2014 Was a Good Year for FDA & Pharma

Transcription

1 Jan 2015 Vol. 14, No. 1 Pharma Marketing Network Was a Good Year for FDA & Pharma Banging Year for Drug Approvals, Wimpy Year for Enforcement Actions Author: John Mack Published by: Pharma Marketing Network publisher@pharma-mkting.com PMN

2 Vol. 14, No. 1: January 2015 Page 1 T he U.S. Food and Drug Administration (FDA) ended 2014 with a bang and a whimper. The Agency approved a total of 41 new molecular entities (NMEs) and biologics (BLAs) in 2014, the greatest number since That was the bang. FDA, however, also ended the year with a whim-per. The Agency sent out only a meager 10 adver-tising enforcement letters in 2014, the fewest ever. Big Bang Approvals! As FDA approached the finish line, it hurried the approval of fifteen new drugs in December Eleven (27%) were approved in final two weeks! 2014 was a particularly good year for biologics and orphan drugs. Ten (10) new licenses to market biologic drugs were issued in That s more than any other year and represents 24% of all new drug approvals. Meanwhile, 15 orphan drugs, which treat rare diseases/disorders affecting fewer than 200,000 people in the U.S., were approved another record (see Figure 1, right). Orphan Drugs Are Where the Money Is The U.S. government has been very kind to pharma companies that develop orphan drugs. Under the 1983 Orphan Drug Act of January 1983, orphan drugs get extended marketing exclusivity, tax breaks, expedited approval by FDA, and require fewer patients in clinical trials. A few years ago, if you asked a pharmaceutical company executive why his or her company developed and marketed an orphan drug you would likely have gotten a response such as because there is an unmet medical need or something similar. Today, however, orphan drugs also meet unmet profit needs and have the potential to turn into blockbusters with annual sales over $1 billion. Banging Drug Prices Of course, to reach that milestone, the price of these drugs has to be very high. The price tag of Amgen s Blincyto (blinatumomab), approved by the FDA on December 2014 as a second-line treatment for Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia, is $178,000 per twocycle course. To reach $1 billion in Blincyto sales, Amgen must find 5,617 patients. It just so happens that the number of people suffering from this form of leukemia in the U.S. is 6,000. In comparison, during Lipitor s heyday, about 1,600,000 patients were needed to generate $1 billion in sales. Just like Willie Sutton, venture capitalists know where the money is in the drug market and venture philanthropists are also getting in the game. Figure 1. FDA Drug Approvals (1994 through 2014). NME=New Molecular Entity, BLA=Biologic License Application; Source: FDA Recently, an investment company (the venture) paid the Cystic Fibrosis Foundation (the philanthropy) $3.3 billion for its future royalties from several cystic fibrosis drugs it helped finance. The foundation, however, did little to lobby for lower prices on the drugs that were developed from the research it funded. As a result, Kalydeco, a cystic fibrosis medication it funded, is one of the most expensive drugs available, at $300,000 a year.

3 Vol. 14, No. 1: January 2015 Page 2 Wimpy Enforcement While FDA hustled in December to reach a record number of drug approvals in 2014, the Agency wasn t as motivated to set any advertising enforcement records last year. For the year, FDA sent out only a meager 9 Untitled Letters and 1 Warning Letter, the fewest ever. This continues a downward trend that began almost as soon as direct-toconsumer (DTC) advertising was sanctioned by the FDA in 1997 (see Figure 2, below). Several explanations for this trend have been suggested, including: 1. Fewer drugs are being marketed 2. Pharmaceutical marketers are getting better at complying with FDA regulations. 3. FDA regulators are too busy dealing with other matters. 4. Staff cuts due to budgetary restraints 5. Fear of being sued by the pharmaceutical industry in the wake of the 2012 Caronia decision that found off-label promotion was protected by free speech 6. DTC marketers are playing it safe. 7. Rising PDUFA fees paid to FDA means less willingness to bite the hand that feeds the Agency. As already noted, 2014 was a banner year for drug approvals. However, 37% of these were for treatment of rare diseases and therefore the marketing budgets for these drugs is expected to be low. Thus, it is conceivable that the downward trend in enforcement actions is due to fewer, non-orphan drugs being marketed. But this can t be the only reason. As for item #2, it s difficult to believe pharma marketers are getting better at complying with regulations when in recent years one drug company after another has agreed to pay multi-million/billion dollar settlements to the DOJ for inappropriately, and in some cases illegally, promoting prescription drugs (see Figure 3, below). Figure 2. Trends in FDA Advertising Enforcement Actions Figure 3. Pharma Criminal & Civil Settlement Planetary System

4 Vol. 14, No. 1: January 2015 Page 3 Busy, Busy FDA Coalition for Healthcare Communication Executive Director John Kamp implied that FDA s Office of Prescription Drug promotion (OPDP) unit has been too busy working diligently on a number of policy documents, including social media guidance for it to have time to deal with such trifling matters as enforcing existing advertising and promotion regulations. He also hinted that FDA is busy contemplating policy items relating to the rights of companies to speak truthfully about the off-label uses of their products under the First Amendment. With regard to Caronia decision/off-label claims, while it is true that none of the letters issued in 2014 mentioned that violation, this is not new. Historically (between 2004 and 2013), only 4% of violations cited in FDA letters was concerned with off-label ( unapproved use ) promotion (see Figure 4, below). So, the 2012 Caronia decision, which stemmed from a 2009 conviction of Orphan Medical sales rep Alfred Caronia probably was not a factor in why fewer and fewer letters have been issued by FDA every year beginning in Note: There was a spike in 2009 and 2010, which was probably in reaction to the VIOXX fiasco during which time the FDA was under extreme pressure from Congress and the public to reign in drug pro-motions and DTC advertising in particular. trying to fit in to hold their jobs. Can anyone really name one DTC campaign that featured really great innovative marketing?, asks Meyer. When was the last time a pharma brand won awards, from outside the industry, on DTC advertising? Meyer blames pharma s conservative legal/regulatory people for preventing DTC marketers from being more innovative. He would like to see more pharma marketers focus more on innovative digital and social media channels, where there is less guidance from the FDA and thus more chance that marketers will inadvertently run afoul of the regulators. Meyer and may have a point. Based on an analysis of FDA Warning letters and 2253 forms (Transmittal of Advertisements and Promotional Labeling for Drugs and Biologics for Human Use) submitted to the FDA by pharma advertisers, I estimate that Internet promotions receive a disproportionate number of violations. This conflicts with a 2013 Eye on FDA analysis that concluded that a Warning Letter was almost three times more likely to be based on a traditional media communications vehicle than on a digital one. Figure 4. Type of Violations Cited by FDA in Warning/NOV Letters Are Pharma Marketers Playing It Safe? World of DTC Marketing blogger Rich Meyer, believes the reason for the decline in NOV letters is because of the lack of really talented marketers and those in DTC who are afraid to rock the boat and just Figure 5. PDUFA Fees Collected by FDA vs. Enforcement Letters Issued

5 Vol. 14, No. 1: January 2015 Page 4 Mark Senak of Eye On FDA looked at 676 violations in the 173 letters sent out by FDA between 2008 and 2012 and found that 57 percent (n=385) of the vehicles cited for violations were nondigital properties compared to 43 percent (n=290) involving digitalbased communications vehicles. How many of these violations are serious? Senak looked at the distribution of Warning Letters for that. Of 45 WLs [Warning Letters] issued by OPDP during this period, wrote Senak, only 12 cited digital communications vehicles while 33 were based on nondigital (traditional) communications, meaning that a Warning Letter was almost three times more likely to be based on a traditional media communications vehicle than on a digital one. Senak cites an obvious limitation of his data: one cannot say whether or not digital is over or under represented. The reason, claims Senak, is that we do not know what proportion of communications by industry is divided between digital and non-digital efforts. Senak contends that the lack of clarity respecting the rules around Internet and social media use does not appear to translate into a greater pattern of enforcement against digital media. I disagree. I believe we DO have some idea of the proportion of pharma marketing communications that are digital and nondigital. Pharma marketers must submit 2253 forms for all ads. Data from these forms suggests digital promotions are very much over represented in terms of violations and warning letters. For fiscal year 2011 (the latest full year of data), the percent of non-internet 2253 pieces reviewed by FDA varies from 72% to 80%, which means the % of Internet pieces is in the 20-28% or let s say 24% for the year o average. So 24% of promotional pieces reviewed by FDA gets 43% of the violations cited and 27% of serious warning letters sent. From this I would have to disagree with Senak and say the lack of clarity respecting the rules around Internet and social media use by pharma DOES appear to translate into a GREATER pattern of enforcement against digital media. PDUFA Fees vs. Enforcement Is it possible that the rise in Prescription Drug User Fee Act (PDUFA) payments to FDA from pharma, which now account for more than 65% of FDA s budget for the regulation of drugs, discourages the FDA from monitoring drug promotion and issuing warning letters? As shown in Figure 5, page 3, since 2002 PDUFA fees collected by the FDA have increased dramatically. This corresponds with the historically flat number of untitled/warning letters issued in the following years (except for the VIOXX spike mentioned above). I contend that in the years prior to the rise in PDUFA fees, FDA issued fewer and fewer warning letters so as not to upset the political process authorizing the future collection of the fees. Since then it has been more or less laissez-faire as the money continues to roll in. Money talks, bullshit walks.