Advanced Therapies: Stakeholders Call for EMA Changes on Regulations, Manufacturing

The European Medicines Agency (EMEA) on Friday issued a report with concerns and regulatory: modifications described by developers, manufacturers and investors in therapy products Medicines (Drugs Advanced Therapy), including gene therapies, products tissue engineering therapies and somatic cell, a UN meeting last week.

Note interpenetration What recurring themes include the need for early interaction with regulators and policy, a larger Executives Transparency larger Managers harmonization between Member States of the EU regulation ATMP, What and a proposal for Industry Being EMA calling more flexible converter Advanced Drug Reservations therapy manufacturing.

"While no decision has been bis prize, European Commission, national authorities and the EMEA and have a conversation Start From The feasibility of these proposals," said EMA.

Context

In the EU regulatory framework Verter Advanced Drug therapy was established by Regulation (EC) No 1394/2007, which has been in force since 2008.

Allows automatic regulation of the UN access to a central line for authorization of advanced therapy products in the EU and created the Committee for Advanced Therapies, which experience gathered and established, among other processes, classification procedures are Certification converter rescuer The early development of advanced therapy.

An EMA convened a meeting covering advanced therapy 27 May, 2016, attended ONT University and researchers, incubators and NGOs Consortium, and NGO representatives Patients and health care, small and large pharmaceutical companies, investors, Technology assessment Bodies health, national competent authorities (ANC) and the European Commission

Manufacturing Proposal

"A mayor proposal at the meeting was to licensing requirements reflect Vierten Special Unique Drugs for manufacturing Advanced Therapy," Specifies rapport. "For example, participants take into account the manufacturing ATMP can take place in different sitios and to pour certain products, further ethical Production may need to take place near the head Involvement What is all the sites concerned. - it including hospitals - would be needed to maintain the manufacturing license. "

However, with the lack of harmonization of the United Nations, only the level of the National Motor Vehicle some Member States allow hospitals hold a manufacturing license, interested parties "a request to the authorities to more flexible requirements pendant principles the years phases of maturation development. specific proposals for adaptation needs under gone up tone based products cells a included (no products handled significantly) could be If you take into account as falling on the border between the grafts and Drug Advanced therapy activate below.

"Similarly, more pragmatic approach should be used converter meet requirements Process Validation converter Many advanced therapy, given the difficulty to produce the required amount of batch conversion to CHARACTERISTICS pharmaceuticals," Specifies rapport.

Other proposals Promotion manufacturing technologies include (for example for manufacturing header and closed systems) and manufacturing models (for example, manufacturing decentralization), which would require more flexibility.

"Regulatory bodies could also stimulate the development of production sites, such as service, the use of small and medium enterprises (SMEs) and other funds, so that capacity is available converter multipart develop different advanced therapy medicinal" said the relationship. "These suggestions were considered timely, given the European Commission What is revising the Directive on GMP requirements Pour advanced therapy and will soon launch the public consultation."

GMO

Another important issue in the meeting focused on a genetically modified (GMO), which had a cool reception in the EU.

Interested parties have indicated that GMO directive (directive 2001/18 / EC) "are not designed specifically for your drugs and aggravated lacunar infarcts compared AEC is by discrepancies in the application in the Member States. From May Different requirements among States members, the integration of assessment of GMOs in the authorization of clinical trials of the United Nations is a challenge, especially in the context of multicenter clinical trials. "

Developers of these gene therapies have called for greater uniformity is suggested as a step premiere, the establishment of a central repository in Spanish, a list of requirements and deadlines for the assessment of GMOs in all Member States.

"Some speakers also called for: modifications directive has the OMG itself," says A good relationship. "Another area that could benefit from further harmonization concern cells and tissues as materials manufacturing EMPLEA premieres Drug Therapy Advanced with contradictory interested Applicant rational application over tissues and cells (Directive 2004/23 / EC) and other relevant laws" .

Other issues

Weighing also called for greater harmonization A "facilitate the movement of materials between the tiles with different requirements (inside and outside the EU) starting and reduce the burden of having a re-tester cells and tissue Before starting the manufacture of drugs . Advanced therapy the test puts itself can then focus on: aspects of the nature of the Advanced therapy Medicinal Products ".

The industry also asked for excipients A SPECIFIC guidelines and a system master file fluent Marketing authorization with respect to excipients and content premieres.

In addition, stakeholders have proposed new tools for Development (organelle eg transferor of extrapolation, modeling / simulation, biomarkers, etc.) to meet the needs of clinical no.

"On the question f risk-benefit ratio product development, stakeholders noted that the current practice is mainly focused on the risks and the call of the additional UN accent is put on the expected advantages, but realistic, particularly when patients have incurable diseases or treatments when appropriate default font "added the relationship.

"If advanced therapy should fulfill its promise to provide innovative treatments for patients, regulators should feed regulatory environment that the UN Promoting Innovation protect public health and, ultimately, the bill easily accessible when patients to appropriate new treatments" says interpenetration.