Should the Best Pharmaceuticals for Children Act be Amended to Accommodate 505(b)(2) NDA Labeling Carve-outs? “YES” . . . According to a New Bill in the U.S. Senate

We’re tickled pink here at the FDA Law Blog when we see an issue raised in one of our posts addressed in legislation. That happened last week when Senators Orrin Hatch (R-UT) and Robert Menendez (D-NJ) introduced the latest iteration of the Orphan Products Extension Now Accelerating Cures and Treatments Act (“OPEN Act”).

The OPEN Act, which draws inspiration from both the Best Pharmaceuticals for Children Act (“BPCA”) (FDC Act § 505A) and the Generating Antibiotic Incentives Now Act (“GAIN Act”) (FDC Act § 505E), would amend the FDC Act to add Section 505G, titled “Extension of Exclusivity Periods For A Drug Approved For A New Indication For A Rare Disease Or Condition,” to authorize FDA to designate a drug (including a biological product) “as a drug approved for a new indication to prevent, diagnose, or treat a rare disease or condition,” provided, among other things, that “prior to approval of an application or supplemental application for the new indication, the drug was approved or licensed for marketing under [FDC Act § 505(c)] or [PHS Act § 351(a)], but was not so approved or licensed for the new indication.”  The designation of a drug approved for a new indication for a rare disease or condition would result in a 6-month extension of various exclusivities provided for under both the FDC Act and the PHS Act.

The OPEN Act of 2017 (S. 1509), is largely a rehash of previous versions of the bill . . . . except for Sections 3 and 4 of the bill.  Of particular interest to this blogger is Section 4 of the OPEN Act of 2017. In March 2017, we put up a post, titled “Should the Best Pharmaceuticals for Children Act be Amended to Accommodate 505(b)(2) NDA Labeling Carve-outs?”  We noted that the BPCA, and FDC Act § 505A(o) in particular, does not address 505(b)(2) NDAs.  “The BPCA neither addresses the carve-out or retention of protected pediatric information from 505(b)(2) product labeling, nor does the BPCA address the use of disclaimers for protected pediatric use information that is carved-out of 505(b)(2) product labeling,” we stated.  This leads to an inequity:

FDC Act § 505A(o) . . . allows an ANDA applicant to omit from its labeling certain patent- and/or exclusivity-protected information concerning the pediatric use of a drug, and to include a disclaimer with respect to the omitted information. . . .

If FDA determines that the protected pediatric information is important safety information, and therefore, must be retained in 505(b)(2) product labeling for reasons of safe use, then a full approval for the affected 505(b)(2) product cannot be issued until pediatric exclusivity has expired. Instead, FDA could issue only a tentative approval, with final approval dependent on the expiration of exclusivity applicable to pediatric labeling information.  This is true regardless of how close a 505(b)(2) NDA product may be to an ANDA drug product (and which ANDA could be approved by operation of FDC Act § 505A(o)).

Section 4 of the OPEN Act of 2017 would remedy the inequity among ANDA and 505(b)(2) applicants that we pointed out by amending FDC Act § 505A(o) to include 505(b)(2) NDAs. Here’s how those proposed changes would appear in the statute if Section 4 of the OPEN Act of 2017 is enacted (deletions shown in strikethrough typeface and additions in bolded and italicized red typeface):

(o) PROMPT APPROVAL OF DRUGS UNDER SECTION 505(j) WHEN PEDIATRIC INFORMATION IS ADDED TO LABELING.—

(1) GENERAL RULE.— A drug for which an application has been submitted or approved under section 505(j) under subsection (b)(2) or (j) of section 505 of this title shall not be considered ineligible for approval under that section or misbranded under section 502 of this title on the basis that the labeling of the drug omits a pediatric indication or any other aspect of labeling pertaining to pediatric use when the omitted indication or other aspect is protected by patent or by exclusivity under clause (iii) or (iv) of section 505(j)(5)(F) of this title , or by exclusivity under clause (iii) or (iv) of section 505(j)(5)(F), clauses (iii) and (iv) of section 505(c)(3)(E), or section 527(a), or by an extension of such exclusivity under this section or section 505E.

(2) LABELING.— Notwithstanding clauses (iii) and (iv) of section 505(j)(5)(F), clauses (iii) and (iv) of section 505(c)(3)(E), or section 527 of this title, the Secretary may require that the labeling of a drug approved under section 505(j) drug approved pursuant to an application submitted under subsection (b)(2) or (j) of section 505 of this title that omits a pediatric indication or other aspect of labeling as described in paragraph (1) include—

(A) a statement that, because of marketing exclusivity for a manufacturer—

(i) the drug is not labeled for pediatric use; or

(ii) in the case of a drug for which there is an additional pediatric use not referred to in paragraph (1), the drug is not labeled for the pediatric use under paragraph (1); and

(B) a statement of any appropriate pediatric contraindications, warnings, or precautions that the Secretary considers necessary.

(3) PRESERVATION OF PEDIATRIC EXCLUSIVITY AND OTHER PROVISIONS.— This subsection does not affect—

(A) the availability or scope of exclusivity under this section;

(B) the availability or scope of exclusivity under section 505 for pediatric formulations;

(C) the question of the eligibility for approval of any application under section 505(j) that omits any other conditions of approval entitled to exclusivity under clause (iii) or (iv) of section 505(j)(5)(F); or 

(D) except as expressly provided in paragraphs (1) and (2), the operation of section 505.

(A) the availability or scope of exclusivity under—

(i) this section;

(ii) section 505 for pediatric formulations; or

(iii) section 527;

(B) the question of the eligibility for approval of any application under subsection (b)(2) or (j) of section 505 that omits any other conditions of approval entitled to exclusivity under—

(i) clause (iii) or (iv) of section 505(j)(5)(F);

(ii) clauses (iii) or (iv) of section 505(c)(3)(E); or

(iii) section 527; or

(C) except as expressly provided in paragraphs (1) and (2), the operation of section 505 or section 527.

We’re honored that Senators Hatch and Menendez incorporated our suggestion into legislation, and we’ll be watching closely to see if Section 4 of the OPEN Act of 2017 makes it into FDARA and is enacted into law.