Policy Update Archive - 2011

December 20, 2011

Gene Patenting and Biological Methods in the Personalized Medicine Space

by Jennifer Giordano-Coltart, Ph.D.
Richard S. Meyer contributed to this post, originally posted on The Age of Personalized Medicine.

Personalized medicine, including the application of genomic and molecular data to better target the delivery of health care and determine a person’s predisposition to a particular disease or condition, continues to expand and become the clinical standard of care for many medical conditions or diseases.  For the past several years, players in the personalized medicine space have seen the federal courts and the legislature debate the extent to which patent protection is available under U.S. law when it comes to biological molecules and methods of assessing biological molecules (e.g., diagnostic testing).

Recently, the debate has heated as a line of lawsuits have made their way through the legal system with sights set on the Supreme Court.  While the technology at issue in these cases is diverse, the questions before the courts always come back to where to draw the line between patent-eligible subject matter (an invention) and patent-ineligible subject matter (an abstract idea, scientific principle or natural phenomenon).  By excluding this type of subject matter from patent eligibility, naturally occurring phenomenon, scientific principles, and laws of nature remain available to all innovators and researchers.

These cases have drawn attention and input not only from biotechnology and diagnostics companies and industry organizations, but also from public interest, policy, legal, and medical organizations.  In particular, the Myriad case[i], focuses on the patent eligibility of isolated genetic sequences associated with breast cancer and diagnostic methods based on detecting those sequences.  How this case is decided will be partly guided by the Supreme Court’s decision in the Prometheus case[ii], for which oral arguments before the Supreme Court were held on December 7, 2011.  In the Prometheus case, the parties are arguing over the patent eligibility of biological methods involving measuring drug breakdown products (metabolites) after administering a drug to a patient to assist in determining appropriate drug dosage.

The courts have been presented with a number of policy arguments on the ramifications of either having or eliminating patent protection for these technologies.  Perhaps the largest policy debate focuses on whether the market exclusivity provided by patents on genetic sequences or diagnostic methods foster commercialization and thus availability of personalized medicine.  Supporters of patent protection for gene patents and diagnostic testing say that this protection enables better patient care by facilitating investment in and commercialization of diagnostic testing and dissemination of data to broaden medical understanding, which in turn leads to new research endeavors and the development of new technologies, and improves long-term public health.  Opponents argue that patent protection for these technologies impair patient health by limiting dissemination of medical knowledge and use thereof, limiting access to diagnostic testing by raising testing costs, and prevents access to second opinion testing to confirm diagnosis.

While important, these policy arguments may be outside the scope of the courts’ patent eligibility analysis of genetic sequences, diagnostic testing, and other biological methods under U.S. patent laws.  The recently implemented patent reform law (America Invents Act)[iii] made no changes to the patent eligibility standards, but did commission the US. Patent & Trademark Office to study access to second opinion testing for genetic testing, indicating that Congress feels further inquiry is needed before legislative action.

---

[i]  Ass’n for Molecular Pathology v. U.S. Patent & Trademark Office, No. 10-1406 (Fed. Cir. July 29, 2011).

[ii]  Mayo Collaborative Services v. Prometheus Laboratories, Inc., 628 F.3d 1347 (Fed. Cir. 2010), cert. granted, 79 U.S.L.W. 3554 (U.S. June 20, 2011)(No. 10–1150).

[iii]Leahy-SmithAmerica Invents Act, Pub. L. No. 112-29, 125 Stat. 284, 325 (2011) (to be codified at 35 U.S.C. § 257)(enacted Sept. 16, 2011).

December 13, 2011

The IOM's Roundtable on Translating Genomic-Based Research for Health Releases Workshop Summary

On July 19, 2011, the IOM’s Roundtable on Translating Genomic-Based Research for Health hosted a workshop to highlight and identify the challenges and opportunities in integrating large-scale genomic information into clinical practice. Challenges range from the analysis, interpretation, and delivery of genetic information to associated workforce, ethical, and legal issues. Additionally, many patients and providers have yet to realize the broad effect genomic discoveries are likely to have on treatment and health.  The main objective of the workshop was to start a discussion on what needs to be done to prepare the necessary infrastructure and to address the various challenges for realizing genomic medicine.  This document summarizes the workshop. Follow the link to read the Workshop Summary: Integrating Large-Scale Genomic Information into Clinical Practice.

November 23, 2011

Comment Request Information Program on the Genetic Testing Registry

NIH has requested comments on thier development of a voluntary registry of genetic tests. The Genetic Testing Registry (GTR) will provide a centralized, online location for test developers, manufacturers, and researchers to submit detailed information about genetic tests. The overarching goal of the GTR is to advance the public health and research in the genetic basis of health and disease. As such, the Registry will have several key functions, including (1) Encouraging providers of genetic tests to enhance transparency by publicly sharing information about the availability and utility of their tests; (2) providing an information resource for the public, including health care providers, patients, and researchers, to locate laboratories that offer particular tests; and (3) facilitating genetic and genomic data-sharing for research and new scientific discoveries. Written comments and/or suggestions regarding the item(s) contained in this notice, especially regarding the estimated public burden and associated response time, should be directed to the: Office of Management and Budget, Office of Regulatory Affairs, OIRA_submission@omb.eop.gov or by fax to (202) 395-6974, Attention: Desk Officer for NIH.

November 21, 2011

Last week, a personalized medicine bill, the MODDERN Cures Act, H.R. 3497, was introduced by Leonard Lance (R-NJ). Drafted by patient organizations that are members of the National Health Council, the bill includes provisions that would improve reimbursement for diagnostic tests and help bring to market treatments that address unmet medical needs. Click here to view the full text of the bill.

November 16, 2011

CMS Official Says Revamped CED Program Could Benefit Molecular Dx Reimbursement

The Centers for Medicare & Medicaid Services is seeking public input on its coverage with evidence development program, a process through which the agency reimburses new medical interventions with the condition that sponsors will collect data showing their products and services are improving outcomes for the Medicare population. To view the full request and comment, please click here.

November 10, 2011

Release of Revised NCI Best Practices for Biospecimen Resources

The National Cancer Institute (NCI) is pleased to announce the release of revised NCI Best Practices for Biospecimen Resources. Originally released in 2007, this document identifies guiding principles defining state-of-the-science practices for biospecimen resources, promoting biospecimen and data quality, and supporting adherence to ethical and legal principles. This revised version incorporates more current and detailed recommendations, and responds to comments received from community stakeholders.

November 7, 2011

Extension of Comment Period on Draft Guidance for De Novo Classification Process

The Food and Drug Administration (FDA) is extending to January 3, 2012, the comment period for the notice entitled Draft Guidance for Industry and Food and Drug Administration Staff - De Novo Classification Process (Evaluation of Automatic Class III Designation). The Agency is taking this action due to a discrepancy in the comment period in the notice as compared to the comment period listed in the guidance document. View the previous PMC update here.

November 3, 2011

FDA Releases Report on 35 Innovative New Drug Approvals in FY2011

Over the past 12 months, the U.S. Food and Drug Administration approved 35 new medicines. This is among the highest number of approvals in the past decade, surpassed only by 2009 (37). Many of the drugs are important advances for patients, including: two new treatments for hepatitis C; a drug for late-stage prostate cancer; the first new drug for Hodgkin’s lymphoma in 30 years; and the first new drug for lupus in 50 years.

In a report released today, FY 2011 Innovative Drug Approvals, the FDA provided details of how it used expedited approval authorities, flexibility in clinical trial requirements and resources collected under the Prescription Drug User Fee Act (PDUFA) to boost the number of innovative drug approvals to 35 for the fiscal year (FY) ending Sept. 30, 2011.

FDA noted that two of the drugs – one for melanoma and one for lung cancer – are breakthroughs in personalized medicine. Each was approved with a diagnostic test that helps identify patients for whom the drug is most likely to bring benefits. To read the full press release, click here.

October 24, 2011

Patient-Centered Outcomes Research Institute Call for Applications for Stakeholder Reviewers

The Agency for Healthcare Research and Quality (AHRQ) Effective Health Care (EHC) Program is assisting the Patient-Centered Outcomes Research Institute (PCORI) with disseminating its call for applications for stakeholder reviewers:

PCORI is looking for patients and other health care system stakeholders to participate in merit reviews of the PCORI Pilot Project Grants Program applications in February 2012. The purpose of the reviews is to determine the significance and quality of submitted research proposals. Each review group will include at least two non-scientist reviewers. The inclusion of non-scientific reviewers, including patients, caregivers, and health care clinicians or providers, helps ensure that the patient perspective is included in PCORI-funded research. Training on how to be a merit reviewer will be provided for those who do not have previous experience. All reviewers will be required to complete a PCORI conflict of interest disclosure form and must be available to attend a one-day review group meeting in Washington, D.C., between February 13-24, 2012. An honorarium of $200 and travel expenses, including food, hotel, and transportation, will be provided for attendance and participation in the review group. Interested reviewers must complete and submit an online application form no later than 12:00 p.m. ET on October 26, 2011.

For more information and to apply to be a reviewer, please visit the following links:

• PCORI Pilot Projects Grants Program
• PCORI Request for Applications for Patient and Stakeholder Reviewers

October 21, 2011

Prescription Drug User Fee Act Meeting

The department of Health and Human Services will hold a public meeting Monday, Oct. 24, 9 a.m. to discuss the Proposed PDUFA V Reauthorization Performance Goals and Procedures for Fiscal Years 2013 through 2017. The section on pharmacogenomics is on page 22. The legislative authority for PDUFA expires in September 2012. At that time, new legislation will be required for FDA to collect prescription drug user fees for future fiscal years. Submit electronic comments to http://www.regulations.gov, identify you comments with the docket number [FR Doc No: 2011-23251].

October 20, 2011

Current Legislation Update

The PMC Legislative Update includes details on the legislation recently introduced and acted on by Congress. These bills relate to personalized medicine, covering areas including health reform, comparative effectiveness, intellectual property, health information technology, and privacy.

Legislative Update as of October 20, 2011 (PDF)

October 14, 2011

House Energy and Commerce Committee introduces ten FDA reform bills

After hearing from patients, inventors, investors, and employers, committee members from both sides of the aisle introduced ten legislative solutions to improve the predictability, consistency, and transparency of FDA’s medical device review and approval process.

• H.R. 3203: Novel Device Regulatory Relief Act
• H.R. 3204: Guidance Accountability and Transparency Act
• H.R. 3205: FDA Renewing Efficiency From Outside Review Management Act
• H.R. 3206: Cultivating Scientific Expertise to Foster Innovation Act
• H.R. 3207: Modernizing Laboratory Test Standards for Patients Act
• H.R. 3208: Patients Come First Act
• H.R 3209: Premarket Predictability Act
• H.R. 3211: Humanitarian Device Reform Act
• H.R. 3214: Food and Drug Administration Mission Reform Act
• H.R. 3230: Keeping American Competitive through Harmonization Act

To read their internal memo, including bill summaries click here.

October 11, 2011

White House seeks ideas for national bioeconomy blueprint

The purpose of this Request for Information (RFI) is to solicit input from all interested parties regarding recommendations for harnessing biological research innovations to meet national challenges in health, food, energy, and the environment while creating high-wage, high-skill jobs.

The public input provided through this Notice will inform the Office of Science and Technology Policy as it works with Federal agencies and other stakeholders to develop a National Bioeconomy Blueprint.

All comments must be submitted electronically to bioeconomy@ostp.gov by December 6, 2011.

October 5, 2011

FDA Commissioner released a blueprint to spur biomedical innovation, improve health of Americans

FDA Commissioner Margaret A. Hamburg, M.D., today released a blueprint containing immediate steps that can be taken to drive biomedical innovation, while improving the health of Americans.

Titled “Driving Biomedical Innovation: Initiatives for Improving Products for Patients,” the blueprint addresses concerns about the sustainability of the medical product development pipeline, which is slowing down despite record investments in research and development.

The blueprint focuses on implementing the following major actions:

• rebuilding FDA’s small business outreach services
• building the infrastructure to drive and support personalized medicine
• creating a rapid drug development pathway for important targeted therapies
• harnessing the potential of data mining and information sharing while protecting patient privacy
• improving consistency and clarity in the medical device review process
• training the next generation of innovators
• streamlining and reforming FDA regulations.

View the FDA's full press release here.

October 4, 2011

CDRH Posted a List of Guidance Documents Under Consideration for Development

CDRH is considering a list of guidance documents for development this year (2012). CDRH plans to update this list every year. CDRH invites interested persons to submit comments on any or all of the guidance documents on the list to docket FDA-2007-N-0270. Comments may include draft language on the proposed topics and/or suggestions for new or different guidance documents. CDRH believes this docket is an important tool for receiving information from interested parties and for making information available to the public.

To view and make comments on the list, click here.

October 3, 2011

FDA Releases De Novo Classification Process Guidance

The FDA released Draft Guidance for Industry and Food and Drug Administration Staff - De Novo Classification Process (Evaluation of Automatic Class III Designation). The purpose of this document is to provide guidance on the process for the submission and review of petitions under section 513(f)(2) of the Federal Food, Drug, and Cosmetic Act (the FD&C Act), also known as the de novo classification process. This process provides a route to market for medical devices that are low to moderate risk, but that have been classified in class III because FDA has found them to be "not substantially equivalent" (NSE) to legally marketed predicate devices. Comments must be submitted to the FDA by January 3, 2012.

September 28, 2011

PCORI Announces $26 million Pilot Projects Grant Program

On September 28, 2011, the Patient-Centered Outcomes Research Institute (PCORI) announced a $26-million Pilot Projects Grants Program that will support about 40 awards. Support may be requested for up to $250,000 in direct costs per year for 2 years. The purposes of the Pilot Projects Grants Program are to:

• Inform PCORI's ongoing development and enhancement of national priorities for patient-centered outcomes research
• Support the collection of preliminary data that can provide a platform for an evolving research agenda
• Support the identification of research methodology to advance patient-centered outcomes research

Information about the Pilot Projects Grants Program and how to apply is available on the PCORI Web site.

September 1, 2011

PMC Develops FDA Guidance Mosaic

At PMC’s last Public Policy Committee meeting, Larry Lesko, Ph.D., F.C.P., Former Director, Office of Clinical Pharmacology, Food and Drug Administration, (FDA), explained that while many in the personalized medicine community expected one guidance document from the FDA to answer all of their questions, what we should expect is a “mosaic of guidances” that will together guide the regulation of personalized medicine products. PMC has begun the task of developing this list of guidances as a reference tool on our website. You may view the current list here. Please e-mail Tim Swope if you have suggestions or additions for this list.

August 23, 2011

Update: Extension of Comment Period on Draft Guidance for Industry and Food and Drug Administration Staff - In Vitro Companion Diagnostic Devices

Originally posted July 13, 2011.

This guidance is intended to assist (1) sponsors who are planning to develop a therapeutic product that depends on the use of an in vitro companion diagnostic device (or test) for its safe and effective use and (2) sponsors planning to develop an in vitro companion diagnostic device that is intended to be used with a corresponding therapeutic product.

To view the draft guidance, click here.

The deadline to submit comments and suggestions regarding this draft document has been extended to October 12, 2011.

August 18, 2011

FDA Releases New Strategic Plan for Regulatory Science

The Food and Drug Administration (FDA) has released its “Strategic Plan for Regulatory Science,” a sweeping initiative to modernize the tools and methods that the agency uses to evaluate whether the products it regulates are effective and safe for consumers. One of its eight new priorities is to "Stimulate Innovation in Clinical Evaluations & Personalized Medicine to Improve Product Development and Patient Outcomes." They plan to implement this through collaborating with others on the following steps:

1. Develop and refine clinical trial designs, endpoints and analysis methods
2. Leverage existing and future clinical trial data
3. Identify and qualify biomarkers and study endpoints
4. Increase the accuracy and consistency, and reduce inter-platform variability of analytical methods to measure biomarkers
5. Develop a virtual physiologic patient

Click here to view the full plan for more details. This new plan provides details for the framework of the regulatory science initiative which was originally released in October 2010.

August 16, 2011

Upcoming American Medical Association CPT Events and Updates

The schedule of events for the October 2011 CPT Editorial Panel meeting and the Advisors Annual meeting is available for your review.  The meetings are being held at the Marriott Chicago Downtown Magnificent Mile hotel. Please contact the Marriott Chicago to make your reservations.  The online registration forms for the CPT Editorial Panel meeting and the Advisors Annual meeting will be available on this website in mid-September.

The Molecular Pathology Coding Workgroup requested feedback this spring on the Molecular Pathology initial Tier 1 and Tier 2 code set. All comments were reviewed and, as appropriate, were submitted to the Tier 1 and Tier 2a Subworkgroups for consideration. An updated code set of descriptors and introductory verbiage, including suggested modifications, will soon be posted to this website for review.

View the Molecular Pathology Workgroup General Session presentation that was shown during the MOPATH workgroup meeting held during the June 2011 CPT Editorial Panel meeting.

A brief history of the Molecular Pathology Coding Workgroup is available. Detailed information can be reviewed in the Request for Molecular Pathology Code Review and Feedback.

Learn about the discussion of the July 20 In Vitro Diagnostic Multivariate Assay Fly-In meeting that was held in Chicago last week.

August 15, 2011

PMC Completes Legislative Specifications
At the suggestion of Congresswoman Anna Eshoo (D-Calif.), PMC's legislative workgroup designed six legislative specifications to help the Congresswoman develop a new version of the Genomics and Personalized Medicine Act that would both foster personalized medicine innovation and enjoy community-wide support. The concepts include:

1. Ensure Adequate Representation of the Personalized Medicine Perspective Through Advisory Committees
2. Incentivize Personalized Medicine by Creating a Transparent and Predictable Regulatory Environment for Personalized Medicine Products
3. Medicare Coverage of Personalized Medicine Diagnostics and Related Items and Services
4. Expedited Exceptions Process for Personalized Medicine Drug/Biologic Under Medicare Part D
5. Personalized Medicine Research and Development Tax Credits
6. Ensure Patient Access to Personalized Medicine Through Delivery System Reforms

To review the specifications, please click here.

August 8, 2011

Update: The Treatment of Certain Complex Diagnostic Laboratory Tests Demonstration; Extension of the Deadline for Submission of Supporting Information

Originally posted July 1, 2011

This notice also serves to notify interested parties that they must obtain a temporary code from CMS for tests currently billed using a “not otherwise classified (NOC)” code but that would otherwise meet the criteria set forth in section 3113 for being a complex diagnostic laboratory test under the Demonstration. The statute requires a Report to Congress that includes an assessment of the impact of the Demonstration on access to care, quality of care, health outcomes, and expenditures. DATES: Supporting information to request a temporary code under the Demonstration has been extended to September 6, 2011.

To view the full notice click here.

July 25, 2011

Notice: Identifying the CDER's Science and Research Needs

The Food and Drug Administration (FDA) is announcing the availability of a draft report entitled "Identifying CDER's Science and Research Needs." This document identifies current priorities in regulatory science related to the mission of the Center for Drug Evaluation and Research (CDER), and will guide strategic planning of internal research efforts. Through external communication of the science and research needs outlined in the report, CDER hopes to stimulate research and foster collaborations with external partners and stakeholders to address these priorities.

To view the report, click here. To view the full notice, click here.

Submit comments by September 25, 2011.

July 15, 2011

Notice: Assessing the Current Research, Policy, and Practice Environment in Public Health Genomics

The Centers for Disease Control and Prevention (CDC) has issued a Request for Information (RFI) for comments, data, and other information helpful to assess the most important steps in research, policy, and practice for the field of public health genomics in the next five years.
Comments must be received by August 1, 2011.  Please submit comments in response to this RFI by clicking here.

June 30, 2011

Notice: Next PCORI Board of Governors Meeting

July 18-19, 2011

Westin Georgetown
2350 M Street NW
Washington, DC

DRAFT Agenda (PDF)

Webcast will be provided for all public sessions.

June 1, 2011

Notice of Upcoming FDA Public Meeting and Draft Guidance
The Food and Drug Administration (FDA) is announcing a public meeting Ultra High Throughput Sequencing for Clinical Diagnostic Applications - Approaches to Assess Analytical Validity. The purpose of the meeting is to discuss challenges in assessing analytical performance for ultra high throughput genomic sequencing-based clinical applications. This meeting will be held June 23, 2011, beginning at 8:00 a.m.

If you wish to attend this Workshop, you must register by June 9, 2011.
Please use either the In-Person Workshop Attendance registration form, or the Online Webcast Viewing registration form.

To view the notice for more details, click here.

Separately, The Food and Drug Administration (FDA) is announcing the availability of the draft guidance entitled "Commercially Distributed In Vitro Diagnostic Products Labeled for Research Use Only or Investigational Use Only: Frequently Asked Questions" (PDF). This draft guidance document is intended for manufacturers and distributors of research use only (RUO) and investigational use only (IUO) in vitro diagnostic (IVD) products and any other entities who label IVD products.

Submit written or electronic comments on the draft guidance by August 30, 2011.

To view the notice for more details, click here.

April 28, 2011

New National Coordinator for HIT Lays Out Principles
The new National Coordinator for Health Information Technology, Dr. Farzad Mostashari, outlined his view for the implementation of HIT policy at the Bipartisan Policy Center last week. He was optimistic about the health care system being on the verge of a “virtuous cycle” where providers and patients will be able to use better information as a tool to improve care. He laid out the core principles behind his office’s work stating, “we believe that the innovation from industry, from the free market, an effective and efficient market, is the biggest driver for innovation.” He noted that government has the role in creating HIT standards to eliminate market inefficiencies. “We have to do the minimum government action necessary, but no less,” he said. HHS Secretary Kathleen Sebelius’ appointment of Dr. Mostashari became effective April 8, 2011.

You may view the video of his speech as well as a discussion of HIT with former Senators Tom Daschle and Bob Bennett here.

Further, the PMC has submitted comments on the ONC's strategic plan, the public comment period has been extended through May 6, 2011. You may submit your own comments by clicking here.

April 14, 2011

Congress Passes a Budget Increasing Funding for FDA, Cutting Funds for NIH
This week, the U.S. House of Representatives passed a continuing resolution for the budget for FY 2011 that strengthens the FDA’s financial foundation, while reducing the NIH budget.

Specifically, FDA’s budget will increase from $2.345 billion to $2.452 billion, or approximately $107 million over FY 2010. Click here for more information about FDA appropriations.

NIH’s budget, on the other hand, will decrease from $31.9 billion to $30.7 billion. This reduction of approximately $260 million represents a 0.8 percent decrease from FY 2010. Click here for more information about NIH appropriations.

With the FY 2011 budget complete, lawmakers will now focus on the FY 2012 budget and resolving differences between Republicans seeking broad budget cuts and Democrats supportive of President Obama’s proposed budget discussed in more detail below.

March 31, 2011

One Year In, Sebelius Discusses Health Care Reform
HHS Secretary Kathleen Sebelius discussed the achievements thus far and those to come from the Patient Protection and Affordable Care Act (PPACA) at Bloomberg Government’s recently hosted conference “The Iron Triangle of Health Care.” Secretary Sebelius focused her remarks on how PPACA makes changes not only to insurance practices, but also to health care delivery. This is a critical issue to address considering that currently, “it takes 17 years for a discovery to become common health care practice.” She also said that PPACA has created two avenues that will significantly improve the system; changing the payment mechanisms to reward better care, not simply abundance of care, and integrating electronic health records as a tool to improve treatment for patients by accruing all of their medical background information, instead of starting from scratch at each visit to a new doctor or hospital. Secretary Sebelius’ remarks coincided with the release of CMS’s proposed regulations for the Accountable Care Organizations created under PPACA, which allow “providers to share risk and savings.” If you would like to comment on the proposed regulations, you must do so by May 31, 2011.

March 21, 2011

Francis Collins Discusses NCATS
NIH Director Francis Collins spoke to representatives from the private sector in a webinar hosted by FasterCures on March 16, 2011 on the creation of the National Center for Advancing Translational Sciences (NCATS).  Dr. Collins sought to reassure industry that NCATS is meant to be a tool for partnerships, not an entity to detract from NIH’s basic science mission, nor a publicly financed drug company.  Rather, he framed the creation of NCATS as a potential solution to the increasing problem of drugs failing in the pre-clinical stage of development often referred to as “the valley of death” because of the high failure-rate. He believes that the current condition of the drug pipeline is “a scientific problem, ripe for examination” and sees NCATS as “a tool for experimentation and process engineering.” 

Dr. Collins invites you to give your feedback on NCATS. You may do so here: http://feedback.nih.gov/index.php/category/ncats.

The archived FasterCures webinar with Dr. Collins is here: http://fastercures.org/train/tools/webs.html.

February 25, 2011

FDA Releases Draft Pharmacogenomics Guidance
The Food and Drug Administration (FDA) in February released draft guidance on the early use of genomic data in clinical studies. The draft guidance, Clinical Pharmacogenomics: Premarketing Evaluation in Early Phase Clinical Studies, stresses that getting genomic information for all trial subjects during early development will make it easier to identify clinically-important genomic differences later in the process. Ideally, the draft guidance says, consent for DNA collection should be obtained from all participants when they are enrolled, to avoid bias that might result from delayed collection. It also suggests considering known pharmacogenomic factors during preclinical assessment, and notes that pharmacogenomic factors for assessing interindividual variability can be integrated into prospective clinical pharmacology studies. For more information or to submit comments, which are due by April 19, 2011, visit http://federalregister.gov/a/2011-3679.

February 15, 2011

President Releases Budget for Fiscal Year 2012
This week, the Obama Administration released its proposed budget for fiscal year 2012. Notable details from the proposed FDA and NIH budgets include:

Food and Drug Administration funding would increase from $2.60 billion to $2.74 billion. The FDA budget includes $48.7 million forAdvancing Regulatory Science and Facilities. “The Regulatory Science and Facilities Initiative will help FDA review and approve products that rely on new and emerging technologies and that offer promising new opportunities to diagnose, treat, cure and prevent disease… The investment in regulatory science will benefit new product development in areas such as personalized medicine and systems biology  including genomics.”

National Institutes of Health funding would increase 2.4 percent to $31.83 billion from $30.78 billion in 2011. One of three main themes will be “Enhancing the Evidence Base for Health Care Decisions: NIH’s aim is not only to spur the development of new treatments, but also to support rigorous programs for assessing and ensuring their effectiveness within populations and for individuals. Research in personalized medicine is essential to fulfilling the agency’s mission and will enhance the evidence base for decision-making in clinical practice.”

Supports Translational Science at the National Institutes of Health (NIH). Through implementation of the National Center for Ad­vancing Translational Sciences (NCATS) and the Cures Acceleration Network (CAN)-$100 million, NIH will increase its fo­cus on bridging the translational divide between basic science and therapeutic applications. The budget for NCATS will be released this Spring, and according to a statement by the NIH, “the overall budget for NCATS will be the sum of the imported programs—an amount much smaller than the several billion dollars currently being spent on translational research by existing Institutes and Centers.”

In addition, the President’s budget calls for research and development incentives, changes to the tax code and USPTO user fee schedule that may affect innovators of personalized medicine products and services. They include:

Increase Investment in Research and Development (R&D) and the Creation of Transformational Technologies. The 2012 Bud­get provides $148 billion for R&D overall.

Simplify, Expand, and Make Permanent the Research and Experimentation (R&E) Tax Credit.The President supports making the R&E tax credit permanent, expanding the credit by about 20 percent, and simplify it so that it is easier for firms to take this credit and make the investments our economy needs to compete.

Improve the Patent System and Protect Intellectual Property. USPTO to gain full access to its fee collections and strengthen USPTO’s efforts to improve the speed and quality of patent examinations through a temporary fee surcharge and regulatory and legislative reforms. In total, this will provide USPTO with more than $2.7 billion in resources in 2012, or more than 34 percent above 2010 levels.

February 9, 2011

New FDA Medical Device Innovation Initiative
DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2011–N–0063] SUMMARY: The Food and Drug Administration (FDA) is announcing the availability of a document for public comment entitled "Medical Device Innovation Initiative" (the report). The report proposes potential actions for FDA's Center for Devices and Radiological Health (CDRH) to facilitate the development, assessment, and regulatory review of innovative medical devices.
DATES: Submit either electronic or written comments on the report by April 11, 2011.
The public meeting will be held on March 15, 2011, from 8 a.m. to 5:30 p.m. Persons interested in attending and/or participating in the meeting must register by 5 p.m. on March 4, 2011.

February 7, 2011

Upcoming FDA Meeting on DTC genetic tests
DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2011–N–0066] Molecular and Clinical Genetics Panel of the Medical Devices Advisory Committee; Notice of Meeting
ACTION: Notice. This notice announces a forthcoming meeting of a public advisory committee of the Food and Drug Administration (FDA). The meeting will be open to the public.
Date and Time: The meeting will be held on March 8 and 9, 2011, from 8 a.m. to 6 p.m.
Agenda: To view the full notice including the agenda, click here.
Addresses: FDA is opening a docket for public comment on this document. The docket will open for public comment on February 7, 2011, and will close on March 1, 2011. Interested persons are encouraged to use the docket to submit either electronic or written comments regarding this meeting. Submit electronic comments to http://www.regulations.gov. Submit written comments to the Division of Dockets Management, Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. It is only necessary to send one set of comments. It is no longer necessary to send two copies of mailed comments. Identify comments with the docket number found in brackets in the heading of this document. Received comments may be seen in the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday.
Location: Holiday Inn, Ballroom, Two Montgomery Village Ave., Gaithersburg, MD.
Contact Person: James Swink, Center for Devices and Radiological Health, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993–0002, 301–796–6313, or FDA Advisory Committee Information Line, 1–800–741–8138 (301–443–0572 in the Washington, DC area), and follow the prompts to the desired center or product area.

January 21, 2011

PCORI Methodology Committee Lacks Private-Sector PM Expert
The Government Accountability Office (GAO) announced the members of the Methodology Committee for the Patient Centered Outcomes Research Institute (PCORI) in January. PMC was disappointed to see that none of the private sector members bring expertise in personalized medicine, particularly since the statute specifically identified experts in genomics as necessary to the Methodology Committee function.

In October, PMC submitted a letter to GAO nominating ten experts in personalized medicine from across academia and industry. Subsequent to the Methodology Committee’s selection, PMC recommended that the PCORI Board of Governors create an ad hoc advisory panel focused on personalized medicine and innovation to ensure that genomic and other personalized medicine considerations are addressed in the development of research methods and priorities.

January 12, 2011

Call for Abstracts to Participate in an AHRQ Research Methods Symposium on Patient-Centered Outcomes (Due March 1, 2011)
The Agency for Healthcare Research and Quality (AHRQ), through its Effective Health Care (EHC) Program, is accepting abstract submissions for an invitational symposium on research methods for comparative effectiveness (CER) and patient-centered outcomes research (PCOR).  The symposium is a follow-up to the 2006 and 2009 AHRQ conferences on Methods in Comparative Effectiveness Research; papers presented at past conferences were published in the journal Medical Care (copies may be freely downloaded from effectivehealthcare.ahrq.gov <http://effectivehealthcare.ahrq.gov>  or  http://tinyurl.com/dl59yw).

The theme of the 2011 conference is “Methods for Developing and Analyzing Clinically Rich Data for Patient-Centered Outcomes Research”.  Authors of abstracts that are selected for the symposium will be invited to present their work at the symposium and to submit a complete manuscript for peer-review and publication in a special journal supplement.

More information and instructions about abstract submission are available at: http://www.effectivehealthcare.ahrq.gov/index.cfm/methods-for-developing-and-analyzing-clinically-rich-data-for-patient-centered-outcomes-research/.  Submissions are due by March 1, 2011.