Cures Act Drug Regulatory Changes Summarized
Ten Hogan Lovells attorneys says the 21st Century Cures Act signed by President Obama unleashes “an expansive suite of measures designed to deliver new cures and treatments to patients.” They highlight key provisions in FDA’s regulation of drugs and biologics:
- require HHS/FDA guidance on novel clinical trial designs;
- require FDA to establish a draft framework to evaluate potential use of real-world evidence in assessing drug safety and efficacy and issue guidance within five years on acceptable uses of real-world evidence to support drug applications;
- require promulgation of provisions in FDA’s informed consent regulations to exempt clinical trials that pose no more than minimal risk to human subjects where appropriate safeguards protecting the rights, safety, and welfare of those subjects are in place;
- require entities conducting clinical trials to submit to ClinicalTrials.gov results of its analysis of the different effects of treatment in women and minorities;
- require FDA to publicly issue a brief statement of patient experience data and related information submitted and reviewed in conjunction with each drug application approved by the agency;
- require FDA to publish a guidance for a framework for qualification of drug development tools, including a multi-step process of submitting a letter of intent, qualification plan, and full qualification package;
- broaden the parameters of healthcare economic information that manufacturers may use to promote their products to formulary committees, payors, and similar entities;
- permit FDA to rely on a “qualified data summary” to approve a supplemental application for a “qualified indication” of a drug or biologic product;
- modify and clarify FDA’s regulation of combination products, including requiring the agency to issue a final guidance on combination products that describes the structured process for managing pre-submission interactions, best practices for the agency in such interactions, and information that sponsors should submit with a combination product meeting request;
- require manufacturers or distributors of investigational drugs to make their expanded access policies publicly accessible;
- clarify FDA authority to facilitate the development, review, and approval of generically targeted drugs and variant protein targeted drugs to address unmet medical needs in one or more patient subgroups for rare diseases or conditions that are serious or life-threatening;
- expand the types of grants and contracts FDA can make for developing drugs for rare diseases and conditions;
- reauthorize for four more years the priority review voucher program for rare pediatric disease product applications;
- accelerate approval for regenerative advanced therapies;
- require FDA to issue guidance within one year for medical devices used in the recovery, isolation, or delivery of regenerative advanced therapies;
- require FDA within two years to initiate and lead a stakeholder engagement process to develop standards for regenerative medicine; and
- require FDA to provide an annual report to Congress on the number and type of regenerative advanced therapy applications filed, approved, licensed, withdrawn, or denied, and on the number of applications granted accelerated approval or priority review.