Download 21st Century Cures Reference Sheet

Reference Sheet: 21st Century Cures
New England Council Healthcare Committee Survey
Title I: Putting Patients First by Incorporating their Perspectives into the Regulatory Process and Addressing
Unmet Needs
Drug Regulatory Processes and Promoting Drug Research & Discovery
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Subtitle A: Patient Focused Drug Development: Require the FDA to establish a structured
framework for the meaningful incorporation of patient experience data into the regulatory decisionmaking process, including the assessment of desired benefits and tolerable risks associated with new
treatments.
Subtitle B: Surrogate Endpoint Qualification and Utilization: Establish a predictable, transparent
process for FDA’s consideration, and possible qualification, of surrogate endpoints. Also allow FDA to
use private-public partnerships to qualify other types of biomarkers.
Subtitle C: Approval of Breakthrough Therapies: Clarify that the FDA may approve a drug that has
received breakthrough designation when early stage clinical data provides sufficient evidence under
current safety and efficacy standards, considering risks and benefits of the drug and the risks
associated with the disease or condition for which unmet needs exist.
Subtitle J: Streamlined Data Review: Streamline review process for adding indications to a drug label
by allowing FDA to accept and review data summaries rather than full data packages.
Subtitle G: Expanded Access: Place transparency requirements on certain drug companies regarding
expanded access programs (programs for patients to access drugs before they are approved). Create
an expanded access task force to provide recommendations to Congress for further reforms of the
program.
Promoting Drug Research & Discovery
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Subtitle D: Antibiotic Drug Development:
o “Antibiotic Development to Advance Patient Treatment Act” to help foster development of
new antibiotics
o Create a new transferable exclusivity program
o “Developing an Innovative Strategy for Antimicrobial Resistance Microorganisms Act” to
incentivize new drug development by improving process of hospital payments
Subtitle K: Cures Acceleration Network: Authorize additional funds for research on repurposing
drugs for new uses.
Subtitle L: Dormant Therapies: Rewards investment in treatments and cures for diseases with unmet
medical needs. Allows innovators to choose pathway and receive fixed year protection period for
these therapies upon FDA approval.
Subtitle M: New Therapeutic Entities: Extend exclusivity for two years for significant improvements
to existing molecules (e.g., new delivery systems, new combinations, new formulations that lead to
les adverse events and increase patient benefits and adherence)
Subtitle N: Orphan Product Extensions Now: Provides six months of additional market exclusivity or
drugs if company establishes that drug treats a rare disease and receives rare disease indication from
FDA on its label.
Reference Sheet: 21st Century Cures
New England Council Healthcare Committee Survey
Breakthrough Devices
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Subtitle E: Priority Review for Breakthrough Devices: Establishes process for designation expedited
review of devices that represent breakthrough technologies with potential to address unmet medical
needs. FDA designation would translate into Medicare and Medicaid transitional coverage benefits
[currently placeholder].
Subtitle F: Accelerated Approval for Breakthrough Devices: Establish accelerated approval pathway
for medical devices.
Manufacturer & FDA Communications
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Subtitle H: Facilitating Responsible Communication of Scientific and Medical Developments:
Proposal to clarify and rationalize rules governing communication and information sharing from
manufacturers to physicians, insurers, researchers, etc [currently placeholder].
Subtitle I: Modernizing the Regulation of Social Media: Regulations of communications on social
media by FDA.
Title II: Building the Foundation for 21st Century Medicine, Including Helping Young Scientists
New Councils/Commissions
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Subtitle A: 21st Century Cures Consortium Act: Establishes public private partnership (“consortium”)
based on the EU’s Innovative Medicines Initiative to accelerate the discovery, development, and
delivery of innovative cures, treatments, and preventive measures.
Subtitle B: Medical Product Innovation Advisory Commission: Creates Medical Product Innovation
Advisory Commission (based on MedPAC) to advise Congress on issues related to discoverydevelopment-delivery cycle.
FDA Regulatory Issues
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Subtitle C: Regenerative Medicine: Requires FDA to update guidance on surrogate and intermediate
endpoints for accelerated approval of regenerative medicine products.
Subtitle D: Genetically Targeted Platform Technologies for Rare Diseases: Clarifies accelerated
approval pathway to enable FDA to rely on data from products that utilize similar genetically
targeted platform technology.
Subtitle E: Sensible Oversight for Technology Which Advances Regulatory Efficiency: Helps provide
regulatory certainty for those developing apps and health information technology.
Subtitle I: Combination Products: Requires FDA to issue guidance on review process for products
that include both drugs and devices.
Subtitle J: Modernizing Regulation of Diagnostics: [Placeholder]
Evidence, Data, and Information Technology
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Subtitle F: Building a 21st Century Data Sharing Framework: Establish data sharing framework to:
o Enable physicians and patients to better identify ongoing clinical trials
Reference Sheet: 21st Century Cures
New England Council Healthcare Committee Survey
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Enable researchers and developers to use Medicare data to improve patient care
Establish a process for Congress to address other issues identified by the President’s Council
of Advisors on Science and Technology
Subtitle G: Utilizing Real World Evidence: Authorize FDA to utilize real world evidence and require
FDA to issue guidance on collecting such evidence.
Subtitle H: Coverage with Evidence Development: Addresses Medicare’s coverage process and
allows Medicare beneficiaries to secure coverage for products that are the subject of the clinical trial
in which they participate.
Subtitle K: Interoperability: National interoperable health information infrastructure [placeholder]
Subtitle L: NIH – Federal Data Sharing: Requires those receiving NIH grants to share data.
Subtitle M: Accessing, Sharing, and Using Health Data for Research Purposes: Enabling research
potential of health facility data.
Other
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Subtitle N: 21st Century Chronic Disease Initiative Act: Requires HHS to carry out longitudinal study
designed to improve outcomes of patients with chronic diseases.
Subtitle O: Helping Young Emerging Scientists: Establishes program at NIH to help young emerging
scientists.
Subtitle P: Fostering High-Risk, High-Reward Science: Requires NIH to support high-risk, high-reward
(potentially breakthrough) projects.
Subtitle Q: Precision Medicine: [Placeholder]
Title III: Modernizing Clinical Trials
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Subtitle A: Clinical Research Modernization Act: Streamline institutional review board process,
particularly for clinical trials conducted at multiple sites, by minimizing regulatory duplication and
unnecessary delays.
Subtitle B: Broader Application of Bayesian Statistics and Adaptive Trial Design: Encourage broader
application of Bayesian statistics and adaptive trial designs.
Subtitle C: Post-Approval Studies and Clinical Trials: Ensure FDA and sponsors periodically evaluate
whether post-approval studies remain scientifically warranted.
Subtitle D: Pediatric Research Improvement Network: Require NIH to implement National Pediatric
Research Network Act
Subtitle E: Global Pediatric Clinical Trial: Sets Sense of Congress that NIH and FDA should work with
the EU, industry, and others to establish a global pediatric clinical trial network.
Title IV: Accelerating the Discovery, Development, and Delivery Cycle and Continuing 21 st Century
Innovation at NIH, FDA, CDC, and CMS
NIH, CDC, and FDA Provisions
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Subtitle A: National Institutes of Health:
o NIH Research Strategic Investment Plan
o Biomedical Research Working Group to Reduce Administrative Burden on Researchers
Reference Sheet: 21st Century Cures
New England Council Healthcare Committee Survey
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o NIH Travel [Placeholder]
o Increasing Accountability at NIH
o GAO Report on Common Fund
o Exemption for NIH from Paperwork Reduction Act Requirements
o Additional Funding for NIH Common Fund
o Additional Funding for NIH Brain Research
o NCATS Phase IIB Restriction
Subtitle B: Advancing Research for Neurological Diseases: Require CDC to set up a surveillance
system for neurological diseases.
Subtitle C: Vaccine Access, Certainty, and Innovation: Provide certainty and transparency with
regard to the regulation of vaccines, including with respect to CMS and CDC.
Subtitle D: Reagan-Udall Improvements: Improve Reagan-Udall Foundation.
Subtitle E: FDA Hiring, Travel, and Training: [Placeholder]
Subtitle F: FDA Succession Planning: Ensure FDA staff has ability to continue to improve expertise
and that FDA develops succession plan for management positions.
Medicare Provisions: Coverage
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Subtitle G: Disposable Medical Technologies: Reform coverage requirements for certain disposable
medical technologies.
Subtitle H: Local and National Coverage Decision Reforms: Reforms Medicare LCD process.
Subtitle P: Medicare Pharmaceutical and Technology Ombudsman: Establish ombudsman at CMS to
allow medical device and pharmaceutical companies to appeal decisions and better understand
rationale behind coverage decisions.
Subtitle Q: Ensure Local MACs Evaluate Data Related to Category III Codes: Ensure that local
Medicare Administrative Contractors (MACs) review all data before making coverage decisions on
Category III codes.
Medicare Provisions: Payment
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Subtitle J: Revise IPPS New Technology Add on Payment Reimbursement Amounts: Provides more
transparency regarding new technology add on payment reimbursement process.
Subtitle K: Lowing Medicare Patients OOP Costs: Allows seniors to better identify the OOP costs
they might face for a given treatment or service.
Subtitle L: Global Surgery Services Rule: Prevent implementation of global surgery services rule,
which does away with bundled payments for surgeons.
Subtitle M: Providers Consolidation and Medicare Payments Examined Through Evaluation:
Requires CMS to analyze and seek public input on how proposed Medicare payment policies would
affect the consolidation of providers and payers.
Miscellaneous Medicare Provisions
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Subtitle I: Telemedicine: Advance opportunities for telemedicine and new technologies to improve
the delivery of quality health care services to Medicare beneficiaries.
Reference Sheet: 21st Century Cures
New England Council Healthcare Committee Survey
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Subtitle N: Medicare Part D Patient Safety and Drug Abuse Prevention: Help prevent high-risk
Medicare beneficiaries from abusing controlled substances.
Subtitle O: Accelerating Innovation in Medicine: Establish a program that allows for patients to
access medical device treatments sooner than otherwise would be available.
Other
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Subtitle R: Advancing Care for Exceptional Kids: Establish Medicaid and CHIP Care Coordination
program for children with medically complex conditions.
Subtitle S: Continuing Medical Education Sunshine Exemption: Clarify that peer reviewed journals,
journal reprints, journal supplements, and medical textbooks are excluded from the reporting
requirement under the Sunshine Act.
Subtitle T: Medical Testing Availability: Clarify law regarding Research Use Only (RUO) labeled
products.
Title V: Modernizing Medical Product Regulation
Manufacturing
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Subtitle A: Manufacturing Incentives: Provide incentives for manufacturing generic drugs here in the
US.
Subtitle B: 21st Century Manufacturing: Require FDA to update guidance regarding novel
manufacturing techniques.
Subtitle C: Controlled Substance Manufacturing and Exports: Provide US pharmaceutical companies
with a level playing field regarding controlled substances exports.
Medical Device Reforms
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Subtitle D: Medical Device Reforms:
 Third Party Quality System Assessment
 Clarify Valid Scientific Evidence as Well-Documented, Real World Evidence Gathered from
Clinical Registries and Studies Published in Peer-Reviewed Journals
 Ensure FDA Reviewers are Trained on Least Burdensome Concept
 Improve Process of Government Recognition of Appropriate Standards Set by Medical
Community
 Streamline Process of Marketing Class I Medical Devices
 Streamline 510(k) Process for Medical Devices
 Allow FDA to Apply the Humanitarian Device Exemption (HDE) to Areas that Impact >4,000
Patients Where Public Health Requires Greater Availability to Treat or Diagnose and there is no
Alternative
 Streamline FDA Committee Advisory Process
 Supply Chain Security for Devices