Topic	HR 6: 21^st Century Cures Act (passed in House July 10, 2015)	Senate HELP Committee bills	Consensus Version (amendment to HR 34 to be taken up in the House Nov. 30)
Patient-Focused Drug Development
Patient experience data	Requires FDA to implement a structured framework to facilitate the incorporation of patient experiences in the consideration of a drug’s benefits and risks	Requires FDA to make a public statement regarding any patient experience data and related information submitted and reviewed as part of an approved drug application	Similar to Senate bill
Patient-focused drug development guidance	Requires FDA to issue guidance on methodological considerations for the collection of patient experience data; methodological approaches that may be used to assess patients’ beliefs with respect to benefits and risks in the management of disease; and methodologies, standards and potential experimental designs for patient-reported outcomes Requires FDA to convene workshops to obtain input regarding methodologies for developing guidance	Requires FDA to issue guidance on how to collect patient experience data, including: appropriate methods to collect data for use by the agency in regulatory decisions, how patients can propose draft guidance to FDA, how FDA will respond to patient experience data submissions, the format and content for patient experience data submissions and how FDA plans to use patient experience data when evaluating the risks and benefits of a drug	Similar to Senate bill
Streamlining patient input	Exempts FDA from going through the Paperwork Reduction Act clearance process when requesting information from patients regarding their disease or treatments	Similar to HR 6	Similar to HR 6
Report on patient experience drug development	Not included	Requires FDA to issue a report every five years – starting in 2021 –assessing the use of patient experience data in regulatory decision-making	Similar to Senate bill
Advancing New Drug Therapies
Qualification of drug development tools	Establishes an FDA review pathway for biomarkers, clinical outcomes assessments and other drug development tools	No specific FDA pathway, but aims to facilitate the use of biomarkers, surrogate endpoints and other drug development tools	Similar to HR 6
Targeted drugs for rare diseases	Authorizes FDA to rely upon data or information previously submitted by the sponsor for a different drug or biological product that incorporates or utilizes the same or similar underlying approach	Allows sponsors of genetically targeted drugs and variant protein targeted drugs to rely on data for the same or similar technology from previously approved applications by the same sponsor	Similar to Senate bill
Rare pediatric disease voucher program	Reauthorizes the pediatric rare disease priority review voucher program until 2019	Reauthorizes the pediatric rare disease priority review voucher program until 2022	Reauthorizes the pediatric rare disease priority review voucher program until 2020
GAO study of voucher programs	Requires Government Accountability Office study on the effectiveness of awarding priority review vouchers in providing incentives for the development of drugs that treat or prevent rare pediatric diseases	Similar to HR 6	Similar to HR 6
Orphan drug grants	Not included	Not included	Amends the orphan drug grant program to clarify that grants may be used for observational studies and other analyses conducted to assist in the understanding of the natural history of a rare disease or condition and in the development of a therapy
Grants for studying continuous drug manufacturing	Allows FDA to award grants to institutions of higher education and nonprofit organizations to further the study of continuous manufacturing for drugs	Not included	Similar to HR 6
Trial Design And Evidence Development
Clinical trial designs	Requires an FDA public meeting, followed by guidance documents, on incorporating adaptive designs and Bayesian statistical modeling into clinical protocols and NDAs/BLAs	Not included	Similar to HR 6 but discussion of statistical methods not limited to Bayesian approaches
Real-world evidence	Requires FDA establish a program to evaluate potential use of evidence from clinical experience to help support approval of a new indication for a previously approved drug and to help support or satisfy post-approval study requirements; requires FDA identify and execute pilot demonstrations to extend use of Sentinel system to support these efforts	Not included	Requires FDA to evaluate the use of real-world evidence to help support the approval of a new indication for a previously approved drug and to help support or satisfy post-approval study requirements; no specific reference to Sentinel demonstration projects
Human research subjects protection	Requires HHS Secretary to harmonize differences between human subject regulations under the Common Rule and FD&C Act to streamline the institutional review board process	Not included	Similar to HR 6
Informed consent waiver or alteration	Allows FDA to waive or alter informed consent requirements for clinical trials with minimal risk	Not included	Similar to HR 6
Patient Access To Therapies
Summary level reviews	Requires FDA to establish a streamlined data review program under which a sponsor may submit qualified data summaries to support approval for a new qualified indication; requires issuance of guidance documents	Allows FDA to rely upon qualified data summaries to support approval of a supplemental application, with respect to a qualified indication for a drug	Similar to Senate bill; no requirement for guidance documents
Expanded access	Requires pharmaceutical companies to make publicly available basic information about their expanded access policies for investigational drugs; requires FDA to finalize Q&A guidance on expanded access	Similar to HR 6	Similar to HR 6 but guidance requirement not included (FDA finalized the Q&A guidance document in June 2016)
Regenerative therapies	Not included	Requires FDA consult with National Institute of Standards and Technology and other stakeholders on development of standards, and to update agency regulations and guidance documents as appropriate REGROW Act (S.2689, not acted on by HELP committee), requires FDA establish a program to conditionally approve a cellular therapeutic product for a five-year period if the sponsor demonstrates preliminary clinical evidence of safety, and a reasonable expectation of effectiveness, without initiation of Phase III trials; requires draft guidance clarifying requirements with respect to cellular therapeutics	Allows FDA to grant accelerated approval for regenerative therapies but does not change the standards of evidence Requires FDA to update guidance and regulations on regenerative therapeutic products and hold a public meeting Requires FDA to consult with NIST and other stakeholders to establish standards to support the development, evaluation, and review of regenerative medicine and advanced therapies products
Health care economic information	Clarifies scope of FDAMA Section 114 on permissible manufacturer communications regarding health care economic information to certain entities; requires FDA draft guidance clarifying how manufacturers can disseminate truthful and non-misleading scientific and medical information that is not included in approved labeling	Not included	Similar to HR 6 but with no guidance requirement
Combination products	Requires FDA to issue final guidance describing the roles of all agency centers when reviewing a combination product	Specifies how FDA shall determine primary mode of action and designate the review center with primary jurisdiction Provides for sponsor submission, and FDA review, of a combination product review plan regarding the standards and requirements applicable to a product's premarket review Requires FDA final guidance describing the role of all agency centers when reviewing a combination product and the process for managing presubmission interactions with sponsors	More closely resembles Senate bill but instead of combination product review plan provides for FDA meetings with sponsors early in development on how best to study the combination product to meet relevant approval standards
Antimicrobial Innovation
Limited population pathway	Allows FDA to approve antimicrobial drugs based on a limited population if drug treats a serious or life-threatening infection; includes special provisions on labeling and promotional material; requires FDA draft guidance on program criteria and process	Similar to HR 6	Similar to HR 6
Medical Countermeasures
Medical countermeasures priority review voucher program	Not included	Creates new voucher program for approval of a medical countermeasures to material threats	Similar to Senate bill; provision sunsets Oct. 1, 2023

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