New Directions for FDA, Part 1 – 21st Century Cures

One of the often stated priorities for lame-duck Congress has been voting on the 21st Century Cures Act. In a press release from Saturday, November 26 from the House Energy and Commerce Committee, the House is set to vote on a new version of the bill following release by House and Senate health committee leaders on a “final” bill.

Originally passed in the House in July 2015 by a lopsided vote of 344-77, all attention then went to the Senate side for passage. And then there was a long wait. This year in April there were statements made that a bill could be out of committee within the week and ready for Senate action. It did not happen. At the beginning of September, there was some talk that it would get attention before the month was over. Now it comes to the lame duck session after the election and media reports that Senate Majority Leader McConnell has termed the bill a priority.

Among other things, the Cures Act seeks to liberalize the way FDA goes about regulating the approval of and access to drugs and biologics through a number of mechanisms. In addition the new final bill has something in it for just about everybody, including funding for the Precision Medicine Initiative for NIH, a boost in funding for FDA, $1 billion in grants to states to combat the opioid epidemic. Many claims are associated with the bill by its proponents – increasing research collaboration, incorporating patient perspective into drug development and the regulatory review process, providing new incentives for the development of drugs for rare diseases and provides support for the “cancer moonshot” initiative. In other words, it would all sound good. For some, too good.

The great milestone in moving FDA to speed approvals occurred during the early days of the AIDS epidemic when drug approvals were long and drawn out and mechanisms to speed approval were lacking. Through the course of activism and effort, FDA reforms allowed for new means to speed the process of approvals though Fast Track and Accelerated Approval programs. Subsequent reforms such as breakthrough therapy designation and orphan drug status designation have intensified the agency’s ability to bring innovative treatments forward for approval.

One might think that AIDS organizations – such a prominent proponent of earlier progress and liberalization of FDA’s approval process – might continue in support for reform offered by the Cures Act. But that is not necessarily the case. Before the 2015 House vote, co-founders of the HIV advocacy organization Treatment Action Group (TAG) joined with former FDA Commissioner Dr. David Kessler to pen an op-ed in the New York Times called “Don’t Weaken the FDA’s Drug Approval Process“. One of the several concerns raised in the piece was that biomarkers could be used to approve a wider set of treatments beyond those that are life-threatening or serious conditions and could allow clinical experience to replace data from well-controlled clinical trials, long considered the gold standard. In addition, TAG has issued an Action Alert for its member related to the lame duck session vote.

It is worth noting that in the wake of liberalizing the FDA approach to approvals in the 1990s, afterwards there were some high profile drug withdrawals that called into question whether or not drugs were being approved too quickly, at a cost to safety. The pendulum, it appears, has now swung again.

For critics it boils down to the issue of safety versus speed and whether or not the Act is even needed. FDA already is the gold standard and approves drugs more quickly than ever. New mechanisms to enhance approval such as Breakthrough Therapy Designation have been put into place and companies are utilizing such means. There were a large number of drugs approved in the past year for rare diseases. And FDA looked to patient experience in approving one drug.

TAG is by no means the only consumer group in opposition, but certainly one of the more interesting given the history. The proposed legislation is certainly one of the most talked about for the lame duck session of Congress and lobbying is reportedly in full swing. The House Energy and Commerce Committee is issuing almost daily press releases on the bill. There is likely a vote this week in the House on the new version and the Senate is said to consider it in December. If passed and signed into law, it represents perhaps only a part of the change that may come in the wake of the elections. More on that in Part 2.

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Posted in Current Affairs, FDA Policy, Legislation | 1 Comment

Local Ballot Initiatives Impacting Health and Healthcare

There is a good deal of commentary about what happened at the nationally in this year’s election cycle. But a good deal happened at the state level as well – things that will impact healthcare and may influence how other states respond. In addition, there were state issues that were voted on that could have an impact on the national debate of some specific topics. All in all, according to Ballotpedia, there were 154 measures available for voters to consider across the states this November, and a number of them touched on healthcare.

In that capacity, here are a few the state ballot initiatives that are of note:

  • Prop 61 – California – Pharma Pricing – This proposition would have required state agencies to pay either the same or a lower price for prescription drugs than the U.S. Department of Veteran Affairs .  The measure was seen as a focal point on the issue of pharmaceutical pricing. The measure failed with 54 percent against to 46 percent in favor.
  • Amendment 69 – Colorado – Single Payer - This measure would have created a single payer healthcare system called ColoradoCare that would provide universal healthcare for residents of the state and financed through a tax on wage and non-wage income. The measure was soundly defeated by nearly 80 percent against to 20 percent in favor.
  • Proposition 60 – California – Condoms in Pornography – Certainly one of the more unusual ballot initiatives voted on this cycle involved a proposal to require actors in pornography films to be given condoms to wear during performances and would further have required the makers of the films to cover the costs of medical screenings and examinations. The measure failed with nearly 54 percent against compared to 46 percent in favor.
  • Proposition 106 — Colorado – End of Life – This ballot measure sets up standards that would allow residents to legally seek and obtain assistance to end their life through the self administration of drugs when facing the circumstance of a terminal illness. The measure passed by nearly 65 percent in favor to 35 percent opposed.
  • Multiple – Medical Marijuana - There were a number of marijuana legalization ballot initiatives, some of which were about recreational use, but several of which were specific to medical need -
    • Arkansas Issue 6 – allows medical use of marijuana for 17 specific qualifying medical conditions, passed 53 percent in favor with nearly 47 percent opposed;
    • Florida Amendment 2 - would allow the regulation of marijuana by the state and use by patients with qualifying conditions such as HIV/AIDS, glaucoma, Parkinson’s Disease, PTSD, ALS, MS, cancer and epilepsy- passed 71 percent to 29 percent;
    • Montana Medical Marijuana Initiative – would amend existing law passed by the legislature to remove existing numerical restrictions on medical marijuana providers who had been limited to the number of patients they could serve, among other things – Passed by nearly 57 percent to 43 percent;
    • North Dakota Medical Marijuana Legalization Initiative – Sets up regulation of cultivation and distribution for medical marijuana for patients of specific medical conditions passed by nearly 64 percent to 36 percent.
  • Soda and Sugary Beverage Taxes - While a significant yes regarding the use of medical marijuana, there was also support for a tax to be placed on soda and beverages containing sugar in four municipalities. The taxes work by leveraging a tax on the ounce for specified types of beverages and the municipalities where the measures passed primarily in California – San Francisco (62 percent in favor, 38 percent opposed), Oakland (61 percent in favor and 39 percent opposed) and Albany (71 percent in favor, 29 percent opposed) and one in Boulder Colorado (don’t have the percents).

All of percentages were gleaned from the site Ballotpedia and were rounded up.

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FDA Seeking Even More Input on Character Space Limitation

This week FDA published a notice in the Federal Register seeking comment on the proposed collection of information regarding research entitled “Character-Space-Limited Online Prescription Drug Communications“. The notice states that the objective of the research is to test whether a link to prescription drug risk information can effectively convey the risks associated with a drug when benefit claims about that drug are made within the character-space-limited communications used in prescription drug promotion.

In other words, if a tweet or small online ad mentions that Drug X Cures Toenail Fungus will a person run out and get their doctor to give them that drug without clicking on the link to warning information? Is this development a good one or one to be concerned about?It sort of depends on the agency’s motivation.

On the one hand, FDA’s curiosity regarding the matter of balancing risk information with benefit information in a character-space-limitation setting is to be commended. It demonstrates a healthy interest in exploring the realm of social and digital media – something the agency has been grappling with since 1996 when it held its first meeting on dealing with the newly emergent Internet. Understanding the implications of social and digital media has not come easy to FDA and the menu of guidance documents promised in 2009 at a two-day public hearing on the topic has produced only a partial response on the part of the agency. In fact, patients, physicians and pharma companies have moved far ahead, and FDA has been struggling to catch up for many years.

The issue of character-space-limitation is one of the areas in which FDA has actually shed some light by producing a guidance document on the topic in June 2014. Is the agency trying to crystallize its thinking in that regard? Are they looking for evidentiary support to go back on the position they took in April 2009 regarding the “one-click rule” – the notion that risk information could be included a click away – when they issued 14 untitled letters in a single day about the use of links to third party information? If so, that would be a good thing.

But on the other hand, one might question whether so much attention to this particular aspect of social media use by pharma is actually warranted.

In the first place, between the patient and the prescription there is a learned intermediary – the doctor. The real world is made up of many variables that will impact whether or not risk information is picked up. And the real world is made up of many attempts to alert patients to risk information, which may or may not be particularly useful. Right now many prescriptions are delivered with pages and pages of information about a drug, but how many people actually read and absorb that information? Are those inclusions really impactful? (I personally doubt it). Do people read them once? Do they read them every time they get a re-fill?  How people get risk information, how they perceive it and act on it and how other factors figure in is complex. In short, a study to discover the use of a single link to risk information is going to tell you a small part of the picture, but it is not going to tell you the whole picture, or anything looking like it.

In the second place, FDA might better spend its time addressing the gaps in guidance around social media – for example the Use of Links to Third Party Sites guidance that was on the guidance agenda not only this year, but last year as well. Or what about adverse event reporting, listed as one of the original five questions posed during the 2009 public meeting on social media?

Finally, in fact, while pharmaceutical companies have many Twitter feeds (I have a database that contains all of the feeds of which I am aware and there are over 300), the overwhelming majority of them are not product specific, but are oriented to corporate news, recruitment and advocacy. You can see the Twitter list here.

The decision to delve further into the question of a link to risk information is important, but the question has been around for a long time. Let’s get it answered and move on.

If so inclined, you can submit comments to the docket on this matter here. And for an overview of FDA activity around social media, there is a tab on the blog devoted to resources located here.

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Posted in FDA Policy, Social Media | 1 Comment

Weekly Roundup 11.4.16

We are almost through it!  Just a few more days.

In the meantime, the more enjoyable aspects of autumn, chilly nights, fires in the fireplace, and brilliant leaves are all upon us. Soon the end of year holidays will be upon us. In the meantime, a few interesting things from the week:

  • OPDP Issues Fifth Warning/Untitled Letter of 2016 – To say that it has been an anemic year from OPDP’s enforcement letters is a bit of an understatement. This week the agency issued its fifth of the year and if it does not double that number in the next few weeks, it will be the lowest level of enforcement seen from this office of FDA. The untitled letter cited a video for Oxtellar XR – an extended release tablet indicated for adjunctive therapy of partial seizures in adults and children 6-17 years. The product has some specific risks associated with use in the label, including warnigns of anaphylactic reactions among others. The video was cited for a lack of adequate diretions for use because of the specific language used in the video that OPDP felt broadened the indication of the compound to include all seizure types and because the presentation did not present risk information in conjunction with the benefits. Of the five letters issued this year, four were Untitled Letters and one was a Warning Letter.
  • AdComm Gives Up Vote on Nocturia Candidate – A meeting of the Bone, Reproductive and Urologic Drugs Advisory Committee voted 14-4 in favor of the benefit/risk profile of SER120 (desmopressin nasal spray) for the treatment of nocturia – a condition in which adults awaken two or more times a night to urinate. If approved, it would be the first treatment approved for nocturia, though there have been previous attempts.  In January 2015, a tablet formulation put forth by a different company and reviewed by a different advisory committee, resulted in a negative recommendation.
  • How Much of the Economy is Regulated by FDA? For many years, many people (including this people) have touted the characterization that FDA regulates one-fourth of the U.S. economy – or in other words, regulates 25 cents of every dollar spent. In a blog posting this week, FDA put that characterization to pasture with a new study by the agency’s staff economists that takes it down a peg – with FDA regulating one-fifth of the economy, or 20 cents of every dollar spent – a level that has apparently held steady over the past five years. The study was done analyzing expenditure data from the Bureau of Economic Analysis (BEA). I stand corrected.

Enjoy the weekend everyone. And get out and vote, if you haven’t already.

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Weekly Roundup 10.28.16

It has been way too long since a Weekly Roundup and way too long since a regular posting. The autumn has turned out to be an exceptionally busy time so apologies on both fronts. But today I’m carving out a little time to at least get out a Roundup and hopefully will have a few new postings next week regarding some things on my mind.

In the meantime, we are getting up in darkness and switching out our closets from summer wear to autumnal clothing. Upstate New York has seen some snow. In the mid-Atlantic, we have had some chilly mornings. And on Monday we will hand out candy to costumed little ones counting on our answering the doorbell. In the meantime, here is a bit of what has transpired at FDA:

  • Male Hormones, Female Sex-drive and Guidance – A couple of things on this front. This week FDA issued class-wide labeling changes for testosterone and other anabolic steroids to include expanded warnings, stating a possible association with abuse and dependence and citing specific potential among adults and adolescents engaged in body building and warning of a number of adverse associations. Also this week, the agency issued guidance entitled “Low Sexual Interest, Desire, and/or Arousal in Women: Developing Drugs for Treatment” to assist sponsors in what has been an often elusive category in the development of treatments for women. The guidance provides overall thinking toward a clinical trials, with specific focus on Phase III trial designs.
  • Accelerated Approval for Soft Tissue Carcinoma Treatment – A new treatment – Lartruvo – with breakthrough therapy status, priority review and fast track designation won accelerated approval this week for the treatment of certain types of soft tissue carcinoma (STS). In the FDA release, Lartruvo is described as a platelet-derived growth factor receptor-alpha blocking antibody which may help slow or stop tumor growth by blocking receptors that cause growth. Lartruvo also has orphan drug status, with the National Cancer Institute estimating that 12,310 new cases and 5,000 deaths will occur in 2016. Lartruvo is approved for use with chemotherapy in STS for patients who cannot be cured with radiation or surgery and who have a type of STS for which chemotherapy is an appropriate treatment. You can read the company press release here.
  • First Line Treatment for Immunotherapy in NSCLC – Keytruda, a compound under study for multiple types of cancer, received FDA approval as a first-line treatment of metastatic non-small cell lung cancer in patients who have a high PD-L1 expression over 50 percent. The approval was granted after studies showed improved survival time over chemotherapy. The company press release can be found here.

That’s it for me this week. Have a great weekend everyone.

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