CDER House Rules

01/25/2016 10:05 AM |

The recent mega-storm allowed many of us drugwonks to curl up with a new work of action non-fiction … from the FDA:

Guidance Agenda: New & Revised Draft Guidances CDER is Planning to Publish During Calendar Year 2016

It’s worth perusing in its entirety, but here are a few items to tickle your regulatory palate:

For those of you following the debate over off-label communications

Manufacturer Communications Regarding Unapproved, Unlicensed, or Uncleared Uses of Approved, Licensed, or Cleared Human Drugs, Biologics, Animal Drugs and Medical Devices

The New Gold Standard

Adaptive Design Clinical Trials for Drugs and Biologics; Revised Draft

Meta-Analysis of Randomized Controlled Clinical Trials to Evaluate the Safety of Human Drugs or Biologic Products

Multiple Endpoints in Clinical Trials

The broader sharing of pharmacoecomonic data

Health Care Economic Information in Promotional Labeling and Advertising for Prescription Drugs Under Section 114 of the Food and Drug Administration Modernization Act

And the continuing saga of “Emerging Electronic Media” (aka: “Social Media”)

Internet/Social Media Advertising and Promotional Labeling of Prescription Drugs and Medical Devices – Use of Links to Third-Party Sites

The evolution of discussing risk information

Presenting Risk Information in Prescription Drugs and Medical Devices Promotion; Revised Draft

For the Biosimilar Brotherhood, three things to consider

Considerations in Demonstrating Interchangeability With a Reference Product

Labeling for Biosimilar Products

Statistical Approaches to Evaluation of Analytical Similarity Data to Support a Demonstration of Biosimilarity

Attention NORD Horde

Guidance for clinical Investigators and Sponsors Natural History Studies for Rare Disease Drug Development

Women and Children First?

Measuring Treatment Benefit in Pediatric Populations: Use of Clinical Outcome Assessments

Pediatric Oncology Product Development; Revised Draft

Pregnant Women in Clinical Trials – Scientific and Ethical Considerations

Pharmacokinetics During Pregnancy and the Postpartum Period – Trial Design, Data Analysis, and Impact on Dosing and Labeling; Revised Draft

Phase Fore!

Postmarketing Safety Reporting for Human Drugs and Biological Products Including Vaccines

REMS Assessment: Planning and Reporting

Waxing Generic

Postmarketing Safety Reporting for Human Drugs and Biological Products Including Vaccines

Updating ANDA Labeling After the Marketing Application for the Reference Listed Drug Has Been Withdrawn

General Principles for Evaluating Abuse - Deterrent Properties of Generic Solid Oral Opioid Drug Products

What’s on CDER’s list is interesting … as well as what is not.

2016 is going to be an interesting year.

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Markey's Mark

01/25/2016 09:14 AM |

Senator Edward Markey (D, MA) is using Senate rules to block the nomination of Rob Califf in an attempt to force the agency to rescind its approval of a prescription opioid for children and change its regulatory practices.

Markey, in Boston Globe interview said he is demanding that the FDA agree to reverse its 2015 decision allowing the pediatric use of the prescription painkiller OxyContin. He also wants the agency to commit to convening expert advisory panels to provide advice whenever considering the approval of an opioid drug, and to ensure that the risks of drug addiction and abuse are taken into account whenever the agency considers approving a prescription opioid.

We call Senator Markey’s attention to the FDA’s new “Guidance Agenda” for 2016.

Prominent on the list are plans to release a new draft guidance on: “General Principles for Evaluating Abuse-Deterrent Properties of Generic Solid Oral Opioid Drug Products.”  That’s important.

Senator Markey is rightly concerned about opioids remaining atop the FDA’s list of “must address” items. But his “hold” on the Califf nomination will, if anything, hold back the agency from making progress on this urgent issue.

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Turingfreude

01/20/2016 09:15 AM |

Terrific article in this week’s BioCentury, “Pricing Politics,” by one of our favorite reporters, Steve Usdin.

Here’s the opening paragraph:

Presidential candidates from both parties are tapping into public anger over prescription drug prices and responding by repeating old proposals, like controlling prices or lowering FDA approval standards, that won’t be enacted and probably wouldn’t work if they were put into practice.

Usdin writes about the “corrosive political environment” and “unrealistic prescriptions (aka: Bernie Sanders). He speaks of schadenfreude over our favorite “Pharma Bro” and how the Turing imbroglio has ignited the always-smoldering fire of public discontent over the way the pharmaceutical industry conducts its business.

How to manage this out-of-control Turingfreude? One way is for the innovative pharmaceutical industry to do a better job communicating the value message (I know, this is a recording) and, per Usdin, both PhRMA and BIO are preparing to ramp up their messaging efforts.

This is important beyond the rhetoric of a political season since many states have transparency initiatives that can only be successfully argued through the lens of relentless science and the potency of value.

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BIO CEO Makes Case For Innovation

01/14/2016 01:29 PM |

In recent days Acorda CEO and BIO CEO Ron Cohen has emerged on the biotech’s sector most effective and articulate voice on medical innovation and drug pricing.

Cohen was quoted in several articles on drug pricing (at least he was taken out of context and misquoted).  And yesterday he was part of a CNBC panel discussion on the cost of medicines. 

The headline for the video blares “Rising drug costs only 'getting worse': Expert”

The expert being Peter Bach.  The definition of an expert is a person who has a comprehensive and authoritative knowledge of or skill in a particular area.

Bach first claimed that drugs are 20 percent of health care spending.  Then he every study shows that drugs like Sovaldi are more expensive than the care it replaces.  He then went on to claim such drugs that Sovaldi aren’t a cure because people can get re-infected with Hepatitis C,

Cohen said:

1. Sovaldi is a medical miracle and do ‘really’ cure.   Beating the virus into submission is the same as NOT having the disease.
2. The insurer funded Institute for Clinical and Economic Review (ICER) shows that even at list prices, Hep C drugs are cost effective.  (Other studies come to the same conclusions.)
3. List price is not what drug companies get. Part of that price become rebates to payers and therefore support a whole posse of PBMs, insurers and oncologists (who get 6 percent of the drug cost as a fee for administering them).

As for the the 20 percent claim, Cohen said that the bigger issue is how to reduce the total cost of care overall and increase value.  Who cares if drugs are 90 percent of the cost of a treatment if it leads to more productivity, longevity and lower spending than would be the case in the absence of such medicines?

It was one of the first times that a media outlet allowed a balanced discussion of drug prices.   And when given the chance to respond, Cohen nailed it.

Bach made claims that belied a lack of authoritative knowledge.  


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STAT and WSJ Stumble On Drug Pricing Reporting

01/13/2016 04:07 PM |

An article by STAT journalist Rebecca Robbins on drug pricing could use a lot of re-editing for balance and depth.  Or maybe should could find another job. 

She contrasts public ‘outrage’ (as represented by some well-dressed protestors)  about drug pricing with an orgy of greedy indifference on the part of biotech CEOs.

Which can also describe Ms. Robbins reporting because of what is NOT included. 

She follows Cohen’s comments (which were taken out of context: Cohen actually said that to smear everyone in the biopharma industry as all being “greedy profiteeers” is an abomination and perverse) with context free narrative about drug prices:

“Many drug makers have raised prices in the past year.  And a slew of new drugs have (sic) hit the market with eye-popping price tags: cancer drugs at more than $11,000 a month; cholesterol drugs at more than $14,000 a year. Then there’s Martin Shkreli, the pharma executive who bought up a decades-old drug and hiked the price 5,000 percent, turning himself into a target of nationwide protests before he was arrested last month on securities fraud charges.”

Yes, linking Turing turd Shkreli who used the Daraprim price hike to short biotech stocks for the sake of his own portfolio, is now lumped in with companies developing new and important medicines that save lives, reduces health care costs and increase well-being is now part of the narrative.

Robbins is not alone in this perversion of reality.  The WSJ’ Peter Loftus runs a me-too story that portrays price hikes as disregarding “mounting criticisms of prescription costs in the U.S”

Loftus claims companies “have raised U.S. prices for dozens of branded drugs since late December, with many of the increases between 9% and 10%, according to equity analysts. The increases are on list prices, before any discounts or rebates that manufacturers sometimes provide insurers and other payers.  Some of the increases add thousands of dollars to the cost of already expensive drugs, and come on top of repeated price hikes in recent years.”

Let’s look at the price ‘hike’ in context.  I will limit this discussion to the deceptive way in which prices are used.   I won’t discuss the fact that neither writer discusses the value of new treatments relative to existing therapies for payers and patients.  


Since Ron Cohen , CEO of Acorda Therapeutics,Inc.  is one of the main characters of these stories, let’s look at how Loftus reports on the pricing of it’s main product Ampyra, which is used to help multiple-sclerosis patients improve walking.  Loftus reports that Acorda raised Ampyra’s price by 11% on Jan. 1, to an annual cost of more than $23,650 a patient. 

Ampyra revenues (unaudited) in 2015 were about $ 436 million.  
When discounts and rebates to PBMs etc are taken into account, Accorda will gain only about 60% of the price increase.

That does NOT take into account that Acorda provides 2 months of free drug to people with new prescriptions, through our First Step program. At this point, 75% of all new prescriptions are First Step (and a higher percent of all commercial Rxs, as we are not allowed by law to give First Step to Medicare/Medicaid patients). The 10-K notes that 38-43% of patients respond to the treamtment, so First Step ensures  the physicians and patients have determined that the patient is a true responder before asking the system to pay for the drug.

It also provides a generous PAP program, giving free drug to a significant portion of the population who are uninsured or underinsured
It also provides co-pay assistance so that no commercially insured patient pays more than $40 for an Rx. 
All this an Acorda is not yet not profitable as a company since it is investing 1/3 of net sales n 6 clinical programs for innovative drugs to treat, Parkinson’s, epilepsy, stroke, MS and migraine.

Which means that the money given to PBMs and insurers aren't spent on more R&D.  Yet neither Loftus or Robbins acknowledge that PBMs and insurers pocket the rebated portion of these prices.  Nor do they note that these organizations then force patients pay to up to 30 percent of the price of the drug which is often marked up by insurers and such pharmacy benefit firms as Express Scripts.  Payers know that companies will – after forking over rebates – also pay a big share of the patient’s drug bill.  (Which explains why per patient sales are way below list price in many cases.)  Indeed, Acorda's copay assistance is provided regardless of where insurers price the drug to patients. 

Robbins points to a Senate report claiming Gilead priced drugs so that many people and Medicaid programs could not afford Solvaldi.  In fact, payers were pocketing the rebates and deny access to the drugs.  The Senate report notes a Gilead memo that states: “While many payers responded to these discounts by opening access broadly, some payers have continued to restrict access despite the discounts. “

Moreover, the Senate report, like Robbins and Loftus, ignored the rebates to the states.  A report based on Medicaid data showed that Medicaid rebates for HCV brand medications “typically increased over time and averaged roughly 60% during 2014 across all brand medications.”  

Medicaid requires companies to provide rebates (to states) of at least “23.1 % of the Average Manufacturer Price (AMP) per unit” or “the difference between the AMP and the best price per unit” to commercial payers if that rakes in more rebates.

On average, AMP is 59 percent lower than Average Wholesale Price, the so-called ‘retail’ price journalists like Robbins and Loftus use.  That means the price used to calculate Sovaldi rebates is $49560 per patient.  ($84000 x 59%).  Gilead then provided rebates of 33 percent of that price according to the Senate report.  That comes out to $33205 per person which is a 60 percent cut from retail price.   Which means that drug prices are a vehicle for redistributing income to private and public payers. 

Finally, neither Robbins or Loftus put drug price increases (net price or otherwise) into perspective.  Loftus states that U.S. prescription-drug spending rose 12.2% in 2014, accelerating from 2.4% growth in 2013. But “price increases for protected brands increased spending by $26.3 billion, contributing 8.2% to total market growth on an invoice price basis; estimated net price growth was substantially lower as rising off-invoice discounts and rebates offset incremental price growth and reduced net price contribution to growth to 3.1%.” 

That’s an increase in spending of about $7.1 billion.  Total US health care spending increased by $100 billion from 2013-2014.  So brand drugs were 7 percent of that amount. 

I believe the incremental benefit of this spending is, a Donald Trump would say, huge.  But first things first.  Reporters should not report on drug prices in a context free zone.  Doing so, especially since the factual context is easily available, is a deliberate perversion of reality. 


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FDA and NBCDs: Do Not Pass GAO

01/13/2016 01:06 PM |

GAO Asked to Assess Viability of FDA’s Complex Generics Pathway

The House Energy & Commerce Committee is calling on the GAO to evaluate whether the FDA’s regulatory pathway for generic versions of complex drugs is sufficient.

Specifically, the committee sent a letter Dec. 10 asking the GAO to assess whether generic versions of nonbiologic complex drugs that are not fully characterized present challenges in meeting generic approval standards.

If the agency concludes that meeting approval standards presents challenges, then the GAO should consult with public and private groups to analyze the following questions:

• What requirements should be established regarding the comparability of the manufacturing process?
• What degree of characterization of the proposed generic version and the reference product should be required to determine safety and efficacy?
• What degree of similarity should be required for the active ingredient of the generic version to be deemed the same as the active ingredient in the reference product?
• What types of evidence should be required to demonstrate bioequivalence?
• How much clinical evidence is needed?

Once those questions are answered, the agency should determine whether current ANDA pathways can address the use of reference products. The study also should make recommendations for the FDA, such as developing policy documents and establishing general principles on evidence needed.

Read the letter here: www.fdanews.com/12-15-15-FDAGenerics.pdf.

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Undermining Patient Values: The ASCO Value in Cancer Care Task Force Framework

01/12/2016 01:32 PM |

Peter and I just published an article in The Journal of Commercial Biology on how the ASCO Value Framework could hurt patients and stifle innovation
Compare the approach ASCO takes to the one shaping the Cancer Moonshot 2020 effort. 


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A New Cure Coalition

01/11/2016 03:53 PM |

A new coalition of biotech companies, insurers, patients, regulators was established with the goal of nothing less than using precision medicine to 'cure' cancer.  (A cure meaning controlling and preventing the progression of tumors so that the disease is within us but can not hurt us) 

Today "leaders from large pharma including Celgene and Amgen, biotech including NantWorks, NantKwest, Etubics, Altor Bioscience, and Precision Biologics, major academic cancer centers and community oncologists announced the launch of ‎The National Immunotherapy Coalition (NIC), a historic alliance--in collaboration with Independence Blue Cross, one of the nation’s largest payers and Bank of America, one of the largest self-insured companies in the U.S.--with a singular focus: accelerating the potential of combination immunotherapies as the next generation standard of care in patients with cancer."

This is perhaps the most important scientific initiative undertaken in human history for reasons I will elaborate in another post.  

For now, here are the three most important features of this initiative:

1.  The QUILT (QUantitative Integrative Lifelong Trial) program is designed to harness and orchestrate all the elements of the immune system (including dendritic cell, T cell and NK cell therapies) by testing novel combinations of vaccines, cell-based immunotherapy, metronomic chemotherapy, low dose radiotherapy and immunomodulators -- including check point inhibitors-- in patients who have undergone next generation whole genome, transcriptome and quantitative proteomic analysis, with the goal of achieving durable, long-lasting remission for patients with cancer.

2.   Multiple randomized Phase 2 trials testing genomically and proteomically informed novel combinations of immunotherapy agents, will pave the way to identifying cancer therapy combinations with the lowest toxicity and the highest quality of life.

3.  Insurers will be covering whole genome transcriptomic tests in patients receiving immunotherapy.    Independence Blue Cross is the first health plan to cover next generation sequencing.   The coalition is talking to others, who I think will have to follow.  

This is good for patients and innovation.  And this approach -- which should be replicated for other illnesses where combinations of treatments - old, new and emerging -- will be gold standard of care. 

That mean groups that seek to measure the value of a single cancer drug based on average response --  like ICER,  the ASCO Value Framework, and Peter Bach's comical Cancer Drug Abacus etc. will become obsolete.  That means Express Scripts effort to use the aforementioned comical Cancer Drug Abacus to establish indication based pricing should be discarded and ignored.  Any entity claiming it wants to establish value that does not seek to promote payment and access to precision medicine to match patients to treatments that control and prevent disease most effectively should be considered outdated and obstructionist.  

This is a great day for those committed to cures.  And that should include everybody.  

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David Bowie's cancer battle :You can neither win nor lose if you don't run the race

01/11/2016 01:02 PM |

The death of the transformative musician, artist and filmaker David Bowie to due to cancer underscores once again the value of innovation.



 

Bowie battled cancer long enough to complete and release his last album on his 69th birthday.  You don't have to be a David Bowie to know that using the retail cost of a cancer drug to discourage it's use would sideline many people with a cancer diagnosis.

As Bowie noted: You can neither win nor lose if you don't run the race. 


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Fab Mab? Look Beyond the Lab.

01/10/2016 10:52 AM |

As we move ahead in the US with more biosimilars being submitted for approval – and for interchangeability – attention must be paid to patient outcomes.

Case in point is research presented at the 2013 ACR/ARHP Annual Meeting that hasn’t as yet received the attention it deserves:

Efficacy and Safety of CT-P13 (Infliximab Biosimilar) over Two Years in Patients with Ankylosing Spondylitis: Comparison Between Continuing with CT-P13 and Switching from Infliximab to CT-P13

Background/Purpose: CT-P13 is an infliximab (INX) biosimilar recently approved by the European Medicine Agency. PLANETAS was a 54-week (wk) randomized double-blind parallel group multicenter Phase I study demonstrating pharmacokinetic equivalence of CT-P13 (5 mg/kg infusion every 8 wks) with INX, in patients (pts) with ankylosing spondylitis (AS) (Park W, ARD 2013;72(S3):516). Here we report results from the extension phase of the Phase I equivalence study, investigating long-term efficacy and safety of extended CT-P13 therapy and switching from INX to CT-P13 in pts with AS.

Methods:

In this open-label extension study, a total of 174/210 pts who completed PLANETAS entered into the extension phase: 88 were continuously treated with CT-P13 (maintenance group) and 86 were switched from INX to CT-P13 (switch group) for 1 additional year.

Results: At wk 54 ASAS20/ASAS40 and ASAS partial remission rates were similar between groups (CT-P13, 70.5%/58.0% and 20.5%; INX, 75.6%/53.5% and 19.8%, respectively). During the extension, ASAS20/ASAS40 rates were similar in the maintenance group (70.1%/57.5% at wk 78 and 80.7%/63.9% at wk 102) and the switch group (77.1%/51.8% at wk 78 and 76.9%/61.5% at wk 102). ASAS partial remission rates were also similar between groups; 21.8% and 21.7% at wk 78, and 27.7% and 28.2% at wk 102, respectively. An overview of the efficacy data are shown in the Table. Anti-drug antibodies (ADA) were comparable between the two groups and positivity was maintained throughout the study (maintenance group, 22.2%, 24.4% and 25.0%; switch group, 26.2%, 31.3% and 30.7%, at wk 54, 78 and 102, respectively). ADA negative pts achieved higher ASAS40 responses (maintenance group, 62.9%/61.5%/66.1%; switch group, 58.1%/60.0%/71.2% at wks 54, 78 and 102, respectively) compared with ADA-positive pts (maintenance group, 38.9%/36.8%/55.0%; switch group, 41.7%/33.3%/45.8% at wks 54, 78 and 102, respectively) with no differences between the maintenance and switch groups. The proportion of pts with ≥1 treatment-emergent adverse event (TEAE) was lower in the maintenance group (48.9%) compared with switch group (71.4%) mainly due to fewer mild and moderate AEs. Serious TEAEs were identical between groups (4 vs 4 pts). Other tolerability and safety outcomes were similar in both groups (Table). TEAEs due to hypersensitivity and infusion-related reactions were similar in both groups (5 pts in maintenance group vs 2 pts in switch group). There was 1 case of TB in each group and 1 report of prostate cancer in the maintenance group (considered unrelated to treatment).

The efficacy data was good. But the safety data is concerning:

Biosimilarity and measurement of efficacy is only one dimension. Attention must be paid to effectiveness relative to real-world patient outcomes data. And that means a much closer pharmacovigilance examination of adverse events for both naïve and switched patients.

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WSJ Context Free Reporting on Drug Costs

01/05/2016 11:37 AM |

I received some puzzled responses WSJ's Joseph Walker and Jonathan Rockoff to a couple of tweets I posted regarding the extent to which WSJ articles on drug costs and prices seek to reinforce a narrative by leaving key contextual facts out of said news items.

I recently referred to a WSJ article on advances in myeloma drugs by tweeting: Will @joewalkerWSJ @JeanneWhalen @jonathanrockoff  ignore PBMs, insurers pocket rebates, make patients pay $1000s http://on.wsj.com/1Oxb69P..  Meaning will these reporters follow up with an article on myeloma treatments hat pins the blame on out of pocket drug costs on drug companies as opposed to or in combination with many other decisions undertaken by PBMs and insurers?  Will they look at the impact of such medicines in reducing hospitalizations, infusions, etc that insurers pay for and are more expensive than the drugs patients must partially cover?

Below is a chart that has a link to a specific article on drug costs by each WSJ reporter and whether or not the articles discuss specific issues that would put the cost and value of drugs in context. 

Maybe this will clear up the confusion.  


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BIO asks, "What's in a name?

01/05/2016 08:48 AM |

The Biotechnology Industry Organization changed its name to the Biotechnology Innovation Organization. The group will continue using the "BIO" acronym.

This is more than just cosmetic. It demonstrates the urgency in both promoting and protecting sustainable innovation in biotechnology. If we are going to argue “value” rather than just “price,” than innovation the most important part of the conversation.

We do what we must, and call it by the best names.

Ralph Waldo Emerson

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CDC's Opioid Reset

01/05/2016 08:43 AM |

CDC sees the light …

This morning the CDC will ask the National Center for Injury Prevention and Control's Board of Scientific Counselors to appoint a new work group to review the agency's draft guideline for prescribing opioids for chronic pain. It's the next step in CDC's effort to address complaints over the secretive way in which the initial draft was developed. Public comments on the draft guideline are due Jan. 13, and the new group will be tasked with reviewing those, too.

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Spotlight vs Blind Spot

01/04/2016 10:23 AM |

Spotlight is a movie about how the Boston Globe's investigative team (called Spotlight) exposed the Boston Diocesan's complicity in protecting child molesters in the priesthood.  The most dramatic moment in the film comes when Spotlight's chief, Walter Robinson (played by Michael Keaton) confesses that he buried a story about multiple priests abusing young boys in the in 1970s.  At the time, pursuing the story -- compared to easier and less controversial investigations of police corruption,  racial discrimination,  government incompetency -- went against the ethos of Boston and the narrative that the Church was an integral part of Boston, where most of the reporters were raised.  

While nowhere near the level of irresponsibiity, the WSJ coverage about drug costs appears to be shaped by the same set of beliefs and emotions.  Every article that the WSJ has written about drug costs in 2015 (with the exception of a blog written by Ed Silverman when Pharmalot was part of the WSJ) ignored the role of pharmacy benefit managers and health insurers in hiking the cost of new drugs used by the most chronically ill patients.  The have ignored the fact that PBMs, in particular, pocket 90 billion in rebates, none of which goes to reducing the cost of drugs.  They have ignored the fact that the discounted drugs are less than 3 percent of total health plan spending or that as the percent increases, the cost of other services decline.  They have ignored the fact that health plans have increasd the number of new targeted medicines -- drugs that work best in specific patients -- by 40 percent since 2014.  (See below.)

There are more oversights as well but the list above suggests that the WSJ -- including the most recent WSJ article -- has a blind spot, not a spotlight on the issue of drug costs.  The WSJ is burying the real story to sustain a narrative, at the expense of patient well-being. 

Our focus in 2016 will be to create greater transparency about how drug prices are set and by who, how those decisions affect patient access to and affordability of such medicines, what affect access has on patient well-being, mortality and health costs and finally, what changes in decisionmaking can reduce spending and increase value (to patients) long term.  

Further,  we will look at how the reactionary defense of the randomized clinical trial is the greatest rate limiting factor in reducing the time and cost of bringing new  medicines, diagnostics and devices to patients.   This will NOT be an FDA pinata festival.   Rather, the RCT is being protected by leading medical journals, academic research centers, major medical specialty societies and health insurers.     Read More & Comment...

It Was a Very Good Year. Really.

12/30/2015 03:58 PM |

Pessimism sells.  As Ben Wattenberg, the late political observer wryly noted: “Worse is better” if you are looking for headlines, grants, attention and want to be deemed an expert.  (Self help gurus being the exceptions.) 

Pessimism is necessary in small doses, allowing us to anticipate, adapt to and often avoid failure or danger.   It is also an adjuvant for change. (I don’t know what that means but it’s pithy enough to sound smart.)  But apocalyptic pessimism, the pessimism that uses every event or fact to confirm certain doom unless we change our ways, is corrosive and unreliable.  How do we know?

As Matt Ridley, another professional optimist observes. “For 200 years pessimists have had all the headlines, even though optimists have far more often been right.”

The steam powered train was predicted to bring ruin to England.
The industrial revolution was supposed to bring enduring misery to millions.
The poor will always be poor.
The sky is falling; the ice caps are melting.
We will run out of oil and natural gas.
Drug prices are unsustainable.

If we had followed the pessimists prescriptions (government control or manipulation of wealth, natural resources, population levels and prices) the world would be more like North Korea than North Dakota. 

I will focus on the last perennial pessimistic prediction. 

We heard and read a lot about drug prices being out of control, skyrocketing, unconscionable, etc. 

In every era where new medicines emerge, the same claim is made.   Te same bunch of pessimists trot out the same proposals: government price controls and rationing.  (Now repackaged as various value frameworks.)

And each time the apocalyptic predictions that new medicines would be unaffordable or would add nothing to our lives  or drive up the cost of health care were wrong. 

2015 was all about drug pricing because there were so many new and expensive (relative to most consumer purchases) medicines hitting the market.   But in fact the high prices – and indeed even the anger about drug prices – are a healthy sign of progress.

It means that diseases that were fatal can be cured for controlled, including Hep C, many cancers and Ebola.  It means that more diagnostics are being developed to match people to the right combination of treatments and to monitor health in real time.  It means that more investment in new treatments for smaller groups of people will be developed.  Nothing sends a signal about the demand for better medicines than high prices. 

At the same time, the high prices are forcing drug companies to do something the rest of the health care system will have to do: change business and financial models to make the case for value and increase the pressure to abandon outdated clinical development tools (randomized clinical trials) and find other ways to demonstrate the health benefits of their goods and services.

Prediction is over-rated when it is too precise.  But the general trend of medical innovation, the digitization of biology, is part of a broader evolution in how we create and use the things that give us joy, insight and well-being.  Our world is better because the footprint for change is getting smaller and faster.  In a WSJ op-ed Andy Kessler wrote that “on Dec. 29, 1959, the physicist Richard Feynman delivered a famous speech at the California Institute of Technology titled “There is Plenty of Room at the Bottom.” He predicted almost limitless possibilities if we could “manipulate and control things on a small scale.” He nailed the next five decades of ever-shrinking realms that ultimately produced trillion-dollar markets in microelectronics, nanotechnology and bioengineering through DNA-level manipulation.…The smaller technology shrinks, the bigger the world can grow. Smaller transistors, faster processors, cheaper sensors will all allow innovators to tackle problems with tremendous precision. It’s as if, because there is plenty of room at the bottom, now there is plenty of room at the top.”

We are increasingly able to navigate and understand what Lee Hood calls “the dark matter of human biology.”  New medicines are the result of our ability to explore health, wellness and disease at a molecular level. 

Here’s a non-pessimistic prediction: the next trillion-dollar market will be new treatments and algorithms that control disease and keep people healthy.   You have to try really hard to despair about that!  Read More & Comment...

Price/Value: A Happy New Year?

12/30/2015 09:19 AM |

NPR/Marketplace story on price/value and PCSK9 approvals:

“The price can’t be considered a stagnant proposition. It needs to be a vibrant, living thing,” said FDA consultant Peter Pitts.

Boston Globe (Ed Silverman) on things to watch (per pharma) in 2016:

A key unknown, however, is whether drug makers will become more transparent about their pricing strategies when negotiating with these payers. This is a “very important” part of determining value for patients, noted Peter Pitts, a former Food and Drug Administration official who now heads the Center for Medicine in the Public Interest.

Happy New Year   Read More & Comment...

Nasty Morons As Master Analysts of Drug Pricing

12/23/2015 10:14 AM |

Did you know that ten of the 80 costliest drugs Medicare pays for had price increases of between 77 and 543 percent?  And did you know that all of them are generic drugs?   Or that the cost per unit of specialty drugs (cancer, MS, psoriasis, etc. ) increased an average of 6-7 percent? 

I bet you didn’t because not one media outlet covering the release of CMS drug spending data mentioned it:  Instead, most of the articles started off like… oh, wait why don’t I just share with  you the highest form of journalistic misdirection by the WSJ's Peter Loftus.

“Hefty price increases for a number of prescription drugs contributed to higher spending by the U.S. Medicare program in 2014, according to new government data released Monday. The Centers for Medicare and Medicaid Services identified at least five drugs that were covered under Medicare’s Part D drug benefit and had increases of 100% or more in cost per unit from 2013 to 2014. At the top of the list was Vimovo, a pain reliever whose cost-per-unit rose more than 500% after Horizon Pharma PLC purchased rights to the drug from AstraZeneca.”

Still waiting for a detailed breakdown of who did what?  Don’t look under the Christmas tree because Santa isn’t delivering it this year.  Neither is the WSJ or Bloomberg (who produced what can only be called a deceitful piece of reporting on the relative price of drugs in Europe and the US).

Instead after providing a description of the new CMS dashboard, Loftus concludes: “The new data emerge amid a rising debate over drug pricing. Cancer doctors have complained that the high costs of new drugs don’t seem to be tied to the benefits provided by the drugs. Presidential candidates Bernie Sanders, Hillary Clinton and Marco Rubio have criticized drug prices on the campaign trail. A U.S. Senate committee recently held a hearing about companies that have dramatically raised prices after acquiring older drugs.”

Just enough ‘information’ to reinforce the narrative that drug prices are wildly out of control because they don’t reflect value if they are new or R&D costs if they are old.   Either way, the real goal of the piece is to imply that drug profits are too high, a view stoked by previous articles by Loftus and others.  Any word about how PBMs and other third parties pocket $90 billion in drug rebates never shared by patients?  Come on. 

The late Christopher Hitchens observed that he had “become too accustomed to the pseudo-Left new style, whereby if your opponent thought he had identified your lowest possible motive, he was quite certain that he had isolated the only real one. This vulgar method, which is now the norm and the standard in much non-Left journalism as well, is designed to have the effect of making any noisy moron into a master analyst.” 

Noisy morons as master analysts.  Now THAT’s accurate reporting.

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Will 2016 Be A Happy Cure Year??

12/22/2015 06:12 PM |

Hillary Clinton plans to announce a plan to increase NIH spending by $2 billion are, all of which would be devoted to finding a cure for Alzheimer's.   

That makes Secretary Clinton the fourth person who is running or almost ran for president this year to call for a national assault on disease.  (Ted Cruz, Mike Huckabee and Joe Biden are the others.  And yes, I am sure if Donald Trump joins them it will be to announce something really, really spectacular.)

That's on the heels of a  "$2 billion funding boost for NIH and a $133 million increase for FDA – levels near what the bipartisan H.R 6, the 21st Century Cures Act, sought for Fiscal Year 2016. Energy and Commerce Committee Chairman Fred Upton (R-MI) commented, “This research boost is an important milestone - we’ve made great progress, but more work remains as we seek to build upon this momentum to deliver cures now. As the Senate continues to do its work, we will continue to seek out every legislative opportunity to improve the future for patients and cures.” The momentum continues as The Hill reports, on the heels of last week’s milestone, Senate Committee on Health, Education, Labor, and Pensions Chairman Lamar Alexander (R-TN) reiterated that the Senate’s cures bill is a priority for the New Year."

And that's not all.  In addition to Senators Roger Wicker and Angus King's EUREKA Act (which would  authorize the Director of the National Institute of Health to work with other federal agencies to establish prize challenges to reach research milestones) there have been several other legislative proposals to encourage and reward initiatives that cure expensive and difficult to treat diseaes.

There can and will be aspects of all these proposals that can be picked apart.   But as my colleague and cure strategist Jim Pinkerton points out: "history tells us that when the country commits to a goal, big things tend to happen."


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Congress Seeks CDC Sunshine

12/22/2015 07:22 AM |

Things are heating up at the CDC.

The agency’s lack of transparency regarding their development of opioid prescribing guidelines has drawn the attention of the House Committee on Oversight and Government Reform.

In a letter to CDC Director, Dr. Tom Frieden both the Chair, Representative Jason Chaffetz (R, UT) and Ranking Member, Representative Elijah Cummings (D, MD) share their displeasure at the secretive nature of the guideline development process. Per the Committee, “We have questions about why a Core Expert Group established by CDC to assist with drafting these guidelines is not considered an advisory committee under the Federal Advisory Committee Act (FACA).”

Those questions and an aggressive documents request  are included in the letter which can be found here. And the Committee wants them by January 8^th. Looks like there will be some people called back from their Christmas vacation at the CDC.

Perhaps the best way to address the CDC’s fairy tale process is to quote from author of Peter Pan:

“Those who bring sunshine into the lives of others cannot keep it from themselves.”

James Barrie   Read More & Comment...

Get on the Omnibus, Gus

12/21/2015 03:19 PM |

Drug Informatiom Daily helps spread the good news --

President Obama Signs Off on Omnibus Bill Upping FDA’s Funding

President Barack Obama signed into law on Friday the fiscal 2016 omnibus spending bill, which contains $4.68 billion in total funding for the FDA.

Congress had wasted no time approving the measure Friday morning, as it sailed through both chambers by wide margins.

The bill – which includes more than $2.7 billion in discretionary funding, a $132 million increase from the previous year – passed the House and the Senate by margins of 316 to 113 and 65 to 33, respectively.

Industry insiders have touted the bill as a positive for the FDA. “[Congress] is beginning to recognize that the FDA, to do its job, needs appropriate funding to be an innovator,” Peter Pitts, president and founder of the Center for Medicine in the Public Interest and a former FDA associate commissioner, tells DID.

Ladd Wiley and Steven Grossman, executive director and deputy executive director, respectively, of the Alliance for a Stronger FDA, describe the funding in a prepared statement as a “victory.” They add that the distribution of funds appears to largely mirror the priorities of the administration’s request.

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