When is science not science? When it's promotion. Maybe.

12/28/2011 08:49 AM |

Just when you thought it might be safe to go on vacation -- something to think about for the remainder of 2011, the free and fair dissemination of scientific and pharmacoeconomic data.

According to a notice in the Federal Register:

The Food and Drug Administration (FDA) is announcing the establishment of a docket to assist with our evaluation of our policies on communications and activities related to off-label uses of marketed products, as well as communications and activities related to use of products that are not yet legally marketed for any use, we would like to obtain comments and information related to scientific exchange.  FDA is interested in obtaining comments and information regarding scientific exchange about both unapproved new uses of products already legally marketed (“off-label” use) and use of products not yet legally marketed for any use. 

And the issue of “scientific exchange” comes front and center. According to the FR notice, To assist with our evaluation of our policies on communications and activities related to off-label uses of marketed products, as well as communications and activities related to use of products that are not yet legally marketed for any use, we would like to obtain comments and information related to scientific exchange.

The FR notice puts this request into perspective:

On July 5, 2011, a citizen petition was submitted by Ropes & Gray and Sidley Austin LLP on behalf of seven product manufacturers (Petitioners):  Allergan, Inc.; Eli Lilly and Co.; Johnson & Johnson; Novartis Pharmaceuticals Corp.; Novo Nordisk, Inc.; Pfizer, Inc.; and sanofi-aventis U.S. LLC under 21 CFR 10.30.  The citizen petition requested that FDA clarify its policies for drug products and devices governing certain communications and activities related to off-label uses of marketed products and use of products that are not yet legally marketed for any use.  Specifically, the petition requests clarification in the following areas:

1.  Manufacturer responses to unsolicited requests; 

2.  Scientific exchange; 

3.  Interactions with formulary committees, payors, and similar entities; and

4.  Dissemination of third-party clinical practice guidelines.

For some time, FDA has been considering these issues and is currently evaluating our policies on sponsor or investigator communications and activities related to off-label uses of marketed products and use of products that are not yet legally marketed for any use.  We have been considering what actions to take in the areas specified by the petitioners with respect to manufacturer responses to unsolicited requests; interactions with formulary committees, payors, and similar entities; and the dissemination of third-party clinical practice guidelines.

Specifically, the FDA asks:

• How should FDA define scientific exchange?  

• What types of activities fall under scientific exchange?

• What types of activities do not fall under scientific exchange?

• Are there particular types and quality of data that may indicate that an activity is, or is not, scientific exchange?

• In what types of forums does scientific exchange typically occur?  Should the use of certain forums be given particular significance in determining whether an activity is scientific exchange or an activity that promotes the drug or device?  If so, which forums?

• What are the distinctions between scientific exchange and promotion?  What are the boundaries between scientific exchange and promotion?

•  Generally, who are the speakers involved in scientific exchange, and who is the audience for their communications?

• Should the identity of the participants (either speakers or audience) be given particular significance in determining whether an activity is scientific exchange or an activity that promotes the drug or device?  If so, which participants would be indicative of scientific exchange and which would be indicative of promotion?

• How do companies generally separate scientific roles and promotional roles within their corporate structures?

• How should the Agency treat scientific exchange concerning off-label uses of already approved drugs and new uses of legally marketed devices? Please address whether there should be any distinctions between communications regarding uses under FDA-regulated investigation (to support potential approval) and communications regarding uses that are not under express FDA-regulated investigation.

• How should the Agency treat scientific exchange concerning use of products that are not yet legally marketed (that is, products that cannot be legally distributed for any use outside of an FDA- or institutional review board (IRB)-approved clinical trial)?

• Should investigational new drugs and investigational devices be treated the same with respect to scientific exchange? Why or why not? 

• Under 21 CFR 812.7(b), an investigational device is considered to be “commercialized” if the price charged for it is more than is necessary to recover the costs of manufacture, research, development, and handling.  Similarly, FDA considers charging a price for an investigational drug that exceeds that permitted under its regulations (generally limited to cost recovery) to constitute “commercialization” of the drug (see 74 FR 40872 at 40890, August 13, 2009; 52 FR 19466 at 19467).  What other actions indicate the commercialization of drug and/or device products?  If there are differences in the steps taken to commercialize drug products and the steps taken to commercialize device products, either before or after approval, please explain these differences.

A lot of questions and, it seems, a lot of potential regulatory mission creep.

Relative to, “Interactions with formulary committees, payors, and similar entities,” the door is now also open for debate on FDAMA Section 114 and health economic data.

There is no on-the-books draft or final guidance on Section 114. It’s been 14 years since the initial language. Health-related quality of life claims are considered under the established "adequate and well-controlled trials" standard.

Some background to put this into perspective:

To address concerns that FDA regulations were limiting the dissemination of outcomes research, Congress added Section 114 to set a new, less stringent standard applicable to promotional dissemination of health care economic information to MCO formulary committees: "competent and reliable scientific evidence."

Even though there is no FDA guidance to explain the agency's understanding "competent and reliable scientific evidence,” PhRMA developed a draft guidance, which was submitted to the FDA in June 1998. In its draft, PhRMA sought input from the International Society for Pharmacoeconomics and Outcomes Research, the Society for Medical Decision Making, the Academy of Managed Care Pharmacy, the American Pharmaceutical Association, and other groups.

In its submission to the FDA, PhRMA explained the history behind Section 114 and proposed guidance on the following terms used in the new law:

• Health care economic information.
• Managed care or other similar organizations.
• Formulary committee or other similar entity.
• Directly related to an approved indication.
• Competent and reliable scientific evidence.

The PhRMA proposal took an approach to interpretation consistent with Congress's intent that Section 114 would increase the dissemination of outcomes research information by product manufacturers to MCOs. PhRMA concluded that the term "health care economic information" should include all forms of economic analysis so the guidance could adapt to new and evolving outcomes research methods.

One of the phrases in Section 114 that is difficult to interpret is that promotion must involve a claim that "directly relates to an indication approved [by the FDA]." In the draft guidance, PhRMA proposed that extrapolation from data included on labeling would be appropriate at least under the following circumstances: from duration of use in labeling to actual duration of use found in pharmacy databases, from dosages included in labeling to actual dosages found in pharmacy databases, and from controlled trial settings to actual practice settings.

The standard set by Section 114, "competent and reliable scientific evidence," is the same standard used by the Federal Trade Commission (FTC) when assessing the adequacy of substantiation for manufacturer claims involving OTC drugs and products affecting environmental health. That standard requires transparency of methods and use of methods accepted by experts in the field. In its proposal, PhRMA recommended that the FDA follow long-established FTC interpretation of the competent and reliable scientific evidence standard.

The full FR Notice on "Communications and Activities Related to Off-Label Uses of Marketed Products and Use of Products Not Yet Legally Marketed; Request for Information and Comments" can be found here.

It’s more than a notice – it’s a broadside and it’s required reading.  The docket should be interesting.  Folks – it’s time to step up to the plate.

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Happy Hannukah! At Long Last FDA Social Media Guidance. (Well, Sort of.)

12/27/2011 01:58 PM |

In time for the last night of Hanukkah – some long awaited (draft) guidance on social media.  Well, sort of.

According to the FDA:

This draft guidance is intended to describe the Food and Drug Administration’s (FDA or Agency) current thinking about how manufacturers and distributors (firms) of prescription human and animal drug products (drugs) and medical devices (devices) can respond to unsolicited requests for information about unapproved or uncleared indications or conditions of use (off-label information) related to their FDA-approved or cleared products. This draft guidance updates and clarifies FDA’s policies on unsolicited requests for off-label information, including those that firms may encounter through emerging electronic media.

“Emerging electronic media.”  You gotta love the lingo.

Further:

The rapid growth of the Internet, including social media tools and other emerging technologies, has made it easier for both consumers and health care professionals to quickly seek information about medical conditions and treatments.  Many firms have also used emerging electronic media to disseminate product information.  As a result, firms may encounter requests for off-label information about their products through product websites, discussion boards, chat rooms, or other public electronic forums that they maintain and over which they have full control.  In addition, third-party sites (i.e., websites and other venues that are either entirely independent of a firm’s control and influence or not fully controlled by a firm) also may reveal questions about off-label uses of a firm’s products.  These questions about off-label uses are typically directed to users of the site at large, rather than directly and privately to firms.  Such posted information is likely to be available to a much broader audience than just the original requester, especially because communication threads (i.e., questions and replies) are often available for an indefinite period of time.

This draft guidance provides FDA’s recommendations to firms wishing to respond to unsolicited requests for off-label information, including both requests made directly and privately to firms and requests made in public forums, including through emerging electronic media.  FDA recognizes that firms are capable of responding to requests about their own named products in a truthful, non-misleading, and accurate manner.  Furthermore, as these firms are regulated by FDA and have robust and current information about their products, FDA recognizes that it can be in the best interest of public health for a firm to respond to unsolicited requests for information about off-label uses of the firm’s products that are addressed to a public forum, as other participants in the forum who offer responses may not provide or have access to the most accurate and up-to-date information about the firm’s products.

Now here’s the important part:

If a firm responds to unsolicited requests for off-label information in the manner described in this draft guidance, FDA does not intend to use such responses as evidence of the firm’s intent that the product be used for an unapproved or uncleared use.  Such responses would also not be expected to comply with the disclosure requirements related to promotional labeling and advertising.  Firms may choose to respond to unsolicited requests for information about off-label uses of their approved or cleared products in a manner other than that recommended in this draft guidance.  Such activity would not constitute a per se violation of the law, but could potentially be introduced as evidence of a new intended use.

(Note: Bolded remarks throughout this discussion are my own emphasis.)

So, what’s the difference between “unsolicited” and “solicited?”  Here’s the FDA view:

Unsolicited Requests

Unsolicited requests are those initiated by persons or entities that are completely independent of the relevant firm.  (This may include many health care professionals, health care organizations, members of the academic community, and formulary committees, as well as consumers such as patients and caregivers).  Requests that are prompted in any way by a manufacturer or its representatives are not unsolicited requests.  Two types of unsolicited requests are addressed in this draft guidance: non-public unsolicited requests and public unsolicited requests.  Responses to unsolicited requests can likewise be non-public (private) or public.

Non-public unsolicited requests

A non-public unsolicited request is an unsolicited request that is directed privately to a firm using a one-on-one communication approach.

Example 1: An individual calls or e-mails the medical information staff at a firm seeking information about an off-label use.  In this case, neither the request nor the response would be visible to the public.

Public unsolicited requests

A public unsolicited request is an unsolicited request made in a public forum, whether directed to a firm specifically or to a forum at large.

Example 2:  During a live presentation, an individual asks a question, directed to a firm’s representative but heard by other attendees, regarding off-label use of a specific product.  This request is a public request. Similarly, a response by the firm that is conveyed to the same audience as the original question would be considered a public response.

Example 3: An individual posts a question about off-label use of a specific product on a firm-controlled website (or a third-party discussion forum) that is visible to a broad audience.  The request could be directed to a firm specifically or posed to users of a discussion forum at large.

This request is a public online request. Similarly, a response by the firm that is visible to the same audience as the original question would be considered a public online response.

Solicited Requests

FDA considers requests for off-label information that are prompted in any way by a manufacturer or its representatives to be solicited.  Such solicited requests may be considered evidence of a firm’s intent that a drug or medical device be used for a use other than that specifically approved or cleared by FDA. Although not exhaustive, the following examples illustrate what FDA generally considers to be solicited requests for off-label information.

Example 4:  If a firm’s sales representative mentions a use of a product that is not reflected in the product’s approved labeling and invites a health care professional to request more information, resulting requests would be considered solicited requests.

Example 5:  If a representative of a firm, such as a medical science liaison or paid speaker (e.g., key opinion leader), presents off-label use data at a company-sponsored promotional event (e.g., a dinner) and attendees then ask or submit requests for more information, these requests would be considered solicited requests.

Example 6:  If a firm issues to health care professionals business reply cards that are intended for use in requesting off-label information, presents statements or contact information in promotional pieces in a manner that solicits requests for off-label medical or scientific information (e.g.,  “Product X continues to be evaluated in more than 50 trials in a broad range of conditions and patients” and “Call 1-800-… for more information”), or displays a commercial exhibit panel suggesting a new indication (e.g., a sign that reads “Coming Soon, a new use for Product X”), requests made in response to these types of prompts would be considered solicited requests.

Example 7:  If a firm provides a phone number, e-mail address, uniform resource locator (URL), or username that is a word, alpha phrase, or alpha representation implying the availability of off-label information for its product, requests using this phone number, e-mail address, URL, or username would be considered solicited requests.

Example 8:  A firm asks or otherwise encourages users to post videos about their own uses of its product on third-party video-sharing sites (e.g., YouTube), which may result in video postings about an off-label use of its product.  If the firm’s initial request for posting of videos results in any questions about off-label uses, or if any off-label video posting made in response to the firm’s encouragement of video postings results in questions about the product’s off-label use, these questions would be considered solicited requests.

Example 9:  If a firm sends out packets of information to known bloggers or online consumer reviewers and encourages them to write about an off-label use of its product on third-party sites and this then provokes a discussion about that off-label use, any requests inquiring about the product’s off-label use as a result of these blogs, whether posted as comments to the third-party site or directed to the firm, would be considered solicited requests.

Example 10:  If a firm announces results of a study via a microblogging service (e.g., Twitter) and suggests that an off-label use of its product is safe and effective, any comments and requests received as a result of the original message about the off-label use would be considered solicited requests.

Example 11:  If a firm sets up a website that enables viewers to read prepared standard responses for the firm’s products that are generated from prefixed pull-down menus naming various disease states, including any standard responses related to off-label uses for the firm’s product, resulting requests for off-label information would be considered solicited.  Moreover, if this website makes it possible to use search terms to generate standard responses that go beyond the scope of the product information being requested, including off-label use information, resulting requests for and responses to such a search would be considered solicited requests.

For all of you out there who thought the FDA was going to answer all your questions and solve all your problems relative to social media and regulatory ambiguity, sorry. Here’s what the agency is telling you – just do what makes sense.

Or, put another way:

FDA has long taken the position that firms can respond to unsolicited requests for information about FDA-regulated medical products by providing truthful, balanced, non-misleading, and non-promotional scientific or medical information that is responsive to the specific request, even if responding to the request requires a firm to provide information on unapproved or uncleared indications or conditions of use. If responses to unsolicited requests fall within these parameters, FDA has not expected those responses to meet regulatory requirements for promotional labeling or advertising and has not considered these responses as evidence of intended use.  This draft guidance sets forth FDA’s current thinking on this topic, consistent with the Agency’s past policy statements about responding to unsolicited requests.

Now, here’s the fun stuff –

RESPONDING TO PUBLIC UNSOLICITED REQUESTS FOR OFF-LABEL INFORMATION, INCLUDING THOSE ENCOUNTERED THROUGH EMERGING ELECTRONIC MEDIA

While it certainly isn’t news, here’s the conclusion the FDA has reached regarding “the Internet” – The Internet has revolutionized communication, information-sharing, information exchange among systems, and collaboration, enabling consumers to become more proactive about their health and safety. Consequently, the Internet has become a widely used medium for manufacturers and distributors of FDA-regulated medical products to disseminate information. 

So stipulated.  Now here’s the interesting part

The Internet has also spawned a variety of social media tools that host online content primarily created and published by users other than the intellectual property owner or product manufacturer.  In some cases, this online content may not be accurate.  Because consumers increasingly use the Internet to search for information about medical conditions and treatments, firms may receive public requests for off-label information about their products through, for example, product websites, discussion boards, chat rooms, or other public electronic forums that they maintain and over which they have full control. Firms may also encounter requests for off-label information on third-party sites (i.e., websites and other venues that are either entirely independent of a firm’s control and influence or not fully controlled by a firm).  Questions about off-label use may be directed to the website users at large, rather than specifically to a firm.

FDA recognizes that firms are capable of responding to requests about their own named products in a truthful, non-misleading, and accurate manner.  Moreover, because firms usually have robust and current information about their products, it can be in the best interest of public health for a firm to respond to unsolicited requests for information about off-label uses of the firm’s products that are made in public forums, especially since other responders may not provide or have access to the most accurate and up-to-date medical product information.

However, because product information posted on websites and other public electronic forums is likely to be available to a broad audience and for an indefinite period of time, FDA is concerned that firms may post detailed public online responses to questions about off-label uses of their products in such a way that they are communicating unapproved or uncleared use information about FDA-regulated medical products to individuals who have not requested such information.  In this circumstance, communications to persons who have not requested information may promote a product for a use or condition for which FDA has not approved or cleared.  FDA is also concerned about the enduring nature of detailed public online responses to off-label questions because specific drug or device information may become outdated (e.g., new risk information may become available).

Here comes the “guidance” part of the draft guidance:

FDA makes the following recommendations to a firm that chooses to respond to public unsolicited requests for off-label information about its product(s), including those encountered through emerging electronic media.

If a firm chooses to respond to public unsolicited requests for off-label information, the firm should respond only when the request pertains specifically to its own named product (and is not solely about a competitor’s product).

The level of specificity of the question posed in a public forum is important in determining the appropriateness of a firm responding to the unsolicited request.

Example 13:  An individual poses the specific question “Can Drug/Device X be used for Condition Y” in a public forum (and this question is not prompted by or on behalf of the firm).  It may be appropriate for the firm to respond as outlined below because the question is unsolicited and specific to the firm’s named drug or device.

However, if an individual poses the non-specific question “What drug/device can be used for Condition Y” in a public communication thread and the firm manufactures or distributes Drug/Device X, which is not FDA-approved or cleared for Condition Y, the firm should not respond to the request because the question is not specific to Drug/Device X.

A firm's public response to public unsolicited requests for off-label information about its named product should be limited to providing the firm’s contact information and should not include any off-label information.

The firm’s public response should convey that the question pertains to an unapproved or uncleared use of the product and state that individuals can contact the medical/scientific representative or medical affairs department with the specific unsolicited request to obtain more information.

The firm’s public response should provide specific contact information for the medical or scientific personnel or department (e.g., e-mail address, telephone number, facsimile) so that individuals can follow up independently with the firm to obtain specific information about the off-label use of the product through a non-public, one-on-one communication.

In other words, a post along the lines of, “Hi Bob.  My name is Susan and I work for DRUG COMPANY NAME.  I saw your post regarding NAME OF PRODUCT.  That is an off-label use of the product.  For FDA-approved indications, here is a link to the official FDA label. If you would like to discuss this product at with our medical personnel, please call this toll-free number”

Not rocket science folks. The draft guidance continues:

After an individual has privately contacted a firm for more information regarding an off-label use of the firm’s product, the firm should provide a detailed response and maintain records following the parameters outlined in Section V of this draft guidance.  Therefore, any substantive communication about off-label uses for the product, in response to the original unsolicited off-label question, should occur solely between the firm and the individual who made the request.  Regardless of the fact that the original, unsolicited off-label question may have been available to a very broad audience, the firm should not make its detailed response with off-label information publicly available within the same forum.  For example, after the requestor has contacted the firm and provided a personal e-mail address to obtain an answer to the off-label question, the firm’s detailed off-label response, within the parameters outlined in Section V of this draft guidance, should be e-mailed to the requestor since this resulting communication will occur solely between the firm and the specific individual making the unsolicited request for the off-label information.

3. Representatives who provide public responses to unsolicited requests for off-label information should clearly disclose their involvement with a particular firm.

FDA recommends that a representative who responds to a public request clearly disclose in his/her public response that he/she is a particular firm’s representative and inform the requestor of the name of the firm representative or department to contact should the individual choose to follow up directly with the firm in a non-public forum for detailed information about the unsolicited request for off-label information.

4.  Public responses to public unsolicited requests for off-label information described in numbers 2 and 3 should not be promotional in nature or tone.

In addition to a firm’s contact and disclosure information, a public response should include a mechanism for providing readily accessible current FDA-required labeling, if any, for the product  (e.g., FDA-approved package insert and, if the response is for a consumer, FDA-approved patient labeling or, for new animal drugs, FDA-approved client information sheet).  The public response should not provide any promotional information.  For example, a public online response should include a direct link to the current FDA-required labeling, if any, but should not include links to any other information (e.g., product websites, product promotional materials, firm websites, third-party websites). Furthermore, the uniform resource locator (URL) or web address where viewers are directed to obtain the FDA-required labeling, if any, should not itself be promotional in tone or content (e.g., should not be www.bestcancercure.com).

If a firm responds to public unsolicited requests for off-label information, including those encountered through emerging electronic media, in the manner described above, FDA does not intend to use such responses as evidence of the firm’s intent that its product be used for an unapproved or uncleared use. Such responses also would not be expected to comply with the disclosure requirements related to promotional labeling and advertising.

What can we learn from this draft guidance?  Well, the most important lesson is that if we just follow our own common sense, social media is a greenfield of opportunity to help market products but – more importantly – to help advance the public health.

If you were waiting to be led by the hand by the "emerging social media" savants at the FDA -- sorry.

The full draft guidance is worth studying and can be found here.

And Happy Hanukkah!

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Ocean's 2011

12/22/2011 08:21 AM |

Well, it certainly has been an eventful 2011.

But the good news is -- you ain't seen nothin' yet.  (Or is that the bad news?)

In either event, thank you for your attention, comments and criticisms during the past year.  And here's to a healthy, happy and successful 2012.

See you all on the other side.

Cheers.

Peter

Peter J. Pitts  Read More & Comment...

Paul's Christmas Epistles

12/21/2011 08:43 AM |

Once upon a time, Paul Kim worked for both Congressman Henry Waxman (on the Energy & Commerce Committee) and Senator Ted Kenendy – giving him the honor of negotiating with FDA on the original PDUFA package and then, subsequently on the reauthorizations both on the House and Senate side.

As a panelist at CMPI’s PDUFA without the Politics conference, he had a lot to say.  (A video of his full comments can be found here.) Specifically, when it came to ornaments to hang on the PDUFA Christmas Tree he had two – both aimed at enhancing and incentivizing innovation.

There are bills that even in a very partisan environment find a lot of bipartisan support and interest in a bicameral way. And there were two I just felt I had to call out. They go to the innovation gap -- an issue and a set of concerns that are so much more resonate now in this economy and the role that the agency plays in promoting innovation. There are two proposals. The first is called the Gain Act. It’s sponsored by Congressman Gingrey - Dr. Gingrey, here in the house – and Senator Blumenthal in the Senate. Wonderful bipartisanship, bicameral support there. It would enhance incentives in terms of exclusivity for sponsors that are trying to generate and create and innovate on microbials. And given the enormous challenge that microbial resistance presents to public health, this is an area where it seems like there’s a classic market failure. There aren’t enough players in the field. The marketing incentives are not strong enough to draw interest, to draw investment. So, that’s a proposal that already has bipartisan support in both chambers. I think it’s the subject of a lot of discussions in the committees of jurisdiction. So, do look out for the GAIN act as a possible ornament somewhere in the upper branches of the PDUFA Christmas tree.

The second proposal is one that has also received bipartisan support, sponsorship and interest on both sides, Senate and House. And that’s to reform the humanitarian device exemption or HDE process.

Little known, it’s kind of the lesser known sibling to the Orphan Drug Act, which has promoted and really generated tremendous innovation for diseases, rare diseases and disorders affecting 30 some million Americans. When it was originally enacted in 1990, there was a profit cap. It said if you develop a device for a very, very small population, you can only charge for cost. It was intended as a safeguard, but instead what’s happened is it’s blocked and chilled interest in using that pathway to market.  The consequence is that we’ve had some 54 HDEs or Humanitarian Devices approved since 1990. In contrast, literally hundreds of orphan drugs have been approved since the enactment of that act. Again, it’s an issue that has bipartisan interest and support, a very small change to existing laws. It’s not overturning the apple cart, but I think you’ll see some significant changes in the marketplace among sponsors, investors, investigators, and scientists as a result of any federal change like that.

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Finally! A Congressional Action to Lower Healthcare Costs!

12/20/2011 08:14 AM |

Some important items courtesy of the good folks at BioCentury:

Finally – a Congressional action that will lower healthcare cost:

IPAB budget slashed

The appropriations bill passed last week by Congress cut 2012 funding for the Independent Payment Advisory Board by $10 million to $5 million. The Patient Protection and Affordable Care Act created IPAB and gives it authority to recommend steps to cut Medicare costs if spending exceeds specific triggers. The recommendations would go into force automatically unless Congress overruled them by a two-thirds vote and enacted other cuts of equivalent size.

IPAB has been criticized by members of both parties. Republicans said IPAB would lead to healthcare rationing. Democrats said they oppose IPAB because it usurps congressional authority.

Understanding the importance of medical technology after-the-fact

Senators introduce device bill

Sens. Chuck Grassley (R-Iowa), Richard Blumenthal (D-Conn.) and Herb Kohl (D-Wis.) introduced the Medical Device Patient Safety Act, which would allow FDA to require postmarketing studies for medical devices approved via the 510(k) process. The proposed legislation would give FDA the authority to revoke a device's approval if postmarketing studies are not conducted. Grassley said the legislation should be included in the reauthorization of MDUFA next year.

And prepare for March Madness

Supreme Court sets March date for healthcare challenges

The U.S. Supreme Court scheduled three days in March to hear challenges to the Patient Protection and Affordable Care Act, including the constitutionality of the law's individual insurance mandate. The court allocated five and a half hours spread across March 26, 27 and 28. On March 26, the court will consider a procedural question on whether the mandate challenge is barred by the Anti-Injunction Act. On March 27, the court will hear arguments on the mandate's constitutionality. On March 28, the court will consider whether the mandate, if found to be unconstitutional, can be severed from the rest of PPACA or if the entire law must be struck down. Later that day, the court will hear a challenge to PPACA's expansion of Medicaid to cover individuals with income of up to 133% of the federal poverty level.

The challenges are taken from three separate cases: National Federation of Independent Business et al. v. Sebelius, et al.; Florida, et al. v. U.S. Department of Health and Human Services, et al.; and U.S. Department of Health and Human Services, et al. v. Florida, et al.

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PDUFA Christmas Tree Ornament for Children

12/19/2011 08:54 AM |

O Tannenbaum, o Tannenbaum,
wie treu sind deine Blätter!

At CMPI’s PDUFA without the Politics conference, each panelist was asked to bring one ornament to hang on the reauthorization Christmas Tree.  Tim Franson (President of the USP Convention and one of the “Fathers of PDUFA”) offered a little lifeboat.

What can we do for those with life threatening unmet needs? One thing, as was done in PDUFA IV, is adding renewal of the Best Pharmaceuticals for Children’s Act. That piece of legislation has done more for pediatric drug development than anything in the past. It’s been good for American children and it should be made permanent. I think the better policy argument is to make it permanent, because companies don’t invest in five-year cycles. (Note to reader: The Act is currently renewable every five years.)

If I’m looking for a return on an investment, I need certainty well in advance of five years on types of studies I should be conducting and I need to know the benefit I’m getting at the end. I think five years is far too short a period of time.

Here’s what Vince Ventimiglia (a former Assistant Secretary for Legislation at the US. Department of Health and Human Services) had to say when asked about renewal vs. permanent authorization:

The argument I heard when we first set the five-year term, and that I heard again when we tried to make it permanent in subsequent years, was that we want to hold these guys accountable. It’s more to use it as a tool against industry -- and hold out the prospect that it could be removed.

To which Father Franson replied:

There’s an accumulated body of evidence, of companies fulfilling their commitments. Maybe its time we make it permanent. I think this point of holding up renewal like the Sword Damocles isn’t very persuasive even to sponsors.

O Tannenbaum, o Tannenbaum!
Das soll dein Kleid
mich lehren.

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Lead or follow?

12/16/2011 09:16 AM |

On August 18th, I wrote that pharmaceutical manufacturers should agree to voluntarily notify the FDA when they become aware of circumstances (manufacturing and otherwise) that might result in drug shortages,

(When I served at FDA we signed an MOU with PhRMA that required its member companies to inform the agency when instances of counterfeit medicines came to the attention of manufacturers. It got the job done – minus additional federal regulation.)

Shortly afterwards, Senators Amy Klobuchar (D, MN) and Bob Casey (D, PA) introduced legislation to require drug manufacturers to give “proper prior notification” to FDA when there is going to be a drug shortage.

Watch the Federal Register on Monday for an interim final rule from the FDA that requires manufacturers that are the only producers of critical drugs to report to FDA all interruptions in manufacturing processes, regardless of whether a drug shortage occurs. Previously, such manufacturers were only required to notify the agency when a drug shortage occurred. The rule goes into effect 30 days after being published.

This would have been a better and more interesting story if industry had taken the initiative rather than being directed to change its behavior.   Read More & Comment...

Do preventive services save money?

12/15/2011 04:28 PM |

The idea that preventive health care saves money is among the most ubiquitous and bipartisan health policy ideas out there. It’s an idea that numerous contenders for president in 2008, Democrat and Republican, endorsed. Prevention, President Obama has argued before Congress, “makes sense, it saves money, and it saves lives.” A recent poll found 77 percent of Americans believe that prevention saves money, with 56 percent believing so strongly.

What if we’re all wrong? What if prevention doesn’t save money?

Read Sarah Kliff’s piece in the Washington Post here.  Read More & Comment...

An UFA by any other name

12/14/2011 07:45 PM |

According to Plato, “They certainly give very strange names to diseases.” Well, he clearly never encountered PDUFA -- otherwise the might have hit the hemlock too.

Ask me and I'll tell you that PDUFA ought to stand for the "Predictability Deposit User Fee Act."  According to Tim Franson, one of the "Fathers of PDUFA," it means “Pharma’s Dollars Underwriting Federal Appropriations.” And when Paul Kim was on the Hill, he wanted to call it the “Giant Omnibus Drug and Medical Device Amendments Act” or GODDMAA.  

Alas – cooler heads prevailed.

But PDUFA is more than just a name -- it's a philosophy.  And as that well-known expert in FDA policy, William Shakespeare said, “There are more things in heaven and earth, Horatio, than are dreamt of in your philosophy.”

Well, neither the Bard nor Horatio could make it to “PDUFA Without the Politics,” CMPI’s Capitol Hill seminar on the fifth iteration of the original UFA, but many hornblowers and FDA cognoscenti did. I was the moderator and was honored to be joined by a panel of peerless nonpareils that included Vincent J. Ventimiglia, Jr (Former Assistant Secretary for Legislation at the US Department of Health and Human Services), Paul T. Kim (Former Deputy Staff Director for health policy for Senator Edward M. Kennedy), Michele J. Orza (Former Assistant Director of the Health Care Team at the Government Accountability Office), and Tim Franson (Former Vice President, Global Regulatory Affairs, Eli Lilly & Co., President, USP Convention).

The Honorable Michael C. Burgess, M.D. (Vice-Chairman, Subcommittee on Health, U.S. House of Representatives) offered remarks as well – specifically on the issue of FDA advisory committee members and conflicts of interest.

But – see for yourself. Full videos of all the presentations can be found here.

Why PDUFA without the politics?  Well – watch the presentations and I challenge you to identify any of the speakers’ politics from their astute commentary on the future of the FDA.  

Which is as it should be.
 

  Read More & Comment...

Ever metaanalysis you didn't like?

12/13/2011 09:40 AM |

A study published in the Journal of the American Medical Association (JAMA) shows that Ritalin, Adderall and other drugs widely used to treat attention disorders didn't increase the risk of serious heart problems. The findings, from an analysis involving nearly 500,000 adults, come on the heels of a separate study that reached a similar conclusion about the medications' effect in 1.2 million children and young adults.

According to an article in the Wall Street Journal, “Researchers and doctors who treat the condition -- known formally as attention deficit-hyperactivity disorder, or ADHD—said that together the reports should generally allay worries about heart risk that have stirred confusion among doctors and patients for several years.”

Fears about potential heart risk for ADHD medicines were sparked in 2005 by reports of about a dozen deaths from sudden cardiac arrest among young users of Adderall. The reports prompted the Canadian government to temporarily remove Adderall from the market, though it later lifted the suspension after it was determined that the patients who had died had heart defects or other underlying heart disease.

The FDA held advisory panels to discuss the matter in 2006 and subsequently required an update to product labeling to mention cardiac risk. The American Heart Association in 2008 urged that children being considered for ADHD drugs undergo a thorough heart exam, possibly including an electrocardiogram, before taking the medicines.

The FDA and other U.S. agencies funded both the JAMA study and an earlier one published last month by the New England Journal of Medicine. The earlier study, by the same research team, reached a similar conclusion among patients 2 to 24 years old.

The study in JAMA compared about 150,000 adult users of ADHD medications between 25 and 64 years old to nearly 300,000 nonusers. Researchers reviewed medical records from four health insurance plans to look at the number of serious cardiovascular events such as sudden cardiac death, heart attack and stroke among both groups.

Despite the findings, the FDA said Monday that patients treated with ADHD medications should continue to be monitored for changes in heart rate or blood pressure as well as for other side effects that include decreased appetite, weight loss and trouble sleeping. The agency also said the drugs "should not be used by patients with serious heart problems, or for whom an increase in blood pressure or heart rate would be problematic."

  Read More & Comment...

Me and Newt and 21st Century Healthcare

12/12/2011 07:57 AM |

In August 2005 I had the opportunity to interview Newt Gingrich on a variety of healthcare issues.  The questions and answers are as fresh as if we spoke this morning – and perhaps even more relevant considering the Speaker’s current position as one of the Republican front-runners.

Peter Pitts:  Hello Mr. Speaker.  Thank you for taking the time to chat with me.  

Speaker Gingrich:  I’m delighted to have the chance to talk with you. 

Peter Pitts:  Mr. Speaker, a lot of people talk about personalized medicine – but what do you think we need to do to make it a reality?

Speaker Gingrich:  In the long run, it’s going to involve DNA data because there are so many things we can learn, particularly with regard to cancer.  In the short run, it’s already starting to evolve.  Take a look at the iHealth record product, for example, where you could keep your personal information and send it to a doctor using encrypted email.  It’s an example of a baby step in the right direction.  I just saw a diabetes cell phone that is being developed in Korea and has actually been submitted to the FDA. It will allow those who have diabetes to monitor their condition on a cell phone, send the data to their personal health record and to their doctor, and even schedule a set time to remind them to check their blood sugar.  It sounds like a bit of magic, but it’s all coming down the road right now. 

Peter Pitts:  Do you think that pharmaceutical companies have the economic models to make personalized medicine a reality, and does the FDA have the tools to review them and approve them?

Speaker Gingrich:  Part of the reason why we are going to see more personalized, narrowly focused drugs by both pharmaceuticals, and even more important, biotechs, is that we are now entering a period where drug companies will be able to reach you in a targeted way. When a drug company rolls out a drug to certain people, it has to go to every doctor’s office in the hope that it will find the 12 doctors that actually need the specific drug. But if it can actually target electronically – for example, because we actually have electronic records now, we could contact directly the 30,000 people who are on Vioxx the morning there was a problem.  That’s the kind of step towards the model that I am describing, where you would know that there are x number of thousand people who have Lou Gehrig’s disease, and you can reach them instantly without an ad campaign.  You could also reach the doctors instantly and therefore, you would actually dramatically increase the net value of certain drugs because you would so dramatically lower both the loss time and the distribution time. 

Peter Pitts:  What would be the broad implications for that in terms of changing the overall health care model – to evolve from an acute care model to more of a chronic care model?  

Speaker Gingrich:  Well there is a difference, let me draw a distinction here. In acute care, you’re going to get more rapidly focused, narrow delivery of precisely what you need for your acute problem. A chronic condition will require lifetime management, which in some cases could mean diet, exercise, and attitude.  In other cases, it will be the application of appropriate medications.  It will be less profitable per unit, but there will be massively more units. Businesses will either use traditional acute care or orphan drugs. 

Peter Pitts:  How do you get physicians and patients in the mindset that they can be cared for individually and precisely, as opposed to waiting until something bad happens, then go for treatment? 

Speaker Gingrich:  Well part of it is an incentivization program.  I’ll give you two examples.  I was just at Des Moines University in Des Moines, Iowa, where they actually gave bonuses to faculty and students for being compliant on exercise, blood pressure, and the management of diabetes.   It’s a terrific program.  And they are beginning to have real impact because people are focused on their own behavior.  I also know that in the Mercy Health system in St. Louis, 76% of the blue-collar workers in a factory signed up for a personalized contract and an incentive plan – 93% of their diabetics are compliant.  So those are examples of focused incentives.

We are working in Georgia with 14 corporations and state government to begin to put together a Bridges to Excellence diabetes program that pays the doctors $100 in advance for managing diabetes.  With the patients in Cincinnati and Mobile, they’ve been saving about $150 a patient for a net of $250 per patient per year by incentivizing the doctor.  We believe that if you combine that with incentivizing the patient, you’ll get an even better response.  And with this new diabetic phone, you are going to get dramatic improvements in compliance.

Peter Pitts:  That’s an interesting point – the convergence of technology on the one side, and drugs and biologics on the other.  On the technology front, what role will medical devices or medical technology inside the body play in the future?

Speaker Gingrich:  I believe almost certainly, in the next decade, you’ll have the equivalent of a pacemaker/cell phone.  Your pacemaker will call 9/11 before you know anything is happening.  It will also send routine monitoring data.  I’m sure you’ve seen the shirts people wear that automatically keep track of vitals.  What will happen at one level is that all of this will become ubiquitous invisibly.   You won’t notice, but it will be constant. It will involve medicine with a wireless capability and a massive bandwidth, matched up with expert systems that analyze data.  Some of this is happening now.  

A facility in Oregon now puts a GPS locator on a band on Alzheimer patients’ arms.  They can walk all over the area without having to worry about getting lost.  They have wired their beds in such a way that the staff knows when they are laying down and sleeping soundly.  If they have a walker, the walker is wired.  If they can go to bed and in the middle of the night, go to the bathroom and later their walker doesn’t move, there is a presumption that they fell down and within two minutes the nurse comes to check.  It’s a very fascinating place and I strongly recommend that you look at it.  It’s an example of some of the things that you are asking about. 

Peter Pitts:  That’s definitely going to avoid a lot of problems and a lot of complications down the road.  What can the FDA, the National Health Institute, or the Centers for Disease Control, or government in general do to help? 

Speaker Gingrich:  Well, you have to remember that government is the largest payer.  It’s up to the government to make purchases intelligently, and to incentivize.  It’s up to the government to help incentivize patients with Type II diabetes.  It’s up to the government to transform Medicaid, and to have the discipline of change.  That’s part one. For part two, it’s up to the FDA to accelerate the introduction of entrepreneurial new products that are not life threatening.  The diabetes phone may be one of these examples.  You want to have very rapid flow of approval for things that are relatively easy to do.  

Peter Pitts:  And for those areas where approval doesn’t exist, how can the FDA reform itself to make that happen?

Speaker Gingrich: Part of it is changing minds and to say, if we don’t believe that there is something significantly dangerous about this, the bias should be in favor of approving it.  When you get to a very sophisticated electronic health record system with expert analysis, you’ll be able to approve things more rapidly because if something goes wrong, you can recall them more decisively.  So that increases the level of risk you can take.  

Peter Pitts:  The pharmaceutical industry is in such a hole right now in terms of their reputation, what can it do to dig itself out of the hole to become a more positive player? 

Speaker Gingrich: I think it should be clear to the pharmaceutical industry that they should focus on reaching and linking to an electronic health system, rather than producing massive commercials.  If you look at the amount of money they spent on commercials in the last two years, you could probably provide an electronic health record for every person in the country.  I think the more people see the pharmaceutical industry genuinely worried about people’s health, the better off we are. Second, I think the more we can move to a Travelocity model of selling drugs, where you have full information, know the strengths and weaknesses of each drug, what the side effects are, and the pricing, you are more in control.  I think that would dramatically improve the image of the drug industry and give people a sense again that they are in charge as customers.  

Peter Pitts:  That’s an interesting concept, to have the pharmaceutical industry adopt and fund electronic records for all Americans.  

Speaker Gingrich: If you look at what Pfizer is doing in Woodbury, Connecticut, they are in fact sponsoring information technology health care in that town. I think if the drug companies are focusing on reaching the customer in an informed way, designed to maximize health rather than profit, people would, in fact, understand that and be responsive to it. 

Peter Pitts: What’s the big idea to reform health care in this country?

Speaker Gingrich:  Well, I don’t know that it’s a single big idea.  We propose a Twenty- first Century Intelligent Health Care System. We propose three very large changes.  First, moving away from acute care to focusing on wellness, prevention, and early detection. Second, from focusing on the provider, to focusing on the individual, so we get you involved in your health before you ever become ill.  Third, that we take advantage of information technology, so that we maximize productivity, accuracy, speed – what people see everyday in other aspects of lives.  We think those changes together really would make a dramatic difference in both saving lives and saving money. 

Peter Pitts: How do you get Americans involved in their health care when they are not ill?

Speaker Gingrich:  By moving to Health Savings Accounts and Health Reimbursement Accounts – when you start getting bills, you start getting directly involved.   Second, if we should have a right to know pricing and quality legislation -- 93 percent of the country likes the idea of knowing the price and quality before making decisions.  I think the country is ready for change, but the politicians aren’t.  And I think 80 percent of the country favors electronic private health insurance for example.  

Peter Pitts:  What does your crystal ball tell you about legislation and price controls?  

Speaker Gingrich:  I think price controls are such a stunningly dumb idea.  It has failed totally almost everywhere in the world.  It has ruined the research components of pharmaceuticals in Canada.  The danger is that if we don’t move to a Travelocity model of individual control of purchasing, somewhere down the road, the government will start setting prices for what it pays for health care just because the prices are so staggering. For four thousand years of recorded history, when politicians have to choose between their own survival and your property, they choose your property and their own survival.  

Peter Pitts:  That’s interesting, so basically replacing the debate over price with a practical movement forward in personal involvement.  Thank you.

  Read More & Comment...

Trade and health: a new agenda for the WTO

12/09/2011 02:28 PM |

At CMPI's second policy round table lunch in Geneva, we were lucky enough to have Fredrik Erixon, the head of Brussels trade think tank ECIPE, as speaker. He spoke on the need for a new international trade compact focused exclusively on health.
 
During the lunch, which was attended by a 25 high ranking Geneva diplomats and officials,   Fredrik set out the case that such a new trade compact would invigorate trade negotiations, while helping countries drive down costs.
 
Fredrik argued that the Doha Round is dead and it looks unlikely that the WTO will ever again prove to be a useful forum from which to negotiate global trade liberalisation. As mercantilism is still a dominant philosophy throughout the world, trade is unlikely to be liberalised further unless it is clear there will be clear gains in trade volumes. Trade negotiations need therefore to become less ambitious and focus on specific, clear issues.
 
Meanwhile, both developed and emerging economies are facing huge fiscal pressures as their populations age. Healthcare already comprises 20 per cent of the service sector of most higher and upper-middle income countries, with costs rising all the time due to factors such as technology, labour costs and so on. In other sectors, these inflationary pressures have been tempered via international trade, as countries are able to trade across borders to exploit their comparative advantage and lower costs.  However, almost no trade exists in healthcare outside of pharmaceuticals and other medical goods.
 
At the same time, emerging economies such as China are developing new healthcare systems. As they are starting from a very low base, it requires enormous amounts of capital. They too would benefit from international trade in healthcare in order to attract investment and capital from overseas.
 
All these factors underline the need for a new trade compact in healthcare.  Richer countries would derive great advantage from utilising communications technologies to have diagnostics and other analysis performed in countries where such things can be done more cheaply. There is also great potential to use such technologies to keep patients at home rather than in capital-intensive hospitals. Emerging markets could tap into this demand to help them mobilise the capitals and skills necessary to help them develop modern healthcare systems.
 
So what can be done? First, there needs to be a thorough reworking of the rules surrounding trade in services. Only a few (mainly developing) countries have made firm commitments under the WTO's General Agreement on Trade in Services (which governs the rules on trade in healthcare). Rich countries therefore need to put healthcare services at the front of their trade strategies. Second, healthcare needs to be included in the WTO’s Government Procurement Agreement to ensure that state healthcare systems are part of this international process. Third, work needs to be done to reduce non tariff barriers to trade in healthcare, such as technological and regulatory barriers.
 
Although medical tourism will become increasingly popular amongst richer people it is unlikely to become a mass market phenomena as it is paid for mainly out of pocket. Rather, using technology to allow patients and their physicians to communicate with low cost, foreign service providers -without having to go to a hospital- could be a potential game changer, driving down costs and improving the quality of care. To make this happen, there needs to be a new global compact for trade in healthcare.

  Read More & Comment...

The Wheels on the Bus go 'Round and 'Round

12/09/2011 01:20 PM |

Solid reporting from Matt Herper over at forbes.com:

Did The Obama Administration Throw The FDA Under The Bus?

Susan Wood quit her job as an assistant commissioner for woman’s health at the Food and Drug Administration in 2005 because she thought political, not scientific, forces were delaying efforts to make the Plan B emergency contraceptive available without a prescription. Four years later, she rejoiced when she was present to watch President Barack Obama sign a statement that said decisions in his administration would be based on science, not politics.

Now she says she is beyond disappointed. Obama’s Secretary of Health and Human Services, a member of his cabinet, Wednesday made a new decision to prevent a further expansion of Plan B’s over-the-counter use, this time to adolescents, overruling not only FDA staffers but also the drug agency’s commissioner, Margaret Hamburg. Although the HHS secretary, Kathleen Sebelius, was within her legal authority under the 1938 law that created the FDA, this is the first time a presidential administration has ever publicly overruled the FDA in this manner.

“This is contrary to the scientific integrity memo signed by Obama,” Wood says. “It’s contrary to the whole principal of making decisions based on science and evidence.”

Wood says the decision sets “a terrible precedent for the whole breadth and depth of the FDA” and that the “medical and scientific expertise to make such decisions and recommendations truly resides in the FDA,” not elsewhere in Sebelius’ department. The only silver lining, Wood says, is that by publicly standing up to Sebelius with a letter of protest posted on the FDA’s web site, Hamburg may have prevented the decision from demoralizing rank-and-file FDA staffers.

President Obama asserted yesterday that he “did not get involved in he process” but backed Sebelius’ decision. Whatever the merits of the plan B decision, using Sebelius’ power in this way may tarnish what has been one of the Obama administration’s great, pro-business achievements: Hamburg’s successful transformation of an embattled FDA, scarred and damaged by the controversies over big pharmaceutical industry scandals like Vioxx and Avandia, into an agency that is approving new medicines and a near-record clip and clearly and efficiently deals with new controversies as they crop up.

The question is whether this decision is a one-off, or a change in the way medicines are regulated. Could future negotiations with drug companies be weakened by the fear that the FDA commissioner would be overruled? And even if Sebelius is always going to back up Hamburg on every other regulatory decision, what about future administrations?

David A. Kessler, who headed the FDA between 1990 and 1997, praises the Sebelius and Hamburg for being transparent by airing their disagreement in public. And he says that for many decisions like Plan B, even if they are based on science, reasonable people can disagree. “There’s no scientific textbook where you can look up the answer,” he says. “You can respect people coming out on different sides.” His worry is not about whether 11-year-olds should need a prescription for Plan B, but about what happens in the future if decisions traditionally made by FDA scientists become cabinet-level political issues.

“My concern is just that it’s lousy precedent,” says Kessler. “The last thing you’d want is to have this be seen as precedent so any new drug applicant sees the decision-maker as the secretary. That’s not the way it’s ever worked or should work.”

Others put their opinions even more bluntly. “The decision on Plan B by the secretary represents an unprecedented level of interference in the FDA process and puts the country on a slippery slope toward politicization of science,” says Steven Nissen, a physician who has been critical of the FDA in the past. “Future political leaders may choose to overrule the FDA on much more critical decisions.” Nissen said he was not speaking for his employer, the Cleveland Clinic, where he is chairman of cardiology.

Peter Pitts, a former FDA associate commissioner who now works at the Center for Medicine in the Public Interest, a right-leaning think tank that receives drug industry funding, echoed those views, calling the decision “outrageous.” Pitts says: “It opens up the hornet’s nest of companies lobbying HHS, of members of Congress lobbying HHS. It’s a Pandora’s box of a decision.”

Not everyone agrees Sebelius’ move will have such far-reaching effects. For one thing, political pressures have in the past been brought more privately on the FDA – it’s not clear that the public argument between Hamburg and Sebelius really changes things. Ramsey Baghdadi, a founder at Prevision Policy, a consultancy, and an editor at the RPM Report, says Plan B is “a unique situation that demonstrates how FDA has to grapple with the intersection of science, politics and policy all at once.”

Baghdadi says it is possible that the Plan B controversy could lead Hamburg to exit the FDA. He calls this “highly speculative,” but notes that Hamburg was appointed when former Senator Tom Daschle was expected to head HHS; she was not a Sebelius pick.

Plan B is thought to generate only about $200 million for its maker, the generic drug giant Teva, and the new regulation was seen as only a minor issue in terms of public health. But the emergency contraceptive, which reduces the chance of pregnancy after unprotected sex by half, is one of a series of issues that have made the FDA into a political football over the past decade, often at times when the agency was without leadership.

The controversy over Merck’s painkiller Vioxx, which increased patients’ risk of heart attacks, resulted in congressional hearings that, in turn, resulted in tougher new drug approvals and voluntary industry limits on drug promotion. Safety issues and badly conducted clinical trials with the antibiotic Ketek helped make antibiotic approvals so tough that drug companies seemed to abandon the field en masse. The backlash over the diabetes drug Avandia led to new hurdles for heart safety for medicines to lower blood sugar. The drama has hurt the drug industry.

Hamburg’s leadership has more or less turned this around. She handled the controversial decision over whether to withdraw Roche’s approval to market Avastin for breast cancer with aplomb, backing her scientists that the data did not support the drug’s use. Although bad for Roche, many in the drug industry saw this as a positive step, because there were fears that not revoking Avastin’s approval would have made it harder for experimental drugs to be approved in the future.

In the fiscal year that ended Sept. 30, the FDA approved 35 new medicines, the second-highest count this decade. New initiatives appear to be making it easier for companies to get clear guidance as to what they need to get new drugs approved. For the first team in years, the FDA seems anything but rudderless – a change that is great for the pharmaceutical industry, which depends on strong, clear, and consistent regulation. The Plan B decision may have set back that turnaround, and, if saner heads don’t prevail in rebuilding the wall around the FDA, it could do long-term damage to medicine in the years to come.

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Making Pharma More Productive

12/09/2011 09:57 AM |

This essay on how to improve R&D productivity rightly focuses on the need to be more targeted and value driven. But it begs the question of what to do about an FDA unwilling to adjust regulatory requirements that reward value driven development? http://www.oliverwyman.com/media/OW_EN_HLS_Publ_2011_Beyond_the_Shadow_of_a_Drought(2).pdf  Read More & Comment...

Katy -- Bar that door!

12/08/2011 01:29 PM |

Whether or not you feel that Plan B (aka -- "The Morning After Pill") should be available OTC to adolescents, the bigger issue (MUCH bigger) is the dangerous precedent of politicizing the FDA process.
 
The morning after Secretary Sebelius' unprecedented overruling of the FDA's decision one thing is clear -- the door is now wide open to anyone who has a beef over any decision rendered by Peggy & Co.
 
The line forms to the left.
 
When asked directly if the White House had weighed in on this matter, the HHS press office refused to comment. In other words, yes.  Can you imagine the hue and cry if this had taken place during the previous administration?
 
Having served as Associate Commissioner at the FDA during the first round of Plan B hysteria, I can personally attest to the heat and scrutiny it generated.  And appropriately so. The significant difference is that debate was internal to the agency. There were differences of opinion -- but the decisions (whether you agree with them or not) were FDA decisions.
 
Leaving aside the peculiar politics of reproductive health, this action by the Secrerary must be reversed. Left standing it will severely undermine the authority of the FDA and embolden those who think that political arm twisting should be used to influence agency decisions.
 
Katy -- bar that door!  Read More & Comment...

FDA Slowing Progress on Progressive Approval

12/07/2011 12:30 PM |

The "word" is that FDA wants to  delay progressive approval by defining clinical benefit on a case by case basis and by front loading Phase I with all sorts of endpoints and metrics that it would be tantamount to going through all the hoops.   The point of the Critical Path initiative was to increase certainty and reduce the need for extensive human experimentation.  Instead, patients and doctors would tell innovators and the FDA how treatments are working in the real world.

The FDA would have a hand in writing regulations and guidances for accelerated approval.  But it seems to me that the agency should not be the one to determine clinical benefit especially when the scientific community and patients can do that.  It should just verify that products are doing what they were designed to do.  The pushback on progressive approval suggests that legislative action may be required to truly bring the FDA into the 21st century.  Read More & Comment...

Social Media, Pharma, and Cats in Sweaters

12/07/2011 07:59 AM |

Yesterday I was pleased to chair Day 1 of the Social Media for Pharma event in New York City.

First off, this and similar events can no longer (and should no longer) be identified as “pharma” events. Witnessing today’s attendance by and active participation of members of the medical technology (née “device”) community along with a full table of dietary supplement marketers, makes it pretty clear this and similar symposia are healthcare events. 

And that’s a good thing.

A few memorable moments:

Greg Kueterman and Amy O’Connor of Eli Lilly & Company gave a splendid presentation on their health policy blog Lillypad (www.lillypad.lilly.com).  One of their most important points (not rocket science, but rarely discussed) is that, when it comes to healthcare in the social media space – it can be about things other than product marketing and that metrics are more than about volume.  It’s not about the top ten, but rather the right ten. Amen.

Greg asserted that he's “the Justin Bieber of healthcare social media.”  Not sure what that means, but it does seem to be an off-label claim.

William Martino (Saatchi & Saatchi Wellness) and Shermon McMillan (Chattem) – henceforth forever known as Shermon/William – painted an interesting portrait of social media and the OTC launch (and subsequent marketing) of Allegra. 

Ned Russell (Saatchi & Saatchi Wellness) pointed out that the oft-quoted FaceBook development maxim of “move fast and break things,” couldn’t be more antithetical to regulated healthcare communicators (note – not marketers, but communicators).  My comment was that this might explain the tone deafness of the world’s largest social media platform has to issues such as moderated user-generated commentaries.  (Also note that the FCC didn’t find FaceBook’s swiftness to cast aside privacy issues so kosher either.)

And, while we’re on the subject, why does FaceBook allow “drugs from Canada” pages.  Did they not get the memo from Google and the DOJ?

Tai Spargo of NORD discussed their new social media efforts in creating a plentitude of orphan disease sites minus any participation of the biopharmaceutical industry.  She also noted (and rightfully so) that there is no rare disease too small for social media.  To illustrate her point she displayed the FaceBook page “Cats in Sweaters.” Meow.

Check it out if you are so inclined at http://www.facebook.com/pages/Cats-in-sweaters/183921988289175. Idle hands indeed do the devil’s work.

Sharon Landa (Johnson & Johnson) and Erica Wittes Schlack (Communispace) discussed the design and utility of private (read, “closed”) social media communities.  Fascinating.  Two comments to pass along, “Feedback is about the brand, but discovery is about the consumer” (utility) and “Diversity is for conversation but similarity is for cohesion (design).

There was also much chatter about how to make social media programs work along side of “traditional communications initiatives.”

Rather than calling them “traditional,” perhaps we should label them as “legacy” programs.

And we all know what happens to those.

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AHIP on HSAs

12/06/2011 05:56 PM |

Politico notes that AHIP’s new report on health-savings accounts contain some interesting findings:
 
America's Health Insurance Plans is out with a new report on the state of health savings accounts. Among the findings: Lower-income individuals open HSAs at a greater rate than do higher income groups - with an average income in the mid-$50,000 range - and the amount of money that individuals have accumulated in their accounts has grown over time, and the amount of money being rolled over from one year to the next also has increased.
 
Click here to read the report.  Read More & Comment...

Just in Time for the Holidays: FDA Approves Hangover Drug

12/05/2011 02:16 PM |

FDA Approved Hangover Medication Hits Market.

The New York Daily News (12/3, Pesce) reported, "A new FDA-approved hangover cure has hit the market." The over-the-counter medication, dubbed Blowfish, "combines 1,000 milligrams of aspirin, 120 milligrams of caffeine and a stomach-soothing agent into two effervescent tablets taken the morning after a night of heavy drinking." Blowfish "costs $2.99 for a single dose, or $11.99 for a six-pack" and "is currently available in Ricky's NYC stores or online at ForHangovers.com."

        The website of WCBS-TV New York (12/5) reports, "Just in time for the holidays, the FDA approves a purported hangover remedy. Blowfish, created in the West Village, is promised to get rid of all your hangover symptoms within 15 to 30 minutes." FOX News Radio (12/4, Rappoport) added that that the drug "combines aspirin, caffeine and a stomach soother into two effervescent pills to be taken the morning after a night of partying." The remedy's creator Brenna Haysom "says the pill is only intended for people who had one too many, not binge drinkers."


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PDUFA: Process or Provision?

12/05/2011 07:15 AM |

At CMPI’s “PDUFA without the Politics” Capital Hill Briefing, Dr. Tim Franson (President, USP Convention, former Vice President, Global Regulatory Affairs, Eli Lilly & Co., and one of the “father’s of PDUFA”) mentioned the Hawthorne Effect – “that which is measured gets accomplished.”

Which begs the question – should more things in PDUFA V be measured or measured with more robust deliverables?

Are “meetings” sufficient promises – or are actions more desirable?  Specifically – are meetings about a benefit/risk grid enough or should there be a date-certain for a regulatory instrument to be operational?

Should PDUFA V be exclusively about process? What about provision?

Speech is conveniently located midway between thought and action, where it often substitutes for both.  – John Andrew Holmes

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