Brief Blog: ICER Releases Value Framework Update For Public Comment

Boston's ICER, the Institute for Clinical and Economic Review, released an updated "value framework" on February 1, 2017, with comment open until April 3.  ICER promises to post finalized updates to its value framework by April 15.   (It gives itself 12 days or less to review and revise based on public comments.)

See:

• ICER press release here.
• Project home page, here.
• 24-page update PDF, here.
• Coverage at Modern Healthcare, here.

For mostly favorable 2016 articles at STAT, here (April) and here (August).  2016 interview with ICER's head, Steven Pearson, at AJMC, here.  2016 blog at Health Affairs by Pearson, here.  A few weeks ago, Pearson published in JAMA Internal Medicine that national guideline committees for hepatitis and cholesterol management did not fully meet IOM conflict of interest criteria (here).

Assuming that favorable aspects of the work speak for themselves, some contrary viewpoints are:

• A disgruntled blogger, here.
• Critical article in Huffington Post, here.  

Some additional notes after the break.

Brief Blog: ACS and Others Ask Hill for LDT Oversight

In November, post-election, the FDA announced it was tabling the release of final guidance documents on LDT regulation (here, here).   Two months later, in mid January, the FDA released a white paper on its latest thinking as a result of two years of back-and-forth on LDT regulation (here, here, here, here).

On January 24, 20-17, 30-plus organizations, including the American Cancer Society, urged that "updating the LDT framework" be a priority of this Congress.   The two-page PDF is online here.

These stakeholders write,

There is no systemic way to be sure of the accuracy and reliability of these tests. Several cases illustrate the challenges with this lack of oversight. Researchers sent samples from the same cancer patients to different LDT providers for cancer testing, and found only 25 percent of the drug recommendations based on test results overlapped.

...The current system of LDT oversight is inadequate and needs updating.

The letter is addressed directly to the Senate Majority Leader and the Senate Minority Leader, rather than to committees in the House or in the Senate.  Coverage at Genomeweb, here.

On February 1, 2017, the trade journal CQ Healthbeat ran an article that LDT regulation is again on the Hill agendas for this year, given the support both of the clinical groups cited above and the joint support for some level of regulation revision at both Advamed and ACLA.

Brief Blog: A Trending Buzzword in Healthcare: Systemness

For its January 30 issue, the back page of the hospital trade journal Modern Healthcare has a full page advertisement for a "Systemness" website.   Turns out, the term has a few years of history in US healthcare and may be a trending buzzword now.

For example, Modern Healthcare ran an opinion piece introducing "systemness" to its readers in 2011.  In a 2013 publication, the ECRI Institute has a cover story on "Systemness," here.  Becker's Hospital Review ran a story in 2014, here.  

A new Modern Healthcare special website is here, sponsored by Dignity Health, with an 11 page PDF white paper here.

Footnote:  In the same vein.   For an interesting new blog on using big data for better healthcare, rather than administrative pains, pointless metrics, and gaming, see Tom Burton of HealthCatalyst, here.

Quinn: Speaking at Diagnostics Reimbursement Course, SF, Feb 19

I'm pleased to participate in a Short Course on Diagnostics Reimbursement held in parallel with the giant annual Molecular Med TriCon in San Francisco.

The Short Course, "Coverage and Reimbursement for Advanced Diagnostics," will be 2-5 pm on Sunday, February 19, in San Francisco.  The TriCon conference is a large, international gathering and runs February 19-24.

The short course agenda is here (or below the break).   The main conference webpage is here.  The course is organized by Katherine Tynan of TynanDX Consulting.

___

The Downing Report, 2017: Innovation at HHS

Gregory Downing, Executive Director for Innovation at HHS, which an early proponent of of precision medicine in the mid-2000s at HHS, researching and calling for more coordination among NIH, CMS, FDA, and other parts of HHS and the government.  Since then, he has had a broader mandate for HHS innovation initiatives.

On January 15, 2017, Downing released a 63-page Innovation Report, along with a blog that frames its genesis and purpose.

Downing's blog entry is here.
The 63-page PDF report is online here.

The report has six sections:

1. Innovation and Entrepreneurship in the US HHS
2. Innovation Management Theory in Government Practice
3. Design, Management, Evolution of HHS Innovation Program
4. Innovation and Entrepreneurship in State and City Governments
5. Models of Sustaining Innovation in Large Private Sector Organizations
6. Strategies for the Future

As an Appendix, there are a couple dozen references cited (including books by Harvard's Clayton Christensen) and several dozen interviewees are listed.

Barack Obama Writes NEJM Article on ACA Repeal

On January 26, 2017, Barack Obama published an article NEJM on current approaches to ACA Repeal.   He views repeal, without replacement, as a bad idea.   The article is online here.

Gulfo and Soon-Shiong Continue to Circulate as Trump Allies

Additional news stories this week on a fourth candidate for head of FDA, Dr. James Gulfo.

Prior potential nominees for FDA have included Republican speechwriter and investor Jim O'Neill, young biotech founder Balaji Srinivasan PhD, and well-respected physician-policy expert Dr. Scott Gottlieb.

In addition, in a speech on January 31, President Trump focused attention not only on pharma prices, but on (A) speeding approval "instead of waiting for many many years" and (B) access for the terminally ill, usually viewed as "right to try" laws (here).  Trump also stated that "We have a fantastic person that I think I'll be naming fairly soon who's gonna streamline the FDA and you're gonna get your products either approved or not approved, but it's gonna be a quick process."  See here.

Dr. Joseph Gulfo

The week of January 23, new attention has focused on Dr. Joseph Gulfo.  For a full listing of his credentials, see his impressive Linked-In page, here.

Gulfo trained in oncology, worked in CROs and biotech startups, and had his longest career segment as CEO of Mela Sciences, which developed the Melafind device for augmented accuracy in selecting atypical moles for biopsy.  Melafind had a difficult multi-year course at the FDA, which Gulfo detailed in his book, Innovation Breakdown (2014).  At Amazon, here.  Book website, here.  A 2011 6-page white paper at the Progressive Policy Institute (here) gives a summary of the FDA's delays and policy issues on Gulfo's product Melafind.

Gulfo, an MD/MBA, has also written a general book on leadership and management (here).

Gulfo is a strong proponent of approvals based on intermediate endpoints, including biomarkers.  More about Gulfo and his book, after the break.

New News on Dr. Soon-Shiong

On January 24, the subscription health news website STAT ran an article that Dr. Patrick Soon-Shiong, who's been shortlisted by NIH watchers as a  potential head of NIH, could also appear in the Trump administration in a novel role.  The role could be closer to health innovation czar, rather than an existing title like Surgeon General, Head of CMS, Head of NIH, etc.  The subscription article is here with a follow-on the next day here.  Aggregated news sources suggest that Soon-Shiong has met at least three times with Trump, one shortly after the election and twice during January.

Soon-Shiong is a very, very strong proponent of molecular medicine, precision medicine, and Cancer Moonshot.  For additional speculation on the health czar role, Managed Care, here; LA Biz, here.

Update: On February 5, a New York Times article by Katie Thomas surveyed the FDA candidate field, dealing at most length with Jim O'Neill, suggesting the Times viewed him as a leading candidate worthy of comment.   O'Neill and Scott Gottlieb declined to comment to the Times.  Gulfo commented at some length.  Times article here.  More on Gulfo after the break.

Ex-CMS Administrator Slavitt to "Stay in Washington, Broker Deal"

Andrew Slavitt, the outgoing acting administrator of CMS, previously worked as a senior executive at Optum.   He announced this week he won't be going back to Optum or United Healthcare, at least for now.  He will be staying in Washington to "help broker a deal" on Fixing Obamacare.

Story and interview at Politico, here.

Update: In March 2017, Slavitt announced plans to join the Bipartisan Policy Center (here).

FDA Publishes Guidance: Manufacturer Communications with Payers

On January 18, 2017, the FDA released a 20 page Draft Guidance on:  Drug and Device Manufacturer Communications with Payors, Formulary Committees, and Similar Entities.

The document is in the form of a Q&A and the FDA will accept comment for 60 days, until about March 15, 2017.  The FDA PDF is here.  The matching comment website is here.

Outgoing Commissioner's Califf's remarks on the document release, one of his last acts in office, is here.

A blog by Amy Dow of Epstein Becker Green is here at NLR; one at RAPS here.

For a detailed blow-by-blow analysis by Tufts policy guru Peter Neumann, see Health Affairs, February 2017, here.

In Amy Dow's brief summary:

FDA states that it will not object to manufacturers providing payors with “unbiased, factual, accurate and non-misleading” information regarding investigational drugs and medical devices, provided that those communications include a clear statement of the investigational status of the product and that its safety and effectiveness have not been established, along with information regarding the stage of product development of the product. 

Information that may be provided by manufacturers in accordance with FDA’s recommendations in the draft guidance includes information about the product such as its drug class or design, the indication sought and the patient population under investigation, a factual presentation of the results of clinical and pre-clinical studies without any conclusions regarding the product’s safety and effectiveness, the anticipated timeline for FDA approval, product pricing information, and anticipated marketing strategies and product-related programs and services, such as patient assistance programs.

More details and links after the break.

National Law Review on "Trump's FDA" - Cites Gottlieb 19 times

The influential journal National Law Review ran an article on January 20, "How Will Trump Change the FDA," focusing almost entirely on Dr. Scott Gottlieb's positions (here).

Despite the generic headline, the article cites Gottlieb by name 19 times.

At the NIH, Francis Collins is remaining as chief for the time being, and possibly long term.  At the FDA, on January 17 a final decision was announced that recently confirmed Administrator Califf was definitely out, putting the FDA's leadership firmly in play for the new administration.

_______

The National Law Review article was authored by Foley & Lardner attorneys Simon Elliott & Courtenay Brinckerhoff.

The Executive Order on Obamacare (January 20, 2017) (Full Text)

On his first day in office, President Trump issued an Executive Order asking the branches of government to do the maximum possible, within the law, to take actions to minimize "the economic and regulatory burdens of the ACA."   The order requires compliance with the Administrative Procedures Act, which generally means that implementation created by agency rulemaking and regulations needs to be rescinded by another round of rulemaking.

The order applies to "all agencies and authorities" and is targeted to those parts of the act that impose economic burdens on states or individuals, providers, payers, or medical manufacturers.

Health order:  Coverage at New York Times, here.  At WSJ, here.  CNN, here.   Forbes, here.  For a rapid post by the prolific Timothy Jost at Health Affairs, here.   NBC highlights "five provisions that could change right away" (here) while The Hill advises that "Hill waits for word from Trump" on what to do next (here).  For discussion by policy expert Nicholas Bagley, here.  For an assessment of the executive order as "very confusing," Vox, here.

General Regulatory Freeze: The White House also issued a freeze on the issuance of any regulations except those required by emergencies (here; at Politico, here.)

Full text after the break.

FDA Writes Very Rapid Report on Drugs that Crashed in Phase III - AKA Why We Need FDA

Since mid November, some of the possible candidates for head of FDA have been long time advocates of a more libertarian stance, "let the market decide," not the FDA.

FDA has just released a detailed and granular 44 page white paper on "22 Case Studies" where drugs worked in Phase 2 but crashed in Phase 3.   That is, FDA argues that rigorous and closely reviewed Phase 3 trials, before market release, kept once-promising drugs from hurting patients through premature access.

The FDA white paper is here.   Coverage at John Carroll's ENDPOINTS is here.

As Carroll notes, the commissioner won't be Califf, as he steps down today.  In contrast, NIH chief Francis Collins remains in place, as least for the near term.

Califf's departure was finalized a few days ago (here, here).

Brief Blog: Nice Idea, But No Takers (CPT 46762 Deleted)

Apparently it was a good idea in theory, but had few takers.

The AMA CPT agenda for February 2017 is out, and Agenda Tab 13 is:
[details after the break]

Brief Blog: Digital Genomics, SAP CoSponsors Precision Medicine Webinar with ASCO

Continuing the them of digital genomics, in February 2017 the interntional enterprise software company SAP will co-sponsor a two part course/webinar with ASCO on precision medicine in oncology.

For an article about the project at Forbes, here.  The course is in a MOOC series branded as OpenSAP.   See more about the course content and registration, here.   It runs February 14-March 15, 2017.

The specific course sounds interesting, and will highlight Big Data and ASCO's CacerLINQ program.
However, it is also part of an emerging advanced software layer in the genomics industry, with other examples being as diverse as IBM Watson's recently announced collaboration with Quest and genomics software/service companies including (e.g.) Syapse, MolecularHealth, Farsight, GenomOncology, MolecularMatch, NofOne, Cellworks, DNA Nexus, Pierian, Translational Software, YouScript, DarwinHealth, and others.

For an archived February 2016 white paper on the advent of "digital genomics industry,"  here.

Brief Blog: Conway = CMS Acting Administrator; New Medicare Law re Appeals, Precedents

Three items of CMS news:

• Slavitt Steps Down
• Conway, Bassano Step Up
• Making Appeals Decisions Precedent-Setting

Slavitt Steps Down
Acting Administrator Andy Slavitt stepped down from his leadership roll at CMS, and leaves behind a blog describing his experiences, here.  Slavitt, a Harvard MBA, and previously worked at McKinsey and as an EVP at Optum.   He gave a 3300-word fireside chat at January's JP Morgan conference in San Francisco (here), which streamed publicly on the JPM website.

Conway, Bassano Step Up
Patrick Conway, currently head of CMMI and also Chief Medical Officer, will act as Acting Administrator until the Senate confirms a Trump Administration appointee (or until a different Acting Administrator is named.)   Amy Bassano will be Acting Director of CMMI.  Some on the Hill have proposed discontinuing CMMI has part of Obamacare repeal (eg here.)

Making Appeals Decisions Precedent-Setting
In other news, CMS has published regulations allowing it to make certain decisions of the Medicare Appeals Council "precedential," meaning they can be more rapidly and easily enforced when similar cases arise in the appeals process.   Lawmaking at 82 FR 4974 (January 17, 2017) is here.  (The 167-page rule has other parts, such as revising the Part D appeals process.)

• The regulations attempt to deal with the colossal delays and backlogs in the appeals process.   See e.g. here.  
• A December blog at this website, here.
• Trade journal article here . 
• Precedential rulings means that a favorable one should be repeated consistently in future hearings on the same topic.  But if an unfavorable one becomes precedential, it may be more difficult to return to the judges with new arguments leading to a favorable decision in future similar cases.

____