UnitedHealthcare Provides Limited NGS Panel Coverage in Advanced Lung Cancer

As reported in some trade journals (e.g. Genomeweb, here), United Healthcare has released a new policy, "Molecular Profiling to Guide Cancer Treatment," effective 10/1/2015.  See UHC Policy 2015T0576A, online here.

The eight-page policy states that multiple or NGS sequencing is "proven and medically necessary to guide systemic chemotherapy in patients with stage IV non small cell lung cancer" when two criteria are met.  First, molecular profiling is used ONLY to test for EGFR, HER2, RET, and ALK gene rearrangements (with a citation to current NCCN practice guidance.)   This sounds like they might expect a "don't ask, don't tell" stance on the several hundred other genes found in some commercial panels.  But although profiling is to be used "only" for the four named genes, the policy refers to 5-50 gene CPT codes, which wouldn't technically apply if only 4 genes were tested.

Second, the laboratory must be approved by the New York State Department of Health (which has explicit standards for setting up NGS assays, orthogonal control quality testing, etc).  The policy then states then indications other than the above are explicitly not covered.

The policy may face challenges in staying up to date; for example, in August 2015 a NEJM article found that NSCLC had good responses to BRAF drugs when V600E mutations were present (here, here) - an indication not yet found in the UHC policy.  In a conference report in September 2015, in a Foundation Medicine study of lung cancer patients under 40, 6% had ROS1 rearrangements, another gene not in the UnitedHealthcare list (here).  FMI reported that 76% of 68 patients under 40 had druggable mutations, 44% of the patients having ALK mutations which are vastly less common in unselected populations.  The report is part of the Genomics of Young Lung Cancer Study (GoYLC).

Noted in Passing: Evidence Based Medicine meets Prescription Drug Coupons

Co-pay support programs for expensive drugs have been in the policy news for at least five years (here, here).  Do they simply help patients with $100 copays, or are they a way of boosting sales of overpriced drugs that have a justifiably poor position on the formulary tier?

Here's an ad for Outcomes and adherence in patient assistance support programs.

Company here.

In Medicare, Can You Appeal a Pricing Decision made by a MAC?

Periodically I am asked, "Can an ALJ making a pricing decision and enforce it on a MAC?"   The answer is a little cryptic and I always have to dig up a Federal Register publication from 2009.  Here is the short answer.

Tom Norton's Insightful Futurism: Pharma Price Escalation, Key Events, and Public Policy

Pharm Exec is an open source trade journal for the biopharma industry.  One regular author, Tom Norton of a communications consultancy, has written a set of three excellent blogs/articles on pharma pricing trends and political forces.

See the following -
September, 2015 - The Pathway to Public Price Controls (here)
May, 2015 - "Take a Breath" on the Specialty Drug Pricing Issues...? (here)
November, 2014 - How Gilead Blew Out the Lights with Sovaldi (here)

See also:
August, 2015 - 2015 Medicaid Drug Price Controls - The Past is Prologue? (here)

Anyone interested in the future of health policy - whether in drugs or other areas - would benefit from seeing how Norton writes, thinks, and assembles a forward-moving story line.

The September, 2015 article contains links to the California Drug Price Relief Act, which will be on November ballots (here).

Trade journal: Hill will not "tinker with" FDA LDT Guidance before it is Final

On September 10, 2015, GRAY SHEET (a subscription trade journal, here) reported that across both political parties and in both House and Senate, the FDA probably will be left to develop a final LDT guidance without interference.  On background, staff indicated the Hill might act after the FDA has acted, but not before.

At a Next Generation Sequencing conference in Washington in mid-August 2015, FDA speakers remarked that public comment on the FDA LDT draft guidance had been considered thoroughly, the draft guidance had been revised, and it was working its way through approval and release channels at the FDA and/or executive branch.

Genomics and RBC Transfusion; Note on Palmetto's LCD.

This week's JAMA has an online-first article on the growth of genomic medicine in the century-old field of RBC transfusion.   The article, "Red Blood Cell Transfusion: Precision vs Imprecision Medicine," by Klein, Flegel, and Natanson, is here.

News Watch: "Medical Testing is So Hot Right Now!"

Two articles within a few days on the theme that medical testing, venture investment, and direct to consumer topics are riding a wave.

From Wired.com, here, September 4, 2015, the article "Medical Testing is So Hot Right Now. This Startup Wants In."  The journalist interviews the founders of CoFactor Genomics and writes,

Why don’t the two of you tell me how you plan to diagnose disease from little rings of RNA that most biologists considered junk three years ago? Let’s talk about your $1.5 million National Institutes of Health grant. While we’re at it, why did you decide to temporarily uproot your company—which is nearly a decade old and already pulling in millions of dollars—to join Y Combinator, the startup boot camp known more for putting nestling consumer tech companies like Instacart, Airbnb, and Dropbox into high orbit?

From Bloomberg Business, here, September 10, 2015, "Consumer Blood Test to Detect Cancer Hits Market Amid Skepticism."  The journalist writes,

Biotechnology startup Pathway Genomics Corp. is introducing the first “liquid biopsy” tests designed to detect cancer-associated mutations in the blood of healthy people who are at high risk of developing tumors....Pathway, a closely held firm backed by investors including International Business Machines Corp. and venture capital firm Founders Fund, is offering its CancerIntercept Detect test for $699, according to Chief Executive Officer Jim Plante. The price tag drops to $299 if the consumer signs up to be tested once a quarter.
“The cost is a big advantage because a liquid biopsy could be one-10th or one-20th the cost of an invasive biopsy,” Plante said. “There’s a lot of value here for the patient and consumer.”

The FDA shared the market's skepticism, asserting that the test was improper for marketing.  For trade press, here.   For the FDA's September 21, 2015 letter, here.  Pathway Genomics has received warning letters from the FDA in prior years (here, here).

The FDA seems polite enough in writing:

 [We] believe you are offering a high risk test that has not received adequate clinical validation and may harm the public health. We would like to discuss with you your offer of the CancerInterceptTM Detect and the associated blood specimen collection device, and any validation strategies you have undertaken beyond those reported in the publications cited in your white paper, including your determination of the test’s clinical sensitivity and specificity and the corresponding positive and negative predictive values for its claimed intended use. Given the importance of your claims, we request an acknowledgement of receipt of this letter and a proposed timeline for meeting with the FDA within 15 business days. 

On September 24, 2015, Pathway Genomics issued a public blog posting commenting on the most recent FDA letter (here):

Pathway Genomics greatly respects and shares the FDA’s concerns about patient safety. We have received a letter from FDA, dated September 21, 2015, requesting certain information regarding the Pathway Genomics CancerIntercept™ Detect testing service.  We are carefully considering the concerns of the FDA as stated in their letter, and we will be responding to that letter.  We assure that there is physician involvement in the ordering, review and follow-up of CancerIntercept™ testing.  We believe that CancerIntercept™ Detect is a  laboratory developed test and, as a CLIA and CAP certified clinical laboratory, we are offering it as such.  While Pathway Genomics is involved in educating and marketing the tests to physicians and consumers, we do not believe this is a direct-to-consumer model. We believe we have performed appropriate validation of the test as a laboratory developed test, and we are in the process of performing additional studies.

WSJ coverage on September 28, 2015, here.

Patient Engagement is a Wellspring of New Trends...from Venture Capital to FDA to CMS.

This blog often starts off with trends in the lab industry but includes a wide range of healthcare topics as well, reflecting the range of health policy consulting work I do.

On the laboratory side, there are many efforts to seek "closer engagement with patients," ranging from Theranos to Labcorp (from here to here).

However, the concept of patient engagement is high in other areas as well.  For background, see Marc Boutin's excellent open access article on Patient Engagement at American Journal of Managed Care earlier in 2015 (here).  Boutin is CEO of the National Health Council.  The Food and Drug Law Institute is offering a webinar on Patient Engagement and the FDA, on September 30, 2015, here.  (The webinar features Pat Furlong of the Parent Project Muscular Dystrophy, Janet Woodcock of the FDA, and is moderated by my colleague Debra Lappin of FaegreBD Consulting).

PMC and BIO host second Personalized Medicine Solutions Summit, October 14, 2015, in DC

The Personalized Medicine Coalition and BIO are holding the second annual Personalized Medicine Solution Summit on October 14, 2015 in Washington, DC.

The home page for the meeting is here.  Topics, following an opening keynote speaker, will be:

• Evidence and integration of personalized medicine into health practice: Issues faced by CMS.
• Education regarding the use of personalized medicine through the health care continuum.
• Integrating a Total Health Solutions Approach.
• Demonstrating the value of personalized medicine in clinical practice.
• Health systems: Infrastructure and information challenges related to personalized medicine.

The first annual summit was attended by several hundred participants.  I have the privilege of being a lead speaker for this year's summit.

Registration site is here and the PDF agenda is here.  Registration is $500; $250 for PMC or BIO members.

FDA Announces NGS Public Policy Workshops on November 12,13, 2015

The FDA has announced public policy workshops on two aspects of next generation sequencing, to be held Thursday November 12 and Friday November 13.  (This was the only Friday the 13th available this year).

The November 12 session is: Standards-Based Approach to Analytical Performance Evaluation of Next Generation Sequencing In Vitro Diagnostic Tests.

The November 13 session is: Use of Databases for Establishing the Clinical Relevance of Human Genetic Variants.

UPDATE (October 22, 2015): FDA has updated the conference webpage with a Discussion Document on the use of databases for variants.  Here.

What is the AMP comment on the MolDX Next Gen Sequencing Analytic Performance Guidance?

The Medicare Palmetto MolDX program has finalized an LCD that provides next generation sequencing panels in patients with lung cancer who have met certain preconditions (here).

Along with this LCD, MolDX published guidance as to its analytical standards for comprehensive gene panel testing, typically by NGS.  The Association of Molecular Pathologists has released a public comment.  Details after the break.

What has AMP recommended for genomic sequencing pricing?

In summer CLFS public meetings of July 2014 and July 2015, the Association for Molecular Pathologists recommended interesting methods by which CMS could price new CPT codes for genomic sequencing procedures, such as 5-50 tumor genes.  AMP also held a webinar on this topic at early 2015 (archived here).

I believe I understand what they've recommended, and helpfully, AMP publicly posted its detailed 12-page public comments to the agency (dated August 6, 2015 (here)).

My sense is that in just a few key places, the AMP document is a bit confusingly worded so the transition from one AMP pricing method to another is not obvious to the reader.   AMP re-presented its methodology to the new CLFS public advisory board which held its first meeting on August 26, 2015 (here).  I haven't yet seen that presentation, the Gray Sheet report on the meeting was a little confusing based on what I already knew about AMP's proposals (Gray Sheet here, subscription).

What follows is, in my own words, my grasp of AMP's proposals.  For the final word, readers should work through AMP's original documentation (here).

Bringing Clear Thinking to Evidence Based Medicine and Evidence Based Policy

Two very recent articles, each discussed in a separate blog, discuss how to bring more lucid and objective insights to two common policy problems.

The question of whether the FDA is "too strict" or "too lax" is perennial for decades.  In a new open access paper, MIT authors Montazaerhodjat and Lo frame this as a "type I" and "type II" error problem, and show an analytical approach to decision-making.  The paper is here - "Is the FDA Too Conservative or Too Aggressive?  A Bayesian Decision Analysis of Clinical Trial Design."  For a summary you can read on your smartphone, see the blog at marginalrevolution.com by Alex Tabarrok (here).  [*]  Tabarrok's blog includes an active discussion chain.

Another very important and perennial question is:  "How Do You Know Which Health Care Effectiveness Research You Can Trust?"  Soumerai, Starr, and Majumdar (from Harvard, Boston University, and University of Alberta) discuss this at the CDC journal Preventing Chronic Disease, here (open access).  Soumerai and Koppel overview the study at Health Affairs Blogs, here.

[*]
Footnote.
Companion piece to Montazerhojdat and Lo.  In July 2015, BIO released a white paper called, "Life Cycle Perspective for Structured Risk Benefit Design."  Here.

CMS Posts Youtube Video of Lab Advisory Expert Panel (August 26, 2015)

At the beginning of August, CMS announced the membership of its new Congressionally required expert panel on laboratory issues, including payment and coverage (article here).  The panel held its first meeting on site at CMS in a public hearing on August 26, 2015.  

Note:  CMS also convened the panel AFTER its preliminary price postings; that second panel meeting was October 19, 2015.

Added 10/2017:  Unofficial uncorrected transcript of the August 26 meeting, here.

Highmark Announces "Coverage with Evidence Development-Like" Program

Highmark announces a program called VITAL for selected new technologies.  VITAL is described as, "a program that aims to accelerate the adoption of new technologies that have received regulatory approval but are not yet covered by most commercial insurance companies."

According to the industry website Healthcare Dive:

     “Technologies that have received regulatory approval from the FDA often lack sufficient scientific data to convince commercial insurers to pay for them,” the company says. “Without support from commercial payers, it is difficult for new innovations to influence the practice of medicine.”
     Highmark Health aims to influence this process through its position as the country’s third-largest integrated healthcare delivery and financing system. It describes the VITAL program as “the missing link between FDA approval of an innovative technology and its full reimbursement.”

For more on Highmark and this program, see Healthcare Dive, here.

Healthcare Dive's home page is here.  See also their BioPharma Dive, here.  Both are good industry news sources.