FDA New Year’s Resolution: Guidances On Payor Communications, Biosimilarity

Agency expects to issue 101 guidances in 2017, including those on REMS assessment, refusal to file NDA and BLA submissions, and amendments to ANDAs.

FDA Center for Drug Evaluation and Research’s 2017 guidance to-do list includes many of the items the agency had planned to get off its plate last year, including two of the most anticipated documents – guidances on biosimilar interchangeability and industry communications with payors.

The guidance agenda for calendar year 2017 includes 101 new and revised draft guidances. FDA posted the agenda on its website Jan. 11 and the following day issued two of the guidances on the list, questions and answers for 180-day exclusivity for abbreviated new drug application filers and guidance for multiple endpoints in clinical trials. (Also see "Multiple Endpoints In Clinical Trials: US FDA Advises How To Avoid False Positives" - Pink Sheet, 14 Jan, 2017.)

On Jan. 12, FDA also issued final guidance on naming of biosimilars. (Also see "Biologic Product Naming: US FDA Sticks With Suffixes ‘Devoid Of Meaning’" - Pink Sheet, 12 Jan, 2017.)

FDA’s guidances for demonstrating biosimilar interchangeability and evaluating analytical similarity data to support biosimilarity have been on agendas for the previous two years. The agency is under pressure to get them done this year as the second Biosimilar User Fee Act agreement requires FDA to issue both by Dec. 31. (See chart for list of key guidances).

The agency has also planned to issue draft guidance on manufacturer communications with payors and formulary committees for the past three years, but this November took a step forward with a public meeting to address its policy towards off-label communications. (Also see "Off-Label Communications: Industry, FDA Debate Role Of Peer Review" - Pink Sheet, 9 Nov, 2016.)

Other items that have been rolled over from FDA’s 2016 agenda include guidances on Risk Evaluation and Mitigation Strategy (REMS) assessment and planning; use of a drug master file for shared system REMS; natural history studies for rare disease drug development; and public disclosure of FDA-sponsored studies.

Among the new entries are four guidances related to generics: amendments to ANDA submissions, meetings with applicants of complex generic drug products, pre-submission facility correspondence for priority ANDAs in GDUFA II, and variations in drug products (ANDAs).

Other items new to the agenda are guidances on rare diseases drug development safety data considerations; refuse to file new drug application and biologics license application submissions; user fee waivers, reductions and refunds for drug and biological products; and fees incurred under the Drug Supply Chain Security Act.

Key Guidances On FDA Agenda For 2017
Category	Title	Background
Advertising	Drug and device manufacturer communications with payors, formulary committees and similar entities	FDA originally said it would issue a draft guidance on industry’s interactions with formulary committees in 2014 and has repeatedly set back the goal.
Biosimilarity	Considerations in demonstrating interchangeability with a reference product	Biosimilar user fee act agreement II requires FDA to issue the draft guidance by Dec. 31, 2017 (Also see "Biosimilar User Fee Agreement Puts FDA On Hook For Delayed Guidances" - Pink Sheet, 22 Sep, 2016.)
Biosimilarity	Statistical approaches to evaluation of analytical similarity data to support a demonstration of biosimilarity	BsUFA II agreement also requires this draft guidance by Dec. 31
Clinical/Medical	Natural history studies for rare disease drug development	21^st Century Cures legislation amended the orphan grant program to clarify that grants may be used for observational studies and other analyses conducted to assist in the understanding of the natural history of a rare disease or condition and in the development of a therapy (Also see "The Evolution Of 21^st Century Cures Legislation" - Pink Sheet, 29 Nov, 2016.)
Clinical/Statistical	Multiple endpoints in clinical trials	The guidance was issued on Jan. 12.
Generics	180-day exclusivity: questions and answers	FDA issued the guidance on Jan. 12. The agency is frequently sued over its exclusivity decisions. In one recent case, Par Pharmaceutical objected to FDA denial of exclusivity for its colchicine ANDA. (Also see "Par Fights FDA Forfeiture Of Generic Colcrys Exclusivity" - Pink Sheet, 3 Oct, 2016.)
Generics	Amendments to abbreviated new drug applications under GDUFA	Under Generic Drug User Fee Act II, ANDA amendments fall within a single, consolidated review goals scheme. (Also see "ANDAs Can Get Priority, Eight-Month Reviews Under User Fee Deal" - Pink Sheet, 24 Sep, 2016.)
Generics	Issuance of ANDA complete response letters before completion of review by one or more disciplines	FDA’s monthly refuse-to-receive numbers for ANDAs declined in the first two months of FY 2017 (Also see "Generic Application Quality Showing Signs Of Improvement" - Pink Sheet, 4 Jan, 2017.)
Generics	Meetings with applicants of complex generic drug products	Under GDUFA II, sponsors can meet with FDA prior to submission of an ANDA targeting complex products (Also see "Complex ANDAs To Be Allowed Pre-Submission Product Meetings" - Pink Sheet, 24 Oct, 2016.)
Procedural	Refuse to file: NDA and BLA submissions	Last year FDA issued a refuse to file letter to PTC Therapeutics saying the NDA for its Duchenne muscular dystrophy drug Translarna (ataluren) was not sufficiently complete to permit a substantive review (Also see "FDA Finds Fault With Third Duchenne Muscular Dystrophy Drug Filing" - Pink Sheet, 23 Feb, 2016.)
Procedural	REMS assessment: planning and reporting	FDA is grappling with how best to assess the program’s effectiveness, as was seen in a two-day review of the REMS for extended-release/long-acting opioids (Also see "REMS Assessment Challenges Shine Through In Opioids Review" - Pink Sheet, 16 May, 2016.)
Procedural	Survey methodologies to assess risk evaluation and mitigation strategies (REMS) goal related to knowledge	Same as above
User Fees	User fee waivers, reductions, and refunds for drug and biological products	GDUFA II agreement indicates ANDA sponsors will be paying higher fees (Also see "Generic Drug User Fees Will Jump More Than 50% In FY 2018" - Pink Sheet, 16 Oct, 2016.)