Office Space

09/19/2011 11:36 AM |

Q: When is an office not an office?

A: When it's a super office.

FR:  Janet Woodcock

 

TO: CDER Staff:

 

The Office of Medical Policy (OMP) has been reorganized into a Super Office.  Within its organizational structure are the Office of Medical Policy Initiatives and the Office of Prescription Drug Promotion. Led by Rachel Sherman and her Deputy, Kathleen Uhl, OMP plays a critical role in directing medical policy programs and strategic initiatives.

 

This includes directing regulation of prescription drug promotion and advertising, providing leadership and scientific advice on clinical trial design, providing consultation and direction in policy issues related to human subject protection and good clinical practices, supporting the recent Health Care Reform Act that provides new legislation for Biosimilars, and developing regulation, guidance documents, and procedures related to medical policy issues.

 

·         Rachel Sherman, Director, Office of Medical Policy

·         Kathleen Uhl, Deputy Director, Office of Medical Policy

·         Janet Norden, Associate Director for Regulatory Affairs

 

The Office of Prescription Drug Promotion

 

The Division of Drug Marketing, Advertising, and Communications has been reorganized and elevated into the Office of Prescription Drug Promotion (OPDP).

This reorganization will leverage OPDP’s resources and processes to provide for the highly effective oversight of prescription drug promotion. 

 

ODPD consists of an Immediate Office, the Division of Professional Promotion (DPP), and the Division of Direct-to-Consumer Promotion (DDTCP). The new structure will help prevent misleading promotion about prescription drugs and enhance the quality of communications about prescription drugs and other health information developed by companies. 

 

Thomas Abrams, Director, Office of Prescription Drug Promotion

Mark Askine, Associate Director, Office of Prescription Drug Promotion

Marci Kiester, Associate Director of Operations, Office of Prescription Drug Promotion

Catherine Gray, Acting Director, Division of Professional Promotion

Robert Dean, Acting Director, Division of Direct-to-Consumer Promotion

 

The Office of Medical Policy Initiatives

 

A newly created Office of Medical Policy Initiatives (OMPI) consists of an Immediate Office, the Division of Medical Policy Programs (DMPP), and the Division of Medical Policy Development (DMPD).

 

This office will develop and coordinate medical policy regulations and guidances that address the policy and program areas covered by the Super Office. The organization of the divisions supports the continued implementation and successful advancement of the Sentinel Initiative, the Clinical Trials Transformation Initiative (CTTI), and the Patient Medication Information (PMI) Initiative. 

 

Within this reorganization, the Patient Labeling Team (PLT) will be moving from the Office of Surveillance and Epidemiology (OSE), Division of Risk Management (DRISK) to OMPI, Division of Medical Policy Programs. The goal of the Patient Labeling Team is to promote the safe and effective use of prescription medications by providing accurate and easily understood patient medication information. OMPI will reach out to affected offices with procedural details on the PLT's transfer.

 

Denise Hinton, Director, Office of Medical Policy Initiatives

Richardae Araojo, Deputy Director, Office of Medical Policy Initiatives & Acting Division Director, Division of Medical Policy Programs

Paula McKeever, Division Director, Division of Medical Policy Development

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Trigger Finger

09/19/2011 09:19 AM |

It seems that the FDA has decided that biosimilar sponsors will not be required to meet with the agency at specific times or for specific reasons during the IND phase of development, allowing them more flexibility early in the process.

 

Product development fees would be collected when a biosimilar sponsor files an IND. It is expected to be about 10% of the cost to file for a marketing application review and be charged annually as long as the product remains in active development stages.

 

FDA wants the earlier infusion of funding to support agency activities during the biosimilar IND stage, which are expected to be more extensive than those for other applications. The agency agreed to discount all product development fee payments from the marketing application fee once it is filed

 

FDA and the brand and generic industry representatives also may have ended their dispute over whether the biosimilar user fee program should be independent of the other user fee programs.

 

At an August 8th meeting the agency circulated draft statutory language authorizing creation of the program independent of other user fees. The minutes did not indicate opposition by GPhA on any portions of the proposal. There also was no mention in the minutes of further discussion of the appropriations trigger.

 

The agency agreed to write a draft commitment letter for discussion at another negotiating session, another indication both sides have moved beyond the issue.

 

 

The biosimilar user fee program is expected to be included in an omnibus bill that also will include the Medical Device User Fee and Modernization Act and PDUFA reauthorizations, as well as the new generic drug user fee program.

  Read More & Comment...

A Challenge to Uncivil Society

09/16/2011 07:10 AM |

On Wednesday I attended the Washington Post’s event on non-communicable diseases (NCDs), “Sharing the Responsibility.” The event was co-sponsored by Eli Lilly & Co.

(Video clips of this event can be found at www.washingtonpostlive.com)

The title of the conference was crucial as world-class speaker after speaker spoke to the need for cooperation between (as Ann Keeling, the CEO of the International Diabetes Federation and Chair of the NCD Alliance put it) “the three P’s – public, private, and people.”

For a change, the discussion of NCDs wasn’t framed as a battle between “good guys” (generally portrayed in the mainstream media as “civil society”) and “bad guys” (private industry).  Rather than being about placing the blame, it was about developing solutions.  This position was stated early and eloquently by the event’s opening speaker, Dr. Julio Frank (Dean of the Faculty, Harvard School of Public Health and the key founding father of the Mexican healthcare system).  Dr. Frank warned that we must avoid and beware of “reductionist solutions.”

When asked about the role of intellectual property rights and their role in addressing the NCD issue, Dr. Frank said that protecting IPRs is crucial to developing new and innovative global healthcare solutions.  While he was answering this question, Ms. Keeling had no comment on the question of IPRs but did comment that, “there are no magic bullets.”

This concept of “shared responsibility” issues many challenges – not the least of which goes out to the “Uncivil Society” movement led by (among others) Jamie Love. Uncivil Society demonizes any role for industry -- except maybe writing checks (which brings to mind Abba Eban’s famous quote about the give-and-take between Israel and the PLO –“We give and they take.”)

As H.L. Mencken famously quipped, “For every complex problem there is an answer that is clear, simple, and wrong.”

Uncivil Society was called out at this event.  “Shared responsibility,” means they must cease repeating and repeating and repeating their incessant falsehood that the majority of the Developing World’s healthcare problems could be solved if only we would do away with patents and intellectual property protection.

The petty agenda of Uncivil Society must not be allowed to hijack the important global mission of combating NCDs.

During the panel on “Public & Private Partnerships,” Herb Riband (VP, External Affairs for Medtronic) spoke about a “confluence of interests.” And John Lechleiter (President and CEO, Eli Lilly & Co.) commented that, “There is no substitute for the power of partnership.” And Lilly is putting its money where its mouth is.  A day before the Post event they announced the Lilly NCD Partnership, a five-year $30 million commitment to fight the rising burden of non-communicable diseases in developing nations. And it’s not about writing a check, but rather combines the company’s resources with the expertise of leading global health organizations, to identify new models of patient care that increase treatment access and improve outcomes for underserved people.

As Mark Kramer (Senior Fellow, Harvard Kennedy School of Business) said, a key role for private industry is to “broker partnerships that propel progress.”

David Brown (a Washington Post journalist and a physician) commented that we must avoid the “false dichotomies” of NCDs (infectious vs. non-infectious, cure vs. prevention, rich vs. poor). So too must we avoid the false dichotomy of “hero vs. villain.”

The Washington Post conference, held in advance of next week’s historic United Nation’s High Level Meeting on NCDs, made it abundantly clear that, to actively, aggressively, and creatively fight NCDs in the Developing World (and, for that matter, the whole world), there must be partnerships rather than partisanship. 

The common ground is shared responsibility.

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Just Shoot Me

09/15/2011 09:21 AM |

Pseudo celebrity Jenny McCarthy has appeared on three episodes of the hit TV show "Just Shoot Me."  Ergo, her rants about the dangers of vaccines must be true, right?

Hm.

Please have a look at this short PSA on why, when it comes to vaccines, celebrity trumps science.

And please, pass this along.  Let's make it a YouTube sensation.
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CME -- No Evil

09/14/2011 06:52 AM |

If you have an interest and no pressing conflicts, two important briefings by the CME Coalition (www.cmecoalition.org) to consider attending:

CME Cancer Policy Briefing

On September 27, from 12:00 – 1:00 in the Rayburn House Office Building “Gold Room” (Room 2168), the Coalition will be presenting a program entitled “Continuing Medical Education:  A Focus on Breast Cancer Breakthroughs.”  This panel will comprise thought leaders in the fields of CME and breast cancer treatment, and will include:

Dr. Jonathan Sackier: Chief Medical Officer, Audax Health Solutions, Visiting Professor of Surgery, University of Virginia, Following positions at Cedars-Sinai and UCLA, he was named Professor of Surgery at George Washington University, in Washington, D.C. where he founded the Washington Institute of Surgical Endoscopy.

Dr. Charles Balch: Professor of Surgery and Oncology and Dermatology, Deputy Director, Johns Hopkins Institute for Clinical and Translational Research, Former CEO of American Society of Clinical Oncology

 

Dr. Dana Simpler: Primary Care Practitioner, Mercy Medical Center, Baltimore, MD, Has appeared in numerous print and broadcast media, locally, regionally and nationally as a health care thought leader.

And …

 

CME HIV Policy Briefing

 

On September 28, from 12:00 – 1:00 in the Senate Commerce Committee Hearing Room (Russell Senate Office Building Room 253), the Coalition will be presenting a program entitled “Continuing Medical Education:  A Focus on HIV Breakthroughs.”  This panel will comprise thought leaders in the fields of CME and HIV treatment, and will include:

Dr. Jonathan Sackier 

Dr. John Bartlett: Professor and Chief of the Division of Infectious Diseases at Johns Hopkins, Internationally renowned authority on AIDS and other infectious diseases and recipient of the prestigious 2005 Maxwell Finland Award for scientific achievement from the National Foundation for Infectious Diseases.

 

Dr. Alan Wasserman: The Eugene Meyer Professor of Medicine and the Chairman of the Department of Medicine at The GWU School of Medicine and Health Sciences, Chairman of the Board of Trustees and President of The George Washington University Medical Faculty Associates.

 

Space is limited, however, so kindly RSVP at by September 16^th to rsvp@cmecoalition.org or call Shea McCarthy at (202) 688-0225.

 

  Read More & Comment...

Separated at Birth?

09/13/2011 06:20 PM |

Michele Bachmann and Jenny McCarthy?
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"One of the great scientific advances in the history of medicine"

09/13/2011 04:29 PM |

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Update from Peyton Place

09/13/2011 03:17 PM |

In advance of next week's UN meeting on non-communicable diseases in the developing world, tomorrow I will attend and participate at a Washington Post forum on this very topic.

 

Today, this relevant news crossed my desk.

Kismet?  I don't think so.

 

Indianapolis, IN – Eli Lilly and Company (NYSE: LLY) announced today a $30 million commitment over five years to fight the rising burden of non-communicable diseases in developing nations. Lilly is launching The Lilly NCD Partnership, which combines the company’s unique resources with the expertise of leading global health organizations, to identify new models of patient care that increase treatment access and improve outcomes for underserved people. The partnership will initially focus on diabetes – a core business area in which Lilly has deep expertise.

 

Non-communicable diseases (NCDs), known as chronic diseases, include cardiovascular diseases, diabetes, cancer and chronic respiratory diseases. The first phase of The Lilly NCD Partnership will focus on improving diabetes care in targeted communities in Brazil, India, Mexico and South Africa.

 

“Non-communicable diseases are afflicting nations, communities and families around the world, with the most vulnerable bearing most of the burden,” said John C. Lechleiter, Ph.D., Lilly chairman, president, and chief executive officer. “We believe we have a responsibility – and are uniquely positioned – to assist in the global fight against these diseases. In partnership with leading health organizations, Lilly will contribute its deep expertise and the company’s broad research capabilities to help find solutions for these pressing societal needs.”

 

Lilly and its partners continue to develop country-specific programs that will launch in early 2012. Lilly and its partners will develop country-specific milestones that, if achieved, will trigger future investments.

 

Partners include:

 

• Brazil: Hospital Israelita Albert Einstein – Diagnostic & Preventive

Medicine and Research Institute

• India: The Public Health Foundation of India, Project HOPE, Population

Services International

• Mexico: The Carlos Slim Health Institute – Casalud

• South Africa: The Donald Woods Foundation, Project HOPE

Congratulations to my friends at Eli Lilly & Co.   Good luck and good karma.

Now if only Peyton Manning was healthy!

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Bachmann Promotes Vaccine Panic

09/12/2011 11:52 PM |

I just wrote (http://spectator.org/archives/2011/09/12/rick-perry-on-science) about how Rick Perry’s position on vaccines was science-based.  Sadly, tonight Republican presidential candidate Michele Bachmann (followed by Congressman Ron Paul and former Senator Rick Santorum) tore into Rick Perry about his executive order mandating that girls entering the 6^th grade receive a vaccine to prevent against human papilloma virus (HPV), the leading cause of cervical cancer as well as mouth cancer, tongue cancer and other cancers of the vagina, penis and anus. 

Yes, HPV is a sexually transmittable disease and the transmission rates for these illnesses have been climbing rapidly.   So too have both the prevalence and incidence of these cancers among young adults.  Which means they are contracting them at around age 12.

Bachmann, Santorum and Ron Paul accused Perry of ordering mandatory immunizations in exchange for a $5000 campaign contribution from a former lobbyist for Merck, the developer of one version of the HPV vaccine called Gardasil.  IN particular, Bachmann alleged that Perry’s decision exposed “little girls” to a dangerous drug.  In doing so, Bachmann – and - by extension Santorum and Paul – gave credence to the canard pushed by the anti-vaccine movement that Merck conspired to market a product that was causing girls to go blind, become paralyzed and die.    

 

First, the science and statistics:

• The most prominent argument against the HPV vaccine Gardasil is that it has been linked to 53 deaths (as of June 2009).  These reports were made to the Vaccine Adverse Event Reporting System (VAERS).  Of these, 30 reported deaths were confirmed to have occurred, but no causal link to the vaccine was found after investigation.  Based on the evidence available, therefore, it does not appear that the vaccine causes death.  More recent evidence demonstrates the same result.
• A post-market surveillance study by the CDC found that the rate of reported deaths (including anaphylaxis) was 0.1 per 100,000 doses distributed.  Their conclusion was that reported adverse events did not differ significantly from vaccines in general.
• Further arguments against Gardasil mention fainting after immunization.  This is a known possible side effect of some vaccines and is included in the package insert.  The insert also includes recommendations to observe the patient for at least 15 minutes after injection to ensure the patient does not fall or suffer injury.

 

Then there is the claim – promoted by an anti-vaccine group claiming that Gardasil contains recombinant HPV DNA that can replicate on it’s own and go haywire on the bodies of young girls.  SANE Vax Inc. Announces the Discovery of Viral HPV DNA Contaminant in Gardasil.   As Respectful Insolence blog points out:

This is utter nonsense. First off…it's not a trivial matter to get recombinant DNA into human cells and expressing the protein that its sequence codes for. It's worth repeating what I described when I first encountered this idiocy in a different context. For rDNA to do what Dr. Lee worries about, the minute amount of rDNA in the HPV vaccine would have to:

• Find its way into human cells in significant quantities, which is highly unlikely given the tiny amount that, even in the worst case, is there.
• Express the protein that it codes for, which would require that the DNA be intact, complete with its promoter and regulatory regions. Again, this is incredibly unlikely, given the amount of DNA we're talking about unlikely.

 

Finally, there is the objection to the vaccination based on the assertion that it promotes sexual activity among pre-teens.   But let’s assume – and hope – that one day a vaccine that prevents HIV is developed.  Would Congresswoman Bachmann also object to requiring immunization in that case because it would similarly encourage boys and girls to engage in sexual activity?  

 

 

 

Meanwhile Congressman Paul has indulged in the vaccine-autism conspiracy.  Congressman Paul, along with Congresswoman Carolyn Maloney (D-NY) and Rep. Maurice Hinchey (D-NY) introduce the "Comprehensive Comparative Study of Vaccinated and Unvaccinated Population Act of 2006," or H.R. 2832.  The bill would have required the National Institutes of Health to complete a study to examine the link between autism and thimerasol.  The bill has never gone anywhere though it did prove that anti-vaccine stupidity is bipartisan. 

 

 

http://www.informationliberation.com/?id=22711

 

Bachmann, Paul and Santorum (to a lesser extent) are spreading vaccine panic with unscientific and untruthful allegations about Gardasil and Merck.    The willingness to foment fear in order to score some political points in the short term is depressing by itself.  I don’t know what’s more demoralizing:  that the fear mongering comes at a time when vaccination rates are dropping because parents believe the same things the candidates espouse or that the media is once again failing to do it’s job and let people know that all vaccines are highly safe and effective.   But I guess that would get in the way of focusing on the catfight between Governor Perry and three politicians who have undermined the public health with misinformed and politically motivated assaults on an important tool in preventing cancer.

 

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Raising (Bio)Shields

09/12/2011 04:13 PM |

Two sobering assessments of the difficulty in bringing forth second generation countermeasures underscore the problems added to the development process when government controls the process. 

www.sciencemag.org/content/333/6047/1216.summary

www.biocenturytv.com/fullplayer.aspx#/BC+Show+52%3A+Biodefense/BioCentury+09.11.11+-+[3]+10+Years+After/608459720001/1150255534001/1153307594001

The need for a commercial application of products is critical to making the nation safer.   The government drains value from bio-defense, it does not create it.
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Nice Package

09/12/2011 06:52 AM |

Nature Biotechnology opines on BIO’s proposal for a renewing and revitalizing the FDA:

 

Keeping innovation on American soil may also prove a Sisyphean task. BIO states that in 2009, 35% of pharma companies outsourced to Asia, primarily China and India, and almost a third of small US biotech firms have been tapped to move their R&D operations abroad of biochemical, genomic, imaging, metabolite, cellular, physiological and clinical scoring-scale information gathered from clinical trial reports, scientific conferences and public literature.

 

The complete article can be found here.

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Nonsense on Brain Stents

09/09/2011 12:00 PM |

When I was at the FDA, we made a conscious effort to stop referring to “compassionate use” because it made it sound like “noblesse oblige.”  We began to call it what it should be – expanded access.

In a house editorial, the New York Times misses the mark not only on this point (where they refer to “humanitarian use” – a nonsensical misnomer), but also as it relates to “comparative effectiveness.”

The Gray Lady opines, “This case raises the question of whether the F.D.A. should demand more rigorous trials before a device is granted a humanitarian exemption. It clearly shows the value of conducting rigorous controlled studies with enough patients to provide meaningful results. This is just the kind of comparative effectiveness research that the national health care reforms seek to promote.”

Well, yes and no. 

Sure, the FDA should always strive to better understand where expanded access programs may lead.  (And, it’s important to note, many patient groups – such as the Abigail Alliance – believe the FDA is already too slow and stingy with such protocols.) But even the best folks at the FDA are only so prescient.  I think it wise to give the folks at White Oak the benefit of the doubt.  Adoption of the Precautionary Principle (where nothing is done until everything is known) only leads to nothing being done and the death of innovation.

And as far as the “comparative effectiveness” statement is concerned, this situation has nothing to do with it whatsoever. A larger scale trial of these stents uncovered the safety problem -- precisely what such trials are designed to do.  It has nothing to do with "comparative effectiveness." When it comes to the FDA -- it's about safety and efficacy.

But when you’ve got a hammer, every problem looks like a nail. 

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Price Controls? OIG Vey!

09/08/2011 10:25 AM |

First it was gun slinging against Forest Labs, now the HHS OIG is gunning for price controls.

 

According to BioCentury, the OIG has recommended that the Obama administration seek authority from Congress to more effectively control Part B drug and biological expenditures. The recommendation came in a report released Wednesday documenting the difference in acquisition cost for Lucentis ranibizumab from Genentech Inc. for wet age-related macular degeneration and Genentech's Avastin bevacizumab, which is used off-label in the indication as a cheaper alternative.

 

The report said using Avastin instead of Lucentis for wet AMD would have saved Medicare Part B $1.1 billion and beneficiaries $275 million in copayments in 2008-09. In those years, HHS OIG said the average sales price for a dose of Lucentis, a mAb fragment against VEGF-A, was about $1,915 compared to about $7 for an intravitreal dose of cancer drug Avastin, a humanized mAb against VEGF.

 

The report noted that CMS, which reimburses for both drugs for AMD, does not have the authority to require price concessions or rebates for products covered under Part B. In a written response including in the report, CMS said it is "evaluating our current authorities and will seek additional authorities as necessary."

 

Genentech said in a statement it will not comment until it finishes reviewing the report. The company added "we do not believe that cost should be the only factor considered when choosing a medicine."

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Rick Perry is NOT Anti-Science

09/07/2011 10:16 PM |

It is likely that Rick Perry's statements about Social Security being a "ponzi scheme" and climate change will make and shape the headlines tomorrow (and tonight).   There will be a steady stream of comments about Perry being anti-science.  But Rick Perry is  not anti-science.

In fact, he made the most sense science-wise of all the candidates.

1.  He did not back down on the need or requirement to have children receive a vaccine that can eliminate many forms of cancer.   I thought it was a courageous and principled stand when he first called for HPV immunizations for all 12 year old girls.   By contrast other presidential candidates -- Bachman, Santorum and Ron Paul in particular -- seem to suggest that parental rights trump immunization requirements in every case.  If that is so, then we need to ask these candidates if they oppose immunization requirements for children and if they believe vaccines cause autism.  Then we will see who is anti-science.

2.  Perry did more to advance medical science in Texas than other governors running for President have done.  Not only did he lead in the establishment of  Cancer Prevention and Research Institute of Texas (CPRIT), a $3 billion, 10-year cancer research fund, Texas in one.  Just recently CPRIT recruited a leading stem cell researcher to establish a pediatric cancer initiative at University of Texas (UT) Southwestern Medical Center at Dallas.  The researcher, Sean Morrison, said this about Texas:

While I have been spending the last five to six years arguing with the Legislature about what kind of research would be permitted in the state, in Texas they were looking for ways to invest billions of dollars into medical research..."Texas is clearly an environment that's more supportive generally of research innovation. Three billion [dollars] for cancer research is going to change the landscape."  

PS.   Perry has never supported proposals to ban stem cell research in Texas either. 

Ignoring these aspects of Governor Perry's record while questioning his position that the  'science' behind predictions of global disaster and requires massive government intervention in the economy  -- which is what the debate is all about -- is anti-science is simply a tactic to silence that individual. Or make him or her look stupid. 

I still would like to know if the candidates who criticized Perry are opposed to mandatory vaccination in any case. 
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Doctors vs. anti-vaccine propaganda

09/07/2011 03:44 AM |

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Not Such Great Expectations

09/06/2011 07:51 AM |

"We spent as much money as we could, and got as little for it as people could make up their minds to give us. We were always more or less miserable, and most of our acquaintance were in the same condition.” -- Great Expectations

In terms of “learning” about healthcare reform from our British cousins, here’s lesson #1:  Don’t believe everything (or anything) you heard on SiCKO.  Lesson #2 is seeing CMS Administrator Dr. Donald Berwick in the role of Pip.

According to an investigation in the Daily Telegraph, at least 10 primary care trusts (PCTs) have told hospitals to increase the length of time before they see patients in order to save money.

In one case a manager said the policy keeps patients in line as “short waiting times also create more demand for treatment due to the expectations this raises."

In some areas, patients endured delays of 12 or 15 weeks after GPs decided they needed surgery, even though hospitals could have seen them sooner.

The maximum permitted time between referral and treatment is 18 weeks.

It comes after an NHS watchdog suggested that if patients are forced to wait a long time, they will remove themselves from lists “either by dying or by paying for their own treatment."

Andrew Lansley, the Health Secretary, said: “This practice is simply unacceptable and one of the many reasons we need to modernise the NHS and put patients’ interests first.

The complete article from The Telegraph can be found here.

“Pause you who read this, and think for a moment of the long chain of iron or gold, of thorns or flowers, that would never have bound you, but for the formation of the first link on one memorable day." -- Great Expectations

Hat tip to Helen Evans at Nurses for Reform.

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What we don't need is a little Christmas right this very minute

09/02/2011 06:44 AM |

A sudden bold and unexpected question doth many times surprise a man and lay him open.  – Francis Bacon

The official PDUFA V technical letter contains no surprises.  There are no “Christmas Tree items.”  It is a document full of incremental improvements. It is, in a word, “clean.”  But don’t let that fool you.  There’s a battle ahead.

(The technical letter can be found here.)

Unlike past reauthorization, when industry and agency reached agreement and Congress rubber-stamped it, this year there are going to be questions. Some relevant (predictability and responsibility), some not (greater regulation of consumer marketing practices), some thorny (should biosimilar reviews be covered under PDUFA through fiscal year 2017). It’s a long list.

For many involved in the reauthorization process, one statement that keeps coming up is -- ”The FDA is broken.” But what does that mean? Rather than making blanket statements that cause friction and promote areas of disagreement, one thing everyone can agree to is that the FDA’s must be both ally and accelerator in the advancement of innovation.

Can that be accomplished within the confines of PDUFA V?

PDUFA IV expires Sept. 30, 2012. Senator Tom Harkin (D, IA -- Chairman of the Health, Education, Labor and Pensions Committee) said he hopes PDUFA V will reach Congress by year-end, so that his committee can mark up the legislation in the spring. Representative Joe Pitts (R, PA -- Chairman of the House Energy and Commerce Committee's health subcommittee.) said he hopes to have PDUFA V enacted by June 30, 2012.

Let the battle begin.

For another interesting view on the “deal on the table,” have a look at what Robert Metcalf, Vice President, Global Regulatory Affairs at Eli Lilly & Company, has to say.  His comments can be found here.

The only thing that should surprise us is that there are still some things that can surprise us.  – Francois de La Rochefocauld  Read More & Comment...

The (Clinical) Trial

09/01/2011 08:18 AM |

Life imitates art.  In Franz Kafka’s unfinished story, “The Trial,” a man is arrested and prosecuted by a remote, inaccessible authority, with the nature of his crime revealed neither to him nor the reader.

 

Well – it’s déjà vu all over again -- new Russian regulations making it harder for the Rodina to become an international destination center for clinical trial excellence.  And nobody knows why.

 

As Dr. Meir Pugatch discusses in a new Journal of Commercial Biotechnology article, “The overall, the strength of national pharmaceutical IP environments provide a good estimate of the level of clinical trials taking place in these countries. Accordingly, countries with a more robust level of pharmaceutical IP protection tend to enjoy a greater level of clinical trial activity by multinational research-based companies. In other words, by choosing to improve their level of protection of pharmaceutical IPRs (together with other factors), developing countries may also be exposed to higher levels of biomedical FDI, not least in the field of clinical trials.”

 

(More on Dr. Pugatch’s article can be found here.)

 

This is bad news for Russia.

 

New data shows that clinical trial activity in Russia is still on the decline following the introduction of new regulations last year. The total number of trials approved in the first half of 2011 fell by 36% to just 200, with trials conducted locally by domestic sponsors most affected, falling by around 80%, according to the Russian clinical trials organization, ACTO.

 

The slowdown is mainly due to the law on circulation of medicines, which came into effect in September last year. Among other things, the law incorporated local clinical trials into the drug registration process, introduced new trial approval and insurance procedures, and imposed a ban on the import of registered products for use in clinical trials.

 

Not only is this bad news for international investment – but it also has the unfortunate side effects of making it more difficult for Russian companies to conduct clinical trials in their own country. Russian companies wanting to conduct local trials (as opposed to Russian arms of multinational studies) can do so only as part of a drug registration procedure, not as freestanding studies.

 

According to ACTO Executive Director Svetlana Zavidova, she did not know why this distinction had been made, but it was possible that those drafting the law had simply forgotten to state that local clinical trials were also a freestanding kind of study.

Kafkaesque – and with unfortunate results.

The number of local safety and efficacy studies approved in Russia fell by 78.4% to 19 in the first half of 2011, while bioequivalence studies were down by 84.4% to just seven. According to ACTO, the fall in bioequivalence studies is probably due to two factors: there are no approved requirements for such studies, and in some cases generic manufacturers cannot refer to the originator's pre-clinical trial results and are having to carry out their own tests, thereby delaying the bioequivalence study.

 

These developments are somewhat worrying given that the government has made much of its plans to build up the Russian pharmaceutical industry and reduce the country's reliance on imported medicines. "It is remarkable that the implementation of the law had a particularly tough impact specifically on the innovation activity of Russian companies, which is in direct contradiction with the governmental desire for import substitution in pharmaceuticals," ACTO commented.

“Remarkable?”  Not really.  Predictable?  Absolutely.

On the positive side, sponsors of international trials fared better than their Russian counterparts in the first half of 2011, gaining 163 trial approvals compared with 160 in the 2010 period. Nonetheless, they had their own problems, including the lack of new rules on insurance, which should have been in place on 1 January this year, and the ban on importing registered drugs (for use as comparators in clinical trials of experimental drugs).

 

Bad News: The import ban affected not only new studies but also those already under way where stocks of the approved drug were running short, says ACTO, which notes that some new studies were cancelled and transferred to other countries.

 

Good News: The ban was finally lifted in June this year when the health ministry took over responsibility for the importation of both approved and unapproved medicines, while the insurance question was dealt with by a May decree implementing the amended rules.

 

Bad News: Another factor dampening trial activity has been the fact that the authorities are not adhering to the new, shorter timeframes for trial approvals stipulated in the new law.

 

In fact, according to ACTO, things are even worse than they were before the law came into force: in the first six months of 2011, the total time taken to gain approval for a trial and obtain import/export permits amounted to an average of 160 days. This is "twice as long as the maximum period allowed by law and 30.5% longer than the all-time worst results.”

 

Other problems remain, says ACTO, including the fact that foreign companies still have to conduct a local trial (free-standing or as part of an IMCT) when seeking new drug registration in Russia. "Unfortunately, this problem is still on the front burner for us", ACTO’s Zavidova commented.

 

That must be some big front burner.

 

Considering the importance of clinical trials as a leading indicator of international pharmaceutical investment, perhaps Moscow should seek expert advice on the subject.  After all, as the Russian proverb says, “With a helper a thousand things are possible.”

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The Four Percent Solution

08/31/2011 01:28 PM |

Is 4% a lot or a little?  Well – it depends whether you’re one of the 4%.

 

As the AP reports, “Pfizer Inc.'s just-approved drug Xalkori, the first new medicine in more than six years for deadly lung cancer, proves the value of precisely targeting rare diseases linked to gene variants

The drug was approved Friday in the U.S. along with a companion diagnostic test for just a small subset of lung cancer patients. It is the poster-child for the 21^st century strategy of developing medicines for relatively few patients to replace (as the AP puts it) “blockbusters for the masses.”

 

"This is a paradigm shift," Dr. Paul A. Bunn Jr., a University of Colorado professor and cancer researcher involved in testing Xalkori, told journalists during a conference call hosted by Pfizer. "It used to be that everybody with cancer was treated the same," with surgery and chemotherapy.

 

Xalkori, a pill with relatively minor side effects compared to the hair loss and nausea that chemotherapy can cause, was approved for the roughly 4 percent of patients with advanced nonsmall cell lung cancer who have what's called the ALK fusion gene.

 

About 6,000 Americans a year develop this cancer, Pfizer said. Those patients, called ALK positive, now can be identified with a $250 molecular diagnostic test developed by Pfizer's partner, Abbott Molecular Oncology. The test was also approved Friday.

 

According to Dr. Mark Kris, head of the thoracic oncology service at Memorial Sloan-Kettering Cancer Center in New York, "You're going to be sparing individuals side effects (and) the waste of resources, time and drug that really isn't going to help them."

Cost efficient and patient-centric. Bingo.

 

Personalized medicine, targeted therapy – call it what you will.  It’s real.  And it’s a giant step towards achieving the 4 rights – the right medicine in the right dose for the right patient at the right time.

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PCORI? Memento Mori.

08/30/2011 08:43 AM |

For those of you who missed Latin class that day, memento mori means, “remember your mortality.”

 

It seems that the American Medical Association has (albeit a little late in the game) woken up to that fact and gotten religion.

 

The AMA has invited other healthcare groups to sign onto a letter to the Patient Centered Outcomes Research Institute (PCORI) on the types of research that should and should not be conducted under ObamaCare. The AMA is concerned about its proposal to "investigate ... optimizing outcomes while addressing burden to individuals, resources, and other stakeholder perspectives."

 

They should have read the bill before they so aggressively supported its passage.

 

Why the turnaround? Could it be that America’s physicians worry that including cost in the equation will open the door to rationing down the line?

 

"We seek further clarification toward the Board's intentions regarding this last component and whether this includes cost analysis," states a sign-on letter scheduled to be sent later this week to PCORI executive director Joseph Selby. "If that is the case, we do not believe that it is consistent with the PCORI's enabling statute."

 

The issue, at its core, is physician disempowerment. With first insurance companies and now Uncle Sam telling doctors how to practice medicine (step therapy, restrictive formularies, more strident and cumbersome preauthorization requirements, and academic detailing), it’s no wonder that physicians are mad and that the AMA is doing a volte-face on PCORI and comparative effectiveness.

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