Latest Drugwonks' Blog

Four More Years

  • 11.07.2012

The next four years are going to be a battle over whether or not Uncle Sam continues to morph into Uncle Sam, MD.

Consider:

Uncle Sam as our nation’s largest payor.

Uncle Sam as funder of comparative effectiveness research.

Uncle Sam as “academic” detailer of comparative effectiveness research.

Uncle Sam as determiner of Essential Health Benefits.

Consider the continued disempowered physicians, patients -- and states (which are the true laboratories for healthcare delivery innovation).

Consider a battle royale over whether or not comparative effectiveness should become a third leg for FDA approvals (in addition to safety and efficacy).

Consider whether CMS reimbursement will move to a value-based reimbursement model, setting up the foundation for a US version of Britain's NICE. (Remember Don Berwick?)

Consider Medicare and Medicaid adopting the NHS model of "expanded access" (formerly known as "risk sharing") wherein reimbursement is granted -- but if predefined patient outcomes aren’t achieved, the pharmaceutical company reimburses CMS. Hm.

Consider FDA Commissioner Peggy Hamburg.  Will she stay or will she go? (I predict she will stay.)

I predict that (regardless of who gets elected) IPAB gets revoked on the Hill. IPAB is a slippery slope to broader government price controls, so it should be good riddance to dangerous rubbish – if the President doesn’t veto this first congressional rebuke to ObamaCare.

And what of the legitimacy of Billy Tauzin’s famous ACA “deal?” Will there be legislative action to revoke the Non-Interference Clause (as mentioned by Vice President Biden -- although not by name -- during the Vice Presidential debate)? Unlikely.

Will the President continue to push for 7 years of data exclusivity for biologics (as opposed to the 12 currently allowed under the ACA?) Will he have the chutzpah to claim he is pro-innovation if he does?

Will we have to endure another foolish waste-of-time debate over drug importation?

And did somebody say “Doc Fix?”

Ladies and Gentlemen, fasten your seatbelts and start your engines.

Those who believe that they are exclusively in the right are generally those who achieve something. -- Aldous Huxley

Rather than speculating as to where the FDA may go in a post-PDUFA V world, it's important to focus on where it has already gone. Without much attention or fanfare, the agency has established a new review board within the Center for Drug Evaluation and Research – the CDER Exclusivity Board. Its goal is to help the agency make consistent findings on whether products should be granted periods of marketing exclusivity.

The board began meeting in November 2011. It is comprised of agency employees from the Office of Regulatory Policy, the Office of Medical Policy, the Office of New Drugs, the Office of Pharmaceutical Sciences, the Office of Executive Programs in CDER and the Office of the Chief Counsel.

The focus is on five-year new chemical entity exclusivity, three-year new clinical trial exclusivity, and exclusivity for biological products, according to a brief description posted on the agency’s website.

“The board will not review or make recommendations with respect to all exclusivity determinations in these areas, but will assist the center in resolving certain matters, including issues that arise in the context of specific requests for exclusivity,” the web page states.

The board generally meets once or twice a month. Members review written submissions from sponsors and board members participate in meetings with sponsors regarding exclusivity determinations for their products.

Some exclusivity matters fall outside the board’s purview: The body generally will not make recommendations for 180-day generic drug exclusivity, seven-year orphan drug exclusivity, or six-month pediatric exclusivity. But it will work with other offices and groups within FDA that are responsible for dealing with these exclusivity determinations as needed.

Your PDUFA dollars at work.

 

Matt Herper of Forbes, in his article, Why Presidents Don't Shape The FDA, writes:

Generally speaking, Democrats like Obama want to ensure that medicines are safe and are less concerned with being friendly to the pharmaceutical industry, and Republicans like Romney believe in lowering regulatory barriers and getting medicines to market faster. But the reality is no president makes the FDA his top priority — which means that the agency is often shaped as much by the opposition as by the commander in chief.

In the bigger picture, this is an illustration that when it comes to reshaping federal agencies, Presidents are not as powerful as you might think — and a reminder that just because a politician campaigns for a change does not mean that he or she will be able to execute it.

The important truth to remember is that the FDA is an agency driven be career staff.  Of the 11,000 or so employees, under a dozen are political appointments (including the Commissioner). And all of those Schedule Cs reside within the Office of the Commissioner. That means 100% of employees in every FDA center are career government workers. Put another way – drugs are being reviewed exclusively by career employees.

To refer to the “Obama FDA” or the “Bush FDA” or a future "Romney FDA" is valid only insofar as the presidentially-appointed  Commissioner sets an agenda. And that is if the Commissioner has an agenda and (most importantly) can enlist senior career officials to buy into it.

During my tenure at the FDA, Commissioner Mark McClellan was able to convince the career leadership that the role of the agency was to be regulator and colleague to industry and, most importantly, change-agent. I believe we had many successes because of this agenda – and the public health was well served.

The most important tool any President has to impact the performance of the FDA is in his choice of an FDA Commissioner. And that appointment must be confirmed by the United States Senate.

Herper concludes:

From an FDA perspective here, the lesson may be that it’s best to insulate the agency from politics as much as possible. One way to do that, according to Peter Pitts of the Center for Medicine in the Public Interest, would be to put FDA commissioners on six-year terms, protecting them from political churn.

More to the point, a six-year term would allow a Commissioner to more fully pursue his or her agenda.

Neither President Obama or Governor Romney has demonstrated any interest in a fixed term for the Commissioner.  At least not yet.

While many on the East Coast are thinking about electrical power, a new global study by the IMS Institute for Healthcare Informatics shows that the use of healthcare IT to increase medication adherence could be a key factor in saving some $500 billion in healthcare spending worldwide -- and that a key factor is the power of information.

“Harnessing available information to set priorities, monitor progress and support behavior change among healthcare stakeholders – including policymakers, payers, clinicians, nurses, pharmacists and patients – is a vital first step,” he said.

Aitken said the increasing use of data in healthcare makes this a good time to apply the levers suggested by the study to lower healthcare costs, which are:

  • Increase medicine adherence by addressing patient beliefs and behaviors at the point of prescription and during medicine intake.
  • Ensure timely medicine use that prevents avoidable and costly consequences among patients with highly prevalent diseases that increase in severity if diagnosis and treatment are delayed.
  • Optimize antibiotic use to turn the tide on rising antimicrobial resistance worldwide due to the misuse and overuse of antibiotics.
  • Prevent medication errors throughout the medicine provision pathway, from prescription to administration.
  • Use low-cost and safe generic drugs where available to leverage the under-exploited opportunity in post-patent expiry markets.
  • Manage polypharmacy where the concurrent use of multiple medicines, particularly among the elderly, risks costly complications and adverse events.

“Not all of this is new. Adherence is not new,” he added. What is new, however, is the ability to use data and predictive modeling to find which patients best respond to what type of medication adherence reminders, he said. Some need a visit from a nurse, which is more costly than using a text or a tweet. Predictive modeling can help an organization use resources wisely to get the most adherence from patients.

The study also focuses on two key factors critical to driving improvement across the six levers: multi-stakeholder engagement and … the power of information.

Knowledge is Power.

The study can be found here.

Advise & Consent

  • 11.01.2012

Dear FDA Colleagues:

I would like to make you all aware of an upcoming transition within the Office of the Commissioner.  Effective November 5, 2012, Jeanne Ireland will be leaving her post as head of the Office of Legislation (OL) to assume a new role as Senior Advisor to the Commissioner within the immediate office.  In that new role, Jeanne will manage high priority projects and, in the short term, will continue to manage the legislative/oversight issues surrounding the recent meningitis outbreak and related pharmacy compounding issues.  Michele Mital has graciously agreed to serve as Acting Associate Commissioner for Legislation and will oversee and manage all other aspects of OL.

For the past three years, Jeanne has been a terrific asset to the Agency in her leadership role in OL, and she has numerous legislative successes under her belt, including most recently the sweeping Food Safety Modernization Act and the Food and Drug Administration Safety and Innovation Act, which included the reauthorization of the prescription drug and medical device user fee programs and for the first time created new user fee programs for generic drugs and biosimilars.  I am sorry to lose Jeanne in this important role, but I am thrilled that she will be assisting us in her new capacity.  I therefore would like to congratulate and thank Jeanne for her prior achievements and to welcome her to the immediate office.  I also would like to thank Michele for taking the reins in OL.

Sincerely,

Margaret A. Hamburg, M.D.

Commissioner of Food and Drugs

It was nice being at a meeting of regulatory professionals where "ED" meant something other than the expected.

Specifically, I was at the annual RAPS meeting in Seattle where the theme of the conference was "Elevate" (as in "raise the level of your game) -- and "ED" referred to "enforcement discretion."

FDA judgment was certainly on the minds of panelists (myself included) during the session on mobile apps as medical devices. At present, there are some 17,828 healthcare and fitness apps and 14,558 that can be deemed “medical.” As Vice President Biden said, “It’s a big f**kin’ deal.”

But when is an app a medical device and when is it not? Not surprisingly, it depends.

Does the app replace paper-based data collection (for example from a blood glucose meter), or does it bring to bear the power of an algorithm that takes raw data and turns it into a diagnosis with treatment recommendations?

What is the level of impact the app might have on a patient's condition. Is the app designed to assist in patient self-management?

Is the app an accessory to a regulated medical device?

Many questions, all of which lead us back to the question of enforcement discretion.

Consider the Draft Guidance for Industry and Food and Drug Administration Staff - Mobile Medical Applications  issued on: July 21, 2011 – specifically, footnotes 12 and 13:

(12) … the FDA intends to exercise its discretion to decline to pursue enforcement actions for violations of the FD&C Act and applicable regulations by a manufacturer of a mobile medical app, as specified in this guidance. This does not constitute a change in the requirements of the FD&C Act or any applicable regulations. 

(13) The FDA's review of these products indicates that the majority of these other mobile apps that may meet the definition of a medical device have functionality either to automate common medical knowledge available in the medical literature or to allow individuals to self-manage their disease or condition. Many of these mobile medical apps also automate common clinician's diagnostic and treatment tasks using simple general purpose tools, including spreadsheets, timers, or other general computer applications, by performing logging and tracking. For example, mobile medical apps that: log, track, and graph manually-entered (keyed in) data that lead to reminders or alarms; act as data viewers for patient education; organize, store, and display personal health data, such as lab results, doctor visits, dosages, calories consumed, etc.; or allow for general dose over the counter (OTC) lookups and use drug labeling to provide information that is typically available on a drug label, e.g., acetaminophen dosage for children and adults.

Now consider the “Mobile medical apps Proposed Scope for Oversight” issued by CDRH. It’s a pyramid divided into three parts:

The top of the pyramid includes mobile medical apps that are traditional medical devices or a part or an extension of a traditional medical device. Clearly within the scope of being regulated as medical devices.

The middle section includes patient self- management apps and simple tracking or trending apps not intended for treating/adjusting medication. This is the area, as defined by CDRH, for enforcement discretion

The bottom section are devices that are not deemed “mobile medical apps” and, as such, have no regulatory requirements.

FYI, per SEC. 201(h)  [321] of FD&C Act:

(a) the term "device" means an instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent, or other similar or related article, including any component, part, or accessory, which is

(1)  recognized in the official National Formulary, or the United  States Pharmacopeia, or any supplement to them,

(2)  intended for use in the diagnosis of disease or other conditions, or in the cure, mitigation, treatment, or prevention of disease, in man or other animals, or

(3)  intended to affect the structure or any function of the body of man or other animals, and which does not achieve any of its principal intended purposes through chemical action within or on the body of man or other animals and which is not dependent upon being metabolized for the achievement of any of its principal intended purposes.

A light regulatory hand allows for innovation to flourish. But will CDRH be flexible enough in its approach to disruptive app-based technologies. "Signs point to yes" -- but developers seeking greater predictability are only somewhat assuaged by such a Magic 8 Ball approach to agency policy.

We're in early days.  

Another "ED" variable is the intended use of an app -- something that is the responsibility of the developer, not the FDA. And discretion is the better part of valor. In short, to thine own self be true.

And what about "human factors" such as user/device interface or the environment in which that interface takes place? Bedroom or operating room? What about user-error mitigation?

And then there's the issue of validation testing.

We've come a long way from the "popsicle-stick-or-tongue-depressor" conversation -- and we've got a long way to go.  As Walter O'Malley (the man who moved the Brooklyn Dodgers to Los Angeles) once opined, "The future is just one damned thing after another."

Something to be thankful for.

Roaring 20

  • 10.26.2012

Arithmetic is being able to count up to twenty without taking off your shoes.

-- Mickey Mouse

To help reviewers better understand patient perspectives, FDA will conduct four public meetings per year on different disease areas during the five-year PDUFA cycle. That equals 20 meetings on 20 different disease areas. The information gleaned from these sessions is expected to help reviewers better understand patient needs and risk tolerance and to help spur further research.

(FDA agreed as part of the reauthorization to better incorporate patient opinions about risk tolerance and other drug development issues into its approval decisions.)

But, under the headline of “no good deed goes unpunished,” FDA is working to allay advocates’ fears that the 20 disease areas selected for public meetings will be the agency’s sole focus over the next five years.

FDA has developed a list of 39 candidate topics for the meetings, about one-third of them rare diseases – but, according reportage in the Pink Sheet, “it drew the ire of some patient groups.” Diane Dorman, NORD’s vice president of public policy said that the agency’s list has pitted disease organizations against each other.

But according to the always thoughtful Theresa Mullin (CDER’s Director of Planning and Informatics), the disease manifest “is not is any sort of priority list for FDA. According to Mullin,the proposed diseases were chosen in part because existing measures are inadequate.

“This is not to try to address all the important diseases, just ones where, in fact, we don’t have very good clinical measures, we don’t have good objective measures now, and we need to develop the best measures we can to encourage drug development,” she said.

Mullin mentioned chronic fatigue syndrome as an example because it does not have a definitive set of symptoms and obtaining more patient information would help develop more drugs for the treatment armamentarium.

The FDA is also convening several “consultation meetings” with patient stakeholders to talk about drug development issues and help formulate the direction of the disease-specific public meetings. Topics will include: resolving conflicting opinions among stakeholders, balancing the priorities of different disease areas within FDA’s limited resources, and ensuring groups outside the Washington D.C. area gain equal access to the talks.

Talk about 20/20 vision!

Yali's Volley

  • 10.25.2012

Yali Friedman, Publisher and Chief Editor of the Journal of Commercial Biotechnology, has an interesting and important essay on how advances in personalized medicine may have the unintended consequence of accelerating the call for US price controls – resulting in fewer new life-saving treatments.

Friedman writes:

Personalized medicine—prescription of drugs most likely to benefit and least likely to harm individual or groups of patients—promises welcome positive changes to healthcare. It may, however, also have negative sequelae originating from incompatibilities with the current healthcare delivery system and the need for regulatory and policy changes to accommodate personalized medicine.

His full article, Will personalized medicine be a driver for widespread price controls, can be found here.

But who's counting?

  • 10.24.2012
Biocentury reports that FDA's 2012 NME approvals near 2011 total.

FDA has approved 28 NMEs since Jan. 1, according to agency records, with at least seven PDUFA dates for NMEs between now and year-end. FDA approved 27 NMEs between Jan. 1 and Oct. 31, 2011, and finished the year with 30, the second highest total of NME approvals in the past 10 years. FDA has averaged about 24 NME approvals each year since 2002, with a high of 36 in 2004 and a low of 17 in 2002.

Good article in the Wall Street Journal about the FDA’s efforts to make not-yet-approved medicines available to patients with life threatening conditions.

If you are a subscriber to the WSJ, the article can be found here.

Kudos to the agency – and well deserved.

Last year, nearly 1,200 patients received treatment with experimental drugs through the compassionate-use program for conditions including hepatitis C, cancer and rare diseases like cystic fibrosis. That is up from about 1,000 patients in 2010, the first year the agency compiled data on the program. The FDA says it has been trying to increase participation, including by helping to set up a Web-based seminar that trains doctors how to make use of the program.

The agency allows manufacturers to make their drugs available, but can't require them to do so, and some companies are reluctant to participate before their products have received marketing approval. The FDA says it has been working to win over more companies. It revised its regulations in 2009 to effectively open the program to a greater number of small drug manufacturers. Most companies that provide drugs for the program do so free of charge, but some seek to recoup their expenses, the FDA says. Health insurers generally don't reimburse patients for the cost of the drugs.

"We get calls from family members and there's this sense these are miracle drugs," says Richard Klein, who heads the FDA's Office of Special Health Issues.

In fact, Mr. Klein says, "you can die faster" if there's an adverse reaction to a drug. And even it if does work, it might only add weeks or months to a patient's life, he says.

OSHI doesn’t get a lot of media coverage, nor do they seek it out. But the work they do is crucial to the mission of the FDA – and to the patients they help.

Bravo.

CMPI

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.

Blog Roll

Alliance for Patient Access Alternative Health Practice
AHRP
Better Health
BigGovHealth
Biotech Blog
BrandweekNRX
CA Medicine man
Cafe Pharma
Campaign for Modern Medicines
Carlat Psychiatry Blog
Clinical Psychology and Psychiatry: A Closer Look
Conservative's Forum
Club For Growth
CNEhealth.org
Diabetes Mine
Disruptive Women
Doctors For Patient Care
Dr. Gov
Drug Channels
DTC Perspectives
eDrugSearch
Envisioning 2.0
EyeOnFDA
FDA Law Blog
Fierce Pharma
fightingdiseases.org
Fresh Air Fund
Furious Seasons
Gooznews
Gel Health News
Hands Off My Health
Health Business Blog
Health Care BS
Health Care for All
Healthy Skepticism
Hooked: Ethics, Medicine, and Pharma
Hugh Hewitt
IgniteBlog
In the Pipeline
In Vivo
Instapundit
Internet Drug News
Jaz'd Healthcare
Jaz'd Pharmaceutical Industry
Jim Edwards' NRx
Kaus Files
KevinMD
Laffer Health Care Report
Little Green Footballs
Med Buzz
Media Research Center
Medrants
More than Medicine
National Review
Neuroethics & Law
Newsbusters
Nurses For Reform
Nurses For Reform Blog
Opinion Journal
Orange Book
PAL
Peter Rost
Pharm Aid
Pharma Blog Review
Pharma Blogsphere
Pharma Marketing Blog
Pharmablogger
Pharmacology Corner
Pharmagossip
Pharmamotion
Pharmalot
Pharmaceutical Business Review
Piper Report
Polipundit
Powerline
Prescription for a Cure
Public Plan Facts
Quackwatch
Real Clear Politics
Remedyhealthcare
Shark Report
Shearlings Got Plowed
StateHouseCall.org
Taking Back America
Terra Sigillata
The Cycle
The Catalyst
The Lonely Conservative
TortsProf
Town Hall
Washington Monthly
World of DTC Marketing
WSJ Health Blog