Welcome to our new website! If this is the first time you are logging in on the new site, you will need to reset your password. Please contact us at firstname.lastname@example.org if you need assistance.
Your membership opens the door to free learning resources on demand. Check out the Member Knowledge Center for free webcasts, publications and online courses.
Communication Strategies. Case Studies. Applied Knowledge.
Hear from leaders around the globe as they share insights about their experiences and lessons learned throughout their certification journey.
Regulatory News | 07 March 2016 | By Michael Mezher
The European Medicines Agency (EMA) on Monday launched its new PRIME (PRIority MEdicines) scheme which aims to streamline the development of promising new therapies that meet unmet medical needs.
Like the breakthrough therapy program in the US, the new EMA program aims to accelerate the development of new drugs to treat rare cancers, Alzheimer's and other neurodegenerative diseases, AIDS and diabetes, among others.
"I think we still see too many patients in desperate need of … more effective treatments," EMA Executive Director Guido Rasi said at a press conference Monday morning in London.
Part of the issue, is that too many drugmakers' development plans are inadequate or fail to generate the data EMA is looking for. "As a consequence, many of these very promising medicines fail to make it to patients … this is particularly true for those developers that have less regulatory experience," Rasi said.
PRIME aims to address that problem by offering sponsors earlier, more tailored engagement with EMA.
Specifically, EMA is looking for relevant in vitro and in vivo data in appropriate preclinical models, with their relevance discussed preferably in the context of the use with other products known to be successfully developed for the condition. EMA also says, if available, established in vivo models for the condition should be used. And unless adequately justified, in vitro evidence alone will generally not be considered sufficient evidence to support eligibility to PRIME support.
Similarly, a new pharmacological target or mechanism of action will not, in and of itself, be viewed as sufficient to justify PRIME support, EMA says.
"Data available to support a request for eligibility in a given indication should support the claim that the product has the potential to bring a major therapeutic advantage to patients, through a clinically meaningful improvement of efficacy, such as having an impact on the prevention, onset or duration of the condition, or improving the morbidity or mortality of the disease," EMA says.
In "exceptional" cases, EMA says, academic and small- and medium-sized enterprises (SMEs) can apply to the program even earlier based on "compelling nonclinical data in a relevant model" and first-in-man studies to demonstrate tolerability.
Alongside today's launch, EMA released a final guideline, Enhanced Early Dialogue to Facilitate Accelerated Assessment of PRIority MEdicines (PRIME), as well as a questions and answers document on the program.
Unlike the agency's adaptive pathways pilot project, which is intended to speed access to medicines where traditional clinical trials are not feasible through either staggered or conditional approval, PRIME is intended to make the development of high priority drugs more efficient by providing early engagement and scientific advice.
When asked how much faster patients will get access to medicines via PRIME, Tomas Salmonson, CHMP chair said, "The big wins, we're convinced, are going to happen in the development phase," noting that while accelerated assessment can shave a couple months off a product's review, a more efficient development plan could save more time and reduce costly delays.
Companies must justify via epidemiological data about the disease (e.g., life expectancy, symptoms and duration, health-related quality of life) that the drug in question may address an unmet medical need and should explain the extent to which the medicinal product is expected to address that need.
"In order to access the breadth of regulatory support during the clinical stages of development, the potential promising activity of the medicinal product should be based on proof of concept in man to justify that clinical benefit can be expected," EMA says.
Applicants accepted to the program will have a rapporteur from EMA's Committee for Medicinal Products for Human Use (CHMP) or the Committee for Advanced Therapies (CAT) appointed at an earlier stage, which is a key feature of the new scheme, according to EMA.
EMA will also provide a kick-off meeting with the rapporteur, experts from other scientific committees and agency staff to provide guidance on a sponsor's drug development plan, discuss key development steps and make recommendations on regulatory strategy.
The agency will then dedicate a contact person within the agency to coordinate work throughout the scheme, and EMA says it will provide scientific advice on a company's overall drug development plan, with the possibility of involving other key stakeholders, such as health technology assessment (HTA) bodies and patients.
Finally, the agency says that medicines accepted to PRIME "are expected to benefit from the accelerated assessment procedure," which allows for a shorter review period once a drug is submitted for marketing authorization.
While formal confirmation of accelerated assessment won't be given until two or three months before a company submits its application for marketing authorization, the criteria a drug must meet to be accepted for PRIME are the same as for accelerated assessment.
EMA Press Release
PRIME Eligibility Requests
Tags: advice, Guido, Medicines, PRIME, Priority, Rapporteur, Rasi, Scientific