EMA Publishes 19-page Guidance on Genomics & Drug Studies

The European Medicines Association (EMA) published a 19 page guidance the use of pharmacogenomics studies in drug development.   Appearing in May, the original PDF document is here and an article summarizing it is available at RAPS, here.  The document includes links to about a dozen prior and related EMA guidances, such as on pharmacogenetics.

EMA is accepting public comments until September 16, 2016.

MolDX Creates Guidelines for Circulating Tumor DNA Validity Studies (Liquid Biopsy)

When will payers have explicit policies covering liquid biopsy...soon?   Possibly, since Medicare's MolDX policy group has issued analytical validity guidelines for circulating DNA somatic tumor mutations.   There wouldn't be much reason to have these test assessment guidelines - which look like a lot of effort and detail - unless there was some foreseeable point when the tests would be covered.

The guidelines 8 pages long and were originally issued May 20, 2016.

The UPDATED V2 was posted by MolDx four days later, on May 24, and is here.

Foundation Medicine Patent Applications

On May 17, 2016, Foundation Medicine announced issuance of a patent, 9340830, entitled "Optimization of Multigene Analysis of Tumor Samples."  For FMI"s press release, here.  For additional trade press, here.   For attached litigation, here.

• The actual patent is online at the USPTO, here.  
• It's lengthy; transferred into Word I get 222 pages and 110,000 words.   
• Faqs.org collates online patent applications for FMI, listing 9, here.   
• 13 listed for GHDX, here.  41 listed for MYGN, here.  

CMS Opens NCD on Leadless Pacemakers; NCD on gender assignment surgery to appear June 3

CMS currently has four open NCD topics, the fourth having been opened on May 18, 2016.

Leadless Pacemakers
The newest topic is a coverage decision on leadless pacemakers.   Leadless pacemakers have some advantages (they are - leadless!) but some disadvantages as well.   CMS is soliciting public comments until June 17, 2016, and then will go into research mode until releasing a draft NCD around November 18, 2016.

Gender Reassignment
Another open NCD process is considering coverage for gender reassignment surgery.  CMS had a negative NCD from 1981 until May 2014, when a judicial decision threw out the old noncoverage as not based on reasonable science (the actual decision here).  The new NCD process was announced in December 2015, and a draft NCD will appear by June 3, 2016 (request letter here; CMS website here).   Between May 2014 and a future finalization date of the in-process NCD (September 1, 2016), gender reassignment surgical coverage is at MAC discretion.

Lumbar Surgery; Hepatitis B Screening
To finish the tally of four open NCDs, an NCD for percutaneous image guided lumbar decompression is underway until a draft decision in October 2016, and an NCD on the Hepatitis B screening test coverage is underway until a draft decision in July 2016.

Theranos Voids Two Years of Test Results (WSJ)

On May 18, 2016, the Wall Street Journal reported that Theranos had voided two years of lab test results based on its Edison device.  WSJ here.   Open access story at Ars Technica, here.

Regarding the latest news, biotech journalist Matthew Herper writes at Forbes, "Theranos isn’t very good at running a diagnostic testing business."

"For a library of links on Theranos, here.

Noridian MolDX Posts, Withdraws, Consolidated Breast Cancer LCD Mirroring ASCO Guidelines

Noridian Jurisdiction E (including California) posted a draft LCD which appears to consolidate coverage for breast cancer prognostic markers, early in the week of May 16, 2016.  

The LCD was posted online at CMS here.  However, according to several sources, Noridian is canceling this LCD for this LCD cycle.

An archival copy of the May 16 LCD is in the cloud, here.

The proposal for a single breast cancer biomarker LCD very closely matched Medicare coverage to February 2016 ASCO guidelines for breast cancer prognostic markers - regardless of whether that guideline is broader or narrower than existing Medicare LCD coverage.  (For the ASCO document, see Harris et al., here.)

A listing of all current draft LCDs in J-E is here.  For this LCD cycle, there is an open comment period from June 2, 2016, to August 8, 2016.

Us Against Alzheimer's Group Posts Detailed Study of Drug Pipeline

For nearly twenty years, since the 1990s, Alzheimer's drugs have had difficult challenges and new drug approvals trickled to a stop despite large numbers of candidates.  For news article see here and for an open access study see here.

A wide variety of drugs, against diverse targets, are under study today.  The Us Against Alzheimer's organization has produced a comprehensive seven page infographic (a highlight screen shot is shown below).  In addition to high-level timelines, the report provides a detailed six page table with further characteristics of each study included.  Press release here; infographic seven page table as PDF, here.

The EU released new draft guidelines for Alzheimer drug approvals in January 2016 (here, here).  In 2013, the Center for Medical Technology Policy (CMPT) produced a 29-page consensus document on evidence for Alzheimer drug trials (here).

Hill Holds Hearing on Part B Nationwide Drug Pricing "Demo"

In March, CMS opened a 60 day comment period on a nationwide drug pricing demonstration, which illustrated the extremely wide-ranging authority granted to the Center for Medicare and Medicaid Innovation (CMMI) under 2010 legislation.   (The CMMI can waive any Medicare law it chooses to, based on a small clause that received little attention at the time.)

On May 17, 2016, the House Energy & Commerce Committee will hold a hearing on the demo proposal.  The House website is here; proposed legislation to stop the implementation of the demo is here; and a House background document on the demo is here.   The hearing will start at 10 ET and will be webcast.

In the backgrounder, House staff write:

...While these concerns are numerous, the mere scope and design of this proposal,including waiving several parts of statute, its nationwide application, the untested application of value based purchasing mechanisms, and the dramatic reduction in reimbursement have led many to call for a withdrawal of the proposed rule. 

Some on the Hill support the CMMI demo (as long as implementation is cautious to concerns); see a 4 page May 16 letter to HHS from Sens. Warren, Franken, and nine others; here.

A coalition against the CMMI proposal has a website, StopCMSCuts.   My original and updated blog on the proposal, here.

FDA releases Next Gen Clinical Sequencing Guidance: For Infectious Disease

On May 13, 2016 FDA released a draft 46 page guidance on how device manufacturers in the next generation sequencing industry can approach approval for diagnostics related to infectious disease.

The guidance is online here.  Comments are accepted for 90 days (until about August 12, 2016).  The guidance follows a workshop on microbial sequencing held two years ago, on April 1, 2014, and also adapts information from an April 2015 colloquium held in conjunction with ASM.

Two high Value Media Hits for Precision Oncology: Sunday NYT Magazine, and Harold Varmus in Science

Two high value media hits for precision oncology in a short interval.

The Sunday, May 15, New York Times magazine is devoted to advances in oncology, with seven lead articles covering topics ranging from from genomic targets in cancer to super-responders to hospice care.   Here.

The April 8 issue of Science was devoted to the new science of metastasis, and featured an editorial by Harold Varmus, former head of NCI and currently holder of an endowed chair at Weill Cornell School of Medicine.  Of note, Varmus calls on CMS to cover molecular profiling in cancer patients:

More after the break.

One Down, Two to Go: CMS Posts Date & Codes for July 2016 Gapfill Meeting

In the near future, CMS is (1) posting information on the July CLFS pricing meeting, (2) posting the "Spring" full list of proposed 2016 gapfill prices from its MACs, and (3) releasing final rulemaking for PAMA Section 216.

One down, two to go.  

The July 2016 CLFS meeting is going to be on Monday, July 18, 2016.  The CMS agenda is online here.  Stated separately in a Federal Register meeting announcement (here) the morning will be crosswalk/gapfill discussions and the afternoon will be a session of the PAMA CMS laboratory advisory board.

(For the advisory board session, look for an eventual agenda here.)

Details after the break.

CMMI Haters Make a Website!

In March 2016, the Centers for Medicare and Medicaid Innovation announced a nationwide program to abruptly change drug payment policy for the agency (article and links here).   CMMI had traditionally run relatively small demo's, but realized it had the legal authority, via a petite phrase tucked into the law, to waive any Medicare law it wants to.   (This is like getting one wish, and the wish is, to have as many wishes as you want.)

At the home page domain STOP CMS CUTS an elaborate website now exists...

NextGxDx: Helping Payers and Providers Manage Genetic Tests

After receiving daily advertisements to attend the AHIP conference in Las Vegas in June (here; Jeb Bush is keynote speaker), I clicked through and browsed around, coming across a May 16 webinar on controlling genetic test utilization sponsored by AHIP and a startup management company called NextGxDx:

More after the break.

Patient centered...medical billing???

In the May 10, 2016, Federal Register, CMS launches a proposal to solicit ideas to make medical billing in the U.S. easier to understand.  Article at HealthcareDive here.   The "challenge" co sponsored by AARP has its own webpage, here.   The Federal Register announcement is here (81 Fed Reg 27783).

There is an article on exactly the same topic - "Drowning in a Sea of Paperwork: Toward a More Patient Centered Billing System in the United States" - by Semigran et al. in the May 3, 2016 issue of Annals of Internal Medicine.  The Semigran article, though almost simultaneous, does not refer to the CMS/AARP effort.   See the article here.

WSJ Lead Article on Advanced Diagnostics in Prostate Cancer

A May 9, 2016 article by Melinda Beck in the Wall Street Journal provides deep-dive coverage for its readers on the boom in more precise prostate cancer testing, and how such tests are targeting different points in the cycle (from handling an abnormal PSA, to deciding whether to do a prostatectomy, to deciding whether to add adjuvant radiation.)   The subscription article is here.   

Tests noted in the article include Prolaris by Myriad Genetics, 4KScore by OPKO Health, Prostate Health Index PHI by Beckman Coulter, SelectMDX by MDX Health, a forthcoming urine-based test from Exosome Diagnostics, the University of Michigan's Michigan Prostate Score (MPS), ConfirmDX from MDxHealth, OncotypeDX Prostate by Genomic Health, ProMark by MetaMark Genetics, and Decipher by GenomeDx Biosciences...ten tests.

The article should be read in context with a current JAMA letter on how badly misinterpreted some RCTs of PSA testing are (here).