unchecked when it comes to procedure or prescription drug prices. On January 1, a new rule forcing hospitals to disclose their prices went into effect, creating greater transparency throughout the system and providing consumers with more information to make informed decisions about their care. In the long term, these disclosures could allow consumers to act rationally and opt for the best value for their care, creating incentives to reduce prices for patients.
 
GOOD IDEA: Enhanced PBM Transparency.
 
Last autumn, the President signed legislation banning “gag clauses” in pharmacy benefit contracts. These clauses stopped pharmacists from disclosing to patients when they could save at the pharmacy counter. By banning these practices, the administration rightly targeted the added costs that middlemen like PBMs add to the system, reducing burdens for patients and shining a light on unfair practices.
 
The best ideas are common property. – Seneca
 
The President wants to protect insurance coverage for pre-existing conditions. Bravo. The President wants to “reduce the price of healthcare and prescription drugs," eradicate HIV-AIDS and defeat pediatric cancers.  Bravo. Now that “the speech” is behind us, the President and Congress should focus and build upon the administration’s successes  in bringing down the real costs of healthcare instead of pursuing fantasy land socialist policies that puts our healthcare system under government control, deters urgent innovation and rations patient care.  Read More & Comment...

AI? Aye. Aye.

02/04/2019 08:50 AM | Peter Pitts

From The Cancer Letter …
 
Gottlieb: FDA to expand real-world data infrastructure to enhance AI capabilities
By Matthew Bin Han Ong
 
FDA is enhancing its ability to handle real-world evidence by training reviewers in data science via a curriculum on machine learning and artificial intelligence, said FDA Commissioner Scott Gottlieb.
 
“We’re working to develop new guidance documents to assist sponsors interested in developing and using real-world evidence,” Gottlieb said at a Jan. 28 panel discussion organized by the Bipartisan Policy Center.
 
“Our ‘Framework for Real-World Evidence Program’ will apply a consistent strategy for harnessing these tools across our drug and biologic review programs,” said Gottlieb, referring to a framework document published last December. The document evaluates the use of RWE to support additional indications for already approved drugs as well as to satisfy drug post-marketing study requirements.
 
“The framework is aimed at leveraging information gathered from patients and the medical community to inform and shape the FDA’s decisions across our drug and biologic development efforts,” Gottlieb said. “The goal is to develop a path for ensuring that RWE solutions can play a more integral role in drug development and regulatory life cycle at the FDA.
 
“Today, I’m announcing four additional activities that’ll help FDA and stakeholders advance these opportunities for the benefit of patients.”
 
FDA plans to:
 
Support the seamless integration of digital technologies in clinical trials by developing a framework on how digital systems can be used to enhance the efficient oversight of clinical trials. These technologies present important opportunities to streamline drug trials and improve data site integrity by remotely monitoring data trends, accrual, and integrity over the course of a trial.

Use digital technologies to bring clinical trials to the patient, rather than always requiring the patient to travel to the investigator. More accessible clinical trials can facilitate participation by more diverse patient populations within div   erse community settings where patient care is delivered, and in the process can generate information that’s more representative of the real world and may help providers and patients make more informed treatment decisions.

Explore how reviewers can have more insight into how labeling changes inform provider prescribing decisions and patient outcomes. The FDA’s Information Exchange and Data Transformation—or INFORMED—is using RWD to examine the impact of a recent FDA labeling change for two approved products from weight-based dosing to flat-dosing of immune checkpoint inhibitors. This project is focused on how community practices are adopting the flat dose after the labeling change, and factors that may affect adoption.

Work with the medical product centers to develop an FDA curriculum on machine learning and artificial intelligence in partnership with external academic partners. The aim of this program is to improve the ability of FDA reviewers and managers to evaluate products that incorporate advanced algorithms and facilitate the FDA’s capacity to develop novel regulatory science tools harnessing these approaches. 

FDA’s Oncology Center of Excellence is working with Friends of Cancer Research, NCI, and others to harmonize reference standards for assessing tumor mutational burden—as determined by multiple proprietary assays—to help identify cancer patients who are more likely to respond to immunotherapy.
 
Harmonizing the measurement of tumor mutational burden across commercial assays used in routine oncology care can help reduce treatment variability, and improve the utility of TMB as a potential biomarker for enriching clinical trials that are designed to test immunotherapies. OCE is also working on a project exploring whether it’s possible to use real world endpoints, such as time to treatment discontinuation (TTD), as a potential real-world endpoint for pragmatic randomized clinical trials, for FDA approved therapies in the postmarket setting.

“Through ‘Project: Switch,’ OCE is investigating whether well-matched contemporaneous synthetic control arms based on prior clinical trials can be used to make inferences regarding the effect of a new drug, or whether a synthetic control could be used to compare data to active control arms in ongoing randomized controlled trials in rare tumor types where the standard of care remained stagnant, and the prognosis is especially poor,” Gottlieb said.
 
FDA’s framework for RWE, created in response to a mandate in the 21st Century Cures, spells out the agency’s thinking on the types of guidances that need to be developed before RWE can be routinely used in regulatory science.
 
“We really need people to weigh in on the guidances, because one thing I did learn at FDA, pretty much if the FDA says something, the industry is going to do it,” former FDA Commissioner Robert Califf said at the meeting Jan. 28. “So, we’d like to get those guidances right.
 
“I’m very excited that Amy Abernethy is coming to the FDA [as principal deputy commissioner]. She is an expert on this, I have every confidence that she’ll help guide us through this.”
 
There is a need to better understand AI algorithms, and whether they generate results that are replicable, said former FDA Commissioner Mark McClellan, who is also a former commissioner for the Centers for Medicare and Medicaid Services.
 
“It’s great to see the progress that’s happening at FDA,” McClellan said at the meeting. “I think Rob [Califf]’s vision for what the future ought to look like, which is a lot of data from a wide variety of sources, including many that a lot of people in the health care industry aren’t really thinking about as important sources of health relevant information—that is the right vision. I think we’re still a long way from getting there. So, great vision, great potential.”

Using real-world data effectively is akin to monitoring jet engines to prevent plane crashes, said Andrew von Eschenbach, former FDA commissioner and former NCI director.
 
“People won’t die, because planes don’t crash. GE has a system in which their jet engines have an incredible number of sensors that are in those engines and they’re sensing and monitoring those engines in real time, and so they know in real time if there’s anything going wrong,” von Eschenbach said at the meeting.
 
“I think what we have is the opportunity with the kinds of tools that are now becoming available, be they sensors in humans, or the opportunity to access the data that’s coming in both real time and retrospectively, we’re going to be able to prevent problems. We’re going to be able to see ahead, just like they can, and not only retrospectively correct what’s going on, but prospectively be able to create what needs to be created to save lives.”  Read More & Comment...