Latest Drugwonks' Blog


  • 05.05.2017
  • Peter Pitts
ICER’s new report, to no one’s great surprise, has found abuse-deterrent opioids provide neither financial nor societal benefits.

GIGO. Garbage In. Garbage Out.

For example:

The ICER analysis specifically does not include diversion – even though the majority of the problem arises from this community.

ICER also changed its model at the last minute to be include all abuse-deterrent formulations (ADFs) and not just Oxycontin -- despite preliminary results that showed that, over 5 years, OxyContin ADF prevented 4,300 cases of abuse, >12,000 abuse years, saved $300 million in medical costs against $387 million in incremental pharmacy costs. These results alone are within the realm of cost effectiveness: $20,500 per abuser avoided, $7100 per abuse year avoided for the most successful ADO introduced into the market.

Societal costs are not included despite ICER’s promise they would be.

Heroin switching is included, despite the fact that it is an incident cohort. Per ICER, “We did not include the effects of increasing heroin use that might result from opioid abusers being switched to ADF opioid, as we are considering only incident and not prevalent opioid abuse in the model.” Hm.

ADF benefit is reduced by 25% because of the author of the original paper (Rossiter) conducted a sensitivity analysis to see how such a reduction would effect the model.

ICER is calling for ADFs to be “cost-neutral.” But how is this possible since the overwhelming cause of the problem are inexpensive, non-ADF generics?

What the ICER report ignores entirely is that one of the factors driving abuse and addiction is the inappropriate use of generic opioids for conditions that have non-opioid, on-label options. (52 percent of patients diagnosed with osteoarthritis receive an opioid pain medicine as first line treatment as do 43 percent of patients diagnosed with fibromyalgia and 42 percent of patients with diabetic peripheral neuropathy.)

Payers often implement barriers to the use of branded, on-label non-opioid medicines, relegating these treatments to second line options – along with new abuse-deterrent opioid formulations. The result is a gateway to abuse and addiction. An unintended consequence of the ICER analysis will be more of this inappropriate behavior.

According to Harvard health economist David Cutler, Virtually every study of medical innovation suggests that changes in the nature of medical care over time are clearly worth the cost.  But, as Aldous Huxley reminds us, ““Most human beings have an almost infinite capacity for taking things for granted.”

Abuse-deterrent opioids are precision medicines. They are not for everyone. As Dr. Charles Inturrisi, professor of pharmacology at the Weill Cornell Medical College, said at a 2013 Center for Medicine in the Public Interest Capitol Hill conference on opioids, “Personalized medicine can reduce the non-responder rate because you can focus in on individuals who are highly associated with being responders and you can eliminate the trial and error inefficiencies that inflate healthcare cost.”

One of the consequences of the ICER report is that it will deter investment in more and more creative abuse deterrent programs. The phrase, “strangling the baby in the crib” comes to mind.

Remember – ICER is the organization that found the new class of Hepatitis C therapeutics failing their cost/benefit litmus test -- another example of this organization being on the wrong side of history. Alas, it’s totally understandable considering that, on September 3, 2016, Dan Ollendorf (ICER Chief Scientific Officer) told the Pink Sheet, “It’s difficult to really understand how these [abuse deterrent opioids] are going to be of benefit if the non abuse-deterrent formulations are still out there.”

Talk about Confirmation Bias!


QunitilesIMS Data to Digest

  • 05.05.2017
  • Peter Pitts
The QuintilesIMS Institute has issued a very interesting new report, “Medicines Use and Spending in the US: A Review of 2016 and Outlook to 2021.” It’s a must-read for many reasons – not the least of which is its plethora of data. Many items to peruse and digest.

Here are four of the reports conclusions – as an appetizer …

* The outlook for spending has been revised downward as expectations for new products and price increases have moderated.
* Invoice price growth for protected brands is projected to be between 7-10% down from 8-11% in the prior outlook.
* Net price growth for protected brands is forecast to be 2-5% through 2021.
* The impact of losses of exclusivity are expected to be 50% greater in the next five years, including the impact of biosimilar introductions.

With the caveat that statistics are like swimwear (what they show you is interesting but what they conceal is essential), have a look and draw your own conclusions.
Representative Morgan Griffith (R/VA) has introduced HR 1703, the Medical Product Communications Act. It’s legislation that would take a lot of the ambiguity out of the FDA’s biggest conundrum – regulatory clarity on the sharing of truthful accurate, and non-misleading off-label information.

As Mr. Griffith writes in a Dear Colleague letter, “Doctors should have the most up-to-date information when caring for their patients and, when done responsibly and in an appropriate context, manufacturers should be able to provide it.”


For more detail on the issue, have a look at this new article from the Therapeutic Innovation and Regulatory Science (the official journal of the Drug Information Association), “Using Off-Label Communications to Responsibly Advance the Public Health.”

The Dubai Declaration

  • 05.03.2017
  • Peter Pitts
When it comes to 21st century pharmacovigilance, sometimes it’s important to look …  backwards. According to the 10th century Arab physician, Ibn Sina, “The time of action must be observed, so that essence and accident are not confused.”

At the United Arab Emirates’ Sixth National Pharmacovigilance Conference” held last month in Dubai, specific recommendations (known as “the Dubai Declaration”) were released by the Ministry of Health and Prevention (MoHP).

According to Dr. Amin Hussain Al Amiri (MoHP- Assistant Undersecretary for Public Health Policy & Licensing Sector, the Declaration will be submitted to the regional Gulf Health Council for Cooperation Council States and the 22-member Arab League with the hope that this “would benefit the Arab World.”

The complete list of recommendations can be found here.

A few stand out from the rest, specifically:

1- Build trust in the critical role of testing medicines following registration and testing random sample from the field.

2- Enhance of pharmacovigilance education programs among HCPs in the region.

3- Include pharmacovigilance education in the academic curricula of medical and scientific schools and making it among the basics of education.

4- ADRs reporting must become a “culture of HCPs” who must not be embarrassed in doing so.

5- Enhance health education to raise public awareness on expected ADRs.

6- Reported ADRs to be categorized and separated according to originator medicine, generic, and the biological view of the difference in chemical compositions.

7- Focus on the educational role of pharmacovigilance officers among the various healthcare establishments in the region.

When it comes to the quality of medicines, remember the words of Dr. Janet Woodcock, (CDER Director, USFDA), “The spark that ignited the flame was when we asked ourselves, Do we know enough about the quality of drugs that are sold in the United States? And the answer was … no.”

We would all do well to learn not just from the past – but from other parts of the world. The West doesn’t have a monopoly on good ideas.

Scott Gottlieb: Hope Over Hate

  • 04.27.2017
  • Robert Goldberg
Today (we hope) the Senate HELP Committee will vote to confirm Dr. Scott Gottlieb as the next FDA commissioner.

Three things stand out about his nomination:

1.    Dr. Gottlieb has garnered the support of a wide range of patient organizations and medical associations.  The same coalition supported Dr. Rob Califf’s nomination in 2016.
2.    The same groups and individuals who oppose Dr. Califf’s nomination are opposing Scott.  Apart from the Senators who opposed both individuals to demonstrate frustration with the FDA’s response to the opioid addiction problem, the critics are the self-important and self-serving hacks from Public Citizen along with smug academics whose names I will not repeat. 
3.    Both Dr. Gottlieb and Dr. Califf are deeply committed to changing the paradigm of FDA approval by working with talented individuals inside the agency.  

Dr. Gottlieb’s nomination reflects a bi-partisan consensus about the role of the FDA that originated with President Obama and the nomination of Dr. Peggy Hamburg:   When it comes to evaluating the risk and benefit of innovations that determine not only whether we live or die but how we live and die, patients and their loved ones have as much of say on the basis for approval and regulation as any expert.  

Dr. Gottlieb will carry on that tradition.  His opponents want to destroy that tradition.  Hope vs. hate. Remember that the next time you see a hateful, angry tweet about the next FDA commissioner.  

Making Drug Manufacturing Great Again

  • 04.21.2017
  • Peter Pitts
From the pages of Investor’s Business Daily

Making Drug Manufacturing Great Again

President Trump wants to lower drug prices and reinvigorate domestic pharmaceutical manufacturing.  Bravo. But standing in the way is the inside-the-Beltway gospel that preaches that regulators love ambiguity.

As a former FDA associate commissioner, I can affirm that's true. Vagueness gives the agency almost unlimited authority to do whatever it wants.
But, when it comes to the FDA, it's predictability in pursuit of the public health that's important. And nowhere is this truer or more timely than when it comes to the oversight of drug manufacturing.

Drug manufacturing isn't sexy to the general public and rarely makes headlines — unless something goes wrong. Recalls make headlines. Adherence to current good manufacturing practices (GMPs) do not.

A few years ago I had the chance to visit Pfizer's Kalamazoo production facility. What impressed me more than the gee-whiz production aspects of the facility (of which there were plenty) was the dedication of the people who work there — top to bottom.

It actually reminded me a lot of the FDA. Long-term employees dedicated to serving the public health through dedication to quality. And they all took it very personally. Just like at the FDA, the Pfizer folks were on personal missions of quality. There was a lot of pride on display.

Mr. President — there hasn't been an exodus of pharma manufacturing to foreign shores. In fact, when I visited the Kalamazoo facility they were exporting (among other things) the active pharmaceutical ingredient (or API, the actual drug substance) for methyl prednisolone (a corticosteroid long off patent) to both China and India.

A U.S. manufacturing facility of an innovative biopharmaceutical company that exports drugs to China and India for profit?  What's wrong with this picture? Well, as it turns out, it's what's right — innovation through manufacturing prowess, organic chemistry smarts and green technology. Better. Faster. Cheaper.

Pharma's always bragging about its ever-growing investment in R&D. But when was the last time you heard about investments in domestic manufacturing? Probably never.

And when was the last time you read about enhanced drug safety through good manufacturing processes and cooperation between industry and the FDA? Not recently. That's a shame because they're both important stories.

The president's nomination of Dr. Scott Gottlieb to be the next FDA commissioner likely portends a more holistic view of drug regulation. I served with Scott for two years at the agency. Not only is he a voice for greater regulatory predictability, he's also a silo-buster.

Having previously served as deputy commissioner, he understands the need for greater interdepartmental cooperation. In other words, it's not just about better utilization of expedited review pathways or better use of real-world data, or enhanced post-market surveillance, or more robust off-label communications. It's about making the process work for patients.

But manufacturing is all about process. Modernized pharmaceutical manufacturing isn't only about ensuring and enhancing quality for finished medicines, it's also about making America's pharmaceutical factories globally competitive players in the production of API (like Pfizer's plant in Kalamazoo) and excipients (the ingredients other than the API that are included in the manufacturing process or are contained in a finished pharmaceutical product.)

And the more complicated the drug, the more complicated the active pharmaceuticals and other ingredients. American know-how and dedication to GMPs present a wonderful opportunity for our domestic facilities to thrive. Quality manufacturing is our unique proposition vis-a-vis less expensive operations overseas.

FDA regulation of medicine manufacturing is also a crucial piece of the solution to preventing future drug shortages. According to a 2012 report from the House Committee on Oversight and Government Reform, by hastily ramping up manufacturing enforcement actions, the FDA "effectively shut down 30% of the total manufacturing capacity at four of the country's largest producers of generic injectable medications."

Resolving this problem will require the FDA to work with manufacturers to find practical, science-based solutions to quality-control issues that neither compromise safety nor slow down production. Regulatory discretion is often the better part of valor.

Enforcement of savvy manufacturing quality control is crucial, but an equally important (and often ignored) aspect of the FDA's mission is to advance America's pharmaceutical production acumen by being both regulator of and partner with industry.

That's a winning combination: the best and the brightest from industry and government together with the best production capabilities in the world. The keys to the kingdom are on the table.

Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.

DTC Fair Balance ... for Lawyers!

  • 04.17.2017
  • Peter Pitts
House Judiciary Committee Chairman, Rep. Bob Goodlatte (R/VA.) wants drug side-effect ads that are run by lawyers to include a warning that patients should talk with their doctors before adjusting medication, the Wall Street Journal reports. Along with the American Medical Association and some drug companies, he contends the ads are to blame for patients suffering harm or even dying after dropping treatment.

PDUFA Reauthorization. Discuss.

  • 04.14.2017
  • Peter Pitts
House, Senate Health Committee Leaders Release Discussion Draft of FDA User Fees Reauthorization

WASHINGTON, DC – The leaders of the Senate and House health committees today released a discussion draft of bipartisan legislation reauthorizing the Food and Drug Administration user fee agreements.

The Food and Drug Administration (FDA) Reauthorization Act of 2017 renews FDA's authority to collect user fees from the makers of prescription brand drugs, medical devices, generic drugs and biosimilars, and several vital programs at the FDA. The 2012 prescription drug user fee amendments (PDUFA), medical device user fee amendments (MDUFA), generic drug user fee amendments (GDUFA), and biosimilar user fee amendments (BsUFA) all must be updated and reauthorized by Congress before the current user fee agreements expire on September 30.

If the agreements are not reauthorized before the August work period, the agency will be forced to send layoff notices to more than 5,000 FDA employees. A delay in reauthorizing these agreements would delay the reviews of critical drugs and devices.

“We are fully committed to a timely reauthorization of the agreements and are well on our way,” said Energy and Commerce Committee Chairman Greg Walden (R-OR). “This represents another opportunity to help expedite the review and approval of new cures and treatments for patients, and we must prioritize it. As this process proceeds, I look forward to continued discussions with my colleagues in the House on other member priorities that could strengthen this important legislation.”

"The swift reauthorization of these user fee agreements is critical to making sure FDA has the resources and personnel it needs to ensure timely review and approval of safe and effective medical treatments,” said Energy and Commerce Committee Ranking Member Frank Pallone, Jr. (D-NJ). “These carefully negotiated agreements encourage innovation, improve our regulatory review process, and provide certainty to both patients and industry. I look forward to working with my colleagues to move this legislation through Congress and to the President's desk.”

“If we do not move quickly to reauthorize these agreements, in late July, the FDA will be forced to begin sending layoff notices to more than 5,000 employees to notify them that they may lose their job in 60 days,” said Senate Health, Education, Labor and Pensions Committee Chairman Lamar Alexander (R-TN). “The sooner we reauthorize the agreements, the better – to give patients, reviewers, and companies certainty. In addition to harming patients and families that rely on medical innovation, a delay in reauthorizing the user fees would threaten biomedical industry jobs and America’s global leadership in biomedical innovation.”

“I’m encouraged by our bipartisan work to reauthorize these agreements to advance safe, effective, and innovative medical products,” said Senate Health, Education, Labor and Pensions Committee Ranking Member Patty Murray (D-WA). “Patients and families nationwide are relying on us to ensure FDA can continue its vital work without interruption. I hope that we can continue to put partisanship aside and advance this important legislation.”

The FDA Reauthorization Act of 2017:

Updates and reauthorizes the user fee programs, which, in Fiscal Year 2016, accounted for 70 percent of the brand drug review budget, 36 percent of the medical device review budget, 75 percent of the generic drug review budget, and 29 percent of the biosimilar review budget.

Reflects the recommendations sent by FDA to Congress in January, which were based on over a year of negotiations and discussions with industry, Congress, patients, and other stakeholders.

Implements the four user fee agreements authorized by FDARA, which support the goals of the 21st Century Cures act and advance key bipartisan priorities:

Prescription Drug User Fee Amendments (PDUFA VI): Enhances patient-focused drug development, supports biomarker development and qualification, dedicates staff to assist in the development and review of rare disease drugs, sets clear timelines and improves guidance for drug and device combination products, and evaluates ways to modernize the clinical trial process.

Medical Device User Fee Amendments (MDUFA IV): Enhances the patient voice in the device development process, supports the collection of real world evidence on the safety and effectiveness of devices, and improves the review process for  “de novo” devices—low- to moderate-risk devices that are the first of their kind.

Generic Drug User Fee Amendments (GDUFA II): Improves the fee structure to support small businesses, provides goal dates for all outstanding generic applications, and establishes priority review timelines.

Biosimilar User Fee Amendments (BsUFA II): Continues to build the biosimilars program, and supports guidance for product developers.

CLICK HERE for the text of the discussion draft, HERE for a section-by-section summary of the discussion draft, and HERE for a one-pager. Health committee leaders ask that stakeholders who wish to comment on the contents of this draft please submit to by April 28th.


  • 04.12.2017
  • Peter Pitts
It's time to go beyond FDAMA 114.

Representative Brett Guthrie (R/KY) introduced the Pharmaceutical Information Exchange (PIE) Act of 2017 (H.R.2026). The bill incorporates the multi-stakeholder consensus recommendations developed during a forum sponsored by the Academy of Managed Care Pharmacy. The legislation would allow biopharmaceutical manufacturers to proactively share clinical and economic information with population health decision makers on emerging therapies in advance of Food and Drug Administration (FDA) approval, an area that is significantly restricted by current federal laws and FDA regulations.

Expediting Complex Geneics

  • 04.06.2017
  • Peter Pitts
It's not about speed, it's about predictability.

The way to expedite complex generics is by making the process predictable. Predictability = more development programs = competition = lower prices.

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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