The Big Red Biotech Blog

Of the FDA, Biosimilars and Elephants

The Patent Doc blog covered a recent FDA presentation (Dr. Rachel Sherman, Feb 15th) on the biosimilar guidance documents issued earlier this month.

This FDA used entertaining analogy to demonstrate the thinking process in this determination.  The presentation contains a line drawing of an elephant (below), where each line represents an analytical or clinical study performed by the biosimilar applicant.  The FDA will have to compare the reference product (figure A) with the biosimilar products (figures B-E) to determine what level of detail is required for a determination of biosimilarity (whether the drawing is an elephant or not).  The biosimilar applicant will attempt to draw the minimum amount of lines such that the FDA can make a finding that the biological product is biosimilar to the reference product (elephant).  The FDA will not permit any "tracing" of the lines in the drawing, as these attributes of the biosimilar are already known.  Each test or study must add something to the whole body of evidence.  This elephant analogy is also used to define "fingerprint" studies, which may tell the biosimilar applicant at an early stage that its product will not qualify for approval under 351(k) (e.g., if early lines in the drawing show a beak or fins).

This got me to thinking about the old Jain proverb of the Six Blind Men and the Elephant.

The story says that six blind men were asked to determine what an elephant looked like by feeling different parts of the elephant's body. The blind man who feels a leg says the elephant is like a pillar; the one who feels the tail says the elephant is like a rope; the one who feels the trunk says the elephant is like a tree branch; the one who feels the ear says the elephant is like a hand fan; the one who feels the belly says the elephant is like a wall; and the one who feels the tusk says the elephant is like a solid pipe.

A king [FDA] explains to them:

"All of you are right. The reason every one of you is telling it differently is because each one of you touched the different part of the elephant. So, actually the elephant has all the features you mentioned."

This resolves the conflict, and is used to illustrate the principle of living in harmony with different beliefs and that truth can be stated in different ways.

 

Here's hoping the FDA can harmonize its biosimilar elephants by using a process "where each analytical study required of biosimilar applicants will advance the field of knowledge regarding the molecule of interest."

Posted by Bruce Lehr Feb 28th 2012.

Ireland Hitting Patent Cliff Too

We hear a lot about various companies in the pharma industry who are nearing their own patent cliff -- where some of their highest selling drugs are reachign the end of their patent life and where they don't have a lot new in their pipeline to replace the revenue that will be lost.  That's a theme that's been discussed ad nauseum for several year now.

But have you ever thought of a country having a patent cliff?  That's something that Ireland may be facing as 5 of the top drugs manufactured in the country are coming off patent altogether or losing protection in the US.  See PharmaGossip.

The drugs in question include (drum roll):

• Pfizer's Lipitor & Enbrel
• Takeda's Actos
• Merck's Singulair
• Lilly's Zyprexa

All are about to experience big revenue drops -- CLUNK!

Posted by Bruce Lehr Feb 27th 2012

 

 

GSK's Witty Fires Salvo at British Government Drug Policies

Among other things, GSK's CEO Andrew Witty says the British goverment is "systematically delaying" the introduction of new cancer drugs, making "false economies", not buying "the next round of innovation", treating drugs as a "simple procurement business" and in general lacking "any kind of strategic thoughtfulness."

Other than that, he thinks the Government is doing a great job in setting UK drug approval and reimbursement policy. He further implies if GSK keeps losing its abiilty to earn a better return that the UK ministers may face a choice between health budgets and drug industry employment. See PharmaGossip and Pharmalot blogs.

Posted by Bruce Lehr Feb 27th 2012.

Vertex Continues to Run its Hep C Race

Vertex, despite the licensing of its well-received new hep C drug Incivek this year, is still in hot pursuit of an oral treatment for hep c that does NOT require interferon. And, it still wants to show elimination of virus in patients by week 12. 

This week it shared results of a small study that combined Incivek, its developmental drug VX-222 and ribavarin to treat virus genotype 1a and 1 b patients with 83% of those treated showing viral loads below lower levels of quantitation by week 12. Now, it will push on to a phase IIb trial based on that success. It is targeting an NDA submission to the FDA by 2014.

Additionally, not one to rest on its laurels, Vertex is also looking at other non-interferon combination therapies using Incivek, VX-222 and two other nucleoside polymerase inhibitor drugs licensed from Alios, ALS-2200 and ALS-2158. Thus, they hope to stay ahead in the hepatitis C treatment market and defend their current share.

Their stock responded yesterday by jumping 3.4% on the news. See Fierce Biotech and Xconomy.

Posted by Bruce Lehr Feb 24th 2012.

BioTech Funding Update

There were a couple of interesting articles on various aspects of biotech funding out there today.  Firstly, Pharmalot related that VC funding for biotechs was actually up 16% in 2011 over 2010 despite all articles about it being unavailable. Q4 was actually up 90% YOY from 2010 and 160 deals closed in 2011 compared to 153 in 2010. What is apparently true are there are a lot more early stage spin-outs from Universities and all of these can't (aren't) be funded with current mechanisms.  The question is do they all deserve funding?  Probably not.  Bottom line, biotechs received $3.5 B in funding in 2011 with an average round raking in $21.8 M versus $19.7 M in 2010. So much for a no funding perception.

Secondly, Life Sci VC blog talks about the milestone payouts that are actually being achieved in these M&A deals with relatively small upfronts and big backend milestone payout schedules. Data for this analysis is hard to come by by the blog's author (Bruce Booth) has estimated the results on 35 deals where public info (or his network) was available for period 2005-2009. The deals represented in $4.3 B in upfront payments with milestones promising up to another $7.0B.  The results?

• At least 24% ($1.7 B) in milestones have paid out
• Approximately 40% ($2.8 B) may still pay out in part or total -- though some milestones may have already been missed
• And 37% of milestones are dead as the projects have been killed. That results in $2.6 B worth of no pay out
• Only 3 of the milestones were actually paid for product approvals (Salmedix, Ovation and Gloucester).

The data is incomplete and the author is asking those in the know to help build a better picture through crowd sourcing.  Until then, the amount being paid on backend milestones in biotech M&A deals will remain somewhat murky.

Posted by Bruce Lehr Feb 23rd 2012.

 

 

 

 

Novartis Hops the Hep C Train

Novartis is the latest big pharma to take the hep C train looking for a chance at a multi-billion dollar payout.  It signed a pact for $440 M with Enanta Pharmaceuticals for its preclinical NS5A drug.  The deal calls for $34 M upfront and a possible milestone-based payout up to $406 M more.

The Enanta drug, EDP-239, is directed to the NS5A target that should provide effects across all three hep C genotypes and does not require the use of interferon.  Avoiding the use of interferon is seen as the goal by most of the leading contenders for the estimated $20 B plus hep C treatment market -- where an ever increasing crowd is now jockeying for position. See Fierce Biotech.

Posted by Bruce Lehr Feb 21st 2012.

GAO Report Places Primary Blame on Manufacturing Failures for Drug Shortages

New supplies of two unavailable cancer drugs will be allowed by the FDA.

Dow Jones Newswires(DJN) reports that the FDA will allow the temporary importation of Lipodox as a substitute for Johnson & Johnson’s Doxil. The drugs have the same active ingredient.  Doxil has been in short supply since about mid-2011. New supplies of Lipodox are “expected to end the shortage and fully meet patient needs in the coming weeks,” the FDA says.

The FDA has also approved a new manufacturer, APP Pharmaceuticals, of preservative-free methotrexate, a drug that is used to treat a type of children’s leukemia and has also been in short supply. The drug should be available in March.  The agency “is actively working with other manufacturers … including Mylan and Sandoz Pharmaceuticals” to meet patient needs.

DJN notes that both shortages can be traced back to the closing of a Ben Venue Laboratories plant in Ohio. Ben Venue, a unit of Boehringer Ingelheim, shut down the plant to deal with manufacturing issues. See here.

The GAO just issued a report that stated the majority of drug shortages now being seen were due to manufacturing problems. See WSJ article. 

While the Pharmalot blog reports that more than two dozen patients who suffered due to drug shortages filed a lawsuit today in DC federal court against the US Dept of Health and Human Services (DHHS), the FDA, and NIH.  The suit argues that FDA licenses to drugmakers that cuase shortages should be invalidated and their patents declared unenforceable (how did they miss the patent office in suit too?).

The poster boy for this suit is Genzyme with its celebrated manufacturing issues at the Allston plant which caused repeated delays in the supply of both Cerezyme and Fabrazyme (which is still not back to normal).

Posted by Bruce Lehr Feb 21st 2012.

 

 

Gilead Still Liking Its $11B Hep C Deal?

Can this be the beginning of an $11 B hepatitis C mistake? Gilead, after shelling out that much for Pharmasset and its purported GS-7977 wonder drug, has experienced its first set back after the purchase. This morning Gilead announced the 6 of 10 patients with the genotype 1 form of the disease had viral relapses within 4 weeks of ending treatment. So much for permanent cures on oral doses without the use of interferon.

Gilead And Its $11B Hep C Deal: So Far, Bad News // Pharmalot.

The effect seen in the market today?  Shares of Achillion and Idenix Pharmaceuticals shot up as they are developing rival treatments for hep C.  Both are looked at as takeover candidates.  Shares of Gilead dropped 15%.  Hep C buyers might take note when putting together their bids for the former two companies.  As Sanofi's CEO Viehbacher noted earlier in the week, the price premiums of these companies in the Hep C market seem overblown.

They used to say nobody gets fired for buying IBM.  How about buyers of Pharmasset?

Posted by Bruce Lehr Feb 17th 2012.

The Costly Myths about Pharmaceutical R&D

I love this back and forth debate about how much drugs cost to bring to market -- focusing ostensibly on the R&D costs.  Problem is -- apparently -- no one can agree on where R&D costs start and finish.  Nor can they agree on which drugs are coming to market.  Is it NMEs?  Is it variations on existing drugs? Both? Neither?

The Costly Myths about Pharmaceutical R&D - Health News Watchdog .

We also get to argue about whether discovery is included or not.  Whether the cost of capital should be considered or not.  Whether the government or public subsidizes the process, to what degree, and whether that should be deducted from the costs in the analysis.  We can't even agree whether we're including biotechs or not in our analyses.

That's how we end up with an estimate of the cost of developing a new drug falling somewhere between $56 M and $11.9 B.  We might be able to just barely slide a piece of paper through that crack.  More like dozens and dozens of reams.

Posted by Bruce Lehr Feb 17th 2012.

Shhh! My Bioproduction Processes are Secret

Mass High Tech today picked up on a theme that is being increasingly bandied about now that biosimilars are becoming a reality in the US -- that being companies may eschew patents in favor of keeping intellectual property information as 'trade secrets'.

The concept is that biosimlars rely much more on production processes as a critical feature to produce the reference molecule -- which a biosimilar maker would like to be able to match or certainly know about in detail.   Patents on processes typically can require disclosure of detailed information that would make "copying" the process or more to the point designing around its patent claims more easily accomplished.  

Trade secrets on the other hand -- are well -- secret.  There is no public disclosure and if you keep your mouth shut (your company's collective mouth shut), you can keep the secret for an eternity in theory.  Patents of course have a finite lifespan of 20 years.

The upshot is that many innovator (or biobetter) companies may now be concluding that to protect themsleves from the expected biosimilar onslaught, they should simply stay quiet on their processes. The "don't ask don't tell" version of the biosimilar world.

Posted by Bruce Lehr Feb 16th 2012.

Not a Good Day for Senior Directors (and others) at Alnylam

Boston Business Journal reports that Alnylam will layoff 52 employees (previously discussed as a restructuring program) on March 23rd.  This 33% trimming of the staff will contribute to savings of approximately $20 M.  Layoffs will include positions for Senior Director of Biotherapics, Senior Director of Business Development, Senior Director of Pharma Operations, and a Senior Director for IT -- along with 9 senior scientists and 14 research associates among the casualties. The one time employee costs of termination will be on order of $4 M.

See what happens when most of Big Pharma gives up on RNAi -- at least for now.

Posted by Bruce Lehr Feb 16th 2012.

Dwindling R&D Returns?

According to Sanofi's Chris Viehbacher studies on R&D productivity in pharma-land show that "on average" if you spend $1 on R&D you will receive a whopping $0.70 in return. See Fierce Biotech.

What if your investment adviser told you, "Hey, if you invest $XX with me today, in 10-15 years I'll give you 70% back guaranteed!"  Wow, such a deal.

No wonder, R&D departments across the industry are under attack, private VC is down, and Big Pharma is looking for partner companies, institutions, etc to invest in to try to get some new products and earn a decent return. Seems strange with the historic profitability of the industry that we would have ended up here.

Posted by Bruce Lehr Feb 16th 2012.

Hep C Companies Rumored as Next Takeovers

Fierce Biotech reported today on the continued hype by investors and analysts for the companies that are pursuing hepatitis C therapies and have gotten good clinical (or preclinical) results.  Two of the leaders who have not yet been swallowed, Achillion and Idenix, are rumored to be takeover targets as big pharma suitors cast an eye toward a purported $20 billion hepatitis C treatment market.

Achillion's CEO, Michael Kishbauch, is out shopping the company to large, as yet unnamed, players. He says he is looking for a premium that would "put a silly grin on all our faces."  He might just get it too.  But other Big Pharma CEO's like Sanofi's Viehbacher are saying the premium's are too high and the value is not there.  This is echoed by GSK's Moncef Slaoui, R&D chief. 

Next thing you know, Sanofi and GSK will be putting in a bid. 

Posted by Bruce Lehr Feb 15th 2012.

Biosimilars are Progressing by the 'Totality of Evidence'

Two more blogs weigh in on the FDA guidelines published for the Biosimilar approval route last Thursday.  The Patent Doc blog seems a bit underwhelmed by the lack of specificity of the guidelines but acknowledges the FDA had a relatively short time line to issue their guidance.  It does acknowledge the guidelines will likely evolve in practice and the FDA gave itself maximum flexibility in interpreting them. The FDA will use a "totality of evidence" standard similar to that adopted in Europe.

The BioProcess Blog for its part seems a little more upbeat that the FDA put guidelines out there to get the process rolling.  In general, it was positive about the fact the FDA will allow the possibility of formula changes and different delivery devices from biosimilar to the reference product without either being an automatic block to taking a biosimilar path. The blog authors were also happy that the FDA will accept data from non-US licensed products in considering the "totality of evidence" in support of a biosimilar application.

To date the FDA has received 31 requests for meetings to discuss company plans for their biosimilars, and 21 of those had been held by the end of December 2011.  There have also been 9 INDs for biosimilars filed to date and the agency is expecting a full 351(k) application soon.  The process is well underway and there is still 60 days for interested parties to comment and suggest changes to the Draft guidelines just issued.

Posted by Bruce Lehr Feb 15th 2012.

Biosimilar Guidance Is A Pandora’s Box: Pan Explains

This is a really interesting short interview from Pharmalot with Jonathan Pan, a manager at the Scientia Advisors consulting firm on his view of what the FDA draft guidelines on biosimilars could mean.

Biosimilar Guidance Is A Pandora’s Box: Pan Explains // Pharmalot.

I excerpted a part I found very interesting concering how the FDA is [apparently] setting up to judge biosimilarity. And how this might open up opportuntities for pharma to get drugs to market as "biosimilars" instead of new entities.

Pharmalot: So was there anything surprising here?
Pan: The major surprise I see here is that FDA is creating a path of similarity versus a path of replication. Effectively, what they’re doing, because of the complex nature of these products, is they’re saying that, for any biologic agent, anybody can create a biosimilar, but that it may not be an identical product. At the end of the day, it’s creating a great way of making a ton of non-inferior products to a particular reference product.

Pharmalot: Aren’t they going go to go on a case-by-case, though?
Pan: Obviously, they leave that language open and, of course, no guidance from FDA is set in stone, but at the end of the day, it’s interesting how they worded this to mitigate the fact that compounds are very complex and because of that, there’s more opportunity for companies to move their products to market. I think if a drugmaker wanted to pursue a biosimilar path versus the new molecular entity, they make take the biosimilar path, because it’s a quicker route to market.

Look, take the rheumatoid arthritis market. If we have one anti-TNF inhibitor, all I have to do is show it’s equivalent to how one of them works… I just have to make a decision to make a biosimilar and go to FDA and say I’ll run a trial to show that it’s safe and my antibody is going to be non-inferior. The FDA, according to the guidance, would say the product is non-inferior and, therefore, it will be approved as a biosimilar.

That’s versus going through a Phase I trial and recruiting patients, and a Phase II trial and recruiting patients, and a Phase III showing superiority for standard of care… They won’t have to go through the regimented process. It’ll be more abridged. As long as they can show in standard pharmacokinetic and pharmacodynamics tests that the product is non-inferior to the reference products. They’ll be going head to head. They’ll still have to go through safety checks and efficacy checks through clinical development plan, but what most pharmaceutical companies do is cherry pick… in this process, they’ll simply try to compare to reference products.

Pharmalot: What, if anything do you see as potentially troubling?
Pan: There is a Pandora’s box here. If implemented in its current state, I imagine there will be some interesting maneuvering. If I’m a pharmaceutical company and any product is at Phase I, I may not want to go through the full development process and instead say that I have a biosimilar and get to market as fast as possible. It’s a new thought process pharma may or may not want to embrace. It’s good for the pharmaceutical industry as a whole because it allows them to get products on the market that may not get on there otherwise.

OK do we have a new way to get similar biologic products to a licensed drug to market?  Or do we have a way to get a new entity to market faster by calling it biosimilar?  Or do we have both?

Posted by Bruce Lehr Feb 10th 2012.

Which Pharma Spent The Most R&D On Each Drug?

Pharmalot just published (via Forbes) an analysis of Big Pharma R&D spending for period 1997-2011.  The data was provided by InnoThink Center For Research In Biomedical Innovation and Thomson Reuters Fundamentals via FactSet Research Systems.

Which Pharma Spent The Most R&D On Each Drug? // Pharmalot.

From this, one could calculate the dollars spent on R&D/new drug approved for each company concerned.  The link above provides a Table of the results for 12 Big Pharma (biotech).  Amgen, it turns out spent the least at $3.7 B per new drug (11) approved during this period.  AstraZeneca, it turns out spent the most at $11.8 B per new drug (5) approved during this period - OUCH!  This coupled with products reaching the patent cliff helps explain why the latter is experiencing really tough times now and cut over 30,000 jobs during the period.

Even $3.7 B per drug at Amgen seems like an astronomical amount of money.  It's no wonder the industry it trying to rethink its business and R&D model.  Clearly something better change when you are spending$3.7 B to $11.8 B PER DRUG.

One can't help but think to some degree this "ranking" over the period has a little to do with luck as well given some of the vaguaries in the approval process and perhaps timing of some projects relative to submission dates in this period.  I'm not certain you can state one company has any better development model than any other based on this data.  You can say Amgen made out slightly better than its peers for the period -- but who's to say that will hold up over the next period?  We're going to have to track some of these "new models" at companies like Novartis, GSK and Sanofi to see if they really see improvements.

Posted by Bruce Lehr Feb 10th 2012.

SEE some more commentary on this subject from In the Pipeline.

FDA Unveils Guidance for Biosimilar Development Today

The FDA posted its draft guidance document for biosimilars on its website today.  You can read a summary below outlined in the PharmTech Talk blog.

PharmTech Talk » It’s Here: FDA Unveils Guidance for Biosimilar Development.

Or you can go see the full document yourself on the Agency's site.

The FDA site notes that comments and suggestions regarding this draft document should be submitted within 60 days of publication in the Federal Register of the availability of the draft guidance. Many comments are expected. Everyone - Have at It.

 

 

Posted by Bruce Lehr Feb 9th 2012.

 

 

GSK DPU Score 4-3 (or was it 6-5)?

GSK management today announced the results from its first 3 year review of its "novel" Discovery Performance Units.   DPUs were formed in 2008 as a means of focusing drug discovery activities in some defined areas and were intended to bolster the pipeline, control costs, and increase ROI in R&D.  GSK has touted them from time to time ever since, so how'd they do in this very first time review?

Drumroll please. Well of the 38 DPUs, 3 will be terminate and 4 new ones will be formed (so now we have 39).  Six (6) DPUs will receive and increased budget and 5 will have their budgets cut. GSK CEO Andrew Witty says he is "quite satisfied" with the results so far.  GSK reports that's its R&D ROI rose from 11% to 12%, while their target remains at 14%. The overall R&D budget meanwhile is supposed to stay flat at $3.7 billon.

More to the bottom line, GSK plans to file for approvals on 4 new drugs and vaccines in 2012, including Relovair for COPD, Promacta for hepatitis C, and trametinib for melanoma.  Six other DPUs have late stage projects. In the next three year cycle, 30 more programs are targeted to move into late stage development. See Fierce Biotech and In Vivo Blog.

Per usual in this industry, the jury will remain out on DPUs until more than a 3 year track record is established and until several high revenue generating drugs actually hit the market.  One might be cautiously optimistic at present.

Posted by Bruce Lehr Feb 7th 2012.

Supporting Orphans Can Be Expensive

 

This post is courtesy of PharmaGossip. It details the 11 most expensive drugs in the USA for an annual prescription.  Not surprisingly, these are all Orphan drugs and mostly biologicals -- aimed at serious genetics diseases that effect relatively small populations.

 

The 11 Most Expensive Medicines in America - Medical Billing and Coding Certification .

Without further adieu, I will just list the top 10 drugs in descending order from most to "least" expensive.  See the article for more information on what conditions these drugs treat.

1. Soliris $409.5 K
2. Elaprase $375 K
3. Naglazyme $365 K
4. Cinryze $350 K
5. Folotyn $300 K
6. ACTH $300 K
7. Myozyme $300 K
8. Arcalyst $250 K
9. Ceredase/Cerezyme $200 K
10. Fabrazyme $200 K
11. Aldurazyme $200

As I indicated, all of these conditions are extremely serious.  The drugs make life or death differences in many cases and certainly make huge impacts on quality of life for many patients. I'm sure the patients are glad to have the drugs regardless of costs.

It is also evident why Orphan Drugs have become attractive to a struggling pharma industry as relatively big sales and good profits can be made from drugs that treat fairly small patient populations.  It was an impetus that caused Sanofi to buy Genzyme (makers of #7, 9, and 10 on the list).  It also sells Aldurazyme in partnership with BioMarin (who also makes Nalazyme). You can see that Genzyme has built a very lucrative Orphan franchise.

Based on competitors comments, this field is likely to become more crowded in the coming decade not less.

NOTE: Fierce Biotech reminds us that Vertex's CF drug Kalydeco ($294 K ) approved this week cracks what is now the top 12.

Posted by Bruce Lehr Feb 7th 2012.

 

 

Is the US Facing a Pharmaceutical Manufacturing Gap?

Here's a post from PharmTech Talk discussing recent testimony by FDA's Margaret Hamburg before the House Committee on Energy and Commerce as it considered extending PDUFA (for the fifth time). 

Is the US Facing a Pharmaceutical Manufacturing Gap?.

Hamburg cited numbers to show that the FDA had approved more new druigs for launch first (70% of last year's 35 new drugs) than other agencies in the world.  In fact, 75% of the new drug approved in US and EMA between 2006 and 2010 hit the US first.

PharmTech noted that the US is the largest drug market at 36% of the world's total in 2010, while the top 5 EU markets accounted for another 17%, and the BRIC countries another 18%.  But the question arose, does the US manufacture enough given its market position and importance?  It turns out that 40% of the drugs consumed in the US are made outside the US, and 80% of the APIs used within are made ex-US. 

The writer questions should the US support its internal manufacturing of drugs more?  I would say, Yes, but with particular regard to supporting biomanufacturing.  A lot of the drugs and APIs cited above are small molecules that have moved to generic status and have therefore graduated to lower cost (at least currently) areas of manufacturing outside the US.  While it may be nice to retain some of that volume, I think it is more critical to keep the bio-drugs as a major focus in the US. 

I think US government policy should look for ways to continue to support biotechnology in both discovery and manufacturing to help maintain our leadership position. 

Posted by Bruce Lehr Feb 6th 2012.

 

FDA Gives Biosimilars Update

Rachel Sherman, Associate Director for Medical Policy, within CDER gave an update on biosimilars today. Protein biosimilars (but not chemically synthesized polypeptides) will be regulated under Public Health Services Act section 351. 

PharmTech Talk » FDA Gives Biosimilars Update.

The 351 (k) approval route will require the FDA to consider:

• Analytical studies to show th ebiosimilar is highly similar to the reference product
• animal studies (including toxicology) as needed
• clinical studies to establish safety, purity and potency in one or more conditions of use for the reference product

To assess biosimilarity, Sherman explained that each study adds a piece of information to understand what a biosimilar product is and is not, and what other information is needed. The FDA may also adopt the European approach to "use the totality of the evidence" to address biosimilarity.

To date, there have been 35 Pre-IND meeting requests for proposed biosimilars to 11 reference products, 21 Pre-IND meetings held and 9 INDs received at FDA. The line is rapidly forming.

FDA guidance has not appeared as yet, and is still in "final stages" at the agency.  No timeline for publication has been issued.

Posted by Bruce Lehr Feb 3rd 2012.

 

Study says Speedy FDA Approvals Associated with More Safety Questions

A recent study by Harvard and Stanford researchers seems to indicate that FDA approvals made closest to the review deadline have higher post-market safety problems.  While no cause and effect is yet claimed, the researchers do note a positive association and suggest the deadlines link to funding may have unintended safety consequences.

FDA Review Deadlines Caused Safety Issues: Study // Pharmalot.

PDUFA which links FDA funding to its ability to meet drug application review deadlines may be altering FDA review behavior.  The study indicates that more reviews now pile up around the 6 month deadline than before PDUFA existed as legislation. Drugs reviewed and approved less than 2 months before the deadline is reached experience higher post-market safety issues than drugs approved more than 3 months before the deadline or after the deadline was reached. A new black box warning is 3.27 times more likely for a drug approved within the 2 month window, and safety related withdrawl is 6.92 times more likely for these drugs.

The study was published at a time when Congress is conducting PDUFA hearings looking at renewal of the legislation. How are we going to choose to balance risk and reward?

 

 

Posted by Bruce Lehr Feb 2nd 2012