§360bb. Designation of drugs for rare diseases or conditions — Inbound Citations

Code of Federal Regulations Citations

Cited by 1 regulation in the Code of Federal Regulations.

• 26 C.F.R. § 1.28-1(c)(1)(ii) — Credit for clinical testing expenses for certain drugs for rare diseases or conditions.

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  (ii) Occurs after the date the drug is designated as a drug for a rare disease or condition under section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb),

Citations to 21 U.S.C. § 360bb as a whole

• 21 U.S.C. § 355a(b)(1)(A)(ii)

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  (ii) if the drug is designated under section 360bb of this title for a rare disease or condition, the period referred to in section 360cc(a) of this title is deemed to be seven years and six months rather than seven years; and

• 21 U.S.C. § 355a(c)(1)(A)(ii)

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  (ii) if the drug is designated under section 360bb of this title for a rare disease or condition, the period referred to in section 360cc(a) of this title is deemed to be seven years and six months rather than seven years; and

• 21 U.S.C. § 355c(k)(1)

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  (1) Unless the Secretary requires otherwise by regulation and except as provided in paragraph (2), this section does not apply to any drug or biological product for an indication for which orphan designation has been granted under section 360bb of this title.

• 21 U.S.C. § 360cc(a)

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  (a) Except as provided in subsection (b), if the Secretary—

  (1) approves an application filed pursuant to section 355 of this title, or

  (2) issues a license under section 262 of title 42

  for a drug designated under section 360bb of this title for a rare disease or condition, the Secretary may not approve another application under section 355 of this title or issue another license under section 262 of title 42 for the same drug for the same approved use or indication within such rare disease or condition for a person who is not the holder of such approved application or of such license until the expiration of seven years from the date of the approval of the approved application or the issuance of the license. Section 355(c)(2)¹ of this title does not apply to the refusal to approve an application under the preceding sentence.

• 21 U.S.C. § 360cc(c)(1)

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  (1) If a sponsor of a drug that is designated under section 360bb of this title and is otherwise the same, as determined by the Secretary, as an already approved or licensed drug is seeking exclusive approval or exclusive licensure described in subsection (a) for the same use or indication for which the already approved or licensed drug was approved or licensed, the Secretary shall require such sponsor, as a condition of such exclusive approval or licensure, to demonstrate that such drug is clinically superior to any already approved or licensed drug that is the same drug.

• 21 U.S.C. § 360cc(c)(3)

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  (3) This subsection applies to any drug designated under section 360bb of this title for which an application was approved under section 355 of this title or licensed under section 262 of title 42 after August 18, 2017, regardless of the date on which such drug was designated under section 360bb of this title.

• 21 U.S.C. § 360cc(e)(1)

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  (1) upon the designation of any drug under section 360bb of this title, shall notify the sponsor of such drug in writing of the basis for the designation, including, as applicable, any plausible hypothesis offered by the sponsor and relied upon by the Secretary that the drug is clinically superior to a previously approved drug; and

• 21 U.S.C. § 360cc(f)

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  (f) In this section, the term “approved use or indication” means the use or indication approved under section 355 of this title or licensed under section 262 of title 42 for a drug designated under section 360bb of this title for a rare disease or condition.

• 21 U.S.C. § 360dd

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  If a drug is designated under section 360bb of this title as a drug for a rare disease or condition and if notice of a claimed exemption under section 355(i) of this title or regulations issued thereunder is filed for such drug, the Secretary shall encourage the sponsor of such drug to design protocols for clinical investigations of the drug which may be conducted under the exemption to permit the addition to the investigations of persons with the disease or condition who need the drug to treat the disease or condition and who cannot be satisfactorily treated by available alternative drugs.

• 21 U.S.C. § 360ee(b)(1)(B)

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  (B) preclinical testing involving a drug for a rare disease or condition which occurs after the date such drug is designated under section 360bb of this title and before the date on which an application with respect to such drug is submitted under section 355(b) of this title or under section 262 of title 42; and

• 21 U.S.C. § 360ee(b)(2)

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  (2) The term “rare disease or condition” means (1) in the case of a drug, any disease or condition which (A) affects less than 200,000 persons in the United States, or (B) affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug, (2) in the case of a medical device, any disease or condition that occurs so infrequently in the United States that there is no reasonable expectation that a medical device for such disease or condition will be developed without assistance under subsection (a), and (3) in the case of a medical food, any disease or condition that occurs so infrequently in the United States that there is no reasonable expectation that a medical food for such disease or condition will be developed without assistance under subsection (a). Determinations under the preceding sentence with respect to any drug shall be made on the basis of the facts and circumstances as of the date the request for designation of the drug under section 360bb of this title is made.

• 21 U.S.C. § 360ff(a)(3)(B)

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  (B) The disease is a rare disease or condition, within the meaning of section 360bb of this title.

• 21 U.S.C. § 360ff(d)(2)

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  (2) The request for a designation under paragraph (1) shall be made at the same time a request for designation of orphan disease status under section 360bb of this title or fast-track designation under section 356 of this title is made. Requesting designation under this subsection is not a prerequisite to receiving a priority review voucher under this section.

• 21 U.S.C. § 360ff–1(c)(1)(A)

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  (A) is the subject of an application under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)] for the treatment of a rare disease or condition (as such term is defined in section 360bb of this title) that is serious or life-threatening;

• 21 U.S.C. § 360ff–1(c)(3)(A)

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  (A) is the subject of an application under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)] for the treatment of a rare disease or condition (as such term is defined in section 360bb of this title) that is serious or life-threatening;

• 21 U.S.C. § 379h(a)(1)(F)

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  (F) A human drug application for a prescription drug product that has been designated as a drug for a rare disease or condition pursuant to section 360bb of this title shall not be subject to a fee under subparagraph (A), unless the human drug application includes an indication for other than a rare disease or condition.

• 21 U.S.C. § 379h(k)(1)

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  (1) A drug designated under section 360bb of this title for a rare disease or condition and approved under section 355 of this title or under section 262 of title 42 shall be exempt from prescription drug program fees under this section, if the drug meets all of the following conditions:

  (A) The drug meets the public health requirements contained in this chapter as such requirements are applied to requests for waivers for prescription drug program fees.

  (B) The drug is owned or licensed and is marketed by a company that had less than $50,000,000 in gross worldwide revenue as determined under paragraph (2).

• 42 U.S.C. § 236(e)(1)

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  (1) identifying the drugs which have been designated under section 526 of the Federal Food, Drug, and Cosmetic Act [21 U.S.C. 360bb] for a rare disease or condition,

• 42 U.S.C. § 256b(e)

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  (e) For covered entities described in subparagraph (M) (other than a children’s hospital described in subparagraph (M)), (N), or (O) of subsection (a)(4), the term “covered outpatient drug” shall not include a drug designated by the Secretary under section 360bb of title 21 for a rare disease or condition.

• 42 U.S.C. § 262(m)(2)(B)

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  (B) if the biological product is designated under section 526¹ [21 U.S.C. 360bb] for a rare disease or condition, the period for such biological product referred to in section 527(a)¹ [21 U.S.C. 360cc(a)] is deemed to be 7 years and 6 months rather than 7 years.

• 42 U.S.C. § 262(m)(3)(B)

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  (B) if the biological product is designated under section 526¹ [21 U.S.C. 360bb] for a rare disease or condition, the period for such biological product referred to in section 527(a)¹ [21 U.S.C. 360cc(a)] is deemed to be 7 years and 6 months rather than 7 years.

• 42 U.S.C. § 287(b)(2)

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  (2) The Center may support clinical trial activities through the end of phase III for a treatment for a rare disease or condition (as defined in section 360bb of title 21) so long as—

  (A) the Center gives public notice for a period of at least 120 days of the Center’s intention to support the clinical trial activities in phase III;

  (B) no public or private organization provides credible written intent to the Center that the organization has timely plans to further the clinical trial activities or conduct clinical trials of a similar nature beyond phase IIB; and

  (C) the Center ensures that support of the clinical trial activities in phase III will not increase the Federal Government’s liability beyond the award value of the Center’s support.

• 42 U.S.C. § 1320f–1(e)(3)(A)

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  (A) A drug that is designated as a drug for only one rare disease or condition under section 360bb of title 21 and for which the only approved indication (or indications) is for such disease or condition.

• 42 U.S.C. § 1395l(t)(2)

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  (2) Under the payment system—

  (A) the Secretary shall develop a classification system for covered OPD services;

  (B) the Secretary may establish groups of covered OPD services, within the classification system described in subparagraph (A), so that services classified within each group are comparable clinically and with respect to the use of resources and so that an implantable item is classified to the group that includes the service to which the item relates;

  (C) the Secretary shall, using data on claims from 1996 and using data from the most recent available cost reports, establish relative payment weights for covered OPD services (and any groups of such services described in subparagraph (B)) based on median (or, at the election of the Secretary, mean) hospital costs and shall determine projections of the frequency of utilization of each such service (or group of services) in 1999;

  (D) subject to paragraph (19), the Secretary shall determine a wage adjustment factor to adjust the portion of payment and coinsurance attributable to labor-related costs for relative differences in labor and labor-related costs across geographic regions in a budget neutral manner;

  (E) the Secretary shall establish, in a budget neutral manner, outlier adjustments under paragraph (5) and transitional pass-through payments under paragraph (6) and temporary additional payments for non-opioid treatments for pain relief under paragraph (16)(G), and other adjustments as determined to be necessary to ensure equitable payments, such as adjustments for certain classes of hospitals;

  (F) the Secretary shall develop a method for controlling unnecessary increases in the volume of covered OPD services;

  (G) the Secretary shall create additional groups of covered OPD services that classify separately those procedures that utilize contrast agents from those that do not; and

  (H) with respect to devices of brachytherapy consisting of a seed or seeds (or radioactive source), the Secretary shall create additional groups of covered OPD services that classify such devices separately from the other services (or group of services) paid for under this subsection in a manner reflecting the number, isotope, and radioactive intensity of such devices furnished, including separate groups for palladium-103 and iodine-125 devices and for stranded and non-stranded devices furnished on or after July 1, 2007.

  For purposes of subparagraph (B), items and services within a group shall not be treated as “comparable with respect to the use of resources” if the highest median cost (or mean cost, if elected by the Secretary under subparagraph (C)) for an item or service within the group is more than 2 times greater than the lowest median cost (or mean cost, if so elected) for an item or service within the group; except that the Secretary may make exceptions in unusual cases, such as low volume items and services, but may not make such an exception in the case of a drug or biological that has been designated as an orphan drug under section 360bb of title 21.

• 42 U.S.C. § 1395l(t)(6)(A)(i)

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  (i) A drug or biological that is used for a rare disease or condition with respect to which the drug or biological has been designated as an orphan drug under section 360bb of title 21 if payment for the drug or biological as an outpatient hospital service under this part was being made on the first date that the system under this subsection is implemented.