Weekly Roundup 12.18.15

It is the rush. I am caught wondering how I got here.That situation that every year I say I am going to avoid, but don’t. The one where I am rushing to get everything done before the holiday that needs to be done and feeling overly stressed. The lack of time meant a lack of posting this week, but I shall use the time over the holiday to get a little more on track. In the meantime, it was a busy week an here are a few notes of interest.

  • Future of Research – For the past few years, the future vision of government-sponsored medical research has sometimes been clouded sending advocates to the Hill to try to persuade policy makers not to cut funding. This week two items of significance provide a degree of clarity for the future. First, NIH announced that it was unveiling a 5 year strategic plan – Turning Discovery into Health. The plan outlines a very long list of aspirations that range from new levels of intensity in the battle against cancer, new wearables that will break new ground and new breakthroughs in vaccines and in HIV. And to make that all possible, with Congress poised to pass a new budget deal, funding for NIH would appear to be on track to receive the biggest boost it has gotten in over a decade.
  • Cascade of Approvals Before the Holidays – A lot of people wonder if there isn’t a big rush at the end of the year to get out approvals before the holidays. For those with that point of view, this week provided some food for thought with the announcement of three new approvals. The first was the approval of Bridion to reverse the effects of neuromuscular blocking drugs during surgery – drugs often used during intubation. The agency said that the drug could be used for faster patient recovery after a procedure but also pointed out that some patients experienced bradycardia and raised the concern of some patients experiencing anaphylaxis. News was made this week in both the diabetes and biosimilars worlds with the announcement of the first insulin approval for a follow on product in the abbreviated regulatory pathway set up for this purpose by FDA. Finally there was an approval announced for the first external defibrillator for children at risk of sudden cardiac arrest who are not candidates for an implant. Heretofore, adult devices had to be used off-label.

And for those wondering if in fact there is a year-end growth in approvals – this month has certainly seen a number of such announcements with a total so far of 9 approvals. In December of 2014, there were 17 approvals or expansions of label announced. By contrast, in 2013, there were only 7.

That’s it for me this week. Off to the post office!  Next week has Friday falling on a holiday and the same for the week after, so Eye on FDA will be on a slightly irregular publication schedule getting ready for some themes to explore in the new year. In the meantime, have a fun, safe and happy holiday season.

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Weekly Roundup 12.11.15

The trees have shrugged off the last of their leaves, allowing twinkle lights to be suspended from their branches. Stockings are hung. Trees are up. And while we may be lulled into thinking that it is going to be a quiet time of the year, in fact, everyone is gearing up to get everything done before the year end and the pace is a little frenetic and we might be busier than we want to be which means that last week, some of us didn’t even get the Weekly Roundup out and can barely get it done today!

That said, here it is and here is some of what happened this week and a little bit of last:

  • Immune Stimulating Therapy Approved – FDA announced this week that it was approving Empliciti in combination with two other treatments for the treatment of multiple myeloma. FDA stated that Empliciti works to activate the body’s own immune system to attack and kill multiple myeloma cells. The two other treatments with which Empliciti are to be administered are Revlimid and dexamethasone and is the second approval this month for multiple myeloma which the National Cancer Institute estimates will have over 26,000 cases diagnosed this year. Empliciti received breakthrough therapy status from FDA along with orphan drug designation and priority review. A company press release can be found here.
  • FDA OKs Marketing of Cooling Cap for Those in Chemotherapy – Ok, when I first saw this I misread the FDA headline on the press release to say that “FDA Allows Marketing of Cool Cap…” But perhaps that should be the headline. For many hair loss can be an unpleasant side effect while undergoing chemotherapy. This week FDA ok’d a product that is meant to minimize that side effect. The way the cap is meant to work is that it circulates a cooled liquid through the cap that reduces blood flow that in turn causes the chemotherapy to have less of an impact on the hair follicles. The FDA press release states that two-thirds of women in studies using the cap reported less hair loss when compared to women who did not use the cap. The release does not state how long one must wear the cap, but did say that common side effects included discomfort to the head, neck and shoulders and chills. The product was approved through FDA’s de novo classification process which is a pathway for low to moderate risk devices that are novel and not substantially equivalent to legally marketed devices.
  • Two Orphan Drug Approvals – In a year that has to be a banner year for orphan drug approvals, two new ones were added this week. First was the approval of Kanuma (sebelipase alpha) for the treatment of lysosomal acid lipase (LAL) deficiency. This condition can cause a harmful build up of fats within cells that can lead to liver and cardiovascular complications and in its more severe form, patients do not survive the first year of life. The therapy provides a protein that replaces the function disrupted by the genetic disorder that causes the deficiency and is manufactured using genetically engineered chickens. In another approval this week, FDA announced action this week to ok the first recombinant von Willebrand factor for the treatment of bleeding episodes caused by protein deficiency that causes von Willebrand disease and affecting about 1 percent of the population.

That’s it for me this week. Have to get back to the rush of the season. In the meantime, have a great weekend.

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Precision Medicine and FDA – Emerging Cloud Platform in Beta

In his State of the Union message this year, President Obama announced a new effort in the way research is done when he unveiled the Precision Medicine Initiative (PMI). The stated goal of this initiative is to begin the transition from a world where we lacked information about how individuals respond to medicine to one where medicine is directed more precisely to individuals. As we have gathered the ability to utilize genetic information and we are amassing large quantities of data on patient experiences, it only makes sense to utilize those abilities to better inform treatment for various types of individuals. In particular, the initiative is aimed at developing more targeted cancer treatments.

The PMI began with some initial investment by the Administration – the bulk going to the National Institutes of Health (NIH) and to the National Cancer Institute (NCI), but there was an allocation made to FDA as well. Part of that investment appears represented in the development of a new cloud based platform by the agency.

Called PrecisionFDA, the platform has entered a phase of closed beta testing. What is it? The cloud-based platform does not appear to fulfill a regulatory function, but rather provides a private space for analysis as well as providing a community for researchers who might be the ones who would do that analysis. In the agency’s own words – it provides a private area where individuals or organizations can conduct genome analysis and comparison against reference material as well as a community area where results can be published and shared along with relevant materials and tools. The agency says that this community is in the process of growth and includes genome test and software providers, standards-making bodies, pharma and biotech companies, healthcare providers, academic medical centers, researchers, patients as well as FDA and other governmental bodies.

You can see a very short (but not very informative) video about the effort here. The agency has also set up a Web Page to overview it. But the most information is contained in a Why, What, Who and How set up here. There is also a link provided for those interested in applying for access to the community, though standards for acceptance did not appear clear.

Interested in following the developments of PrecisionFDA?  If so, FDA has started a new Twitter feed for the project – @precisionfda, joining the other 16 FDA Twitter feeds of which I am aware. If you are interested – you can subscribe to a page seeing only FDA tweets across their feeds here on the Eye on FDA Twitter Feed or just see the entire FDA Twitter population.

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The Growing World of Do It Yourself Health

Sparked by an inquiry regarding a speaking gig around the future of do-it-yourself (DIY) medicine got me to thinking about how much the concept has changed over the years, and most certainly at an accelerating pace.

Back in 2011, I did a posting about a wonderful exhibit put on by the Philadelphia Art Museum called “Health for Sale“. It was a collection of poster art in pre-regulatory days used to market lotions, cough medicines, elixirs and all sorts of promises to cure what ails you, or might ail you. The exhibit provided a peek into the world of Do It Yourself health as it existed before there was any regulation – when any mixture put in a bottle could be sold as a cure for almost anything.

A lot has changed in self-care or DIY healthcare since then and some things have not. On the one hand, there is a whole agency now seeing to it that claims that are put to medicines are accurate. But on the other hand, the very concept of DIY healthcare is transforming and in many respects, involves new expressions where the regulatory side of things may have trouble keeping up.

People are becoming increasingly involved in their own care on a number of fronts since the days of Health for Sale.  From seeking knowledge about a condition to treating and preventing it, there are new levels of involvement for consumers/patients in their healthcare. While once we were passive listeners in the doctor’s office, today we come in more engaged participants.

For starters, the patient, some might say, became more enlightened with the advent of direct-to-consumer advertising – others might not. Whichever way you fall on that, it is a means of spreading information about conditions and treatments that was not available prior.

In addition to DTC, there is a huge industry in supplements – the ability to self-prescribe and self-treat and seek to prevent conditions with products that are not drugs. And for those that prefer drugs, in the 1990s there was a pretty large migration of products from RX to OTC status – drugs which treated symptoms like allergies and rashes and heartburn to drugs that treated conditions like GERD – a switch pipeline that would appear to have slowed somewhat these days by comparison.

And then there are home remedies. In the early days of the AIDS epidemic before there were any antivirals on the market, people tried growing mushrooms in their homes to make tea or concocting formulas that were passed around for homemade treatments that would do something in the face of a growing epidemic where medicine offered nothing.

Today it is technology that has come to play a much bigger role. First of all, there is Dr. Google – the ability to look up information in whatever form and from whatever source, either Facebook, Wikipedia or the treatment guidelines of a medical society, at the very least giving a patient something more to talk about during the medical visit than in 1985.

Social media allows one to crowd-source - to share symptoms and get opinions and help from friends and from strangers alike. It allows for an enhanced referral process and rankings to assess a provider. And patients can find much needed support in guidance from other patients and in discovering the existence of much needed resources.

Today, one can use one’s phone for a phone call, but is much more likely to be used for other things – many of them health related. With the advent of apps, you can check blood pressure, monitor activity, count calories, check on your sleep patterns or a host of other medically related endeavors. Or if you choose, you can get a wearable device to handle some of that load. At some point, you may use it to monitor more than functions. Perhaps in the future you will be able to do things like monitor your own liver function or cholesterol level -which might open the way for the RX to OTC switch of cholesterol lowering medications.

In the past 25 years, there has been a tremendous evolution of DIY care. Some of the change does not eliminate the medical presence, but makes it more convenient and accessible and which rely more heavily on the patient in some respects – such as Telemedicine.

Through all of this, the regulatory aspect has struggled to keep up. FDA still struggles in completing its task set out in 2009 regarding guidance in the use of digital and social media platforms.  Many apps were on the market before FDA began addressing that category. Change does not occur in a vacuum. New developments and their uptake are occurring at a much faster rate than their regulation and when you think about it, DIY is an entire evolving health industry segment signifying perhaps how far we have come from the days of Health for Sale.

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Up Scope! Pharma, Periscope and Some Regulatory Questions

Back in June in a posting about the new (relatively) live streaming capability brought to use by Periscope. The app, acquired by Twitter prior to its launch early in 2015, allows you to live stream content from where you are to your followers who can provide commentary – and to allow your Twitter followers to see it afterwards, including the comments. Periscope, in essence, allows you to be your own reporter. In the June posting, I talked about how it might be used to film AdComm outcomes and that it had great utility in healthcare for developments and announcements at medical meetings.

But would a pharma company use Periscope? Are there special regulatory concerns that might be in play given this particular digital venue? Here are thoughts on each question.

So are pharma companies using Periscope?  You bet.

I checked out this list of the top 25 pharma companies by global sales.  I found that 10 of them – or 40 percent of the top 25 pharma companies – appeared to have established Periscope feeds and some of them are even following the Eye on FDA Periscope feed (thank you!). They ranged in the size of followership from 0 (though for each of these, I became a follower so now they have at least 1 follower) to 717. I have heard of at least one company holding an event using their Periscope feed.

Which brings us to the second question – are there concerns that regulatory might bring up associated with Periscope use?

What if a company were running a broadcast of a patient group at a medical meeting and someone mentioned an off-label use? What if they stated something that was misinformation about the product? What if they mentioned an adverse event?

So what if someone mentioned an off label use or other misinformation during a pharma Periscope session?  Of course, FDA/OPDP does not issue platform specific guidance documents. But in the June 2014 Draft Guidance document (page 5) on correcting misinformation, FDA had this to say about third party content on a company hosted site when they provided an example:

“A firm hosts a discussion forum about its drug or devices’ FDA-approved use on its corporate website and does not participate in the discussion, but it does monitor the forum for profanity and obscenity. The forum includes an overarching and clear and conspicuous statement that the firm did not create the content of the forum. The firm is not responsible for the information that is posted by an independent third party and can, if it so chooses, correct misinformation according to this guidance.”

This might seem to suggest that a third party speaking on Periscope session – analogistic to the corporate website in this case – might convey misinformation and the company would not be responsible – as long as the company were not participating in the interview and was able to communicate a conspicuous statement that the firm was not creating or prompting this content. Or it might not.

Following that example, FDA also states:

“However, a firm’s control over, involvement with, or influence over a product-related communication, even when generated by a third party, may result in the firm being responsible for the information as a promotional communication. Thus, firms might be responsible for UGC that they solicit or influence, regardless of the forum.”

So perhaps if a company asked for an interview with a specific entity, as opposed to benign broadcasting of such an interview by a third party (with a disclaimer), responsibility might ensue.

That is as it pertains to misinformation. Regarding an adverse event – that is one of the five question areas raised in the 2009 public hearing about which we have had no guidance from FDA.  We would only know by further FDA explanation, usually through the issuance of untitled letters, which OPDP has issued only rarely this year. In the meantime, it is up to individual companies to decide how, and if, to proceed.

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