As part of ef­forts to pre­pare for the con­tin­ued rise in da­ta-rich ap­pli­ca­tions and to help fill the gap sep­a­rat­ing sci­en­tif­ic ad­vances and new ther­a­pies, the FDA on Wednes­day un­veiled its Tech­nol­o­gy Mod­ern­iza­tion Ac­tion Plan (TMAP).

Near-term mod­ern­iza­tion in com­put­er hard­ware and soft­ware tech­nolo­gies are the fo­cus of the FDA’s TMAP, which of­fers a broad overview of how the agency needs to adapt. For in­stance, the TMAP re­port notes that the FDA will need to have a tech­ni­cal in­fra­struc­ture that can ac­cept, eval­u­ate and an­a­lyze nov­el sources of da­ta (e.g., re­al-world da­ta) and ap­ply that da­ta to reg­u­la­to­ry de­ci­sion mak­ing over the next sev­er­al years.

“FDA’s ac­tion plan has three el­e­ments: (1) mod­ern­iza­tion of FDA’s tech­ni­cal in­fra­struc­ture; (2) en­hanced ca­pa­bil­i­ties at FDA to de­vel­op tech­nol­o­gy prod­ucts to sup­port its reg­u­la­to­ry mis­sion; and (3) com­mu­ni­ca­tion and col­lab­o­ra­tion be­tween FDA and stake­hold­ers, in­clud­ing the tech­nol­o­gy in­dus­try and oth­er gov­ern­ment agen­cies, to dri­ve tech­no­log­i­cal progress that is in­ter­op­er­a­ble across the sys­tem and de­liv­ers val­ue to con­sumers and pa­tients,” the re­port says.

Over­seen by the FDA’s Of­fice of In­for­ma­tion Man­age­ment and Tech­nol­o­gy (OIMT), in the com­ing months the FDA will en­gage with stake­hold­ers to de­vel­op a strat­e­gy and un­der­stand­ing of com­mon pri­or­i­ties for the agency’s ap­proach to da­ta.

“Is­sues to ad­dress in this con­text will in­clude: How FDA will en­sure that reg­u­la­to­ry de­ci­sions at the Agency con­tin­ue to be in­formed by high-qual­i­ty da­ta; How FDA will pro­mote ef­fi­cient da­ta use and da­ta stew­ard­ship across the Agency; How FDA will con­tin­ue its on­go­ing com­mit­ment to a high­ly se­cure da­ta and IT en­vi­ron­ment,” the re­port notes.

The FDA al­so ex­plains how it will start with “small pi­lot use case projects” to link reg­u­la­to­ry ex­per­tise with tech­nol­o­gy know-how by bring­ing FDA re­view­ers and tech­nol­o­gists to­geth­er to tar­get prob­lems and op­por­tu­ni­ties that will ad­vance mod­ern­iza­tion ef­forts.

“These so­lu­tions high­light what is pos­si­ble with­in FDA’s cur­rent en­vi­ron­ment. Ex­am­ples for con­sid­er­a­tion in­clude Pre­ci­sionF­DA, 7- and 15-day safe­ty re­port­ing for In­ves­ti­ga­tion­al New Drug (IND) ap­pli­ca­tions, and up­dat­ing the PRE­DICT mod­el with ma­chine learn­ing,” the re­port notes.

The agency will al­so look to build new tech­nolo­gies to en­able re­al-time FDA op­er­a­tional man­age­ment dash­boards, a col­lab­o­ra­tive project with Of­fice of the Na­tion­al Co­or­di­na­tor for Health In­for­ma­tion Tech­nol­o­gy on the “life of a da­ta el­e­ment” and tech­no­log­i­cal so­lu­tions for the FDA’s im­port op­er­a­tions.

Plan

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Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”

Lexicon Pharmaceutical is not giving up on its Type I diabetes candidate, despite FDA’s repeated rejections. This week the company laid out is argument again for a hearing on sotagliflozin in response to the FDA’s most recent denial.

The issue goes back to March 2019 when the FDA made very clear to Lexicon and its now departed partner Sanofi that it would not approve their application for a potential Type I diabetes drug because it does not appear to be safe.