EMA lauds PRIME priority meds scheme and outlines potential revisions

Regulatory NewsRegulatory News | 08 March 2022 |  By 

Eighteen medicines for previously unmet needs are available due of the PRIME (PRIority MEdicines) scheme according to the EMA, which plans to optimise the programme to ensure developers get the support they need.
The EU regulator made the comments in a review published this week that looked at the impact of the scheme since its launch in 2016 (RELATED: EMA Looks to Accelerate New Therapies Targeting Unmet Needs, Regulatory Focus 07 March 2016).
Ten of the PRIME designated products covered by the scheme received a conditional marketing authorisation (CMA), meaning they were brought to market earlier than they otherwise would have been, according to the report.
In addition, seven of the products approved through PRIME were advanced therapy medicinal products (ATMPs), medicines deemed to have the potential to reshape the treatment of the condition they are designed to treat.
Enhanced communication
PRIME “has had a positive impact on the authorisation of new medicines that address patients’ unmet medical needs,” according to EMA. “PRIME medicines represent significant progress in their therapeutic areas. They include innovative technologies such as CAR T-cells therapies, one-time curative gene therapies, treatments for rare cancers and a vaccine to protect against the Ebola virus.”
The agency pointed to increased communication and information exchange between biopharmaceutical developers and regulators among the most positive impacts of the scheme.
“Enhanced interaction with EU regulators through PRIME is particularly useful for developers of more complex products and for applications that rely on smaller datasets. This includes for example ATMPs and orphan diseases, which often present new scientific and regulatory challenges,” EMA officials said in a statement.
According to the report, the higher level of interaction reduced so-called clock stop – the time developers need to answer questions asked by the EMA during the review process – resulting in faster reviews and earlier patient access.
Optimise PRIME
EMA will be looking at when to admit drugs to the PRIME scheme, as part of its efforts to improve the program, according to the report.
Products granted PRIME support must have the potential to address an unmet medical need. The challenge is determining when during development process this potential can be demonstrated according to the agency.
“Very few early entries in the scheme were granted in the first 5 years of the operation of PRIME, as it can be difficult at this stage to substantiate the promising nature of the medicine, partly due to the fact that few animal models with good predictability of efficacy in the human body exist,” the report states. “Nevertheless, later entry in the scheme limits the possibility of input (particularly in the CMC, dose finding and nonclinical areas), and the opportunity for global alignment.”
To address this, the agency plans to strengthen the need for applicants to outline the expected benefit from PRIME when seeking entry to the scheme and to make clear what types of support they will seek.
The EMA also said it will leverage experience gained from the orphan drug programme to inform on the suitability of nonclinical data to support PRIME applications earlier in the development process.
Patient input
Virginie Hivert, therapeutic director of EURODIS, a European rare-disease organization, suggested the EMA should involve patients in efforts to improve PRIME.
“Strengthening the involvement of patients in this process, as well as reinforcing the collaboration with [health technology assessment] bodies and payers, would allow for the generation of relevant data all along the medicines life cycle, for tackling even more efficiently the unmet medical needs and for PRIME to truly fulfil its potential,” she said in an interview with Focus. She added that even with PRIME, more needs to be done to improve access to medicines.
“An adequate path to access needs to be set up as PRIME alone doesn't automatically mean availability to patients across Europe. The case of Zynteglo is in this case emblematic of the shortcomings of a fragmented European healthcare system,” Hivert said.  
Zynteglo (betibeglogene autotemcel), bluebird bio’s gene therapy for patients 12 years and older with transfusion-dependent beta-thalassaemia, was one of the PRIME products granted marketing authorisation in the first five years of the scheme.
The product was launched in Europe in 2020. However, last summer bluebird said that as a result of challenges “achieving appropriate value recognition and market access for Zynteglo in Europe,” it had decided to focus on the US market, according to a statement.
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