Research into Pediatric Uses of Drugs; Additional Authorities of Food and Drug Administration Regarding Molecularly Targeted Cancer Drugs
Section 6601 · Sec. 6601 ·
What this chapter is about
This part widens what a maker may be told to study in children with cancer. The study can now cover the new drug used together with another drug. Guidance is due within a year, the changes start three years out, and two reports go to Congress.
The document says “is amended”Who acts: CongressHow: statuteSec. 6601 in the PDF
What the document says
“the investigation described in this paragraph is a molecularly targeted pediatric cancer investigation of--
``(i) the drug or biological product for which the application referred to in such paragraph is submitted; or”
Section 6601 rewrites section 505B(a)(3)(A) of the Federal Food, Drug, and Cosmetic Act. The required investigation may cover the drug in the application, or that drug used with another active ingredient. The other ingredient must either have an approved generic or biosimilar application and be found by the Secretary, after consulting the applicant, to be part of the standard of care for a pediatric cancer, or have an approved application to treat an adult cancer held by the same applicant and be directed at a molecular target the Secretary finds substantially relevant to a pediatric cancer.
What the document actually says
“the investigation described in this paragraph is a molecularly targeted pediatric cancer investigation of--
``(i) the drug or biological product for which the application referred to in such paragraph is submitted; or”
That sentence, in plain words
The study must look at a drug aimed at a target in a cancer cell. It may look at the drug in the application on its own.
What this is about
Cancer in children is often treated with drugs used together. A study of one drug alone may not answer much. This lets the study cover the pairing.
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The document says “shall”Who acts: drug and biological product sponsorsHow: statuteSec. 6601 in the PDF
What the document says
“A molecularly targeted pediatric cancer investigation referred to in subparagraph (A) shall be designed to yield clinically meaningful pediatric study data that is gathered using appropriate formulations for each age group for which the study is required, regarding dosing, safety, and preliminary efficacy to inform potential pediatric labeling.”
The rewritten paragraph requires the investigation to be designed to produce clinically meaningful pediatric data on dosing, safety and preliminary effectiveness, using appropriate formulations for each age group, to inform potential pediatric labeling. A combination study may be required only where the application drug has a single new active ingredient, or more than one where the combination has not been approved before but each ingredient is in a product already approved for an adult cancer.
What the document actually says
“A molecularly targeted pediatric cancer investigation referred to in subparagraph (A) shall be designed to yield clinically meaningful pediatric study data that is gathered using appropriate formulations for each age group for which the study is required, regarding dosing, safety, and preliminary efficacy to inform potential pediatric labeling.”
That sentence, in plain words
The study must give real answers about children. It must use a form of the drug fit for each age group. It must cover dose, safety and early results.
What this is about
A drug made for adults may not come in a form a child can take. Data from a study that ignores that is of little use. The rule says the study must fit the child.
No action is recorded against this proposal. That is not evidence that none has been taken, and nobody has yet read it against the record. See what the tracker does not yet cover.
The document says “shall”Who acts: Secretary of Health and Human ServicesHow: statuteSec. 6601 in the PDF
What the document says
“``The Secretary shall determine whether subparagraph (A) or (B) of subsection (a)(1) applies with respect to an application before the date on which the applicant is required to submit the initial pediatric study plan under paragraph (2)(A).''.”
Section 6601 requires the Secretary to decide which of the two requirements in section 505B(a)(1) applies to an application before the initial pediatric study plan is due. It also adds a rule of construction: an application subject to one of those subparagraphs is not subject to the other. The Secretary may require the results of completed preclinical studies relevant to the plan to be submitted at the same time.
What the document actually says
“``The Secretary shall determine whether subparagraph (A) or (B) of subsection (a)(1) applies with respect to an application before the date on which the applicant is required to submit the initial pediatric study plan under paragraph (2)(A).''.”
That sentence, in plain words
The health chief must decide which of two rules covers an application. That must happen before the first study plan is due.
What this is about
A maker cannot plan a study without knowing the rule. Deciding first saves wasted work. An application falls under one rule, not both.
No action is recorded against this proposal. That is not evidence that none has been taken, and nobody has yet read it against the record. See what the tracker does not yet cover.
The document says “shall”Who acts: Secretary of Health and Human Services, Commissioner of Food and DrugsHow: statuteSec. 6601 in the PDF
What the document says
“not later than 12 months after the date of enactment of this Act, issue draft guidance on the implementation of the amendments made by subsection (a); and”
Subsection (b) of section 6601 requires the Secretary, acting through the Commissioner of Food and Drugs, to issue draft guidance on implementing the amendments within 12 months of enactment, and to finalize it within 12 months of the close of the comment period on the draft.
What the document actually says
“not later than 12 months after the date of enactment of this Act, issue draft guidance on the implementation of the amendments made by subsection (a); and”
That sentence, in plain words
Within 12 months of the day this law passed, put out draft guidance. It explains how the changes will work.
What this is about
Draft guidance goes out for comment first. The final version comes within a year after comments close. Guidance is advice, not a rule.
No action is recorded against this proposal. That is not evidence that none has been taken, and nobody has yet read it against the record. See what the tracker does not yet cover.
The document says “shall apply”Who acts: Secretary of Health and Human ServicesHow: statuteSec. 6601 in the PDF
What the document says
“The amendments made by this section apply with respect to any application under section 505(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(b)) and any application under section 351(a) of the Public Health Service Act (42 U.S.C. 262(a)), that is submitted on or after the date that is 3 years after the date of enactment of this Act.”
Subsection (c) of section 6601 makes the amendments apply to applications under section 505(b) of the Federal Food, Drug, and Cosmetic Act and section 351(a) of the Public Health Service Act submitted on or after three years from enactment.
What the document actually says
“The amendments made by this section apply with respect to any application under section 505(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(b)) and any application under section 351(a) of the Public Health Service Act (42 U.S.C. 262(a)), that is submitted on or after the date that is 3 years after the date of enactment of this Act.”
That sentence, in plain words
The changes cover an application sent in later. It must come in three years after the day this law passed, or after that.
What this is about
Drug work takes years to plan. A rule that hit at once would catch studies already under way. The delay gives makers time.
No action is recorded against this proposal. That is not evidence that none has been taken, and nobody has yet read it against the record. See what the tracker does not yet cover.
The document says “shall”Who acts: Secretary of Health and Human Services, Comptroller General of the United StatesHow: statuteSec. 6601 in the PDF
What the document says
“Not later than 6 years after the date of enactment of this Act, the Secretary of Health and Human Services shall submit to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor, and Pensions of the Senate a report on the Secretary's efforts, in coordination with industry, to ensure implementation of the amendments made by subsection (a).”
Subsection (d) of section 6601 requires the Secretary to report to two committees within six years of enactment on efforts to ensure the amendments are put into practice. The Comptroller General must study, within eight years, how effective the required assessments and investigations have been in developing drugs for pediatric cancer, including benefits and burdens, and report the findings to the same committees within ten years.
What the document actually says
“Not later than 6 years after the date of enactment of this Act, the Secretary of Health and Human Services shall submit to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor, and Pensions of the Senate a report on the Secretary's efforts, in coordination with industry, to ensure implementation of the amendments made by subsection (a).”
That sentence, in plain words
Within six years the health chief must send a report to two groups in Congress. It covers work done to put these changes in place.
What this is about
A watchdog office must also study whether the rules worked. That study comes in eight years. Its findings go to Congress two years later.
No action is recorded against this proposal. That is not evidence that none has been taken, and nobody has yet read it against the record. See what the tracker does not yet cover.
The main things the section does: rewrite what a molecularly targeted pediatric cancer investigation may cover, set requirements for its design and the limitation on combination studies, require the Secretary to decide which requirement applies before the study plan is due, add a rule of construction keeping the two tracks apart, require draft and final guidance, set the applicability date, and require a report from the Secretary and a study and report from the Comptroller General.
The conforming amendments that only redirect cross references within section 505B.
The section amends the Federal Food, Drug, and Cosmetic Act and points at the Public Health Service Act. Neither is indexed here, so what section 505B otherwise requires is not recorded on this site.
