The Food & Drug Administration has apparently decided that "early communications" are best delivered late in the week.Today its Botox.
Last week was Chantix.
Two weeks ago, it was Vytorin and ENHANCE.
Keep your Friday afternoons open.
Showing posts with label FDA. Show all posts
Showing posts with label FDA. Show all posts
Friday, 8 February 2008
Parallel reviews by the Food & Drug Administration and the Centers for Medicare & Medicaid Services may be just around the corner.According to a research note put out by Stanford Group’s David Blaszczak, Greg Frykman and Jan Wald earlier this week, the agencies are close to issuing a Federal Register notice that will solicit comments on a voluntary program that would allow a manufacturer to receive an approval from FDA and a national coverage decision from CMS at approximately the same time.
The idea for a parallel review process was first proposed after Mark McClellan left his post as commissioner of FDA to become administrator of CMS in 2004. Given his experience at the heads of both agencies, it seemed natural that McClellan would be interested in a closer relationship between FDA and CMS, but plans for a demonstration project were eventually scrapped.
Questions about whether the agencies were starting to work more closely together resurfaced during the erythropoietin safety debate. Rather than wait for FDA to conclude its safety review of EPO, CMS conducted a simultaneous assessment and issued a national coverage decision before FDA had reached a final conclusion on restricted labeling. We have covered that story extensively in The RPM Report; subscribers can click here and here to read all about it.
So what would parallel reviews mean for industry? The majority of manufacturers certainly won’t be pleased about the potential for FDA and CMS to work more closely together: the Stanford team notes that drug and biologic sponsors are likely to complain about a more extensive FDA review process, which could slow down drug approvals. Confidentiality is also likely to be a top complaint.
But industry’s queasiness also stems from a fear that parallel reviews could blur the line between two agencies with two very different missions: FDA’s review of safety and efficacy, and CMS’ determination of whether coverage is “reasonable and necessary.” Inevitably, the conversation turns to whether cost would start to become a factor in either decision—the same reason that most of industry remains uneasy about a national center on comparative effectiveness.
Since the proposed process would be voluntary, it’s likely most manufacturers won’t take advantage of it. But Stanford believes that “forward-thinking” companies should consider it, under the following circumstances:
• they have a potentially successful product anticipated for, or in registrational development,
• that is likely to cause a paradigm shift in the management of one or more serious and life-threatening diseases and;
• for which premium pricing is under internal consideration.
If nothing else, the proposal should serve as a reminder that sponsors should avoid waiting until after FDA approval to open coverage discussions with CMS. On the contrary, that exchange needs to take place early and often.
Wednesday, 6 February 2008
We know all of you have been passing the time following the Presidential Primaries when the race you’re really interested in is who the next head of FDA’s drug center will be, right? Right. A number of agency watchers and former officials agree that the longer the search goes on, the more likely FDA will be forced to look inside for a replacement. We talked to one former high-ranking FDA official with some knowledge of the search and here are some of the things we found out.
First, FDA Commissioner Andrew von Eschenbach is “concerned” that the recruitment for a director of the agency’s Center for Drug Evaluation & Research is taking so long and “not looking fruitful.” FDA chief medical officer Janet Woodcock is temporarily overseeing the center’s day-to-day operations until a permanent replacement is named. The absence of a CDER director has been magnified by the vacant director position at the Center for Food Safety and Applied Nutrition.
“He didn’t want the two largest centers without permanent leaders for a long time,” the former official says.
That’s why von Eschenbach made filling the CFSAN vacancy a priority. On January 4, FDA announced Center for Veterinary Medicine director Steven Sundloff would take over the food center.
There’s been some serious difficulty getting a large number of qualified external candidates, the ex-FDAer maintains, since the October 2007 departure of former CDER director Steve Galson due to his appointment as acting US Surgeon General. That’s understandable, according to the individual: “Who’d want a job when you won't know who your boss will be a few months out, where public perception is on the down slope, there’s little likelihood of getting substantial new appropriations, and you won't even be allowed to say you need more resources.”
That’s a convincing argument against wanting the job, wouldn’t you agree? When the search began in October 2007, an outsider taking over the drug center appeared to be a slam dunk. But now, as the French would say, c’est pas tout a fait evident. (Note:
It appears that a serious internal candidate has, in fact, emerged.
We don’t know who it is, but we can speculate can’t we? Sure we can.
1) Center for Biologics Evaluation & Research Director Jesse Goodman:
As we noted in our earlier CDER search story, Goodman was the only FDA insider garnering serious consideration for the job at the outset. Goodman, a virologist, was named CBER director at the end of 2002, replacing Kathy Zoon, who moved to the National Cancer Institute.
The CBER head came to FDA in 1998, originally in the Office of the Commissioner directing the US Interagency Task Force on Antimicrobial Resistance. He later moved to CBER, where he worked on bioterrorism preparedness and blood and vaccine safety, eventually becoming deputy director before taking over for Zoon.
Most importantly for Goodman’s chances, he’s shown that he can run a large center within the agency’s organizational structure that operates in a similar fashion to CDER.
Odds: 3 to 1
2) CDER Deputy Director Douglas Throckmorton:
Time and time again, when the CDER search is brought up to FDA observers and former agency officials, I hear: “Why not Throckmorton?” Well, why not? Throckmorton would appear to be the obvious choice considering he has been the center’s number two for the last two and a half years. He served in an acting capacity for a year prior to that.
Before his promotion to deputy director, Throckmorton headed up the cardio-renal drugs division—a very high profile review group within FDA.
He also serves as chair of FDA’s Drug Safety Oversight Board, CDER’s liaison to the agency’s human subjects’ research review board, and chair of the center’s research coordinating committee.
Throckmorton has been involved in some interesting FDA meetings as of late. On January 9, “acting for” Woodcock, Throckmorton made a presentation to students and faculty at Duke University’s Fuqua School of Business along with Office of Medical Policy director Robert Temple. We know, we know, not the strongest evidence that he’s next, but still.
On January 17, the CDER deputy was involved in a senior CDER management meeting with PhRMA board representatives, including soon-to-be-retired Eli Lilly CEO Sidney Taurel, Lilly VP-global regulatory affairs, and PhRMA deputy VP for scientific and regulatory affairs Alan Goldhammer.
Meetings aside, Throckmorton has been viewed as somewhat of a rising star at FDA. He joined the cardio-renal drugs group in 1997 as a medical reviewer. Three years later, he was named deputy director of the division, and director two years after that.
So what’s the problem? One theory is that Throckmorton is too young. That seems a bit dubious considering all of his current responsibilities and fast rise up the career ladder. But if FDA were going to name Throckmorton as Galson’s replacement, they would have done it by now. After all, they passed on giving him the “acting” title altogether in favor of Woodcock, who previously ran the drug center.
Despite initially getting passed over, I still think he’s a very strong candidate and running right behind Goodman.
Odds: 5 to 1
3) Office of New Drugs Director John Jenkins:
Okay, we admit we are a bit in the tank for Jenkins. He recently took the time to speak at length with The RPM Report about the state of new drug approvals (aka the drug approval drought), drug safety, and the new drug reform regulations under the FDA Amendments Act. You can read it here if you haven’t already.
Before the interview, we thought Jenkins was a smart thinker and effective spokesman on regulatory issues. We are more convinced than ever afterwards.
Jenkins’ position alone as FDA’s top drug reviewer is enough for him to warrant consideration. He oversees 17 drug divisions and a number of important office-level groups within CDER. To see them all, click here.
You could argue that Jenkins’ ability to keep drug review times in check—actually reducing priority and standard review times in 2006—while reviewers are spread increasingly thin under a climate of poor morale makes him the most prepared to take over the drug center.
Case in point, FDA/Sponsor meetings have more than doubled over the last five years and Jenkins has calculated in the past that drug reviewers have nine industry meetings every working day of the year. In addition, new drug applications have increased in the 10% range while FDA’s budget has remained flat.
Publicly, Jenkins has been even more impressive. During a June House Oversight & Government Reform Committee hearing looking into the handling of heart risks linked to Avandia, Jenkins performed admirably before a host of Congressional lawmakers looking for blood.
He was equally impressive during a media briefing on January 25 to discuss how FDA planned on handling data from Schering-Plough/Merck’s ENHANCE study on the statin Vytorin. Click here to read our take on FDA’s response to questions about the Zocor/Zetia combination.
Jenkins is also an FDA veteran with 16 years under his belt, six as the head of the Office of New Drugs, and considered to be one of Woodcock’s “people.” One thing that could preclude him from getting the job is that the whole Vioxx debacle occurred while he was in charge of OND. But if that’s the case, none of the internal candidates listed here stand a chance of getting the CDER job.
Odds: 7 to 1
4) Office of Medical Policy Deputy Director Rachel Behrman:
Ah, the Dark Horse. There’s always a dark horse candidate. When you flip through the obvious internal candidates, Behrman doesn’t come to mind. However, one former FDA official says Behrman could be the most serious internal candidate.
Behrman was picked by Woodcock to head the Office of Critical Path Initiatives in the Commissioner's Office--an important priority for the agency in general and Woodcock in particular. That is in addition to her role as second-in-command to the dean of drug development Robert Temple in the medical policy office. And in her spare time, Behrman also serves as Director of the Cross-Centers Initiatives Task Force. That’s just the kind of collaborative position FDA Commissioner Andrew von Eschenbach has tried to promote within the agency.
Behrman joined FDA in 1989 and has some experience in the spotlight that the CDER director operates under. For instance, she testified before the Senate Special Committee on Aging regarding in 2005 regarding the impact of direct-to-consumer advertising on seniors. The full testimony is right here. The fact that the Division for Drug Marketing and Communication reports into the Office of Medical Policy probably serves as an advantage as DTC continues to remain a public and political focus.
Behrman’s obvious drawback as a candidate is that she has less direct experience with product reviews than the other candidates. Of course, if FDA's first choice would be an outsider, that drawback could actually be a selling point to the search committee.
Odds: 15 to 1
Tuesday, 5 February 2008
Welcome to the battle of the budget numbers.To hear it from the Bush Administration, the president’s fiscal 2009 budget request for FDA is quite generous. The $2.4 billion request is a 5.7% increase over the previous year—a nice little boost in an otherwise tight budget environment. And it really looks good when you compare it to the 3% budget reduction the president is recommending for the Department of Health & Human Services overall.
As FDA chief operating officer Jim Dyer put it yesterday, the president’s budget request will “maintain momentum” over 2008 funding levels, allowing FDA to “target the critical areas that have been identified by the Commissioner and the Secretary.” The increase, he said, “gives us a real good start and lays the groundwork of where we need to go.”
Others would dispute that analysis. The Alliance for a Stronger FDA, which has been advocating for greater funding for the agency, notes that the Administration’s budget is an increase of just 2.9% in appropriated funding (from $1.72 billion to $1.77 billion). The balance comes from user fees paid to FDA by industry, which would increase 14.4%.
“FDA is in critical need of significant new resources,” William Hubbard, a former deputy commissioner and Alliance member said. “The amount in the Administration’s proposed budget is not only inadequate, it is barely half of what FDA needs just to keep pace with inflation.” What FDA really needs, the Alliance says, is an additional $380 million in appropriations, or seven times the Administration’s request.
The battle over funding for FDA was previewed during a House Oversight & Investigations subcommittee hearing last week. The topic was an FDA Science Board report that found the agency to be so deficient in its scientific and technological capacities that it is unable to meet its regulatory responsibilities. You can find our earlier post on that here.
And money is the biggest obstacle: while FDA has received generous raises in user fees over the years for activities like new drug reviews, other areas—like drug safety, information technology and guidance development—have largely been funded by appropriated dollars, which haven’t kept up with inflation.
Last week’s House hearing demonstrated the conundrum FDA faces. Congress loves to beat up on FDA for not asking for more money, but then doesn’t actually give the agency any more money to do its job. It’s a vicious cycle: the agency can’t do its job without more money, and it can’t get more money because it’s not doing its job. Once the dust clears, that’s an awful lot of time and money spent on getting nowhere.
The Bush Administration seems to think it’s time for FDA and Congress to get back to work on developing a follow-on biologics approval pathway.
The president’s $2.4 billion fiscal year 2009 budget request for FDA lays out an agenda for an abbreviated approval process on follow-on, or “generic,” biologics. “The budget proposes a new authority for FDA to approve follow-on protein products through a new regulatory pathway that protects patient safety, promotes innovation, and includes a financing structure to cover the costs of this activity through user fees,” the request says.
During a conference call, FDA deputy commissioner for operations Jim Dyer said the agency would work with Congress to submit legislation authorizing an abbreviated pathway for follow-on biologics. That’s not really news; the agency has been in discussions with Capitol Hill and industry stakeholders for some time, and has testified in congressional hearings that it has the scientific expertise to support an approval process.
Legislation authorizing a follow-on biologics pathway came close to being attached to the drug safety/user fee bill last year, but was pulled at the 11th hour. For more coverage on the bipartisan negotiations—and what the final deal looked like—click here and here. Subscribers to The RPM Report can read the content for free, or you can sign up for a 30-day free trial.
Practically speaking, the mention of follow-on biologics in the budget request won’t result in much. Finalizing something as controversial as an abbreviated approval process for follow-on biologics during an election year is more than a little optimistic; any real work probably won’t get underway until there is a new president in the White House in 2009.
But the budget request does set the president’s agenda for FDA for the next fiscal year, and lays out what the White House hopes to see the agency accomplish. At the very least, the mention of follow-on biologic user fees will trigger a score from the Congressional Budget Office, which, should it demonstrate savings to the health care system, would be handy during the next round of negotiations.
The request also includes a call to revive user fees for pre-reviews of direct-to-consumer advertising television commercials. A program was enacted as part of the FDA Amendments Act, but Congress killed it off by refusing to fund it in the omnibus appropriations bill. (For more analysis of that story, click here.)
So the Administration, at least, hasn’t given up on that program. Of course, we already knew FDA and industry were eager to make it work—it is Congress that has been of two minds on the DTC program—enacting it in September and killing it in December.
So don’t get too excited about follow-on biologics or DTC user fees. The President’s budget keeps hope alive for action on each this year. But it doesn’t change the basic truth that both issues depend primarily on the priorities of a Democratic Congress in an election year—not the final budget from the outgoing Administration.
The president’s $2.4 billion fiscal year 2009 budget request for FDA lays out an agenda for an abbreviated approval process on follow-on, or “generic,” biologics. “The budget proposes a new authority for FDA to approve follow-on protein products through a new regulatory pathway that protects patient safety, promotes innovation, and includes a financing structure to cover the costs of this activity through user fees,” the request says.
During a conference call, FDA deputy commissioner for operations Jim Dyer said the agency would work with Congress to submit legislation authorizing an abbreviated pathway for follow-on biologics. That’s not really news; the agency has been in discussions with Capitol Hill and industry stakeholders for some time, and has testified in congressional hearings that it has the scientific expertise to support an approval process.
Legislation authorizing a follow-on biologics pathway came close to being attached to the drug safety/user fee bill last year, but was pulled at the 11th hour. For more coverage on the bipartisan negotiations—and what the final deal looked like—click here and here. Subscribers to The RPM Report can read the content for free, or you can sign up for a 30-day free trial.
Practically speaking, the mention of follow-on biologics in the budget request won’t result in much. Finalizing something as controversial as an abbreviated approval process for follow-on biologics during an election year is more than a little optimistic; any real work probably won’t get underway until there is a new president in the White House in 2009.
But the budget request does set the president’s agenda for FDA for the next fiscal year, and lays out what the White House hopes to see the agency accomplish. At the very least, the mention of follow-on biologic user fees will trigger a score from the Congressional Budget Office, which, should it demonstrate savings to the health care system, would be handy during the next round of negotiations.
The request also includes a call to revive user fees for pre-reviews of direct-to-consumer advertising television commercials. A program was enacted as part of the FDA Amendments Act, but Congress killed it off by refusing to fund it in the omnibus appropriations bill. (For more analysis of that story, click here.)
So the Administration, at least, hasn’t given up on that program. Of course, we already knew FDA and industry were eager to make it work—it is Congress that has been of two minds on the DTC program—enacting it in September and killing it in December.
So don’t get too excited about follow-on biologics or DTC user fees. The President’s budget keeps hope alive for action on each this year. But it doesn’t change the basic truth that both issues depend primarily on the priorities of a Democratic Congress in an election year—not the final budget from the outgoing Administration.
Monday, 4 February 2008
Sometimes coming up with a headline for our weekend roundup can be difficult. This was not one of those times.While we went with what we figured was the most universal of Super Sunday experiences, we had choices. Also considered: While You Were Watching It For the Ads, While You Were Giants, While You Were Eli-d, While You Weren't Going Undefeated, While You Were Choking, While You Were Jumping Off the Pats' Bandwagon, While You Were Participating in the Unauthorized Use of Game Footage Without the Express Written Consent of the NFL, and While You Were Praying There Would Be No Tom Petty Wardrobe Malfunction.
Some of us here at the IN VIVO Blog are Eagles fans (no, really, we probably haven't mentioned it before) and so the only suitable Superbowl outcome would have been for both teams to get disqualified somehow. But alas, at least it was a tight game.
Believe it or not there was more than Super Sunday and Super Tuesday prognosticating going on this weekend. Or so we heard.
- Amgen and Takeda inked a broad alliance to develop and commercialize 13 Amgen products in Japan. In a second and related deal, Takeda becomes Amgen's worldwide partner for the Phase II oncology candidate AMG-706 (motesanib). Amgen gets up-front payments of $200 million and $100 million respectively, and a further hundreds of millions in milestones and R&D expense sharing, plus royalties on Japan sales (and worldwide 50/50 profit sharing on motesanib). Takeda is also acquiring Amgen's Japanese subsidiary Amgen KK.
- From the Financial Times: a preview of GSK's forthcoming results this week with one key question--can the Big Pharma get FDA approval for Cervarix? We weighed in on this question on Friday.
- [Self Promotion Alert] The line-up for our Pharmaceutical Strategic Outlook conference in New York (March 18-20) keeps on getting better.
Photo from Flickr user jwinfred used under a Creative Commons license
Thursday, 31 January 2008
Drug companies aren't the only ones worried about the sinking rate of new drug approvals. Food & Drug Administration officials are equally concerned over the innovation drought. After all, the number of new drugs making it to market is at its lowest since 1983.FDA's Office of New Drugs Director John Jenkins, who oversees all new drug applications within the drug center, is especially preoccupied with the lack of results from the drug development process. "We agree that it’s very disheartening that despite the rather massive expenditure of research dollars, we’re not seeing a growth in the number of NMEs submitted to the agency for review," Jenkins says of new molecular entities getting aproved by the agency. "We are seeing a continued growth in the number of new commercial INDs submitted, so there still seems to be a lot of innovation. It’s a question of how to get them out the other end of the pipeline."
Jenkins also addressed other issues ranging from drug safety to how FDA plans to prioritize implementing the new drug reform regulations under the FDA Amendments Act. In particular, he addressed the public disagreement between the drug review and drug safety groups during the Avandia advisory committee meeting last July.
"Consensus is not our goal," Jenkins says. "That strikes some people as odd when they first hear me say that, but I think that if you’re in a regulatory organization and people think that consensus is the goal, that leads to a subtle pressure to conform to the prevailing viewpoint even though you may not agree with the prevailing viewpoint and you may in fact be right."
You can read the whole interview in The RPM Report by clicking here. Free registration for non-subscribers is on the left side; subscribers should just log in.I would love to hear your comments on Jenkins' views on FDA, drug companies and drug development.
Wednesday, 30 January 2008
It’s déjà vu all over again.“Either we’re going to make sure this agency has the resources necessary to do its job, or we’re going to watch it continue to deteriorate.”
That’s Rep. Henry Waxman, speaking yesterday at a hearing of the Oversight & Investigations subcommittee of the House Energy & Commerce Committee. The topic: an FDA Science Board report released late last year that found FDA to be so deficient in its scientific and technological capacities that it is unable to meet its regulatory responsibilities.
Wonder why? It comes down to one simple reason, and it shouldn’t be a surprise to anyone: the regulatory demands on FDA have increased exponentially as funding for the agency has deteriorated.
But wait—didn’t the Prescription Drug User Fee program, reauthorized as part of the drug safety overhaul, fix this problem?
Alas, no. Over the years, FDA’s new drug review activities have received relatively generous funding thanks to PDUFA. It’s everything else—information technology infrastructure, scientific research to support regulatory standards, the crisis du jour that drains resources from everything FDA does—that is the problem.
The modest increase in FDA’s budget for non user fee programs is nowhere near enough to solve that problem, much less support the new authorities the agency received in drug safety. But, hey, we could have told you that—check out our earlier story on FDA’s funding crisis in The RPM Report.
In the words of former FDA chief counsel Peter Barton Hutt, who helped put the Science Board report together, that chronic underfunding has left FDA “barely hanging on by its fingertips.” What’s needed, Hutt recommended, is a doubling of FDA’s budget over the next two years, a 50% increase in employees, and a 5.8% cost of living adjustment every year thereafter—numbers that had chairman Bart Stupak’s head spinning.
Maybe that is why Stupak seemed more interested in bashing von Eschenbach over specifics in the Science Board report. One highlight was a long exchange between the two over what von Eschenbach had requested for funding in the President’s fiscal 2009 budget. Stupak wanted specifics he could compare to President Bush’s numbers; von Eschenbach demurred until the budget is released next week.
You might have thought that after the passage of the drug safety law, Congress would have left FDA alone for a while to implement it. But it doesn’t look like that’s in the cards this year: Stupak promised Commissioner Andrew von Eschenbach at least five more appearances in front of his Oversight & Investigations Subcommittee alone this year. And that’s on the heels of a packed 2007 Hill schedule for von Eschenbach and career FDAers.
So what does this mean for drug companies? Well, it’s not good news: when FDA officials are hauled up to Capitol Hill and bashed over the head for doing a poor job, that doesn’t reflect kindly on the industries it regulates. And as Gail Cassell, Lilly’s VP-scientific affairs testified, when advances in science outstrip FDA’s ability to regulate, that means new therapies don’t get approved in a timely manner.
Perhaps the saddest thing about the FDA Science Board report is that none of the findings are new. The Institute of Medicine and, most recently, the Government Accountability Office, have issued similar reports on the chronic underfunding of the agency. You can find them here and here.
Waxman closed his remarks with this comment: “I hope the Science Board report will be the last report we have to read about the desperation at the Food & Drug Administration.” You can bet Waxman isn’t alone in feeling that way.
Friday, 25 January 2008
The Food & Drug Administration is weighing in on the Vytorin controversy via an "early communication" on the study results.Given the hue and cry over the drug since Merck and Schering-Plough finally disclosed disappointing data from the ENHANCE trial January 14, many may wonder how FDA can say it is "early" with its response. After all, didn't Steve Nissen, the United States Congress, and seemingly every media outlet in the country already tell us what to think?
As far as Vytorin goes, the FDA announcement indeed is decidedly anti-climactic. The agency summarized the results already made public by the company, and said it would review the data fully once it is submitted by the sponsor. On a media call, Office of New Drugs Director John Jenkins said it may be another couple of months before the data is submitted, and up to six months before the agency completes its review.
And, Jenkins added, it is not like there is much to expect from the regulatory review itself. Vytorin is already clearly labeled as not showing any demonstrated benefits in long term outcomes data. The agency will be looking carefully for any new safety information in the data, Jenkins said, but it is not aware of any red flags at this time.
So not much new there.
But there is an important way in which FDA is getting out in front of the Vytorin story. One of the primary messages of the early communication and the media call was to try to diminish speculation that the ENHANCE trial calls into question one of the basic tenents of cholesterol therapy: the focus on LDL levels as a key marker for treatment.
The agency's main message: it remains confident that LDL reduction is a fully validated surrogate endpoint as a basis of approval for cholesterol treatments--and that LDL reduction is an appropriate therapeutic goal for physicians and patients. (Recall that the Schering-ENHANCE study failed to show the expected benefit of combination therapy with Vytorin versus single-agent simvastatin, despite showing significantly better LDL reductions.)
"It would be premature to embark on any systematic change in how we approve lipid-lowering drugs," Jenkins said. "We have a long track record" of success in basing approvals on LDL, he noted.
In fact, Jenkins added, LDL reduction is a much more fully validated than the arterial plaque endpoint used in ENHANCE.
"It is tempting to think of" changes in arterial plaque "as a direct measure of what these drugs do," Office of Medical Policy Director Robert Temple added. But "that may not be the best measure." He noted that statin drugs show improved outcomes in a matter of months--before any measurable changes in arterial plaque are observed.
"The surrogate that really works is LDL cholesterol," Temple stressed. "I'm very concerned that this will lead to people becoming indifferent" to their LDL levels, rather than seeking treatment.
That message may or may not help Vytorin in the marketplace--but it is great news for all cholesterol drug development companies.
Friday, 18 January 2008
No one really wants to shout it from the rooftops, but for FDA and the drug industry, Monday's Supreme Court’s decision not to consider whether terminally ill patients have a legal right to access unapproved drugs is a little more sweet than bitter.The decision by the high court ends a long, topsy-turvy, and often emotional legal fight by the Abigail Alliance to allow patients access to investigational drugs and biologics outside the clinical trial setting. (The alliance is named for Abigail Burroughs, who passed away in 2001 while trying to gain access to the then-investigational cancer drug cetuximab, now marketed by ImClone and Bristol-Myers Squibb as Erbitux.)
Along with the Washington Legal Foundation, the Abigail Alliance sued FDA in 2003 to allow access to unapproved drugs after the completion of Phase I studies. A Washington, DC district court initially dismissed the case, but the decision was overturned by a three-member panel of the US Court of Appeals. This August, an en banc appeals court reversed the decision, prompting WLF to ask the Supreme Court to consider the case.
Appealing to the Supreme Court is always a long-shot legal strategy, but the high court’s consideration of the Abigail Alliance lawsuit could have resulted in significant changes for FDA and industry. Check out full coverage of the Abigail Alliance lawsuit in The RPM Report here and here. If you don’t yet subscribe, you can sign up for a free trial at TheRPMReport.com.
For FDA, a win by Abigail Alliance would have meant changing its regulations to make it easier for patients to access investigational medicines. FDA has had mechanisms in place for patients to access unapproved drugs outside the clinical trial setting since the 1970s, including “treatment use” and “emergency use” INDs—mechanisms that the Abigail Alliance argued are insufficient and overly bureaucratic.
But FDA argued that it has a “compelling interest” to restrict some patients from getting investigational drugs—namely protecting them from what might be an unsafe product. And given the intense congressional scrutiny over drug safety since the Vioxx withdrawal, you can bet FDA doesn’t have warm and fuzzy feelings about giving really sick patients a drug that hasn’t yet passed the approval hurdle.
For manufacturers, expanding access would have presented a tricky dilemma: balancing the goodwill gesture of granting a patient a dying wish against the threat of litigation should something go wrong. That’s not a comfortable place to be: Amgen’s outside attorney Mark Gately (Hogan & Hartson) said he “dreaded the day” that FDA changed its regulations—or a court ordered it to do so.
But it doesn’t look like that day will come anytime soon. FDA obviously isn’t budging, and it’s unclear whether a new commissioner would change its legal interpretation of the issue. Congress could pass legislation to expand patient access to unapproved drugs, but the Abigail Alliance’s big advocate on Capitol Hill, Sen. Sam Brownback (R-Kansas), doesn’t seem to be making it a top priority this session.
The Washington Legal Foundation’s press release has an air of finality to it, but it isn’t conceding defeat: “We will continue our effort to persuade FDA that terminally ill patients deserve better access to drugs that FDA has deemed suitable for large-scale clinical trials.” But we’re not going out on a limb to say that’s a long-shot proposition.
Thursday, 10 January 2008
Lots of interesting responses to our post on the historically bad year for new drug approvals this year.One top R&D exec at a big pharma company focused on the last line: “Something needs to change.”
“It does and it is. If we can steer clear of major disruptions I am convinced that we can turn this round. A lot of change needs to happen, but directed at improving the process of choosing targets, getting them into man and to proof-of-concept quickly then streamlining a clumsy development engine.”Another reader in a Big Pharma R&D organization writes somewhat less optimistically:
“I am sure everyone in the industry is thinking of ways to ‘innovate’ out of this situation and I believe the next year or two is going to be interesting for us all.”
And then there is this pragmatic response from the VC side:
“Depressing but very interesting. This shows why venture capitalists should leave it to the pharmas to try to get drugs approved!”Speaking of depressing, we also received congratulations from a colleague at another publication for managing to work in a Philadelphia sports reference in a post about NME approval statistics. The depressing part is that it has been 25 years since a major Philly sports team won a championship. Yikes.
There is lot’s more to say about the state of R&D productivity. We have taken a deeper dive into the numbers on TheRPMReport.com, and coupled it with some of the observations of top industry executives at The RPM Report's FDA/CMS Summit.
There is one surprise: as bad as the past several years have been by all conventional measures of R&D output, there is a glimmer of hope. The optimistic view, that we are on the brink of an unprecedented flow of innovative new products—just might be right. (You do have to be a subscriber to The RPM Report to read our complete analysis, or sign up for a 30-day trial to get a taste of what you are missing.)
Please Note: our initial count of drug approvals in the IN VIVO Blog was off by one. It turns out there were 17 new molecular entity approvals, not 16—Fresenius Kabi’s hypovolemia agent Voluven (hydroxyethyl starch) was approved December 27.
Voluven was approved under the 505(b)(2) mechanism as equivalent to other blood volume enhancers, so it definitely does not add to our benchmark statistics (innovative commercial therapies, or ICTs). The extra NME also doesn’t change anything else in our analysis: 17 NMEs is still the lowest total since 1983, as is 19 novel molecules (NMEs plus novel biologics).
Voluven is nevertheless an interesting approval: it is the latest example of the emerging follow-on biologics pathway at FDA.
What’s that? You think there is no such pathway? Not so. Congress has yet to enact a legislative pathway for follow-on versions of biologics regulated under the Public Health Service Act. But for biologics that happen to be regulated under the FD&C Act (like human growth hormone, insulin, insulin-like growth factor, etc. etc.) follow-on approvals keep trickling out of the agency. Look for more on that topic as well, coming soon in The RPM Report.
Wednesday, 9 January 2008
Let’s hear it for the United States Congress. They aren’t too proud to change their minds—at least, not when it comes to tackling the question of how best to respond to those pesky TV commercials for prescription drugs.In September, Congress enacted a new user fee program to fund pre-reviews of direct-to-consumer television ads, on the premise that both industry and society would benefit by ensuring that the Food & Drug Administration could offer constructive feedback on ads before they air.
The program, part of the FDA Amendments Act, set some tight timelines for FDA and industry to get the system up and running. Together, they got their acts together, crossed all the Ts and dotted the Is, and got the program up and running. FDA even began doing pre-reviews pursuant to the guidelines.
All for naught. In December, Congress changed its mind. In the omnibus appropriations bill signed the day after Christmas, Congress did not fund the new user fee program, and instead gave the agency $4 million in additional money from the Treasury to cover the cost of pre-reviews.
Since FDAAA sets a hard stop to the user fee program—FDA must collect the first round of fees before the end of January—there is now essentially no chance that the Pay TV program (as we liked to call it) will happen.
You have to feel bad for the industry and FDA negotiators who had to herd all the cats to hammer out the new user fee agreement.
Still, on paper at least, this turn of events is great news for advertisers. Rather than paying a fee of over $80,000 per commercial to get FDA’s feedback, they can get it for free. And, in theory at least, FDA can hire just as many new reviewers, but at the taxpayer’s cost—not industry’s. So the agency should be able to provide timeline and predictable responses as planned under Pay TV.
What’s not to like?
Quite a lot in fact. First, there is the thorny question of what happens next year and beyond. Unlike the user fee program, which was intended to run for five years and would have built a reserve fund to ensure stable funding for the ad review group, there is no guarantee that Congress will continue to provide additional funding to support the pre-review program.
That in turn may make it hard for FDA to follow through on its hiring plans. The agency doesn’t want to hire new reviewers this year only to have to lay them off in September when the current fiscal year ends. And even if FDA decides to take that chance, will they agency be able to recruit enough people willing to take a job that could turn out to be short term?
The agency has not decided yet how it will proceed, but promises it will explain its plans soon. (Our guess: FDA will wait until the FDAAA deadline to collect the fees—January 28—and then make the announcement as part of a formal withdrawal of the notices creating the new fees.)
Bear in mind that while pre-reviews are technically voluntary, we think advertisers would be very wise to use that process rather than risk facing the new enforcement actions Congress gave FDA under FDAAA.
Consider the dilemma advertisers will be in if FDA cannot or simply does not provide timely responses. Run an ad and risk a hefty fine? Or wait for an answer—and in effect surrender the right to advertiser that industry fought so hard to protect during the FDAAA debate.
The collapse of the user fee program doesn’t change two important facts for advertisers. First, FDA now has a much stronger hand in shaping TV ads—both in determining if products are advertised as well as what ads look like. (Subscribers to The RPM Report can read more here. Not a subscriber? Sign up for a free trial.)
More importantly, no matter what FDA does in 2008, you can bet that this issue is coming back in 2009. President Obama (didn’t I read yesterday that he had won?) or whomever takes over the White House will select a new FDA commissioner and you can bet DTC will remain a hot button issue.
The Energy & Commerce Committee offered a timely reminder of that fact by opening an investigation into Pfizer’s Lipitor commercials. Those commercials have been held up as examples of the new, more responsible approach industry is taking to DTC. (You have to ask yourself: has the committee seen Pfizer’s new “Viva Viagra” ad?)
Friday, 4 January 2008
The old adage comparing the unattractiveness of political decision-making with sausage making may apply to Food & Drug Administration drug approval decisions in the future.If Iowa Senator Chuck Grassley has his way, every FDA drug advisory committee may soon replicate the Avandia debate from last summer -- when the head of the new drug review office in charge of the original Avandia approval debated the head of FDA's drug safety office in an open airing of differing opinions from within the agency. My colleague Ramsey Baghdadi wrote compelling about the drama at the Avandia meeting last summer. (Click here to read the story.)
In the most recent version of what has become an annual year-end epistle to FDA on the agency's handling of the approval of Sanofi-Aventis' antibiotic Ketek (telithromycin), Grassley demands that FDA start bringing the wide range of different staff opinions on new drug applications out in the open for the advisory committee and the public to hear.
Until recently, FDA has tried to present its advisory committees with consensus positions on drug applications and scientific issues in the applications. But after investigating Ketek, Grassley finds that approach too glossy and a way to avoid public oversight of tough scientific decisions.
The Avandia meeting should be the new model. Grassley prodded FDA to adopt the same format where “FDA reviewers were allowed to express their professional opinions and recommendations regarding those drugs.” In case FDA missed the gist of his message, the senator repeated: “It is important not only that advisory committees be provided with complete and accurate data but also any differing scientific opinions and/or assessments regarding the data from FDA reviewers.”
And NDA sponsors should watch how FDA responds to Grassley's pressure. If he succeeds in opening up the FDA advisory committees to more disputes between FDA staff, he could well bring the all the blood and guts of an Iowa sausage factory to the drug review process. (See TheRPMReport.com for more coverage of the prospects, implications and background on more open debates at FDA advisory committees.)
Thursday, 3 January 2008
When does a drought stop being a drought, and just become a desert?That question has to be raised when contemplating yet another disappointing year for innovative pharmaceutical launches in the US.
FDA approved just 17 new molecular entities in 2007—the lowest single year total since 1983, when there were 14 NME approvals.
FDA’s official tally will probably be 19, including two therapeutic biologics approved by the Center for Drug Evaluation & Research. FDA began including biologics in its total in 2004, so that makes historical comparisons difficult. But even if you count all 19, this was still the worst year since 1983.
That’s 25 years ago, folks. Gandhi won best picture. Toto won album of the year. A Philadelphia sports team actually won a championship.
That’s 25 years ago, folks. Gandhi won best picture. Toto won album of the year. A Philadelphia sports team actually won a championship.
If you want to understand the decline in productivity industry wide, consider this: total R&D spending by brand-name companies in 1983 was $3.2 billion, compared to $43 billion in 2007. In other words, the industry spent $228 million per NME approved in 1983, compared to $2.5billion each in 2007. Or, if you prefer, the extra $40 billion in R&D spending brought with it a total of five additional therapies.
Big Pharma didn’t have anywhere near as many mouths to feed in 1983 either. The entire domestic brand business was just under $17 billion, according to data reported by the Pharmaceutical Research & Manufacturers of America trade association. Domestic sales of brand companies today are ten times higher. (If you haven’t read Roger Longman’s post yesterday about the importance of adjusting industry infrastructures, please do so now.)
Of course, looking at any single year doesn’t tell you anything about the overall health of the new product flow in the industry. Pharmaceuticals do have a relatively long commercial life, so as long as there is a health bolus of new products every few years, things should be fine.
Unfortunately, looking across multiple years doesn’t make the picture any brighter. Last year was the worst for new product launches since 1983. The second worst? 2005. Third worst? 2006. Fourth? 2002. In fact, FDA has approved more than 30 novel molecules only once this decade, when it cleared 36 in 2004. FDA approved more than 30 every year in the second half of the 1990s.
Or consider this: over the past three years, FDA has approved a total of 61 new molecular entities and novel biologics. The agency approved 60 in 1996 alone.
We like to track our own statistic, Innovative Commercial Therapies. That represents our attempt to measure the number of truly novel molecules (no enantiomers, metabolites or pro-drugs, where the basic question—is it safe and effective in humans?—has already been answered; no diagnostics; and no non-commercial products like biodefense agents developed by the Department of Defense.)
We think that gives a more accurate indication of the real output of big pharma and biotech pipelines. That only makes the picture that much grimmer: there were just 14 ICTs in 2007. Below are the statistics over the past decade.
If you divide that chart in half, you can see that FDA approved a total of 199 ICTs in the six years from 1996 through 2001. That compares to just 122 in the six years from 2002 through 2007, a decline of 39%.
Okay, enough gloom and doom. Looking on the bright side, at least there were fewer first time generic launches in 2007 than there were new molecular entity approvals. As we reported last year, for the first time in memory the industry suffered a net loss of patented medicines in 2006.
Of course, it was a close race. By our count, there were 14 first time generic launches in 2007, balanced against the 16 NMEs. And boy is it hard to imagine the crop of new drugs launched in 2007 matching the commercial peaks of the brands that lost exclusivity—products Norvasc, Ambien, Lamisil, Coreg, and Protonix.
And, since the industry suffered a net loss of two patented molecules in 2006, that means that the entire pharmaceutical industry has only stayed even in the number of patented medicines on the market for the past two years. That, to put it mildly, is not a recipe for sustained growth in the industry in the years ahead.
Bear in mind that, not only is the absolute number of new product approvals declining, so is the likely peak market size for new products. In other words, at a time when the industry desperately needs the pipeline to pump out more new products than ever, it is getting only a trickle.
Something needs to change.
Please Note: This post has been updated.
Our initial count of drug approvals in the IN VIVO Blog was off by one. It turns out there were 17 new molecular entity approvals, not 16—Fresenius Kabi’s hypovolemia agent Voluven (hydroxyethyl starch) was approved December 27.
Voluven was approved under the 505(b)(2) mechanism as equivalent to other blood volume enhancers, so it definitely does not add to our benchmark statistics (innovative commercial therapies, or ICTs). The extra NME also doesn’t change anything else in our analysis: 17 NMEs is still the lowest total since 1983, as is 19 novel molecules (NMEs plus novel biologics). Voluven is nevertheless an interesting approval: it is the latest example of the emerging follow-on biologics pathway at FDA.
What’s that? You think there is no such pathway? Not so. Congress has yet to enact a legislative pathway for follow-on versions of biologics regulated under the Public Health Service Act. But for biologics that happen to be regulated under the FD&C Act (like human growth hormone, insulin, insulin-like growth factor, etc. etc.) follow-on approvals keep trickling out of the agency. Look for more on that topic as well, coming soon in The RPM Report.
Wednesday, 2 January 2008
Congress finished some important work before leaving town for the holidays, finally enacting new funding legislation for the federal government for fiscal 2008. The bill, signed by President Bush December 26, is critical for the Food & Drug Administration, because it allows the agency to start using the new, higher user fees it was authorized to collect by the drug safety law signed in September.That means the agency can continue its full-steam ahead approach to implementing the user fee provisions of the FDA Amendments Act—the one piece of the legislation that both FDA and industry share unreserved enthusiasm for.
But the new funding comes with a big hitch: a provision stipulating that FDA cannot transfer any money to its other initial priority in implementing the law: the new Reagan-Udall Foundation, a public-private partnership that is supposed to help FDA develop new scientific tools to enhance its regulatory mission.
That may not sound like much to worry about. The new foundation is broadly supported by industry, which welcomes any opportunity to advance drug development science—but it seems like an afterthought in the context of the big changes in FDA’s regulatory authority over drug safety that are the centerpiece of the new law.
There are, however, very significant implications to the dispute, which could ultimately be critical to determining what role industry will have in the development of a new active surveillance system for pharmaceuticals.
Representative Rosa DeLauro, who chairs the House Agriculture Appropriations Subcommittee that oversees FDA, is concerned that the foundation may be an avenue for the pharmaceutical industry to exert more influence over the agency. She registered her concerns in a letter to FDA November 1.
FDA didn’t help the situation when it selected the board members for the foundation. The agency chose a very distinguished group to oversee the foundation, including former GlaxoSmithKline R&D President Tachi Yamada, who now heads the Gates Foundation’s global health program. On paper, Yamada is a perfect choice—his industry R&D background and current philanthropic position are hard to beat when considering the type of people who should oversee the foundation.
But the timing of the choice was disastrous. FDA announced the board selections on November 15—the same day that the Senate Finance Committee released a report on its investigation of claims that GSK intimidated an academic researcher who questioned the safety profile of Avandia. The report reviews a number of communications between GSK and the researcher, including emails from Yamada, and concludes that “the documents in the Committee’s possession raise serious concerns about the culture of leadership at GSK.”
Given DeLauro’s position that the foundation is an avenue to give industry undue influence over FDA, that made the selection of Yamada seem like a blunder.
Industry should hope that cooler heads prevail, and that DeLauro’s concerns can be assuaged. The Reagan-Udall Foundation should be a positive for drug development, and so pharma wants it to get off the ground.
But it is also the most viable short-cut to getting moving on a new active surveillance system for pharmaceuticals. The new law directs FDA to set up a public-private partnership to help build the database and develop the tools for analyzing signals to make regulatory decisions. The agency is leaning towards giving that mission to the new foundation—McClellan was one of the champions of including the provision in the FDA law in the first place.
Putting the new foundation in charge of developing the active surveillance system would address industry’s biggest concern with the project—whether product sponsors will have an appropriate role in shaping the new system.
That’s where things get really tough. DeLauro is already concerned about industry influence in the foundation. And Chuck Grassley, the ranking Republican on the Senate Finance Committee who oversaw the Avandia report, wants to play a role in shaping how the active surveillance project develops—at least to the extent it involves Medicare claims data. And Grassley’s view is that industry should have no role in analyzing that data.
So the fight over Reagan-Udall is probably just beginning. For industry the stakes may be higher than they seem.
Tuesday, 18 December 2007
This was the email that went out to Shortly after arriving at UCSF as Dean, I discovered a series of financial irregularities that predated my appointment. I reported these issues to appropriate university officials at the time, and have endeavored to work with the university ever since to solve these problems. The university characterized me as a whistleblower. During the summer, Chancellor Bishop requested my resignation. I continued to try to solve these problems. Yesterday, Chancellor Bishop terminated my appointment as Dean, effective immediately. Over the course of the past four years, it has been my pleasure and honor to work with the outstanding faculty, staff, students and donors of this remarkable school and institution. I want to thank all of you for the opportunity to foster and nurture outstanding programs on behalf of UCSF.
Sincerely,
David A. Kessler, M.D.
Professor of
Pediatrics, and Epidemiology and Biostatistics
The news came as somewhat of a shock to the academic community and Washington policy observers who followed Kessler when he was FDA commissioner under the Bush I and Clinton Administrations from 1990 to 1997. UCSF disputes Kessler’s account of the school’s finances.
It’s not the first time Kessler has stirred up controversy or been the target of an investigation into his handling of financial issues. Prior to his voluntary resignation as FDA commissioner in 1996, Rep. Joe Barton (R-Tex.) accused Kessler of overbilling the government for expenses of just under a $1,000. Kessler eventually wrote a check to FDA for the full amount.
After stepping down from FDA, Kessler subsequently took a position as Dean of Yale Medical School in 1997. After a six-year run at Yale, where it, in September 2003, Kessler was appointed Dean of the UCSF School of Medicine.
Kessler is not the first former FDA commissioner to have been involved in a dispute over the use of institutional funds. Former commissioner Donald Kennedy was forced to resign as president of
Kessler says he will remain at UCSF as a professor but it’s hard to imagine that could last very long considering the nature of Kessler’s firing as dean. The RPM Report noted earlier this year that Kessler had reemerged from his quiet life as an academic and into the public spotlight as a very vocal observer in the wake of criticisms against FDA.
Kessler participated in a roundtable discussion of former FDA commissioners organized by the George Washington University School of Public Health in February. Kessler showed up a few months later at a May 7 House Oversight & Government Reform hearing on food safety.
At the time, we thought Kessler was angling for an appointment in a possible Democratic administration in 2008—maybe as head of NIH. But this latest spat with UCSF almost assuredly quashes any possibility of that happening. A new administration won’t be looking for a nomination fight early on in what looks to be another closely divided election.
But Kessler could still end up in
However, it’s unlikely Kessler will find a position as prominent as dean of the UCSF medical school. And it’s all but certain that Kessler will be left out of the running for a coveted appointment if the Democrats take the White House.
Thursday, 13 December 2007
If you thought the battle over Lucentis and Avastin was confined to FDA, CMS and NIH, you’re wrong. Now Congress is jumping into the fray in significant fashion as is typically the case when the government feels they are overpaying for something.The letter was in response to Genentech’s decision to cut off distribution of Avastin to compounding pharmacies, which are responsible for creating micro-doses of the drug that are tolerable in the eye. For more on Genentech's strategy, see this feature in the December RPM Report.
The Office of the General Counsel justified its decision by saying it was obvious the demo project would improve the quality of the clinical trial and would benefit from having Medicare beneficiaries participate in it, therefore a demonstration project was not needed to prove it.
The Senate Special Committee on Aging will wait to receive more documents over the next several weeks, and after review, decide whether to hold an oversight hearing. “We have enough to hold a Q&A hearing right now, but we want to wait until we have all the documents,” the staffer says. “The story is far from over.”
Monday, 10 December 2007
The primary concern of Day 1 of the FDA/CMS Summit was just how bad for the industry are the unofficial tollgates of a more safety-conscious FDA and the official post-approval burdens imposed by the FDA Amendments Act.Day 2 focused on a perhaps more inchoate fear – the chance that payors of all stripes will take the savings they need out of the hide of the drug industry.
And they will need them. Amgen VP of Global Coverage and Reimbursement Josh Ofman noted the variety of ways drugs cut overall health costs – but ultimately acknowledged, as did a variety of other speakers, that cost-containment was going to hit drugs hard – either, says Ofman, through controlling market access, restricting coverage (e.g., through formulary controls), or by imposing conditional coverage.
Most speakers did not expect the industry nightmare of a single-payor to soon take flesh. And the popularity of unfettered drug choice makes dispensing restrictions political and economic non-starters. But without them, the easiest (and most politically popular) target for cutting costs, separately noted Eli Lilly’s top politico Alex Azar and Rob Seidman, the influential former pharmacy chief at Wellpoint, is the biopharma industry—through price reductions and rebates (which fatten PBM profits as much as they cut drug expenses). Drugs covered by Part D—where consumer choice is most obvious (“what do you mean I can’t have Lipitor?” the consumer bellows at the pharmacist)—will likely face the brunt of the pricing assault.
Unfair? Absolutely. As much BS as drug companies hand out about their R&D spend, the fact is they can’t invest in new medicines if they can’t charge enough for the ones they get to market. Investors, for one, won’t allow it. Rob Seidman somewhat cynically commented that if the drug companies can’t create new products for less than $1 billion, they “need to build a better mousetrap.”
So—some mousetrap suggestions. First, there are ways of cutting that cost. Lilly’s Chorus division has shown it can get products to proof-of-concept much quicker than traditional development programs, giving their late-stage clinical colleagues a much broader choice of likely programs to push forward (and their business development colleagues a slew of out-licensing candidates that would otherwise have been dust-collectors on lab shelves).
Second, former Pfizer exec Stephen Williams, now with a new firm he founded called Decisionability, offered an interesting solution to the rising reimbursement risks – securitizing them in much the same way private equity firms and royalty buyers have learned to securitize drug-development programs. Instead of packaging development-stage products into tradeable securities (e.g., Morgan Stanley’s Pharmaceutical Royalty Monetization Assets—click here for more on just how clever the financial community can be), theoretically one could bundle approved products into packages that could cut a sponsor’s risk of reimbursement problems and permit investors to share in the upside of a positive outcome.
But drug companies also need to figure out ways of cutting reimbursement risk without turning to Wall Street. And one very practical solution is to start exploring how to create at least two formulations, one Part D and one Part B, for a molecule entering development (it’s a point we’ve mentioned before—here for example). That means exploring how a small-molecule headed for Part D might be useful in an IV infusion…and doing so at the earliest stages of planning for proof-of-concept. Take those biotechs, for example, developing small-molecule therapies for cancer or as replacements for niche drugs in orphan diseases, like lysosomal storage disorders: oral is convenient, sure. But IV can have both therapeutic and reimbursement advantages, too.
It’s reimbursement-oriented portfolio management – and it’s why R&D executives need to be au courant on the challenges their commercial colleagues face with payors (and why the marketing guys should focus on more than just market size when they kibbitz on drug development issues).
Final suggestion: there were roughly 200 attendees at the FDA/CMS Summit. More than half were the drug companies’ policy mavens. It’s clear, at least to this blogger, that what was once inside-the-beltway wonk material is now central to financial and drug-development strategy. If senior marketing, finance and R&D execs are leaving this stuff to their Washington groups, they’re leaving themselves wide open to the competitors who aren’t.
Final suggestion: there were roughly 200 attendees at the FDA/CMS Summit. More than half were the drug companies’ policy mavens. It’s clear, at least to this blogger, that what was once inside-the-beltway wonk material is now central to financial and drug-development strategy. If senior marketing, finance and R&D execs are leaving this stuff to their Washington groups, they’re leaving themselves wide open to the competitors who aren’t.
If you attended our FDA/CMS Summit last week, it was great seeing you. If you missed it, shame on you. The keynote address by Cleveland Clinic cardiologist Steve Nissen on the state of the FDA stirred up a lot of controversy, not surprisingly.However, one thing we didn't hear in his remarks could be more worrisome to the biopharmaceutical industry than what he actually said. A little birdie told us at the meeting to prepare "for something big" to be released this week.
We have no idea what the "something" could be. Do you? I will say that the last time a little birdie told us at The RPM Report that Steve Nissen was up to "something big," the "something" turned out to be Avandia.
We ventured down to Washington for the FDA/CMS Summit and while our RPM Report colleagues will undoubtedly share their own thoughts over the next few days, this auslander wants to set down a few of his own.In perhaps the most entertaining of talks, Regulator-without-Portfolio and Cleveland Clinic’s cardiovascular boss Steve Nissen blasted the FDA leadership for soulless toadying to industry. He started at the top with Andrew von Eschenbach – damned as a close Bush friend (admittedly, from our point of view, hardly a recommendation) and a “urologist” (an apparent reference to some religio-medical schema that blesses the cardiovascular as kosher and condemns the urological as treif).
He was even rougher with Scott Gottlieb, who, disguised as a regulator, apparently secretly serves the Dark Lord. The sign of his sin: pre-FDA, he wrote a newsletter on biotech stocks. That the Satanist Gottlieb no longer ran the newsletter; that as an FDA employee he couldn’t invest in those stocks; that having written about biotechs might actually have helpfully informed a more realistic policy toward the companies who actually create the products on which FDA passes judgment, Nissen did not consider – or at least did not mention.
But parallel Nissenian logic would shackle Alan Greenspan to the economic consulting hell out of which he crept to run the Fed while Robert Rubin would have been stuck arb’ing at Goldman Sachs rather than shaping and running Clintonomics – and few would have said that either man had behaved, once in government, as the corporate lackey he was clearly trained to become.
Don’t get us wrong. We’re as disgusted as most Beltway outsiders by the industry/regulator revolving door, and we nearly joined an atheist chorale when Bush allowed his Zealots (taking a break from bashing stem cell research) to reach into the FDA and, pun intended, neuter Plan B.
But Gottlieb – whose politics we ourselves continue to endeavor to change – was in our view exactly what the Agency needed, helping Mark McClellan open it up, even if just a crack, to the possibilities of adaptive trial design; making the social science of risk communication into a reality in a group which doesn’t much believe in anything but FDA-defined hard science; and—weirdly, given the Secret Industry Program he’s been charged by Nissen with promoting—trying to get some guidelines around conflicts of interest on advisory committees.
And while we can’t say that we regularly carouse with the FDA’s senior careerists, we’ve certainly never heard them bash Gottlieb for trying to impose some right-wing agenda on them. [Disclaimer alert: Scott is a friend of ours and was once very nearly a Windhover employee …which must make us second-order Satanites?]
There is also this odd fact: the Mafia which, the accusation goes, ran/runs FDA for the benefit of Republicans and Big Pharma, approved a mere trickle of new products while wrapping lots of existing ones with new warnings and restrictions. Were these Mammonites merely incompetent at serving their Master?
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