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Thursday, January 29, 2015

The Obama "Precision Medicine Inititative" and the Congressional "21st Century Cures Act."

During his recent 2015 SOTU Address, the President alluded to his forthcoming proposal to spur "Precision Medicine" -- a.k.a. "Personalized Medicine," focused in significant measure on basic and applied "omics" science for development of significantly more accurate and effective diagnostics and therapeutics.

Relatedly, now comes word of the release of  a Congressional "discussion draft" (pdf) of the "21st Century Cures Act."


The partisan bickering ensued before the toner was dry.
Dems balk on medical technology bill
By Darius Tahir, January 28, 2015


Democrats on the House Energy & Commerce Committee have withdrawn support from legislation that would loosen regulatory requirements on a broad range of new healthcare technologies, sources close to the legislative discussions say.

The sprawling 21st Century Cures Act, whose 393-page first draft was released on Jan. 27, is aimed at speeding up the adoption of next generation medical technologies. It seeks to foster drug development, including of antibiotics, by requiring the Food and Drug Administration to allow more “flexible” clinical trial design and gives medical-device makers more “innovative device review pathways.”

It also incorporates the Sensible Oversight for Technology which Advances Regulatory Efficiency (Software) Act sponsored by Rep. Marsha Blackburn (R-Tenn.), which would allow the FDA to take a risk-based regulatory approach to new healthcare information technologies. Its goal is to foster faster adoption of mobile medical applications, clinical decision-support tools and other healthcare-related software.

Rep. Frank Pallone (D-N.J.), the ranking member of the committee, said the draft legislation disappointed him because it “does not reflect true bipartisan collaboration.”

“In its current form, I am concerned that the nearly 400-page draft could create more problems for our healthcare system than it solves,” he said. “Further, the draft does not include any real dollars to fund additional basic research at the National Institutes of Health.”...
Below, I've stripped out the topical headings and sub-headings to give you a sense of where all of this might be headed.
TITLE I—PUTTING PATIENTS FIRST BY INCORPORATING THEIR PERSPECTIVES INTO THE REGULATORY PROCESS AND ADDRESSING UNMET NEEDS

Subtitle A—Patient-Focused Drug Development

Sec. 1001. Development and use of patient experience data To enhance structured risk-Benefit assessment framework.

Subtitle B—Surrogate Endpoint Qualification and Utilization

Sec. 1021. Evidentiary standards for the review of requests for the qualification of surrogate endpoints; Biomarkers partnership.

Sec. 1022. Enhancing the process for qualification of surrogate endpoints.
Sec. 1023. Transitional provisions for previous submissions for qualification of biomarkers as surrogate endpoints. 

Sec. 1024. Biannual reports to Congress.

Subtitle C—Approval of Breakthrough Therapies

Sec. 1041. Approval of breakthrough therapies.

Subtitle D—Antibiotic Drug Development

Sec. 1061. Approval of certain drugs for use in a limited population of patients. 

Sec. 1062. Susceptibility test interpretive criteria for microbial organisms.
Sec. 1063. Election to convey a portion of extended exclusivity period applicable to qualified infectious disease products.

Sec. 1064. Encouraging the development and use of new antimicrobial drugs.

Subtitle E—Priority Review for Breakthrough Devices 


Sec. 1081. Priority review for breakthrough devices.
Sec. 1082. CMS coverage of breakthrough devices to be supplied. 

Subtitle F—Accelerated Approval for Breakthrough Devices

Sec. 1101. Accelerated approval for breakthrough devices.

Subtitle G—Expanded Access

Sec. 1121. Expanded access policy as condition of expedited approval.
Sec. 1122. Notification of submitters of expanded access requests.
Sec. 1123. GAO qualitative analysis on individual patient access to unapproved therapies and diagnostics.

Sec. 1124. Expanded Access Task Force.
Sec. 1125. Finalizing draft guidance on expanded access.

Subtitle H—Facilitating Responsible Communication of Scientific and Medical Developments

Sec. 1141. to be supplied.

Subtitle I—Modernizing the Regulation of Social Media

Sec. 1161. Dissemination of information about medical products using the Internet.

Subtitle J—Streamlined Data Review

Sec. 1181. Streamlined data review program.

Subtitle K—Cures Acceleration Network

Sec. 1201. Flexible research authority.
Sec. 1202. Repurposing drugs.

Subtitle L—Dormant Therapies

Sec. 1221. Definitions.
Sec. 1222. Capturing lost opportunities and creating new cures for patients. 

Sec. 1223. Implementation and effect.

Subtitle M—New Therapeutic Entities

Sec. 1241. Extended exclusivity period for certain new drug applications and abbreviated new drug applications.

Subtitle N—Orphan Product Extensions Now

Sec. 1261. Extension of exclusivity periods for a drug approved for a new indi- cation for a rare disease or condition.

TITLE II—BUILDING THE FOUNDATION FOR 21ST CENTURY MEDICINE, INCLUDING HELPING YOUNG SCIENTISTS

Subtitle A—21st Century Cures Consortium Act

Sec. 2001. Innovative Cures Consortium.

Subtitle B—Medical Product Innovation Advisory Commission

Sec. 2021. Medical Product Innovation Advisory Commission.

Subtitle C—Regenerative Medicine

Sec. 2041. Issuance of guidance on surrogate and intermediate endpoints for accelerated approval of regenerative medicine products.

Subtitle D—Genetically Targeted Platform Technologies for Rare Diseases


Sec. 2051. Genetically targeted platform technologies for rare diseases.

Subtitle E—Sensible Oversight for Technology Which Advances Regulatory Efficiency

Sec. 2061. Medical and health software defined.
Sec. 2062. Applicability and inapplicability of regulation.
Sec. 2063. Exclusion from definition of device.

Subtitle F—Building a 21st Century Data Sharing Framework

PART 1—IMPROVING CLINICAL TRIAL DATA OPPORTUNITIES FOR PATIENTS

Sec. 2081. Standardization of data in Clinical Trial Registry Data Bank on eli- gibility for clinical trials.
Sec. 2082. Clinical Trial Data System.

PART 2—IMPROVING CLINICAL OUTCOMES FOR PATIENTS AND PROGRAM INTEGRITY THROUGH CMS DATA

Sec. 2085. Expanding availability of Medicare data.
Sec. 2086. Empowering patient research and better outcomes through CMS data.
Sec. 2087. Allowing clinical data registries To comply with HIPAA privacy and security law in lieu of complying with the privacy and security provisions of the Common Rule.
Sec. 2088. Access to CMS claims data for purposes of fraud analytics.

PART 3—BUILDING A 21ST CENTURY CLINICAL DATA SHARING SYSTEM

Sec. 2091. Commission on Data Sharing for Research and Development.
Sec. 2092. Recommendations for development and use of clinical data registries.

Subtitle G—Utilizing Real-World Evidence

Sec. 2101. Utilizing real-World evidence.

Subtitle H—Coverage With Evidence Development

Sec. 2121. Authority for coverage with evidence development for medical de- vices under the Medicare program.

Subtitle I—Combination Products

Sec. 2141. Regulation of combination products by the Food and Drug Administration.
Sec. 2142. GAO report on FDA regulation of combination products. Subtitle J—Modernizing Regulation of Diagnostics

Sec. 2161. to be supplied.

Subtitle K—Interoperability

Sec. 2181. to be supplied.

Subtitle L—NIH–Federal Data Sharing

Sec. 2201. Sharing of data generated through NIH-funded research.

Subtitle M—Accessing, Sharing, and Using Health Data for Research Purposes

Sec. 2221. Accessing, sharing, and using health data for research purposes.

Subtitle N—21st Century Chronic Disease Initiative Act

Sec. 2241. Plan for longitudinal study on outcomes of patients with a chronic disease.

Subtitle O—Helping Young Emerging Scientists

Sec. 2261. Funding research by emerging scientists through Common Fund.
Sec. 2262. Report on trends in age of recipients of NIH-funded major research grants.

Subtitle P—Fostering High-Risk, High-Reward Science

High-risk, high-reward research program.

Subtitle Q—Precision Medicine to be supplied.

TITLE III—MODERNIZING CLINICAL TRIALS

Subtitle A—Clinical Research Modernization

Protection of human subjects in research; applicability of rules. Use of institutional review boards for review of investigational device exemptions.

Subtitle B—Broader Application of Bayesian Statistics and Adaptive Trial Designs

Sec. 3021. Clinical trial modernization.

Subtitle C—Postapproval Studies and Clinical Trials


Sec. 3031. Evaluations of required postapproval studies and clinical trials.

Subtitle D—Pediatric Research Network Improvement

Sec. 3041. National Pediatric Research Network.

Subtitle E—Global Pediatric Clinical Trial

Sec. 3061. Sense of Congress.

TITLE IV—ACCELERATING THE DISCOVERY, DEVELOPMENT, AND DELIVERY CYCLE AND CONTINUING 21ST CENTURY INNOVATION AT NIH, FDA, CDC, AND CMS

Subtitle A—National Institutes of Health

Sec. 4001. NIH research strategic investment plan.
Sec. 4002. Biomedical research working group to reduce administrative burden on researchers.

Sec. 4003. NIH travel.

Sec. 4004. Increasing accountability at the National Institutes of Health.
Sec. 4005. GAO report on Common Fund.
Sec. 4006. Exemption for the National Institutes of Health from the Paperwork Reduction Act requirements.
Sec. 4007. Additional funding for NIH Common Fund.
Sec. 4008. Additional funding for NIH brain research.
Sec. 4009. NCATS Phase IIB Restriction.

Subtitle B—Advancing Research for Neurological Diseases

Sec. 4021. National neurological diseases surveillance system.

Subtitle C—Vaccine Access, Certainty, and Innovation PART 1—DEVELOPMENT, LICENSURE, AND RECOMMENDATIONS

Sec. 4041. Prompt review of vaccines by the Advisory Committee on Immuniza- tion Practices.
Sec. 4042. Review of transparency and consistency of ACIP recommendation process.

Sec. 4043. Guidance on vaccine development.
Sec. 4044. Meetings between CDC and vaccine developers.
Sec. 4045. Modifications to priority review voucher program for tropical diseases.
Sec. 4046. Guidance on changes to an approved application for biological products.
Sec. 4047. Expediting the process for export certifications for vaccines.
Sec. 4048. NIH vaccine research.

PART 2—MEDICARE, MEDICAID, AND OTHER PROVISIONS
Sec. 4061. Requiring prompt updates to Medicare program upon issuance of ACIP recommendations.

Sec. 4062. Encouraging health plans to establish programs to increase adult immunization.

Subtitle D—Reagan-Udall Improvements Bill


Sec. 4081. Reagan-Udall Foundation for the Food and Drug Administration.

Subtitle E—FDA Hiring, Travel, and Training

Sec. 4101. TO BE SUPPLIED.

Subtitle F—FDA Succession Planning

Sec. 4121. Professional development of FDA staff.

Sec. 4122. FDA management succession planning.

Subtitle G—Disposable Medical Technologies

Sec. 4141. Coverage of certain disposable medical technologies under the Medicare program.

Subtitle H—Local and National Coverage Decision Reforms

Sec. 4161. Improvements in the Medicare local coverage determination (LCD) process.

Subtitle I—Telemedicine


Sec. 4181. Advancing telehealth opportunities in Medicare.

Subtitle J—Revise IPPS New Technology Add-On Payment (NTAP) Reimbursement Amounts

Sec. 4201. Coding and reimbursement reforms.

Subtitle K—Lowering Medicare Patients OOP Costs

Sec. 4221. Medicare site-of-service price transparency.

Subtitle L—Global Surgery Services Rule

Sec. 4241. Treatment of global surgery services rule.

Subtitle M—Providers Consolidation and Medicare Payments Examined Through Evaluation

Sec. 4261. Rulemaking that implements certain Medicare payment changes to consider effects on provider consolidation.

Subtitle N—Medicare Part D Patient Safety and Drug Abuse Prevention

Sec. 4281. Establishing PDP safety program to prevent fraud and abuse in Medicare prescription drug plans.

Sec. 4282. Part D suspension of claims payment.
Sec. 4283. Improving activities of Medicare Drug Integrity Contractors (MEDICs).

Sec. 4284. Requiring e-prescribing for coverage of covered part D controlled substances.

Subtitle O—Accelerating Innovation in Medicine


Sec. 4301. Establishment of manufacturer opt-out program for medical devices.

Subtitle P—Medicare Pharmaceutical and Technology Ombudsman

Sec. 4321. Medicare pharmaceutical and technology ombudsman.

Subtitle Q—Ensuring Local Medicare Administrative Contractors Evaluate Data Related to Category III Codes

Sec. 4341. Ensuring local medicare administrative contractors evaluate data re- lated to Category III codes.

Subtitle R—Advancing Care for Exceptional Kids

Sec. 4361. Findings.
Sec. 4362. Establishment of Medicaid and CHIP Care Coordination program for children with medically complex conditions as Medicaid State option.

Subtitle S—Continuing Medical Education Sunshine Exemption

Sec. 4381. Exempting from manufacturer transparency reporting certain transfers used for educational purposes.

Subtitle T—Medical Testing Availability

Sec. 4401. Clarification regarding research use only products.

TITLE V—MODERNIZING MEDICAL PRODUCT REGULATION

Subtitle A—Manufacturing Incentives

Sec. 5001. Extension of exclusivity period for American-manufactured generic drugs and biosimilars.

Subtitle B—21st Century Manufacturing

Sec. 5021. Updating regulations and guidance on current good manufacturing practice requirements.

Subtitle C—Controlled Substance Manufacturing and Exports

Sec. 5041. Re-exportation among members of the European Economic Area.

Subtitle D—Medical Device Reforms

Sec. 5061. Third-party quality system assessment.
Sec. 5062. Valid scientific evidence.
Sec. 5063. Training and oversight in least burdensome means concept.
Sec. 5064. Recognition of standards.
Sec. 5065. Notification of marketing of certain class I devices.
Sec. 5066. General and specific uses.
Sec. 5067. Humanitarian device exemption application to in vitro diagnostics. Sec. 5068. Advisory committee process.

Subtitle E—Supply Chain Security for Devices

Sec. 5081. Short title.
Sec. 5082. Device distribution supply chain.
Sec. 5083. Authorized trading partners.
Sec. 5084. National licensing standards for wholesale device distributors. 

Sec. 5085. National licensing standards for third-party logistics providers. 
Sec. 5086. Waivers and exemptions.
Sec. 5087. Uniform national policy.

Sec. 5088. Penalties.
The legislative sausage-making process. Thus far only miniscule and vague references therein alluding to "health information technology" and "interoperability."
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SPEAKING OF DC
ONC Annual Meeting
Interoperable Health IT for a Healthy Nation


February 2 - 3, 2015
Washington Hilton
1919 Connecticut Avenue
Washington, D.C. 20009

The Office of the National Coordinator for Health IT (ONC) will hold the 2015 Annual Meeting February 2-3, 2015, at the Washington Hilton in Washington, D.C. The meeting will gather over 1,000 health IT partners for two days of plenaries and breakout sessions. Online registration has closed; however onsite registration will be available. The Annual Meeting will also be webcasted on February 2, 2015 and the morning plenary on February 3, 2015.
What has SHARP accomplished for EHRs?
'I would say in general that the SHARP programs were successful, but some were more successful than others'

January 29, 2015


After four years and $60 million, the ONC's Strategic Health IT Advanced Research Projects program is essentially done, and each project has made strides in improving various elements of EHRs. Those involved in SHARP believe their work has advanced the health IT field, but it remains to be seen if any of their innovations find their way into widespread use.

"I would say in general that the SHARP programs were successful, but some were more successful than others," said Douglas Fridsma, MD, former ONC chief scientist who departed in September to become president and CEO of the American Medical Informatics Association. Fridsma was effectively the point person for SHARP while at ONC.

With money appropriated by the 2009 HITECH Act, ONC issued grants in 2010 to four major institutions, to "address well-documented problems that impede the adoption of health IT," according to the program's website...
JAN 30th UPDATE
Medscape Medical News
President Requests $215M for 'Precision Medicine'

Alicia Ault, January 30, 2015


Washington, DC — Putting his money where his mouth is, President Obama will seek $215 million to finance the Precision Medicine Initiative he first mentioned in his State of the Union speech on January 20.

The funding request will be included in his fiscal 2016 budget, which will be sent to Congress on February 5.

"The time is right to unleash a new wave of advances in this area — precision medicine," said Obama at today at the East Wing of the White House. And he said he knew the time was right, in part, because "there is bipartisan support for the idea."

A centerpiece of the president's proposal is a million-patient research cohort that will depend largely on physicians and hospitals to help recruit participants and ensure there is continuous and long-term data collection and follow-up, Francis S. Collins, MD, PhD, director of the National Institutes of Health (NIH), told Medscape Medical News.

He likened the Precision Medicine Initiative to the Human Genome Project...
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More to come...

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