RockHealth's Report on Digital Biomarkers in Healthcare

RockHealth is a San Francisco based organization that funds and supports innovative ventures in digital health.  (For their portfolio of 60 funded and/or affiliated company, see here.)

RockHealth has released an online essay that provides an overview of digital biomarkers in healthcare.  (For the full report, $99, click their "Download Report" button.)

FDA Panel Supports Landmark CGM Indication for Insulin Management; Medicare Needs to Change

For years, Medicare has held a bizarre position that continuous glucose meters (CGM) are not covered because they are "precautionary" medical devices.  For recent examples of how administrative judges have treated this policy posture harshly, see here.

Meanwhile, the industry took a big step forward on July 21, 2016, when the FDA's clinical chemistry advisory panel voted to support a new indication for the Dexcom CGM, one that directly points to its use in insulin dosing.

Hopefully, this is a stage that will leading directly to FDA approval of this indication -- and long overdue fixes at CMS.

FDA Issues Two Guidances on Real World Evidence; NEJM Article

Better use of pragmatic and real world evidence is critical for health care delivery.   While the gold standard for effectiveness is held to be a tightly controlled randomized trial, what really happens when patients are not so carefully screened, or when drug compliance is not monitored daily or weekly?   What would have happened to the data if the control arm was ordinary care with all its flaws and not an IRB-approved, carefully monitored standard of care at an academic center?

Reform efforts like 21st Century Cures legislation (drafted; not enacted) contain provisions about adaptive trials and real-world evidence.  FDA is taking strides in this area with two new guidances released in July 2016, one on "Adaptive Designs for Medical Device Clinical Studies" and one on "Use of Real World Evidence to Support Regulatory Decision Making for Medical Devices."

See the Federal Register announcements here (for Adaptive Designs) and here (for RWE).   See the FDA guidance documents here (for Adaptive Designs) and here (for RWE).  Comments through October 27.

Trade journal article here.  For publications on RWE at Pubmed, here.

For discussion of a June 2016 paper by the Center for Bipartisan Policy on RWE by Mark McClellan, Patrick Soon-Shiong, and others, here.

For an August 2016 NEJM article on Pragmatic Trials by Ford & Norrie, here.

Aetna's Mike Kolodziej MD: Flatiron's National Medical Director

Update:  MK moved to the ADVI Consultancy in October 2017.
For info on the 2018 acquisition of Flatiron by Roche, here.

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Press release from Flatiron Health announces Michael Kolodziej MD is joining them as National Medical Director for Managed Care Strategy.   Kolodziej has been a senior medical director at Aetna and has often spoken at national conferences on payer policy in oncology.  Earlier, he had been a senior physician leader US Oncology.  See two of his recent articles here and here, a detailed interview here, and June 2016 Youtube remarks here.

Regarding health policy, during Kolodziej's tenure, Aetna recently announced it would favor cancer-risk testing at genetic labs that share data into ClinVar (here).  Kolodziej has strongly supported ASCO's TAPUR registry for off label precision medicine (here).

Flatiron Health is discussed in my Spring 2016 white paper on digital health and genomics (here).  They raised $130M in Series B funding in May 2014 (Google Ventures, First Round Capital, Labcorp).  They raised $175M in Series C funding in January 2016 (Roche, Casdin Capital, Allen & Company, Baillie Gifford).

Press release here.  Clipped after the break.

Germline Genetic Testing: Evidence and Accuracy

Two articles in the June 2016 issue of the American Journal of Human Genetics address evidence and accuracy for germline sequencing.

The first article is Green et al., "Clinical Sequencing Exploratory Research Consortium: Accelerating Evidence-based Practice of Genomic Medicine," available here.

The second is Amendola et al., "Performance of ACMG-AMP Variant Interpretation Guidelines among Nine Laboratoris in the Clinical Sequencing Exploratory Research Consortium," available here.

Abstracts are clipped below the break.

In August 2016, AMP is running open registration for a program called VITAL, Variant Interpretation Testing Across Laboratories - details here.

Transcript for Medicare's July 18 2016 CLFS Public Meeting

In the cloud, find a 77 page transcript of Medicare's July 18, 2016 all-day public workshop on pricing new CPT codes for 2017...here.

CMS will announce its proposed pricing for new codes around September 1; take public comment; and announce final prices for CY2017 in November 2016.

(Those final prices are subject to appeal; if appealed, CMS will reconsider in July 2017).

For powerpoint files presented at the meeting, here.

For a discussion of a statistical error from one of the PAMA expert panelists, here.

For the full meeting archived on Youtube, here and here.

Commercialization of Molecular Diagnostics: NextGenDX Conference in DC, August 24-25

The multi-track Next Generation Diagnostics Conference is holdings its eighth annual event in Washington, August 23-26, 2016.   The conference website is here.

Within the conference, I have the chance to chair a panel on PAMA.   The panel includes Sylvia Trujillo of the AMA, Carol Blackford, Acting Director of the CMS Hospital and Ambulatory Policy Group, Brian Carey of Foley Hoag LLP and Rick Zimmerer, Life Sciences, KPMG.

I am planning to release, "An Innovator's Guide to PAMA" around the time of this panel.

This panel will kick off the conference track "Commercialization of Molecular Diagnostics," which runs August 24-25.   The see this track's special webpage, here.   Registration for the conference is open.

Details after the break.

Real World Evidence, Bipartisan Policy, and the FDA

In June 2016, the Regulatory Focus newsletter at RAPS published an article, "New Report Calls on FDA to Clarify Use of Real World Evidence," (here).

The news article summarizes findings in a 32 page report issued by Washington's Bipartisan Policy Center.  The report discusses FDA evidence requirements from 1962 forward, and urges more pragmatic approaches to evidence evaluation.   Prestigious authors include Mark McClellan, Andrew von Eschenbach, and Patrick Soon-Shiong.   The report is online, here, and underscores themes we've heard over the past year or two in the 21st Century Cures effort.

For a 2016 academic article on a "certified data" approach to RWE, see here.
For a July 2016 FDA draft guidance on RWE in medical device decision-making, here.
For an August 2016 NEJM article on Pragmatic Trials, here.

A Very Brief History of Pricing By Indication

After running into several white papers on Pricing By Indication (PBI), and recalling a 2014 article in JAMA by Peter Bach on the topic, I pulled together some internet research into a Very Brief Index (VBI) on PBI.

CMS July 2018 CLFS Crosswalk/Gapfill Meeting: Powerpoint Files

On July 18, 2016, CMS held the annual summer "crosswalk/gapfill" meeting for new AMC CPT codes to be used in CY2017.   CMS posts the full day's video online, here and here.  (For a moment, I had both morning and afternoon videos playing simultaneously on my browser, which was interesting.)   

Powerpoint and other files presented at the meeting are in the cloud, here.  This is a zip file; click the download arrow near the top.

Note that some presenters verbally corrected errors in their presentations.   For example, relative to the archived deck, AMP changed its  recommended crosswalk for 813X7X (methylated Sept9) to 81288.

PAMA Lab Panel Member Makes Gross Error; Nobody Corrects Him

On July 18, 2016, CMS held its annual meeting on policy for the Clinical Laboratory Fee Schedule, paired with an annual meeting of the Clinical Laboratory Advisory Panel created by PAMA.  Sessions are archived on Youtube, here and here.

The agenda walked through new laboratory codes for 2017, so stakeholders, CMS staff, and the PAMA panelists could discuss crosswalk/gapfill recommendations.  This is not necessarily the most exciting day of the year.

However, I was amazed by statistical errors made repeatedly by one of the PAMA advisory board panelists - and even more amazed that none of the other dozen panels corrected him.  More after the break.

CMS Proposes Pre-Diabetic Preventive Benefit - Raises Concern about Dhealth Delivery of DPP

Last March, CMS announced successful program metrics for a pilot diabetes prevention program (DPP) targeted to patients meeting prediabetic criteria.

At the time, I noted the support of myself and many organizations for DPP services.

However, I also noted, in some detail, that the actual support for the particular Medicare DPP pilot program was thin and that it had some contradictory results.  For example, it reported large cost savings of several thousand dollars per patient, whereas these programs are usually rated as cost-effective, not cost saving.

The project is not published in a peer reviewed journal, and to achieve its metrics, CMS allowed the program evaluator to make some statistical waivers, like waiving the "p<.05" concept that bedevils most scientific publications.

CMS gave the DPP pilot data a "pass," in order to meet larger program goals, whereas other coverage and policy decisions get nitpicked to death by policy gurus at CMS.

CMS has now incorporated the DPP benefit in summer rulemaking, and is requesting public comment.   CMS will include virtual or remote delivery of DPP.   CMS writes,

CDC recognized DPP organizations deliver DPP services in-person or virtually via a telecommunications system or other remote technology. The majority of current DPP organizations provide DPP services in-person, but an emerging body of literature supports the effectiveness of virtual sessions delivered remotely. We propose to allow MDPP suppliers to provide MDPP services via remote technologies.

However, CMS states that:

We recognize that the provision of MDPP services by such virtual methods may introduce additional risks for fraud and abuse, and if this proposal is finalized, we would propose specific policies in future rulemaking to mitigate these risks. We thus seek comment on whether there are quality or program integrity concerns regarding the use of virtual sessions, or whether they offer comparable or higher quality MDPP services when compared to in-person services. We seek comment on strategies to strengthen program integrity and minimize the potential for fraud and abuse in virtual sessions.

More detail, including some in's and out's of the legal implementation tactics, below the break.

CMS Posts Agenda for Annual Lab Policy Meeting (Monday, July 18).

CMS posts the agenda for the annual laboratory policy meeting.  PDF is online at CMS, here.  I have also clipped the text of the PDF below the break.

There are 15 presentations from the usual suspects - labs which have new codes under review, as well as ACLA, CAP, AMA, AMP, and so on.

Note that CMS promises a live stream of the proceedings.  Expect to find the day archived at the CMS Youtube channel, as well.

After a lunch break, CMS hosts a meeting of the PAMA Clin Lab Advisory Panel, from 1 to 4 pm, but no specific agenda is given.   Panelists will, at a minimum, discuss their take on the new codes that are up for payment proposals.

The actual list of new CPT codes to be discussed was posted a month ago and is online at CMS, here.

CMS Releases CY2017 Proposed Physician Fee Schedule Rule: Start Hearing about PAMA 220

On Friday, July 8, CMS released the Proposed Physician Fee Schedule Rule for CY2017.

The CMS website for the rule, and its extensive attachments, is here.
The rule is online here (July 15, 2016; 81:46162-746; Tracking number CMS-1654-P).

For Medscape's early summary, see here.
For high points in Becker's Hospital Review, see here.

More after the break.

CMS Releases CY2017 Proposed Outpatient Rulemaking: Implementing New Law for Hospital Place of Service

Each summer, around July 1, CMS releases hundreds of pages of proposed policy updates, separately for hospital outpatient services and for physician/laboratory services.

The hospital outpatient proposals are out.

• CMS press release, here (800 words).
• Nerdier CMS fact sheet, here (4500 words).
• Federal Register Publication, HERE.   (186 pp,  81 Fed Reg 45604-788).
• AHA News, here.
• MedCity News, here.

A few details below the break.

FDA Releases Guidance for NGS Testing

On July 6, 2016, "in support of the President's Precision Medicine Initiative," FDA released a pair of draft guidances for next generation sequencing (NGS).

The guidance aim to reflect a modern, "flexible and streamlined" approached to genomic test regulation.  They also help the agency deal with the shift from single specified analytes (e.g. cholesterol in mg/ml) to very broad based testing with limitless variants in possible results.

One guidance focuses on standards for public genetic variant databases to "support claims for their tests" on the way to "marketing clearance or approval" by the FDA.  The second guidance focuses on the use of reference standards (genome in the bottle) for NGS tests, especially for rare diseases and for demonstrating analytical validity.

The FDA's press release webpage is here.   The database guidance is here.   The standards for germline diseases guidance is here.  The guidances are 14 and 29 pages, respectively.  Public comment runs for 90 days, or until about October 5.  The guidances aren't for virus or microbial NGS testing, but a May 2016 guidance already addressed that, here.  Early remarks in Forbes, here.  For a 15 minute interview with FDA's Liz Mansfield on Mendelspod, here.

The start date for the FDA regulation of lab developed tests is here - whoops, that hasn't been released yet.

Licking and Garfield's Creative Work on Pharma Strategy & Payer Strategy

In 2011, I had the chance to write an article on personalized medicine with Susan Garfield, then at Bridgehead Consulting.   I am currently writing an article on the likely global reception for next-generation Alzheimer drugs, and during the research I ran across Susan again.

I highly recommend the article by Ellen Licking and Susan Garfield, "A Road Map to Strategic Drug Pricing," which appeared in the trade journal IN VIVO in 2015.  A copy is available open-access at her current company, EY (here).  They write:

• Products come to market with clinical trial data and not real-world evidence.  Stakeholders may see them as "having potential" but "not proven value" at the time of launch...many products enter the market with a "value gap."
• To accelerate the shift to proven value and bridge the value gap, biopharma should consider multi-stakeholder collaborations aimed at co-creating data to support innovative pricing models.

That's the teaser; see the full article.

The EY website also features an interesting two-pager, "The myth of the 'payer,' " by Roger Longman of Real Endpoints (company website here.)  Real Endpoints helps pharmas implement some of the ideas discussed by Licking & Garfield; its logo is "Preparing health care companies for today's value based economy."   Longman points out that there is no monolithic "payer" but different payers have different interests.  At the extremes, a consolidated payer/entity like the VA would view savings on drug costs and hospitalization equally.  On the other hand, a Medicare Part D plan and some other carved-out PBMs are only incented to save costs on drugs, regardless of the spillover on health or on hospital costs.   

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For another take on strategic gaps in the way we've set up PBMs in the US, see here.

Continuous Glucose Monitors: Coverage Possible After an LCD Challenge in 2016?

This blog details an important case where Medicare administrative judges rule that Medicare non coverage of continuous glucose monitors is wrong.   Details after the break.

AMP/CAP Guidelines for Lung Cancer MDX: Open Comment on New Guidelines

Guidelines are increasingly impactful on payers, from the non invasive prenatal testing field (NIPT) to cancer in the elderly.  A new edition of the Lung Biomarkers guideline is up for public comment.   This guideline was last released in 2013 (as Lindeman et al.), and is a joint work product of the College of American Pathologists, the Association for Molecular Pathology, and the International Association for the Study of Lung Cancer (CAP, AMP, IASLC).

Access the relevant draft documents and enter your comment via the AMP website, here.
Comments are open til August 2, 2016.

Key questions clipped and pasted below the break.

Medicare Proposes Bigger Changes to Appeals Backlog

On June 28, 2016, HHS issued a press release and a lengthy proposal for major changes to the severely backlogged claims appeal process for Parts A and B.

• The press release is here.
• The National Proposed Rulemaking (NPRM) is here  - and 103 pages long. (81 FR 43790, July 5, 2016).
• The 11-page PDF document is here.

• Coverage at DIVE Healthcare is here.
• Coverage at HME News is here.

The comments-due date is given as August 29, 2016.

More after the break.