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European Regulatory Roundup: EMA to Open Secure Portal for Orphan Drug Designations

Posted 14 June 2018 | By Nick Paul Taylor 

European Regulatory Roundup: EMA to Open Secure Portal for Orphan Drug Designations

Welcome to our EU Regulatory Roundup, our weekly overview of the top regulatory news in Europe.
EMA to Open Secure Portal for Orphan Drug Designation Filings, Management
The European Medicines Agency (EMA) is launching an online portal for orphan drug designations next week. EMA wants sponsors to start using the portal from 19 June in anticipation of it becoming mandatory three months from now.
Once live, the portal will provide sponsors with a new way to file for orphan drug status and manage their existing designations. EMA thinks creating a single platform for the submission of, and access to, orphan drug designation data and documents will result in quicker, easier access to information and improved data quality. Sponsors will also receive automatic status updates.
Sponsors will need to invest some time upfront to realize these benefits. EMA thinks sponsors will be able to navigate the system after reviewing a simple guidance document it will release next week, but first they will need to register as portal users.
EMA is phasing in the use of the portal over the next three months, over which time sponsors can continue to apply for orphan drug designations via existing processes. To ensure the portal is ready for the September end to the transition, EMA is gathering feedback from 26 organizations through a user acceptance testing program. The initiative will source feedback on the guidance and user interface.
The rollout of the portal is part of an EMA-wide program to improve its interaction with industry and management of master data, the reusable, non-transactional information that is key to the core operation of businesses.
EMA Notice
Ireland Rejects Industry Request for Further Medical Device Fee Reductions
The Health Products Regulatory Authority (HPRA) of Ireland has rejected industry requests to further cut its medical device fees. HPRA said it is unable to improve on the proposed 20% to 75% reductions over the much-contested 2017 fees.
Ireland introduced medical device regulatory fees for the first time in 2017. Despite gathering feedback before adopting the fee structure, HPRA received criticism, particularly from small companies. Armed with this feedback and a clearer picture of the composition of the Irish medical device industry, HPRA proposed to slash most of its fees in a consultation document last month.
Having reviewed feedback from two trade groups and 22 medical device companies, HPRA has learned some businesses see the cuts as insufficient. These businesses view the 2018 fees as excessive. Some companies want to see HPRA’s fees benchmarked against other European member states. Other commenters want a lower fee for virtual manufacturers and micro enterprises.
HPRA, while acknowledging the demands, is sticking to the fee structure it proposed in May. That reflects a belief that the amount recouped from the proposed fees will already be too little to fully cover HPRA’s medical device costs. These costs are set to rise as HPRA implements the European Union medical device rules, adopts an IT system and prepares for Brexit.
While HPRA rejected the prospect of across-the-board changes to the fee structure, it is willing to talk to companies with specific problems related to the charges. The feedback suggests some companies are worried about receiving penalty fees as a result of their use of an operating model not anticipated by HPRA’s fee structure.
HPRA will submit the proposed fee structure to the Minister of Health for approval.
HPRA Response
EMA Holds Public Hearing on Risks and Benefits of Quinolone and Fluoroquinolone
EMA has held its second public hearing on the risks and benefits of medicines. The half-day event attracted 55 requests to speak about  quinolones and fluoroquinolones, which EMA whittled down to 23 contributors.
In total, 115 people applied to attend the meeting. A little under half of the requests came from patients, healthcare professionals, academics and other people with experience of quinolones and fluoroquinolones who wanted to speak at the event. EMA ultimately decided to give five minutes to 23 of the applicants, which came from 11 countries.
EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) held the meeting to support its probe into the safety of quinolones and fluoroquinolones, antibiotics linked to adverse reactions that affect the muscles, joints and nervous system. PRAC heard directly from patients suffering from these side effects, giving it an insight into their lives and interactions with the healthcare system.
“More than anything we need to restore the chain of trust,” PRAC chair June Raine saId in her closing remarks. “It has been a repeated message we’ve heard that one of the hardest things to accept with this set of adverse reactions is a lack of recognition that the patient is right and is telling something that needs to be listened to and not dismissed. Let’s commit to playing our part in restoring the chain of trust.”
Those concerns are in line with those expressed by women affected by complications related to vaginal mesh implants, many of whom criticized healthcare professionals and regulators for failing to take their problems seriously.
Hearing Video
Dutch MEB Revises Policy on Sharing Risk Minimization Measures with Healthcare Professionals
The Dutch Medicines Evaluation Board (MEB) has revised its policy on the direct communication of risk minimization measures to healthcare professionals. MEB sees the changes cutting the risk that a healthcare professional will inadvertently overlook a risk minimization notice.
Dutch policy already called for the use of an orange hand symbol on the envelopes of particularly important or urgent risk minimization notices, such as those about new contraindications. However, there was no means to differentiate more routine risk minimization notices from other types of mail.
To ensure all risk minimization notices are easily identifiable, MEB has introduced a white hand logo. Routine risk minimization notices will carry the white hand, while urgent updates will continue to display the orange hand.
MEB introduced the changes alongside other revisions to its policies. The changes allow one marketing authorization holder to act on behalf of other providers of the same medicine. MEB has also added information about the obligations of parallel distributors and amended the template for materials aimed at healthcare professionals.
The new policy came into force on 11 June, but does not apply to ongoing procedures.
MEB Notice, More (Dutch)
Council of Europe Starts Construction of Pharmaceutical Reference Standard Building
The Council of Europe (CoE) has begun construction of a secondary site for the European Directorate for the Quality of Medicines and HealthCare (EDQM). CoE will use the site in Metz, France to store its portfolio of more than 3,000 pharmaceutical reference standards.
EDQM already operates out of a site in Strasbourg, where it stores its reference standards. Opening a second site will increase EDQM’s capacity to store reference standards and medicine ingredients. As importantly, it will provide a backup source of reference standards in the event of problems at the primary facility in Strasbourg. The reference standards support the batch release of medicines.
“EDQM currently has the capacity to produce 1.6 million vials per year and these reference standards are shipped to over 100 countries worldwide every day. The new EDQM site ... meets this need, by enabling us to create a secondary stock so as to ensure an uninterrupted service in the event that the main building in Strasbourg should suffer a major incident,” a CoE representative said.
To ensure the site fulfills this function, the building will have areas dedicated to logistics, informatics and technical matters. The building will store around 12 million vials at temperatures ranging from +5°C to -80°C.
Press Release, Inauguration Speech


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