Quotes of the week

10/29/2010 11:13 AM |

“Even when voters emphasize the economy, they generally do so in a way that very much involves ObamaCare. Voters aren't so much angry that their representatives haven't fixed the economy but that they haven't prioritized the economy -- that they passed a $787 billion "stimulus" that merely stimulated the National Debt Clock and then turned their attention to what they cared about most: passing a huge health-care entitlement that a clear majority of Americans opposed.”


  Read More & Comment...

NICE -- but not necessary

10/28/2010 08:35 AM |

Yes Minister.

The British Health Minister, Lord Howe, has affirmed his government’s position that NICE has become “redundant” and would be “moved gradually away from single health technology assessments towards the formulation of quality standards.”

NHS bodies are currently obliged to follow (or at least consider) NICE guidance but the changes will mean its cost-effectiveness reports will in future be purely advisory.

According to his Lordship, “In terms of cost-effectiveness, even though we will rely on NICE’s advise, we will move onto our own value-based pricing system.” He added that the coalition government wants to ensure patients and the NHS are allowed access to the medicines they both want and require.

Lord Howe said: “So that there is no doubt, the current terms of the PPRS (Pharmaceutical Price Regulation Scheme) pricing system will be upheld in full until it comes to an end in 2013,” but after this it will be replaced by the government’s evolving definition of value.

Lord Howe:  “We need a much closer link to the price the NHS pays and the value that a new medicine delivers.”

For more on value-based pricing, see here.

When the new Congress begins its hearings on ways to really reform the US healthcare system, the British decision to denude NICE should be raised relative to many issues – not the least of which is the role and responsibility of PCORI (the Patient-Centered Outcomes Research Institute). 

This isn’t the end of QALY-based cost-effectiveness, nor is it the beginning of the end of CATIE/ALLHAT/STAR-D comparative effectiveness – but it is the end of the beginning of a cost-based care philosophy that places the short-term needs of payers (be they public or private) over the long-term interests of patients.

Outcomes-based clinical effectiveness – now that would be (lower case) nice.

  Read More & Comment...

Location. Location. Location.

10/28/2010 07:59 AM |

Food & Beverage Industry Announces Front-of-Pack Nutrition Labeling Initiative to Inform Consumers and Combat Obesity

(Washington, D.C.)  America’s leading food and beverage manufacturers and retailers joined forces today in the fight against obesity and announced their commitment to develop a new front-of-package nutrition labeling system.  The unprecedented consumer initiative will make it easier for busy consumers to make informed choices when they shop.

This program will add important nutrition information on calories and other nutrients to limit to the front of the packages of many of the country’s most popular food and beverage products.  To appeal to busy consumers, the information will be presented in a fact-based, simple and easy-to-use format.  In the coming months, the Grocery Manufacturers Association (GMA) and the Food Marketing Institute (FMI) will finalize the details of the initiative, including the technical and design elements.  In addition, details will be finalized on how to provide consumers with information on nutrients needed to build a “nutrient-dense” diet and on “shortfall nutrients” that are under-consumed in the diets of most Americans.  GMA and FMI will continue to consult stakeholders on these and other details in the coming weeks.

Consumers will begin to see the new label in the marketplace early next year. 

To build consumer awareness and promote use of the new label, America’s food and beverage manufacturers and retailers have agreed to support the change to their product labels with a $50 million consumer education campaign.  The campaign, to be launched in 2011, will be aimed at parents who are primary household shoppers. 

  Read More & Comment...

IRS: Breast Feeding Doesn't Count As Healthcare

10/27/2010 11:47 AM |

Remember how Obamacare proponents said the IRS would not meddle in medical decisions...

Acne Cream? Tax-Sheltered. Breast Pump? No.
By DAVID KOCIENIEWSKI
Published: October 26, 2010


Denture wearers will get a tax break on the cost of adhesives to keep their false teeth in place. So will acne sufferers who buy pimple creams
A breast pump and various accessories can run about $500 to $1,000 for most mothers a year.
People whose children have severe allergies might even be allowed the break for replacing grass with artificial turf since it could be considered a medical expense.
But nursing mothers will not be allowed to use their tax-sheltered health care accounts to pay for breast pumps and other supplies.
That is because the Internal Revenue Service has ruled that breast-feeding does not have enough health benefits to quality as a form of medical care.

You can't make this stuff up...


www.nytimes.com/2010/10/27/business/27breast.html


  Read More & Comment...

Risk Communications: You make the call

10/27/2010 09:20 AM |

“Anyone who isn’t confused really doesn’t understand the situation.”

--  Edward R. Murrow

On November 2^nd, from 12:15-1pm the FDA is holding a teleconference to discuss the agency’s risk communications research agenda.

45 minutes? 

The teleconference will begin with a presentation by the FDA on its research agenda and its role in supporting the agency’s strategic plan for risk communications by “strengthening the science of risk communications.”

This will be followed by a panel of FDA’s social scientists and a Q&A.

FDA and social science?  Maybe 45 minutes begins to make more sense.

Be there or be square.

“The speed of communications is wondrous to behold.  It is also true that speed can multiply the distribution of information that we know to be untrue.”

 -- Edward R. Murrow

  Read More & Comment...

Showing Pharma the puerto

10/26/2010 10:24 AM |

Talk about biting the hand that feeds you.  Puerto Rico, long home to pharmaceutical manufacturing facilities, has just passed a new law (Law 154) that imposes “special taxes” on non-resident companies.  That means all of the “Big Pharma” firms doing business on the island.

According to a 2006 survey, the biopharma sector supports over 94,000 jobs in Puerto Rico and conducted 425 clinical trials on the island commonwealth.

 It should also be noted that Law 154 was enacted minus any public input or comment.

 Adios.

  Read More & Comment...

Grassley is Criminalizing the Doctor-Patient Relationship

10/25/2010 03:27 PM |

Now it appears that Senator Chuck Grassley believes that he has the right to claim individual doctors are guilty of Medicaid fraud because they prescribe more medications than other doctors.  And when Grassley targets someone or a company,  it is hunted down by the media and the Feds. 

From Business Week:

"A key U.S. Senator is asking federal officials to investigate after reviewing data that shows doctors across the country prescribing alarmingly high numbers of powerful mental health drugs paid for by Medicare and Medicaid..

Grassley did not name the doctor, but state records obtained by The AP show Dr. Fernando Mendez-Villamil wrote an average of 153 prescriptions a day for 18 months ending in March 2009. That's nearly twice the number of the second highest prescriber in Florida, who wrote a little more than 53,000 prescriptions, according to a list compiled by state officials.

The doctor's attorney, Robert Pelier, said his client tried to contact Grassley to explain the data but got no response.

"Dr. Mendez-Villamil is a specialist that has treated the most afflicted and poor in the area" and has never acted improperly, Pelier said. He filed a lawsuit against the state alleging authorities tried to terminate him from being a Medicaid provider "without cause."

Federal officials said they stopped reimbursing Villamil after Grassley inquired about the doctor to HHS in December."

Nice work.  And welcome to another aspect of Obamacare:  Targeting and terminating payment of doctors who are fingered by the feds for providing "too much" treatment..  Having Grassley serve as a bounty hunter is optional.

I thought it would be nice to hear Dr. Mendez-Villami's side of the story since most media accounts gloss over it.   I found this letter he wrote to HHS. You tell me if this sounds like a crook or just one more honest and hardworking doctor who will ultimately bag Medicaid because of the reimbursement cuts and threat of retaliation.   

PS.. Here's something CMS and Grassley overlooked:  No one gets rich writing prescriptions..  Where is the fraud.  Is it a criminal activity to prescribe more than what a Senator regards as "normal?"

Why isn't anyone raising hell about this abuse of power?

Dear Senator Grassley,

I am writing to add a human face -- along with some background information and context -- to whatever image you may have of the doctor in Miami who wrote all those prescriptions for mental health drugs.

I am that doctor. My name is Fernando Mendez-Villamil and I have practiced psychiatry in South Florida since 1998. I received my medical degree from the Universidad Central del Caribe in Puerto Rico and performed my residency in psychiatry in Jackson Memorial Hospital, the public medical center serving metropolitan Miami.

I can only imagine what you thought when you were given information by the Florida Agency for Health Care Administration showing that one doctor wrote close to 97,000 prescriptions for mental health drugs over 21 months. This came to 153 prescriptions a day, 7 days a week for that entire period; twice as many prescriptions in that period as the number 2 Medicaid prescriber in the state.

These numbers may have struck you as an indication something was wrong. Frankly, since I have never stopped to do such a calculation, it seemed a little peculiar to me too. I never felt I was treating an extraordinary number of people or writing an exceptional number of prescriptions.

At the same time, I never thought I would be faulted for working hard or for being very organized and efficient. But now - with this publicity and the questions it has engendered - I have looked at the matter carefully and would like to provide you and the public with some context within which to consider these facts.

First, I want you to know that I take very good care of my patients. My top priority is to improve their conditions.  In more then ten years of practice I have worked with thousands of afflicted individuals. Their conditions have ranged between mild emotional upset to extreme debilitating psychosis. 

By every indication, my patients and their families are pleased with my care.

I may be an oddity as a physician because I do not play golf, I do not have a boat and I seldom leave my practice for extended vacations and am involved in my practice on a daily basis. That is not to solicit sympathy or to appear “noble.” I am simply committed to my patients, profession and enjoy what I do and do not seek distractions.

As to my patients, they share some characteristics.

* Many receive a combination of medications with each drug addressing specific aspects of their condition identified by the use of objective clinical findings along with subjective complaints. Therefore, when I see such patients I must write or renew an average of three or four prescriptions.

* Some patients come to me after having been under the care of other medical providers who prescribed some of these medications. I must evaluate whether those drugs are effective in achieving their treatment objectives, whether some medications should be discontinued and whether additional medications are required. This can be a very difficult process when I am dealing with a mentally ill patient who may not be able to assist fully in the examination process.  I would like you to know that these efforts are in combination with initial and ongoing psychiatric assessments of my patients.

* Ongoing treatment plans with the use of atypical antipsychotic medication is necessary in order to maintain patients stable and effectively responsive to their mental health needs.  I firmly believe that a person’s health should not be placed in danger by erratic or ineffective treatment.

* As a result of my assessments and treatment plans I have initiated; most of my patients are very stable and experience very few changes in behavior or in their medical condition over time.  Of great importance is the fact that hospitalization rates of my patient population are less than 1%.  Hospitalization can be costly and otherwise interrupt the mental health and wellbeing of the patients. While I seek to be alert of subtle variations in my patient’s responsiveness to the treatment or behavior that might indicate a need to alter their regimen,  you may appreciate that my patients are not always able to reliably explain how a drug is affecting them. Chronic mental health patients require complex combinations of medications based on their past treatment, physiological and psychological needs.

As to the specific prescriptions I have written, I have asked the Florida Agency for Healthcare Administration to provide me the information that was presumably provided to you and consequently to media outlets. The department has responded to my request by saying they have not received any recent requests for information about me or my practice and could therefore not furnish me with such information.

The information received from this agency advised that I am not under any sort of investigation. It also appears that the circumstances surrounding the release of the information to you supports this also. This was good to hear because I would hate to think that the government would discourage hard work and efficiency in the delivery of medical care.

In a similar vein, I was very surprised to read in the Miami Herald that Medicare had supposedly stopped payment for my services; and I am very pleased to confirm with Medicare officials that this was NOT true.

As to the numbers of prescriptions that have been cited, let us look at them on a hypothetical basis and see what they show.

Assuming the numbers are correct, I wrote 97,000 prescriptions in 21 months.

AHCA does not indicate how many patients were involved. However, again assuming those numbers are correct I believe the following would outline the pertinent facts in this matter.

Spread over 21 months, on an average six day, sixty to sixty-three hour work week that comes to approximately 4619 prescriptions per month. It is also not uncommon for me to actually work beyond these hours if there patients that need to be seen; as my practice is mainly a walk in, first come first served office.

At my average work schedule of 22-24 days per month, that means I saw between 47-55 patients a day and wrote an average of 210 prescriptions.  That averages to 2-3 prescriptions per patient per appointment.  Please remember that most of my patients are seen every other month; are given refills and my services are focused on the ongoing administration, effectiveness and responsiveness of the medication treatment plan I have prescribed for these patients.  Time wise, I average 10-15 minutes per patient. 

Generally, I work an average workday of 11-12 hours.  Not counting one hour in the aggregate for lunch and other non patient related activities, I see roughly four patients per hour.

Given the stable nature of my patients –these efforts are reasonable and well within the norms of modern psychiatric practice and in treating my patient population. This is especially true given that mine is a psychiatric practice that rarely involves time-consuming psychotherapy, physical contact or examination of the full range of vital signs.  Please keep in mind that the interaction I have with my patients is a combination of patient assessment, monitoring and evaluation of a continuing course of treatment as to the effectiveness of the patient’s medication regimen and/or treatment. 

Senator, I certainly appreciate your efforts to monitor matters such as this. Our country’s health care system is vast and complicated and seemingly includes much inefficiency. I hope you continue your good work with vigilance and determination.

However, in my case I assure you there is absolutely no cause for concern or alarm.

And I probably COULD use a nice vacation.

Respectfully,

Fernando Mendez-Villamil, M.D.

EMAIL: mendezvillamildr@gmail.com

 

cc: The Honorable Kathleen Sebelius

 Secretary, U.S. Department of Health and Human Services

 
Charlene Frizzera
Acting Administrator, Centers for Medicare and Medicaid Services
U.S. Department of Health and Human Services

The Honorable Thomas W. Arnold
Secretary, Agency for Health Care Administration
State of Florida

  Read More & Comment...

Of “Time Outs” and the “Think About It Chair”

10/25/2010 09:22 AM |

And speaking of PDUFA reauthorization – wait, time out.

FDA has proposed a new application review model that would allow it to call a time-out, which could be used to resolve mid-cycle issues.

The idea seems to be an admission by the agency that the six and ten month review times for priority and standard applications, respectively, are no longer sufficient to actually complete a review.

So, it’s not really an issue of “time out” as it is “time on” – as in “time added” at the agency’s discretion.

Minutes of the Sept. 27 industry and FDA Prescription Drug User Fee Act reauthorization negotiation revealed the agency proposed the time-out solution in an effort to create more application review time that will, among other things, allow more feedback to sponsors.

The proposal also could be used to address industry requests for a formal mid-cycle meeting to gain agency feedback on an application’s direction.

But will more feedback result in greater predictability? The length of the review clock stoppage would be fixed, but was not specified in the minutes of the 9/27 meeting.

Industry representatives called for a mid-cycle review in a previous PDUFA negotiating session, but FDA said comprehensive feedback likely was not possible at the midpoint of an application review because its process is not complete. Hm.

An agency willingness to add more meetings to the process is a change from previous discussions with industry, where the agency argued allowing for more meetings could lead to more sponsor questions and create more work for reviewers.

But FDA maintains its previous position that it cannot provide additional application support with the resources it has -- and so its offer for more sponsor-requested meetings means the review time must be extended. Hm.

  Read More & Comment...

Acronyms and Unintended Consequences

10/25/2010 09:04 AM |

REMS, FDA, FDAAA, FTC, ANDA ...

The Federal Trade Commission is looking into whether companies are using REMS to thwart generic competition.

Markus Meier, assistant director of the Health Care Division in FTC’s Bureau of Competition, said he had seen two ways a company may use REMS to block generics: preventing generic companies from getting samples of the drug for bioequivalence testing, or making it difficult for a generic to participate in its REMS program.

“Some systems are so restricted that there is nowhere to buy the product except to buy it from the branded company,” he said. If the branded company refuses to sell it then the generic cannot do bioequivalence testing and therefore cannot file an ANDA and “potentially there will never be generic competition for the product.”

Hm.  “Spirit” of FDAAA notwithstanding, can a company be “forced” to sell its product to a competitor?  Interesting IP question.

Meier said FTC has also seen some instances in which the generic company got sufficient samples and filed an ANDA and then had to come within the branded company’s REMS to start marketing their product. At that point “the branded company tries to impose onerous requirements for participation in the program so that it makes it difficult for the generic to actually qualify,” he stated.

Hm.  But don’t all REMS have to be approved by the FDA?  Minus more directive practices from the agency, well, if you’re not part of the solution …

 “I’m not saying that’s been proven,” Meier added. “I’m not saying we’ve made that accusation of anybody but we’re looking at it. And one of the reasons we’re looking at is because the statute (FDAAA) expressly prohibits participants in these programs from using REMS to erect barriers to competition.”

As for dealing with potential REMS abuse, Meier said FDA does not believe it has any mechanism to enforce the FDAAA provision prohibiting a company from using a REMS to block or delay approval of an ANDA.  But “onerously prescriptive” is certainly in the eyes of the beholder.  Is a REMS that’s tough for the proper public health reasons somehow wrong if it restricts entry of a generic and/or follow-on product? 

Certainly not.   Read More & Comment...

The Same Game

10/22/2010 09:21 AM |

Will FDA tighten standards for generic drugs?  According to CDER Director, Dr. Janet Woodcock – the answer is “yes.”  Good call.

At a meeting of the Generic Pharmaceutical Association in Bethesda, Maryland, Janet said the agency is discussing tightening the limits "so there is less variability.”

Less variability equals better predictability.

Dr. Woodcock commented that patients have complained about generic anti- seizure medications not working as well as brand-name counterparts.

This strikes a very personal note for me.  One of my sons has Juvenile Myoclonic Epilepsy.  His condition is wonderfully controlled via his meds – and I’d like it to stay that way.

Woodcock said that industry executives have approached her with concerns that some generic drugs have spurred quality concerns that went unnoticed in the approval process because clinical testing includes too few patients.

“They say, ‘I know there are products out there that aren’t equivalent,’ and typically they’re manufacturing folks,” Woodcock said in her speech. “I’ve heard it enough times from enough people to believe that there are a few products that aren’t meeting quality standards.”

Woodcock said she didn’t know when the agency would come to any conclusions about generic standards of equivalence. The standards assure the generic is absorbed at the same rate and extent as the brand-name version.

The absorption problems aren’t necessarily harmful, Woodcock said. FDA permits generic drugs to absorb at a 25 percent different rate and extent than the originals they copy.

In April, a group of outside FDA advisers, the Pharmaceutical Science and Clinical Pharmacology Advisory Committee, voted 11-2 that the agency’s equivalence requirements aren’ sufficient for certain medicines. They didn’t offer an alternative, and suggested the FDA list “critical dose drugs,” or drugs where a small difference in concentration can change patients’ reaction, that may need new standards.

The Pharmaceutical Science and Clinical Pharmacology Advisory Committee voted unanimously, with one abstention, that critical dose drugs do constitute a distinct group and voted unanimously that FDA should develop a formal list of those drugs - although the terminology of "narrow therapeutic index" may be more appropriate. And in an 11-2 vote, the committee concluded that current bioequivalence standards are not sufficient for drugs in the narrow therapeutic index group.

Critical dose drugs have a narrow therapeutic index, meaning that "small changes in blood concentration have the potential to result in serious therapeutic failures and/or serious adverse drug reactions." FDA is consulting the committee on the need to establish separate bioequivalence criteria for these drugs given continuing debate about whether critical-dose drugs require special consideration, the agency explained.

Currently, the "sameness" of a brand product and a generic version is evaluated based on two-treatment crossover study to prove bioequivalence, the aim being to show that the 90 percent confidence intervals of the geometric mean test/reference ratios for both maximum plasma concentration and the area under the plasma concentration-time curve fall within a range of 80 percent to 125 percent.

It’s good news that the FDA is taking a strong stance in favor of the public health – because there will be many who seek to undermine this important initiative.

  Read More & Comment...

En Attendant Gudiance

10/21/2010 08:59 AM |

For those of you waiting for FDA to reveal the magical regulations that will unleash the power of healthcare social media – don’t hold your breath.

According to DDMAC Director Thomas Abrams, some social media guidance might be released by the end the year and will address issues related to Internet drug promotion --not how to use specific social media tools like Facebook, YouTube and Twitter

“What we’re going to do is address questions like how to respond to unsolicited requests for information, what’s the corporate responsibility for correcting and monitoring third party sites for misinformation, what’s the corporate accountability for having people post promotional videos and things like that,” Abrams said during a Food and Drug Law Institute conference on enforcement.

And things like that.

 

In other words, and as predicted, the low hanging fruit.

 

Since last November’s Part 15 hearing, DDMAC has communicated that efficacy claims must be accompanied by risk information and cast doubt on the concept of a “One-Click Rule.”  So much for regulations setting pharma free.

 

“In an instant all will vanish and we'll be alone once more, in the midst of nothingness!"

                        -- Waiting for Godot

 

For more on why industry shouldn’t wait for regulatory holy writ, see here.

  Read More & Comment...

Bach Festival

10/20/2010 07:49 PM |

My friend Peter Bach has been one of the busiest and arguably most thoughtful health care policy experts in recent months.  (Then again, as  Red Sox fan, Peter has lots of time on his hands this fall!)   As he knows, I don't always agree with his conclusions but they are always built on scientific research and always thoughtful.  He is also a physician who puts his patients first.   His editorial on the Women's Health Initiative study demonstrating a slight increase in risk of death from cancer in women ages 50-70 who took PremPro is an example of this approach.  He calls for research to investigate the relationship between the likely mechanism and pathways that may be implicated in this reported risk and outcomes in a prospective study.   No one can argue with that.  Or should they.

Peter has written a lot on comparative effectiveness research and has some suggestions about how to encourage it and conduct it in ways that promote better care and less cost.    As the Economix blog put it:  "using the research not to decide whether to cover a procedure but instead to decide how much to pay for it. If there isn’t research showing that a more expensive treatment is more effective than a cheaper treatment, then the reimbursement rate for the cheaper treatment applies to both."  Of course if a more expensive treatment is more effective than it should be paid for. 

I don't trust the motives of many who make up the CER community and believe that CER research contributes nothing to the goal of improving clinical decisionmaking.  Uwe Reinhardt's fippant assertion that differences in clinical pratice can't be controlled for is completely wrong and self serving since Reinhardt endorses using average outcomes of average populations with minimal risk adjustment to decide who gets what.  For Reinhardt's smug analysis:  economix.blogs.nytimes.com/2010/10/15/basing-pay-for-performance-on-outcomes/


  I believe Peter is the exception.   The links to his articles and those written about his work are below.   He offers a sensible starting point for developing an alternative to the CER slush fund.


jama.ama-assn.org/cgi/content/short/304/15/1719

www.theledger.com/article/20101020/ZNYT01/10203007

economix.blogs.nytimes.com/tag/us-health-care-costs/

content.healthaffairs.org/cgi/content/abstract/29/10/1796
  Read More & Comment...

Risk Management Management

10/20/2010 10:09 AM |

Day 2 of the Third Annual Risk Management and Drug Safety Summit focused on the future from a policy perspective -- and why.

 

A few points to share and consider:

 

After the 65 presentations made at the FDA's public meeting and over 2000 docket submissions, it's becoming increasingly clear that not only is the agency's REMS initiative confusing to industry and stakeholders -- but that the problem is, in many ways, one of the FDA's own making.

 

Not that the there's anything wrong with the REMS concept, to contrary, the recent Avandia decision (and, notably, Tysabri) shows the value of a program that provides avenues for both approving and maintaining products with high risk profiles on the market. The problem is that the excesses of the post-Vioxx inquisition have inured many -- specifically practicing physicians -- to any kind of safety warnings.  

 

When important safety warnings become ambient noise, we're facing a dangerous public health situation.

 

Consider the rash of black box warnings proliferated by the agency since the recall of Vioxx. It's the Precautionary Principle come home to roost. Despite the best intentions, early safety signal communications has resulted in a rash of negative unintended consequences: tabloid media reporting, patients scared into non-compliance, labels being increasingly "lawyered-up," and ever-more defensive medical practices that add costs to our already over-burdened healthcare system and birth ever-more (and more obnoxious) 1-800 ambulance-chaser ads. At a certain point, prescribers just stop listening. And that's not good.

 

Someone raised the issue of REMS and federal preemption. The laughter took a few minutes to subside. Bad decisions have negative unintended consequences. The implications of Wyeth v. Levine will be with us for years to come.

 

A related issue is that of product knowledge. What is the real level of understanding physicians have about the products they prescribe -- especially since they are spending less and less time with pharmacy field staff (often by politically-correct dictate).

 

Isn't it time that sales reps begin to "detail the label" to their physician audience? That would be a real public health service -- and also has positive marketing potential. A solid double play.

 

And perhaps it's time for the FDA to measure how the New Labeling Rule (not so new anymore) has impacted prescriber understanding. Such a study would be a whole lot more useful to protecting and promoting the public health than yet another study on the viewing public's understanding of fair balance and adequate provision. (And maybe its time for industry to step up to the plate and rewrite their existing labels in the new format.

 

Relative to PDUFA V reauthorization, it's going to be a tough slog for the FDA to ask for new things when it still has much to deliver on from PDUF IV.  And that certainly includes many risk management issues, REMS included.

 

This becomes even more interesting when you consider the likelihood of different members of Congress wielding the gavel on some committees pretty crucial to the reauthorization process. One issue raised, relative to reauthorization, is the need for the FDA to clarify the when, where and how of the OSE in the review process.

 

The conference ended on a positive and thoughtful note. Rather than a laser beam focus on REMS, perhaps what is required (from both regulator and regulated) is a broader focus on the appropriate spirit of risk management -- of which REMS is only a piece.

 

Perspective.  It's a wonderful thing.

  Read More & Comment...

Let 1000 inititives bloom

10/19/2010 07:03 AM |

CDER Director, Dr. Janet Woodcock gave the opening keynote at yesterday’s third annual Risk Management and Drug Safety Summit.  And it was an address to reckon with.

Some selected comments:

“Safety issues aren’t headlines – they’re biology; a scientific puzzle.”

“The CDER mission is to promote and protect the public health by assuring that safe and effective drugs are available to Americans.”

“Safe does not mean risk-free. Effective does not mean equally for all. We do not have a legislative mandate to consider pricing issues and I doubt we ever will.”

Her four-part definition of “safety”: (1) risks are managed, (2) quality is assured, (3) health fraud is pursued, and (4) advertising is appropriate.

She announced that in “the Fall,” FDA would release several FDAAA-related guidance documents:

–      Guidance that should reduce burdens on the healthcare system of “Medication Guide-only” REMS, while preserving use of Medication Guides to present important information to patients as part of patent labeling

–      Guidance on safety related labeling changes

–      Guidance on Postmarketing Studies and Clinical Trials

Relative to advancing the science of drug safety Janet discussed:

• In silico modeling

• Systems biology

• Animal models

• In-vitro models

• Benefit-risk assessment

She also announced that the agency was creating a new group within CDER’s existing biostatistician office to focus on safety.

She discussed failure modes and effects analysis (FMEA).  Some in the audience whispered that, hopefully, FMEA would do a better job than FEMA.

Janet also shared that morale within CDER “despite what you may read in the press,” is high and has improved since more staff has been hired to more fairly address the workload. 

She addressed the FDA’s participation in the Observational Medical Outcomes Partnership, and many more issues -- all which can all be found in her PowerPoint here. 

And it’s definitely worth a look to any and all interested and/or concerned about the future of REMS specifically and risk management more generally.

Next up was Sir Alasdair, who shared the MHRA’s “pharmacovigilance toolkit. (Note, this toolkit can be found as part of his PowerPoint presentation here.)  It’s elegance and simplicity is in stark contrast to the FDA’s approach, which seems to be “let 1000 initiatives bloom.” 

He made an interesting observation that, in the 1950s, thalidomide was drug safety’s first historical moment and that Vioxx was it’s “second coming.”

He spoke to the Avandia issue by stating that EMA and FDA had reached the same conclusion by reviewing the same data simultaneously.  However, since the regulatory tools of MHRA (as part of EMA) and FDA are different, the results were different.  To wit, while Avandia remains on the market in the US with a REMS, in the EU the drugs license has been suspended.

Sir Alasdair also adroitly addressed the need to move from data management to risk management – a finesse that many in the audience also viewed in contrast to the current FDA approach.

Specifically, he discussed the need to move from reactive to proactive methodology and harness more robust sources of data, moving “up the evidence hierarchy.  And that “regulatory actions should be linked with measurable public health benefit.”

 

It was an information-packed presentation that is worth careful examination by those interested in how the EU is approaching various risk management issues.

The highlight of the morning was a brief, heated (but friendly) Q&A debate Drs Woodcock and Breckenridge on the topic of HbA1c as a legitimate biomarker for Avandia. (Sir Alasdair’s point was that, if Avandia was being reviewed for approval today, HbA1c would not be an acceptable marker.) It was a collegial exchange that should only slightly impact the Special Relationship.

A video of this point/counterpoint will soon be available on www.drugwonks.com.

Juergen Schmider (Corporate Safety Officer and Vice President of Global Pharmacovigilance & Epidemiology at Cephalon) was up next and made some very interesting points about, among other things, off-label promotion – about which he commented, “It used to be a gentleman’s offense, but no longer.

Indeed.

Herr Schmider’s presentation can be found here.

There were a series of other presentations, all of which can be found at www.fdanews.com/RMDSSpresentations.

The day ended on a hopeful and thoughtful note with the following comment from Gary Appio (US Safety Director, Novartis): “We need to advance towards thinking not about risk management, but benefit/risk management.

Amen.

  Read More & Comment...

Today I am a REMS plan

10/18/2010 07:52 PM |

Today I chaired the third annual Risk Management and Drug Safety Summit where I was joined by (among others) CDER Director, Dr. Janet Woodcock and MHRA Chairman, Sir Alasdair Breckenridge.

It was a risk management bar mitzvah in the sense that it felt like REMS was finally coming-of-age – although not yet entirely mature.

Here’s how I kicked off the conference and introduced Janet:

It’s been quite a year on the REMS watch. As Walter O’Malley – the man who moved the Brooklyn Dodgers to Los Angeles once commented, “The future is just one damn thing after another.”

During the course of 2010 there have been both bouquets and brickbats.

Last month the Infectious Diseases Society of America suggested that overuse of antibiotics could be controlled via a REMS-like approach – or even an actual REMS.  If it takes a REMS to drive the safe and appropriate use of antibiotics, then so be it.  Burdensome on docs, yes – but you know the drill.

In July -- Janet Woodcock said “We don't have the kind of standardization and consistency of REMS programs that would be ideal."  Then she announced that the FDA would begin to develop a standard REMS system. Perhaps this will even include a Periodic Table of the Elements to Assure Safe Use.

In June -- John Jenkins, director of the Office of New Drugs -- and the best dressed man at the FDA – admitted that having to develop and then assess the impact of medication guides as part of the REMS program is a burdensome administrative task for the agency.

He said, "We are looking to try and be creative in how we interpret that part of the statute, so stay tuned to see if we're able to find some creative ways around this," he said.

In the meantime, said Dr. Jenkins, "until we work through this further, there are a lot of medication guide-only REMS - a lot of burden on us, a lot of burden on you - that we'd like to try to get out of."

As of June 3^rd, FDA had listed 123 REMS on its website. Eighty-four consist only of a MedGuide, while another 25 involve a medguide and communication plan. The other 14 REMS require sponsors to adopt elements to ensure safe use. Five of those also involve a MedGuide; three also have a communication plan; and the other six also require both a MedGuide and a communication plan.

If FDA does not find a solution to the current MedGuide Malaise, the next reauthorization of the Prescription Drug User Fee Act in 2012 offers an opportunity for legislative change. Industry and other stakeholders already have cited REMS as an area for focus during PDUFA V.

In May -- FDA began designing a five-item grid as a management tool to explain its risk-benefit decisions in a new more concise format.

The grid has five basic factors that need to be addressed. The top two are the seriousness of the condition addressed and the need for a new treatment of the condition. Then comes the traditional heart of the NDA package: analyses of clinical data on the benefits of the drug and the risks associated with its use.

Significantly, the fifth fundamental factor is explicitly the level of risk management associated with the product. FDA is going to take it into consideration in every decision; and sponsors who ignore or underplay the identification of who should use the product and who might use it will have a gap in their filings.

The grid proposal does not call for a fixed mathematical formula behind each approval. Net/Net, the agency has not tried to reduce the role of judgment in approval decisions.

Judgment?  You mean FDA decisions aren’t black and white?  Egad! Someone had better tell Congress.

In the words of John Jenkins, disagreement "happens a lot in the decisions that we have to make. Very few of the decisions that we make on drugs are easy. Very few of the drugs we see have a dramatic overwhelming benefit with relatively no risk. We see that most drugs have marginal to moderate benefits on a population basis and they have general safety but they have the risks of serious toxicities at some low levels." In other words, every decision is "very complex."

Really?

Key take-away is that the FDA is officially moving risk management into the list of key factors affecting new products. And, for better or worse, "judgment" is in the eye of the beholder.

In April -- Biogen Idec announced that it is developing a test that can tell patients their odds of getting PML from Tysabri.

The screening tool could be marketed as early as 2011 if clinical trials involving 9,000 people show a low rate of false findings.

With a false-negative rate of 2 percent, patients would lower their risk of getting the brain disorder PML to 1 in 25,000 for the first three years of their Tysabri therapy. That’s 21^st century risk management.  REMS par excellence.

In March -- Josh Sharfstein suggested, during a House Energy and Commerce Health Subcommittee hearing, that FDA could use more authority to bring negotiations over a drug's Risk Evaluation and Mitigation Strategy to a swifter conclusion.

Josh said, "It's very important for us to work with companies to come up with something that works. There's no question there's a lot we learn from the interchange with companies, but it sometimes can take a long time to come to agreement. Well – that’s a bit of the pot calling the kettle black – but at least it’s an acknowledgement of the problem.

And, of course, there was Avandia.

Let me introduce our first keynote speaker, CDER Director, Dr. Janet Woodcock, by reminding you of what she told this conference last year. Janet said that, “Safety means doing the right things for patients. FDA must consider post-approval issues as part of a drug’s lifecycle.”

Importantly, Janet understands that there’s a real difference between “headlines and help.”  In other words, REMS and other safety mechanisms can be viewed as either “headlines” about “unsafe” drugs or in a more appropriate context of “safe use.”

According to Janet, “FDA does not control the health care system, so our improving the use of marketed drugs, to a great extent, is going to involve influence rather than control.”

“Influence rather than control” is a savvy and sophisticated concept -- one that many of our elected members of Congress could learn from, and one in which REMS plays an important role.

I believe we can also hear the voice of Janet Woodcock in the white paper the agency released a few weeks ago on “Advancing Regulatory Science for the Public Health.

“There is no single discovery — no magic bullet — to address our unique set of modern scientific regulatory challenges. But one thing is clear: if we are to solve the most pressing public health problems we face today, we need new approaches, new collaborations and new ways to take advantage of 21st century technologies. And we need them now.

Ladies and Gentlemen, I am pleased to introduce Dr. Janet Woodcock.

  Read More & Comment...

Chile and The Least Costly Alternative

10/18/2010 10:32 AM |

How would comparative effectiveness be used to justify the Least Costly Alternative in the case of the Chilean miners.

It cost $33 million to rescue 33 Chilean miners.  Each made $12000 a year.   If we use the QALY approach and assume $50000 per QALY it was obviously a waste of Chile's time and money to undertake the successful operation.  Couldn't that money be better spent on disease management programs?  There would be money left over to pay the families of the miners after the mine had been sealed up.  Too bad that Health Dialog didnt have a shared decision making tool to discourage the miners from asking for such an expensive, invasive and untested procedure.   At least Chile should have waited to let AHRQ conduct a CER review before deciding to pay for the rescue. 


  Read More & Comment...

Exchange Rates

10/18/2010 07:07 AM |

When it comes to mandated health insurance exchanges, state officials must strive to ensure that they don’t crowd out free market mechanisms.  Preserving a vibrant private insurance market will maximize choice and enable people to find the insurance plan that best fits their needs.

Healthcare coverage isn’t a one-size-fits-all proposition. 

When President Obama said that people who are happy with their insurance “can keep it,” we should keep him to his word.  Policymakers and private healthcare stakeholders need to work together so that state exchanges don’t become the only way to get health insurance.

Choice is crucial.

See here for a more detailed examination of the slippery slope towards a single payer system.

  Read More & Comment...

What Chile Can Teach Us About Obamacare

10/17/2010 09:27 PM |

  Read More & Comment...

What does Pharma want?

10/15/2010 08:04 AM |

During a past episode of Mad Men, the creative team at Sterling/Cooper is hard at work ideating on a “women’s product” campaign when someone asks, “What do women want?”

Strolling by, Roger Sterling quips, “Who cares!”

Well, when it comes to social media, what does pharma want -- and who cares?

Many will say “regulation from the FDA -- in fact, a great many.  But is that really what pharma wants?

Yesterday I participated in a small roundtable (sponsored by AstraZeneca) on “Examining the Roles of the FDA and the Pharmaceutical Industry in Social Media.” 

(Full disclosure: I ate two small eggplant and tomato tea sandwiches and drank 2.5 cups of organic coffee. I did not offer to reimburse AZ for the “gift.”)

What does pharma want? One of the opening comments was that pharma wants the “ability to engage” in social media.  My response to that was to ask whether pharma has the “will” to engage – because they certainly have the ability if they choose to use it.  And where there’s a will, there’s a way.

Another issue that came up early – and that generated a lot of conversation – was the need to bifurcate the discussion of digital advertising from that of social media. There are rules for digital advertising, paid digital advertising. Social media, on the other hand, is the New Frontier.  It’s the crucial gray zone that exists between regulated speech and user-generated content.  It’s where the rubber meets the road.

What pharma wants (or should want) is specific areas of clarification from the FDA on this new and exciting zone of opportunity.

What of the empowered digital healthcare consumer that we hear so much about?  Well – there were a few of them in attendance at the AZ confab and they had some interesting things to say.

What struck a chord for me was when one of the civilians in the room (by which I mean a patient) said that she really had no idea why pharmaceutical companies chose to absent themselves from disease-related social media conversations.  She assumed it was because Big Pharma is afraid of mixing it up with real people in real time dialogue.

And she’s right, of course – but for reasons she didn’t suspect. The ensuing explanations of adverse event reporting and other compliance-related issues didn’t cause her to nod her head, but rather to say (indeed, almost insist) that “pharma should explain to people why they’re not there.”

Blame the FDA! was the knee-jerk reaction.  But that’s not fair and it’s not true.  How can the agency be blamed for industry’s reluctance to push the boundaries – even a little?   Fear of warning letters?  Fear of unearthing adverse events? I say, where there’s a will, there’s a way. If you won’t blaze the path – even a little -- then don’t expect anyone to know where you want to go.

Unfortunately, blazing new territory through real-time learning is not, shall we say, historically a tradition of the pharmaceutical industry.  Everyone wants to do new and exciting things – second.

Here’s an even more basic question – what’s the right thing to do?  I submit that it’s irresponsible to actively avoid participating in the social media healthcare conversation.  It is, to directly quote CDER Director Dr. Janet Woodcock, “where the people are."  Healthcare begins at search.

 

But, someone pushed back, that’s why we need more directive regulation from the FDA.  I fundamentally disagree (1) that’s what’s needed and (2) that’s what’s coming.  Let me explain.

(1)  IMHO, “We need more regulation” just doesn’t cut it. Since there is no direct “ask” from industry, it’s impossible to expect the FDA to offer direct guidance. It’s not like requesting guidance for DTC advertisements.  That was a precise request for a tangible deliverable that resulted in direct and specific rules and regulation. More regulation?  Be careful, that may be precisely what you get.  Also, “more” guidance means nothing without a more precise reference.  “More” relative to what aspects of social media?  These details were lacking at last November’s Part 15 hearing and (alas) equally so in the lengthier (but equally non-specific) docket submissions.

 

(2)  What are the odds, lacking direction, expertise and experience, that DDMAC will deliver some kind of deus ex machina solution?  Expecting the Holy Grail will only lead to disappointment and frustration.  And blaming the FDA when that happens won’t make anything better or move the social media agenda any further ahead.  If industry is expecting to climb the steps of White Oak on its knees, kiss an FDA relic and miraculously throw away the crutches hobbling their ability to participate in social media, well, there had better be a Plan B.

 

Where there’s a will there’s a way.

 

Then there’s the question of language and syntax.  For example, what does “sponsored” mean? Let’s do a brief thought experiment.  Consider a televised PGA tour event.  When a product logo for an erectile dysfunction medicine appears on the screen and the announcer intones, “This portion of the Masters is sponsored by DRUG NAME HERE,” nobody out there in the viewing audience takes that to mean the “sponsor” has chosen the speed of the greens, the height of the rough, or the pairing of golfers in the tournament.  But say “sponsored” on a social media site and watch the sparks fly at internal regulatory review. Fore! This also leads to the still vague regulatory distinction between property owner and property user – an issue in dire need of FDA clarification.  Discussion of this important social media issue in FDA docket submissions?  Try and find it.

 

Of course, there’s the subtle but crucial differentiation between “permissible” and “appropriate.”  And this returns us to where we started.  What does pharma want?  Do they want social media, primarily, as a new channel for marketing or do they see it as a new and exciting and robust and dynamic mechanism for advancing the public health through real-time interactive communications?

Indeed – why not both?  Where there’s a will, there’s a way.

 

And AZ – thanks for the sandwiches and kudos for a job well done.

  Read More & Comment...

A new wrinkle

10/14/2010 05:02 PM |

The FDA has approved a clinical trial of Botox as a treatment for vaginal spasms that can block sexual intercourse and gynecological examinations.

 

clinicaltrials.gov also lists a similar study that already is under way at the University of Tehran in Iran.

 

The full article can be found here.

I leave the rest to your imagination.

  Read More & Comment...