Policy Update

May 16, 2013

CMS Releases 2013 Gapfill Payment Amounts for Comments

For Calendar Year (CY) 2013, the Centers for Medicare and Medicaid Services (CMS) determined it was most appropriate to use the gapfilling methodology to establish payment rates for the approximately 114 new Molecular Pathology Current Procedural Terminology (CPT) codes as well as 1 additional test code that fell into another category. As outlined at 42 CFR 414.509(b)(2), the public has 60 days from the date of this web posting to comment to CMS about the Medicare Administrative Contractors (MACs) payment rates.  In order to be considered by CMS, comments must be received by CMS by July 8, 2013.  After the 60-day comment period has ended, CMS will share the comments with the MACs as part of developing final prices under the gap-filling methodology. Send all comments directly to CMS at MoPathGapfillInquiries@cms.hhs.gov. To learn more, click here.

May 2, 2013

IOM Roundtable Workshop Registration Now Open

The Roundtable on Translating Genomic-Based Research for Health is hosting a workshop on June 5 that will examine the impact of conflict of interest policies on medical innovation. Presentations and discussions will explore best practices and potential solutions for facilitating innovation while ensuring scientific integrity and public trust. To learn more and register, click here.

May 1, 2013

PCORI Board Meeting May 6

The Patient-Centered Outcomes Research Institute (PCORI) will hold a public meeting of its Board of Governors in Chicago on May 6. The meeting will take place from 8:00 a.m. to 5:00 p.m. CT and will include a 30-minute public comment period at 2:45 p.m. CT.

A webcast will be available for those who cannot attend in person. Click here to register for the webcast and to provide public comment.

• PCORI Board of Governors Meeting
• WHEN: Monday, May 6, 2013, from 8:00 a.m. - 5:00 p.m. CT
• WHERE: Eaglewood Conference Center, 1401 Nordic Road, Itasca, IL 60143
• ONLINE and by PHONE:  Click here for webcast, dial-in and meeting materials.

April 24, 2013

FDA Invites Patient Participation

The U.S. Food and Drug Administration (FDA) has launched the third phase of an initiative to increase patient participation in the regulation of drugs and medical devices. On Wednesday, the agency went live with a new website aimed at demystifying the regulatory process for consumers and patient advocates. The new FDA website, called FDA Patient Network, is designed to educate patients on the process of getting medical devices and drugs from the idea stage to pharmacy and hospital shelves. In addition, the FDA Patient Network will be a gateway for caregivers, patients and patient advocates to email their views and hear live chats, webinars and open meetings, including those of the scientific advisory panels that meet to recommend for or against new drugs' approval by the agency.

April 23, 2013

PCORI Announces Funding Opportunity for National Patient-Centered Clinical Research Network

PCORI today issued two funding announcements for up to $68 million to improve the nation's capacity to efficiently conduct patient-centered comparative clinical effectiveness research (CER). The two linked cooperative agreement funding announcements will support development of a National Patient-Centered Clinical Research Network designed to unite millions of patients through a coordinated collaboration with researchers and healthcare delivery organizations. Complete funding announcements, application guidelines and other related material are available in the here. Required letters of intent (LOI) from applicants are due June 19 and complete applications are due September 27.

In addition, on April 25 and 26 of this week, PCORI will partner with the Institute of Medicine for a workshop entitled, "Observational Studies in a Learning Healthcare System." Click here to learn more and view the webcast.

April 18, 2013

IOM Roundtable on Translating Genomic-Based Research for Health

The Roundtable on Translating Genomic-Based Research for Health is hosting a workshop on June 5 that will examine the impact of conflict of interest policies on medical innovation. Presentations and discussions will explore best practices and potential solutions for facilitating innovation while ensuring scientific integrity and public trust. Further information will be available on the roundtable webpage.

On June 24, the roundtable will host a public workshop to assess the current landscape of drug repurposing and repositioning, examine enabling tools and technology, evaluate the business models and economic incentives for pursuing a repurposing approach, and discuss how genomic and genetic research can be positioned to better enable this paradigm in drug development. Further information about the workshop can be found here.

April 16, 2013

NHGRI to Fund New Centers of Excellence in Genomic Sciences

The National Human Genome Research Institute plans to fund new Centers of Excellence in Genomic Sciences, or CEGS, to create interdisciplinary teams that pursue innovative genome-based approaches to address biomedical problems and to understanding the basis of biological systems.

NHGRI, along with support from the National Institute of Mental Health, expects to provide up to $2 million per year for each of the new CEGS it funds, and plans to award up to four new awards each year. Learn more here.

April 15, 2013

AHRQ Releases New Report on Progression-Free Survival in Drug Evaluation

Progression-free survival (PFS), defined as the time from random assignment in a clinical trial to disease progression or death from any cause, has recently become an endpoint of considerable interest in the study of new oncology drugs. In comparison to overall survival (OS), the gold standard for cancer drug evaluation, PFS can be evaluated using shorter, smaller and less costly studies. Its use as a primary endpoint, however, can be challenging, as it is subject to a wide range of potential biases, and its use as a surrogate for OS has been demonstrated only for certain disease and treatment scenarios. The objective of this methods project is to address whether PFS is an outcome related to psychological well-being or quality of life (QOL).

View the full report here.

April 9, 2013

FDA Issues Draft Guidance, "Molecular Diagnostic Instruments with Combined Functions"

The Food and Drug Administration (FDA) is announcing the availability of the draft guidance entitled “Molecular Diagnostic Instruments with Combined Functions.” This draft guidance document provides industry and Agency staff with FDA's current thinking on regulation of molecular diagnostic instruments that have both device functions and non-device functions, and on the type of information that FDA recommends that applicants include in a submission for a molecular diagnostic instrument that measures or characterizes nucleic acid analytes and has combined functions. View the Draft Guidance here. Submit comments by July 8, 2013. View the notice here.

April 9, 2013

UPDATE-Comment Period Open: AHRQ Draft Technology Assessment Announcement-Update on Genetic Tests Currently Available for Clinical Use in Common Cancers

The Agency for Healthcare Research and Quality's (AHRQ) Technology Assessment Program has posted the draft Technology Assessment report, Update on Genetic Tests Currently Available for Clinical Use in Common Cancers. View the draft report here. Submit a public review here. Comment period closes April 22, 2013 at 5:00 p.m. ET.

April 2, 2013

U.S. Preventive Services Task Force Releases Draft Recommendation Statement on BRCA Testing, Open for Comment

The U.S. Preventive Services Task Force (USPSTF) recommends that primary care providers screen women who have family members with breast or ovarian cancer with one of several screening tools designed to identify a family history that may be associated with an increased risk for potentially harmful mutations in breast cancer susceptibility genes (BRCA1 or BRCA2). Women with a positive screen should receive genetic counseling and, if indicated after counseling, BRCA testing. The USPSTF recommends against routine genetic counseling or routine BRCA testing for women whose family history is not associated with an increased risk for potentially harmful mutations in the BRCA1 or BRCA2 genes. The draft recommendation may be viewed here. You may submit comments until April 29, 2013.

March 28, 2013

AHRQ Draft Technology Assessment Announcement-Update on Genetic Tests Currently Available for Clinical Use in Common Cancers

The Agency for Healthcare Research and Quality's (AHRQ) Technology Assessment Program will be posting a draft technology assessment for review on April 3, 2013. This draft is entitled “Update on Genetic Tests Currently Available for Clinical Use in Common Cancers.” If you are interested in reviewing this document, please visit: http://www.ahrq.gov/research/findings/ta/call-for-public-review.html. The document will be available for review from 9:00 am April 3, 2013 to 5 pm on April 17. If you have any questions, please contact ahrqtap@ahrq.hhs.gov.

March 27, 2013

PMC Members Submit Comments on FDA's Enrichment Guidance Document

Last week, the comment period closed on FDA's draft guidance, "Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products." The purpose of this document is to provide guidance to industry on enrichment strategies that can be used in clinical trials intended to support effectiveness and safety claims in new drug applications (NDAs) and biologics license applications (BLAs). A number of PMC members have submitted detailed public comments on this guidance which you may view here. View the guidance document here.

March 26, 2013

AMP Releases Proposal to Address CPT Coding for Genomic Sequencing Procedures

The Association for Molecular Pathology (AMP) has submitted to the American Medical Association’s CPT Editorial Panel its proposal addressing CPT coding for genomic sequencing assays.

AMP stated "the ability to so closely examine the human genome has and will continue to result in the need for countless new codes. The report includes two proposed schemes for coding genomic sequencing assays; 1) the Technology-Agnostic, Single Code Strategy, and 2) the Code Mate Strategy. Option 1 relies on a single, methodology-agnostic code that includes both technical and interpretive work whereas Option 2 introduces separate codes for technical vs. interpretive processes." Read the full AMP proposal here.

The AMP Economic Affairs Committee (EAC) invites you to contribute your input to the AMA's CPT Editorial Panel regarding the proposed coding for genomic sequencing assays. Please submit your feedback to AMP via email at NGScomments@amp.org no later than April 15, 2013. The feedback will be compiled and forwarded to the AMA CPT Editorial Panel for its consideration.

March 26, 2013

IOM Releases The Economics of Genomic Medicine Workshop Summary

To better understand the health economic issues that may arise in the course of integrating genomic data into health care, the IOM Roundtable on Translating Genomic-Based Research for Health hosted a workshop July 17-18, 2012, that brought together economists, regulators, payers, biomedical researchers, patients, providers, and others to discuss the many factors that may influence this implementation. Read the workshop summary released today here.

March 7, 2013

IOM ASCO Workshop Videos and Slides Available

The Institute of Medicine and the American Society of Clinical Oncology (ASCO) held a workshop February 11-12, 2013 to examine ongoing activities in the implementation of the recommendations put forth in the IOM consensus report, A National Cancer Clinical Trials System for the 21st Century. Workshop sessions and discussions will highlighted progress made by NCI and the Clinical Trials Network, funding needs and opportunities, strategies for prioritizing trial concepts, addressing ongoing and future implementation challenges, opportunities for the development of effective partnerships to accelerate innovation, and current regulatory issues.

View the workshop videos here. Learn more here.

February 14, 2013

MEDCAC to Hold Meeting on Genetic Tests for Cancer Diagnosis

The Secretary's Advisory Committee on Genetics, Health and Society (SACGHS) has defined genetic testing as "…any test performed using molecular biology methods to test DNA or RNA, including germline, heritable, and acquired somatic variations." CMS has commissioned technology assessments (TAs) on two types of genetic tests. The first TA examined DNA- or RNA-based tests which are likely to predict the tissue of origin in patients presenting with a cancer of unknown primary site (CUP). The second TA examined fluorescent in-situ hybridization (FISH) tests for cancer/pre-cancer in patients with atypical squamous cells of unknown significance (ASCUS) or low-grade squamous intraepithelial lesions (LSIL) in cytological specimens from the uterine cervix.

CMS will convene the panel May 1, 2013 to consider the evidence about these two types of genetic tests for cancer diagnosis.

February 13, 2013

IOM Releases Cancer Care Workshop Summary

On October 8-9, 2012, the IOM’s National Cancer Policy Forum held a workshop to examine the drivers of current and projected cancer care costs, as well as potential ways to curb these costs while maintaining or improving the quality of care. This document summarizes the workshop, entitlted Delivering Affordable Cancer Care in the 21st Century. Click here to download the report.

February 12, 2013

FDA to Release Long-Awaited Guidance on CMO Agreements, Biosimilars in 2013

January 31, 2013

Science Board to the FDA to Meet Next Month

On February 27, 2013, the Food and Drug Administration (FDA) will host a meeting of its Science Board, which provides advice on specific complex scientific and technical issues important to the FDA and its mission, including emerging issues within the scientific community. Plans for an Agency-wide working group to address cross-cutting genomics activities will be presented. To view the full meeting notice for more information, click here.

January 29, 2013

PCORI Seeks Advisory Applications for Advisory Panels

The Patient-Centered Outcomes Research Institute (PCORI) today began inviting applications for its first four PCORI Advisory Panels as part of its ongoing effort to engage a broad range of healthcare stakeholders as partners in its research agenda.

PCORI is seeking patients, caregivers, clinicians, researchers, other members of the healthcare community and the general public to serve on advisory panels on "Assessment of Prevention, Diagnosis, and Treatment Options; Improving Healthcare Systems; Addressing Disparities; and Patient Engagement."

Charters for each panel, including the scope of work for panelists, are available on the PCORI website. Applications can be submitted online until 5:00 p.m. ET, Monday, March 4. They will be reviewed by PCORI Staff and panelists selected based on experience, background, ability to contribute to the scope of work described in panel charters, and commitment to advancing PCORI’s mission.

January 28, 2013

PMC Submits Public Comments to CMS on Draft Coverage with Evidence Development Guidance

PMC has submitted comments to CMS regarding its Draft Guidance, "Draft Guidance for the Public, Industry, and CMS Staff Coverage with Evidence Development in the context of coverage decisions." PMC urged CMS to consider the following recommendations:

• CMS should adopt the principles included in the 2006 CED guidance document.
• CMS should ensure that review of evidence and decisions about evidence development are clearly explained and transparent.
• CMS should clarify the role of the Agency for Healthcare Research and Quality (“AHRQ”) in the application of CED.

You may view PMC's full comment letter here.

January 28, 2013

New FDA Guidance on Clinical Pharmacogenomics

FDA has released new guidance, "Clinical Pharmacogenomics: Premarket Evaluation in Early-Phase Clinical Studies and Recommendations for Labeling." The guidance provides recommendations on when and how genomic principles should be considered and applied in early-phase clinical studies to address questions arising during drug development and regulatory review. The guidance does not address trial design or statistical analysis considerations for later-phase randomized controlled clinical trials that are intended to draw definitive conclusions about treatment effects in a genomic subgroup or codevelopment of a drug and in vitro diagnostic. Rather, the considerations here are more relevant for exploratory and observational studies intended to generate genomic hypotheses that may then be tested in confirmatory trials. View the Federal Register notice here. View the Guidance Document here.

January 25, 2013

FDA Public Hearing: "Creating an Alternative Approval Pathway for Certain Drugs Intended to Address Unmet Medical Need"

The Food and Drug Administration (FDA) is announcing a public hearing to obtain input on a potential new pathway to expedite the development of drugs, including biological products, for serious or life-threatening conditions that would address an unmet medical need. The drug’s safety and effectiveness would be studied in a smaller subpopulation of patients with more serious manifestations of a condition. Such a pathway could involve smaller and more rapid clinical trials than would occur if the drug were studied in a broader group of patients with a wide range of clinical manifestations. The labeling of drugs approved using this pathway would make clear that the drug is narrowly indicated for use in limited, well-defined subpopulations in which the drug’s benefits have been shown to outweigh its risks. The purpose of the public hearing is to obtain information and comments from the public on the need for and feasibility of this pathway and its potential advantages and disadvantages. The meeting will be held February 4, 2013 from 9:00 am to 5:30 pm. To view more meeting information click here. To view the webcast on February 4, click here.

January 23, 2013

IOM Workshop: Refining Processes for the Co-Development of Genome-Based Therapeutics and Companion Diagnostic Tests

There has been a major alteration in the way that drugs are being developed with traditionally separate entities, pharmaceutical and diagnostic companies, now working in close collaboration. However, questions remain regarding the processes for partnering, the economic viability of co-development for diagnostics, payer reimbursement for companion tests and off-label use of these drugs, the substitution of non-FDA approved tests, the challenges in changing technologies for an existing companion diagnostic, the need for multiple tests for ever smaller diagnostic tissue specimens, and many others. The Institute of Medicine Roundtable on Translating Genomic-Based Research for Health will host a workshop to examine the challenges of and potential solutions for co-development of molecular tests and targeted therapeutics on February 27, 2013. To learn more and register for this meeting, click here.

January 15, 2013

CMS Releases Draft Guidance on Coverage with Evidence Development

CMS sought public input with a goal to improve the application of CED, which they had last released a guidance document on in 2006.  In November of 2011, CMS asked particularly for comment on the following areas.

• Implementation of CED through the national coverage determination (NCD) or other avenues under Part A and Part B;
• Potential impact of CED on the Medicare program and its beneficiaries; and
• Suggested approach to CED to maximize benefit to Medicare beneficiaries.

CMS has released the draft guidance that considered the public comments gathered which you can access here. Public comment is due by January 28, 2013.

For reference purposes, you may view the 2006 CMS CED guidance here.

January 8, 2013

IOM Genomics Roundtable Workshop Summary Available Online

The publication of the Institute of Medicine’s workshop summary, Genome-Based Therapeutics: Targeted Drug Discovery and Development. The report may be accessed on-line where a PDF copy may be downloaded for free here.

You may go to the meeting page to view the presentations and hear the audio files for this workshop here.

January 7, 2013

Recalcitrant Cancer Research Act Signed into Law

Language derived from the Recalcitrant Cancer Research Act of 2012 was signed into law as part of the much larger National Defense Reauthorization Act of 2013. From the official summary, section 1083 of the law: "Amends the Public Health Service Act to require the Director of the National Cancer Institute (NCI) to develop a scientific framework for research on recalcitrant cancers (cancer with a 5-year relative survival rate below 50%), which includes: (1) a review of the status of research, such as a summary of findings, identification of promising scientific advances, a description of the availability of qualified scientific researchers, and the identification of resources available to facilitate research; (2) identification of research questions that have not been adequately addressed; and (3) recommendations for actions to advance research and for appropriate benchmarks to measure progress on achieving such actions. Requires the Director to develop the framework within 18 months and review and update it every 5 years.

"Requires the Director to identify within 6 months 2 or more recalcitrant cancers that have a 5-year relative survival rate of less than 20%, and are estimated to cause the death of at least 30,000 individuals in the United States per year. Authorizes the Director to identify additional such cancers and to consider additional metrics of progress (such as incidence and mortality rates) against such cancer.

"Requires the Director to convene a working group for each identified cancer to provide expertise on, and assist in developing, a scientific framework under this Act.

"Requires the Director to consider each relevant scientific framework developed under this Act when making recommendations for exception funding for grant applications."

January 7, 2013

Advisory Committee on Heritable Disorders in Newborns and Children Webinar

HHS Secretary's Advisory Committee on Heritable Disorders in Newborns and Children will host a webinar January 31 and February 1, 2013, regarding the development of newborn screening activities, technologies, policies, guidelines, and programs for effectively reducing morbidity and mortality in newborns and children having, or at risk for, heritable disorders. View the Federal Register notice including the agenda here. Register for the webinar by January 17 here.

January 4, 2013

FDA CDRH Experts Discuss Ongoing Regulatory Efforts in Personalized Medicine

Elizabeth Mansfield and Alberto Gutierrez of the Food and Drug Administration Center for Devices and Radiological Health (CDRH) were interviewed in GenomeWeb's Pharmacogenomics Reporter yesterday, and highlighted a few pertinent personalized medicine guidances:

• CDRH has prioritized releasing the final companion diagnostics guidance by March 31
• CDRH plans on issuing the codevelopment draft guidelines by September 30
• Also by September 30, CDRH, in collaboration with the National Institute of Standards and Technology, plans to develop genomics standards — specifically to "select and sequence the human DNA and microbial DNA reference materials to support analytical validation of genetic sequencing technologies."

The interview sheds light on a number of other pertinent personalized medicine regulatory initiatives at CDRH. They also highlighted their plan to make their information more publicly available. They recently published a list of the genetic tests that they have cleared or approved. Last year, they pulled all the companion diagnostics out on one page.