the 21st Century Cures Act and Innovations in Clinical Trials

After the bill passed through house and senator, the president Obama signed the bill into law on December 13, 2016. The 21st Century Cures Act is now officially a law in effect.

If you have trouble to find the final version of the cures act, Here is the one signed into the law by the President Obama.

H.R.34 - 21st Century Cures Act

The NPR has a good summary of "who wins and who loses with the 21st Century Cures Act".
The 21st Century Cures Act will greatly benefit the NIH and NCI. Here is a latest article published in New England Journal of Medicine regarding NIH's perspectives on the Act.

The 21st Century Cures Act — A View from the NIH

Some sections in this act are very relevant to design and analysis of clinical trials. The section 3021 calls out the adaptive design and other novel clinical trial designs. 

SEC. 3021. Novel clinical trial designs.(a) Proposals for use of novel clinical trial designs for drugs and biological products.—For purposes of assisting sponsors in incorporating complex adaptive and other novel trial designs into proposed clinical protocols and applications for new drugs under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355) and biological products under section 351 of the Public Health Service Act (42 U.S.C. 262), the Secretary of Health and Human Services (referred to in this section as the “Secretary”) shall conduct a public meeting and issue guidance in accordance with subsection (b).
(b) Guidance addressing use of novel clinical trial designs.—
(1) IN GENERAL.—The Secretary, acting through the Commissioner of Food and Drugs, shall update or issue guidance addressing the use of complex adaptive and other novel trial design in the development and regulatory review and approval or licensure for drugs and biological products.
(2) CONTENTS.—The guidance under paragraph (1) shall address—
(A) the use of complex adaptive and other novel trial designs, including how such clinical trials proposed or submitted help to satisfy the substantial evidence standard under section 505(d) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(d));
(B) how sponsors may obtain feedback from the Secretary on technical issues related to modeling and simulations prior to—
(i) completion of such modeling or simulations; or
(ii) the submission of resulting information to the Secretary;
(C) the types of quantitative and qualitative information that should be submitted for review; and
(D) recommended analysis methodologies.
(3) PUBLIC MEETING.—Prior to updating or issuing the guidance required by paragraph (1), the Secretary shall consult with stakeholders, including representatives of regulated industry, academia, patient advocacy organizations, consumer groups, and disease research foundations, through a public meeting to be held not later than 18 months after the date of enactment of this Act.
(4) TIMING.—The Secretary shall update or issue a draft version of the guidance required by paragraph (1) not later than 18 months after the date of the public meeting required by paragraph (3) and finalize such guidance not later than 1 year after the date on which the public comment period for the draft guidance closes.

The act specifies that the real world evidence can be used to support the drug approval where Real world evidence means data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials.

The act creates a new pathway for medical device - breakthrough medical device - a similar pathway for breakthrough drug. 

The act specified the importance of the biomarker in drug approval process where biomarker “(A) means a characteristic (such as a physiologic, pathologic, or anatomic characteristic or measurement) that is objectively measured and evaluated as an indicator of normal biologic processes, pathologic processes, or biological responses to a therapeutic intervention; and

“(B) includes a surrogate endpoint.

The act contains a section for "targeted drugs for rare diseases"

SEC. 3012. Targeted drugs for rare diseases.Subchapter B of chapter V of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360aa et seq.) is amended by inserting after section 529 the following:
“SEC. 529A. Targeted drugs for rare diseases.“(a) Purpose.—The purpose of this section, through the approach provided for in subsection (b), is to—
“(1) facilitate the development, review, and approval of genetically targeted drugs and variant protein targeted drugs to address an unmet medical need in one or more patient subgroups, including subgroups of patients with different mutations of a gene, with respect to rare diseases or conditions that are serious or life-threatening; and
“(2) maximize the use of scientific tools or methods, including surrogate endpoints and other biomarkers, for such purposes.
“(b) Leveraging of data from previously approved drug application or applications.—The Secretary may, consistent with applicable standards for approval under this Act or section 351(a) of the Public Health Service Act, allow the sponsor of an application under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act for a genetically targeted drug or a variant protein targeted drug to rely upon data and information—
“(1) previously developed by the same sponsor (or another sponsor that has provided the sponsor with a contractual right of reference to such data and information); and
“(2) submitted by a sponsor described in paragraph (1) in support of one or more previously approved applications that were submitted under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act,
for a drug that incorporates or utilizes the same or similar genetically targeted technology as the drug or drugs that are the subject of an application or applications described in paragraph (2) or for a variant protein targeted drug that is the same or incorporates or utilizes the same variant protein targeted drug, as the drug or drugs that are the subject of an application or applications described in paragraph (2).“(c) Definitions.—For purposes of this section—
“(1) the term ‘genetically targeted drug’ means a drug that—
“(A) is the subject of an application under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act for the treatment of a rare disease or condition (as such term is defined in section 526) that is serious or life-threatening;
“(B) may result in the modulation (including suppression, up-regulation, or activation) of the function of a gene or its associated gene product; and
“(C) incorporates or utilizes a genetically targeted technology;
“(2) the term ‘genetically targeted technology’ means a technology comprising non-replicating nucleic acid or analogous compounds with a common or similar chemistry that is intended to treat one or more patient subgroups, including subgroups of patients with different mutations of a gene, with the same disease or condition, including a disease or condition due to other variants in the same gene; and
“(3) the term ‘variant protein targeted drug’ means a drug that—
“(A) is the subject of an application under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act for the treatment of a rare disease or condition (as such term is defined in section 526) that is serious or life-threatening;
“(B) modulates the function of a product of a mutated gene where such mutation is responsible in whole or in part for a given disease or condition; and
“(C) is intended to treat one or more patient subgroups, including subgroups of patients with different mutations of a gene, with the same disease or condition.
“(d) Rule of construction.—Nothing in this section shall be construed to—
“(1) alter the authority of the Secretary to approve drugs pursuant to this Act or section 351 of the Public Health Service Act (as authorized prior to the date of enactment of the 21st Century Cures Act), including the standards of evidence, and applicable conditions, for approval under such applicable Act; or
“(2) confer any new rights, beyond those authorized under this Act or the Public Health Service Act prior to enactment of this section, with respect to the permissibility of a sponsor referencing information contained in another application submitted under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act.”.