Weekly Roundup 4.27.18

Another busy week that did not permit me time to get to some of the postings I have been working on between Roundups, but next week look for some. There has certainly been a lot going on and a lot to talk about. In the meantime, some of the many interesting things that occurred this week:

  • Opioid Crisis Response Act of 2018 Moves Out of Committee –  Upon a unanimous vote, the Senate HELP Committee advanced new legislation – the Opioid Crisis Response Act of 2018 – aimed at multiple aspects of the opioid crisis.  The bill represents a culmination of several hearings on the topic, some of which directly involve FDA. Specifically the bill has built into it a number of clarifications to make clear that FDA has authority to require specific packaging for opioids (such as pill-limited blister packs, often a tool mentioned by Commissioner Gottlieb) to address over-prescribing; ability of the agency to require disposal programs by manufacturers as part of the packaging; and providing FDA and border patrol with greater detection equipment and technology to screen for illegal imports. On the drug development side, FDA would be tasked with facilitating development of non-opioid/non-addictive investigative compounds by navigating existing expedited approval pathways and to provide new guidance related to the risk-benefit assessment of products with a potential for misuse/abuse; on label claims respective to products on pain control and opioid reductino of use; and on appropriate use of endpoints in clinical trials for non-addictive and non-opioid pain treatments. This brief summary focuses on FDA, but there are also a number of provisions that address responsibilities of various stakeholders. 
  • Second Installment by Gottlieb on FDA Budget – Last week in remarks before the House Committee on Appropriations, Commissioner Gottlieb took a short victory lap around some FDA achievements and outlined some future moves related to smoking and nutrition and food labeling. This week on the other side of the Hill before the Senate Committee on Appropriations, he continued in the same vein, but with added content that had a dual focus. First he said the additional money being provided in the budget for FDA would go to modernizing the review process for generic drugs, improving on what was already a banner year in new generic approvals, with an aim to (among other things) bring the costs of drugs down through the increase in competition as a result. This will include a drive by FDA to bring drug labels of generics up to date where the original product on which label updates rely has been withdrawn. The second area of focus was to provide up to date information on the safety and efficacy of drugs in use on the market to help patients make informed decisions. The full text of remarks can be found here
  • FDA Takes Action on Multiple Fronts Against E-Cigs and Youth – FDA outlined a plan of action last year aimed at reducing the introduction of nicotine products to young people. Often plans remain just that, or their implementation is sketchy. This week FDA announced broad action on e-cigarettes focusing on both retailers and manufacturers. With respect to the former, the agency cited retailers for sales to young people and are seeking to address online sales. And on the manufacturing side, the agency is seeking information about marketing and related documents in a bid to discover what has made the products so popular among kids and the agency will also be looking for ways that companies have sought to mislead young consumers. 

Things to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices

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Weekly Roundup 4.20.2018

In some recent postings, it has been noted that the FDA Commissioner has made liberal use of the vehicle of “Commissioner Statements”. Last month, there were 9 – on average 2 a week. Then suddenly this month, the entire first half of the month went by with no word. Finally this week, there was a statement.  But it’s about more than numbers, it is about the substance and this week there was a good deal of activity, a few things of which are noted below. 

  • Gottlieb on FY 19 FDA Budget – Scott Gottlieb delivered remarks regarding the increases for FDA’s budget for the upcoming fiscal year where he outlined his pathway to developing an approval process that relies on cross-disciplinary experience to build a “knowledge management platform” to build a more empirical approach to review and approval, one that would ostensibly re-tool the process to be more effective in a modern world and to enhance the speed and efficiency of review. He also was able to relate that FDA had just come off an extremely productive year in terms of approvals for new molecular entities, devices and generic drugs and that two public health initiatives – on smoking and nutrition and food labeling – were moving forward. He got a lot into a short set of remarks. 
  • Advisory Committee Unanimous Go on Cannabidiol – The Peripheral and Central Nervous System Drugs Advisory Committee this week gave a unanimous approval vote respecting a candidate cannabidiol compound seeking approval for the treatment of two serious, rare forms of epilepsy. If approved, it would be the first drug approval involving a compound derived from marijuana. The PDUFA date set for a decision from FDA is June 27. The company release can be found here
  • FDA Issues Guidance on Caffeine Supplements – Following two deaths after consumption of highly concentrated caffeine supplements, FDA issued a new guidance  with immediate effect to outline the circumstances by which supplements may pose a serious risk. The release regarding the guidance notes that a single teaspoon of highly concentrated powdered caffeine can contain the equivalent of 20-28 cups of coffee. The guidance addresses both powdered and liquid formulations sold in bulk. 

Things to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices

Photograph by Lynne Bertram

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Perspective on Pharma Support for Patient Advocacy Organizations

Last week Kaiser Health News (KHN) released a database called “Pre$cription for Power” that tracked pharmaceutical company donations made to patient advocacy groups, with an extensive accompanying article.  The database is an important collection of information and serves to enhance transparency about the relationship between the industry that gives us medicine and the patients who need it. Looking at the year 2015, the KHN story revealed that the amount of money pharmaceutical companies donated to patient advocacy organizations in nearly twice the amount they paid for lobbying services. 

However, the nature of the presentation, even by its name, clearly implies heavily that (1) the funding from pharmaceutical companies is solely tied to advocacy efforts, and (2) that a financial relationship between pharmaceutical funding and patient advocacy group necessarily compromises the integrity of the latter. That merits a focused look. 

As someone who worked for many years in two of the largest HIV/AIDS non-profit organizations in the U.S. during the very formative years in the 1980s and also in the 1990s, I was witness to the evolution in thinking regarding acceptance of pharmaceutical funding by a large HIV/AIDS patient advocacy organization. There was certainly debate, with some degree of passion on both sides. The ultimate question was whether acceptance of such funding would skew in any way the commitment to patient interests. In the end, the consensus on the part of most involved was that the acceptance of such funding would not be a conflict. In the accompanying article that announces the KHN database, HIV/AIDS organizations were singled out as a particularly potent force for advocacy that other patient groups sought to emulate. As I have often said, HIV organizations took the mold of healthcare advocacy, broke it, and recast it in their own image. The success of that advocacy changed the landscape for all kinds of patient organizations. 

But while effective at advocacy, HIV/AIDS organizations did much more.  It is important to note that while many patient organizations are effective advocates, they also provide a range of essential services. In fact, for many, service provision is the primary mission and they are often a lifeline for many patients, particularly in HIV.  In many cases, those services rely on philanthropy for survival. 

HIV services developed to fill the void that existed when the mainstream infrastructure failed. When healthcare professionals refused to attend people with AIDS in hospital rooms, when families turned family members away, when dentists refused to provide care, when landlords kicked people out, when people lost their jobs – it is a long list. There is still such a void in the spectrum of services today, though in other important ways. There are doctors who will not prescribe pre-exposure prophylaxis medications (PrEP) to patients. Many minority people at higher risk for HIV and many transgender people at higher risk are located in geographical areas where there is very little support or access to information. It is often pharmaceutical money that makes support where there otherwise would be none – support that can keep people infected from transmitting the virus and that can keep the uninfected from exposure. It is unlikely that HIV organizations are unique in this regard – funding from donors – including pharmaceutical donors – funds many key services for patients. 

The database provided in Pre$cription for Power provides total amounts, but actually does not parse out what portion of the donations made by pharmaceutical companies to patient advocacy organizations are for the funding of important programs and what actually goes to advocacy. By comparing the amount in donations to the amount spent in lobbying, there is an implication that the companies have purchased lobby services in these amounts from non-profits. Unless the dollars tracked in the database are known to be solely for advocacy projects, that would not be the case.

When it comes to advocacy, patient organizations often have much in common with industry and so the presence of lobby days, as noted in the Pre$scription for Power materials should not be much of a surprise. For example, NIH funding for research is in everyone’s best interest. If patient organizations did not receive any funding from pharmaceutical companies, they would still likely join forces in pursuit of that goal. Moreover, in reviewing the database, there are examples of organizations who have received funding from pharmaceutical companies who have been active critics and in some cases, litigants vis a vis pharmaceutical companies and interests. In other words, the mere presence of financial support does not, by itself, serve as notice that an organization’s independence has been compromised. The dollars are not damned. 

Finally, donor support comes to non-profit patient organizations in many ways. It can take the form of support for fundraising events such as walk-a-thons, sponsorship of tables at dinners, or the result of competitive grants, and as noted above, not exclusive to advocacy. It is possible that for some organizations, the level of philanthropy may be a factor in their approach to advocacy, but it cannot and should not be assumed. It is a question that deserves an answer – patients are deserving of that. But as the assessment of that question should be balanced. The patients who rely on the services that are often provided by these donations deserve that as well.  

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Weekly Roundup 4.13.18

For those of superstition, it is the first of two Friday the 13ths for this year. For the rest of us, we are assessing our bad luck with regard to the fact that if you live in the mid-Atlantic it appears that we will have our annual late wintery weather, followed by 20 minutes of Spring, followed by a long, hot summer. In any case, as folks returned from Easter vacation, Congress was back in session and when not focused on Facebook, was having hearings on opioids. And FDA, who did not take a break, was busy too.

Here is a bit of what happened this week.

  • FDA Finalizes  Two Guidance Documents on Next Generation Sequencing  (NGS) Tests – FDA has been charged with enhancing innovation via, among other means, of reduced regulatory burden. This week FDA issued two guidance documents on NGS Test development that the agency said in its release would enhance the ability to deliver targeted treatment. According to the Commissioner, the guidance documents provide a more modern framework for the development and evaluation of such tests. The first guidance allows for developers to rely on a clinical evidence from FDA recognized databases to support claims in relations to efficacy. The second guidance makes recommendations for designing, developing and validating tests for genetic diseases. In a speech given the same day of the release before the 2018 Community Oncology Conference, Dr. Gottlieb provided additional perspective on the two guidance documents and their impact. 
  • FDA Guidance on Moderate Risk Devices to Utilize 510(k) Clearance  – To gain marketing clearance for a moderate risk medical device, manufacturers have been required to test the device under development against an existing (predicate) device already approved to demonstrate safety and efficacy.  But under a new guidance proposed by FDA this week, such a manufacturer might instead be permitted to volunteer to use a different pathway which would allow clearance if the manufacturer demonstrated that the device meets a set of objective, established criteria instead. According to the FDA, the predicate device and the new device are often very different given advances in technology, presumably setting up the comparison of an apple to another really different apple. This has been another in a line of new policies which are termed “modernizing” FDA’s regulatory approach by easing the regulatory burden on manufacturers. The objective is to do so without compromising FDA’s gold standard on safety and efficacy. 
  • First Generic Approvals – FDA updated its list of 2018 First Generic Approvals. In 2017 there were 80 such approvals during the year which had been a banner year for generic approvals overall. It has been said that this year will also see a large number of approvals. The 2018 list has 12 such approvals so far for the year, while in 2017 by the end of the first quarter there were also 12 approvals. This year, therapeutic categories touched by the approvals included secondary parahyperthyroidism and HIV. 

Things to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices

Photo by Atul Vinayak on Unsplash


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FDA Enforcement Lags, Focus on Promotion of Unapproved Drugs

It goes without saying that enforcement by FDA’s Office of Prescription Drug Promotion (OPDP) as identified through regulatory action letters (warning or untitled) over the past few years is a mere shadow of its former self. Enforcement was once robust, peaking at 156 letters issued 20 years ago in 1998. The year before last, the number had dropped to 5, only increased to 11 by a December surprise when 6 letters were suddenly issued within the month. So far this year, there has been only one.

Many have speculated on the reasons for the drop, and FDA has provided nothing specific in response to inquiry. With enforcement actions so few, it pays to look closely at the circumstances that have moved FDA into action. 

Of all the major violations that generally occur with pharmaceutical product promotion – Omission or Minimization of Risk, Superiority Claim, Broadening of Indication, Unsubstantiated Claims, Promotion for an Unapproved Use and Promotion of an Unapproved Drug, the one that ranks the highest in terms of number of violations is by far risk, the one that has ranked the smallest has been Promotion of an Unapproved Drug. FDA regulations state that a drug sponsor, or investigator, or anyone working on their behalf, should not indicate that a drug is safe or effective prior to its approval by the agency.

How uncommon is it? If you look all the way back to 2004, there have been only 14 letters issued covering 15 different communications vehicles for promotion of an unapproved drug, which averages out to one per year.

However, they did not come out at the rate of one per year – in fact, over one-third of them came out during 2016 and 2017. While comprising only 4.6 percent of all the letters issued since 2004 have involved promotion of an unapproved drug, 28 percent of the letters issued since 2015 have involved promotion of an unapproved drug. Either it is happening more often, or it is something about which FDA still cares a great deal about, even in an era of diminished enforcement actions. 

It may be worth noting that, with one exception, nearly all of the letters were sent to entities that are lesser known.

What trips the regulatory wire? FDA tends to look at the totality of a presentation when assessing promotional communications – meaning the agency looks to see if there are factors which contribute to an overall impression rather than just an individual statement. In other words, use of the brand name, in conjunction with other aspects of the way the drug is being talked about (such as being displayed next to already approved drugs) could create the impression that a drug has been approved. That said, of course, specific language – like indicating that a product has fewer safety issues than the current treatment (“the drug is safe” and “the drug is efficacious”) – could also be a cause of concern.  Common to the violations cited in recent letters were present tense “conclusory” language such as “accurately detects” and ” is a unique long-acting formulation”. 

Are the violations predominant in one kind of media over another?  Over the span of time, violations have occurred as oral statement from clinical investigators during medical meetings, on exhibit panels at medical meetings, in brochures and in videos. Of the total number of 15 letters, 9  involved a website, making it the most common communications vehicle involved in promotion of an unapproved drug.  That is a place one might think it would have been the easiest to prevent, especially given the fact that oral statements are harder to guard against when it comes to any violation. It may just be an indication also of the fact that websites are easier to monitor and detect. 

There has been some speculation that FDA is centering its enforcement in areas where there is highest risk – which would include things like a product with serious potential adverse events being promoted in a way that omits risk information. When it comes to pre-approval promotion, not only has the proportion increased, but the rate of such violations as well. Those two facts combined might lend the impression that this is a violation in which FDA is more interested. And given the prominence of the category as a subject of FDA enforcement, communicators should take note. 

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