1. Pink Sheet
  2. >>Product Reviews

Do Fast Track Regulatory Pathways Mean Quicker Reimbursement For Gene Therapies?

 
• By Francesca Bruce

The norms on securing market access are being rewritten for cell and gene therapies.

Fast Track
Accelerated regulatory pathyways can throw up evidentiary challenges for reimbursement • Source: Alamy

Without caution, fast track regulatory paths can be the “route to non-reimbursement” for cell and gene therapies, according to Eric Faulkner, vice president of global real-world-evidence at Novartis Gene Therapies. He advised that to succeed with payers, companies selling cell and gene therapies need to prove transformative effect and think “outside the box” when it comes to generating evidence and communicating with payers.

Novartis Gene Therapies

Novartis is the company behind the gene therapy Zolgesnma (onasemnogene abeparvovec) for the rare disease spinal muscular atrophy. It won approval from the US Food and Drug Administration in May 2019 after being awarded fast track, breakthrough therapy and priority review designations.

It was conditionally approved in the EU in May 2020 after receiving support through the EU’s priority medicines (PRIME) Scheme. Prior to its approval, it was initially accepted for regulatory review under the European Medicines Agency’s accelerated access mechanism but later revered to the standard review timetable.

Novartis also holds the ex-US rights to Luxturna, a gene therapy for inherited retinal dystrophy developed by Spark Theraputics. Luxturna won EU approval in November 2018. It was approved in the US in December 2017 after the FDA granted the application priority review and breakthrough therapy designations.

Securing reimbursement for gene and cell therapies is challenging for a number of reasons, including high prices for products, small...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Product Reviews

‘Unprecedented Story’: Revoked EU Marketing Authorization Fails To Thwart Translarna Sales

 
• By Francesca Bruce

PTC Therapeutics is continuing to supply its Duchenne muscular dystrophy treatment, Translarna, in some European markets, despite EU revocation of the drug’s conditional marketing authorization earlier this year.

US FDA’s Second Half 2025 User Fee Goals Swing Toward Respiratory Disease

 
• By Bridget Silverman

The 43 novel agents with user fee goal dates in the remainder of 2025 have fewer oncologics and infectious disease drugs than the first half of the year

Otsuka/Lundbeck’s Rexulti PTSD Claim Will Test US FDA AdComm’s Supportive Evidence Flexibility

 
• By Bridget Silverman

Rexulti’s post-traumatic stress disorder sNDA tries to make up for a failed Phase III study by placing a Phase II study into a key evidentiary role, raising questions for the July 18 advisory committee meeting about assessing discordant results and post-hoc analyses.

US Return Of GSK’s Blenrep Threatened By Ocular Toxicity, Dosage Optimization Questions

 
• By Sue Sutter

The FDA's Oncologic Drugs Advisory Committee will consider whether appropriate dosages of belantamab mafodotin have been identified for two second-line, multiple myeloma indications. Ocular toxicities and large number of dose modifications in trials raised FDA concerns about benefit-risk.

More from Pink Sheet

Otsuka/Lundbeck’s Rexulti PTSD Claim Will Test US FDA AdComm’s Supportive Evidence Flexibility

 
• By Bridget Silverman

Rexulti’s post-traumatic stress disorder sNDA tries to make up for a failed Phase III study by placing a Phase II study into a key evidentiary role, raising questions for the July 18 advisory committee meeting about assessing discordant results and post-hoc analyses.

Organ-On-A-Chip: Upcoming US FDA Guidance Could Help Flesh Out ‘Roadmap’

 
• By Michael McCaughan

The FDA is pushing the use of novel alternatives to animal models, but the Government Accountability Office said sponsors need more clarity and hopes an upcoming guidance will help.

UK’s Plan To Become World’s Third Top Life Sciences Economy Falls Short, Says ABPI

 
• By Neena Brizmohun

The Association of the British Pharmaceutical Industry says that while the government’s newly published Life Sciences Sector Plan includes many positive commitments, they are not enough.