Meeting on the Meds | Ultra-Orphan Drugs Should Be First Area of Focus for Regulatory Alignment, Say Drug Regulators

Date: April 13, 2021 | Country: JAPANUNITED KINGDOMUNITED STATES | Region: ASIA & SOUTH PACIFICEUROPENORTH AMERICA | Type: Regulation | Keywords: #access #accessconsortium #allianceforregenerativemedicine #atmp #cellandgenetherapy 
#druglaunch #ema #fda #mhra #negotiation #orphandrug #projectorbis #raredisease #realworldevidence 
#regulatoryharmonization #regulatorysubmission #ultrararedisease

PRICENTRIC BRIEF:

  • During the virtual 2021 Cell & Gene Therapy Meeting on the Mediterranean organized by the Alliance for Regenerative Medicine (ARM), national drug regulators from the United States, European Union, United Kingdom, and Japan concluded that regulatory harmonization is crucial to help patients with ultra-rare diseases access advanced therapy medicinal products (ATMPs), including cell and gene therapies
  • Due to very small patient populations (sometimes 100 to 200 people worldwide) and national-level idiosyncrasies, making an ATMP commercially available for an ultra-rare disease is not viable for pharmaceutical companies; therefore, regulatory harmonization would combine markets to make launching a drug more viable for companies
  • ATMPs should be the first area of focus for regulatory harmonization, and early dialogue between drug regulators and pharmaceutical companies would greatly benefit relevant patients—already, some drug regulators have teamed up for early conversations with companies (for example, the ACCESS Consortium and Project Orbis) 

 

THE DETAILS

BARCELONA, Spain – During the virtual 2021 Cell & Gene Therapy Meeting on the Mediterranean organized by the Alliance for Regenerative Medicine (ARM), national drug regulators from the United States, European Union, United Kingdom, and Japan concluded that regulatory harmonization is crucial to help patients with ultra-rare diseases access advanced therapy medicinal products (ATMPs), including cell and gene therapies.

Due to very small patient populations (sometimes 100 to 200 people worldwide) and national-level idiosyncrasies, making an ATMP commercially available for an ultra-rare disease is not viable for pharmaceutical companies, which means patients can miss out on access due to regulatory hurdles.

As explained by Peter Marks, Director of the Center for Biologics Evaluation and Research at the United States Food and Drug Administration (FDA), “The problem is that if the sponsor has to negotiate different regulatory requirements in four different locations, that becomes untenable. We’re not talking about indications that have thousands of people, we’re talking about indications that have 100 to 200 people globally treated a year.”

For the drug regulators, regulatory harmonization would combine markets to make launching a drug more viable for companies. There is a need, therefore, to align and harmonize regulatory requirements for these products so ATMPs for ultra-rare conditions can reach patients who still benefit.

ATMPs should be the first area of focus for regulatory harmonization, conference attendees agreed.

Already, some drug regulators have teamed up for early conversations with companies—for example, the ACCESS Consortium and Project Orbis allows different drug regulators and companies to connect and align for simultaneous regulatory submissions, thereby accelerating time to access for patients.

Overall, however, full regulatory alignment, including using real-world evidence (RWE) and patient reported outcomes, needs stringent guidelines, and it’ll take some time for these to manifest.

Learn more about Pricentric ONE and our Global Pricing Solutions!

Contact us with your questions and global pricing needs, and an expert will follow up shortly.