Regulatory support for development of orphan medicines: virtual workshop from the European Medicines Agency

A live online event from the European Medicines Agency on 30 November combined short talks with Q & A sessions and was intended to encourage early and efficient interactions with  regulators by highlighting pre-marketing support in medicine development for treating rare diseases.

The meeting aimed in particular to explain existing tools relevant to early product development strategy. 

These include:​

• orphan designation;
• protocol assistance;
• the PRIority MEdicines (PRIME) scheme.

Stakeholders were given an opportunity to express their views in relation to early medicine development and regulatory interactions. ​

The workshop was targeted at small to medium enterprise medicine developers, academia, patients, healthcare professionals and European Reference Networks (ERNs), often at the forefront of medicine development to treat rare and neglected diseases.

High level points include:

There are estimated to be 6-8000 orphan conditions of which around half concern diseases with an incidence of less than 1 in 10000.

183 orphan medical products have been registered in the EU since 2000. ~120 of these have active orphan status for treating ~130 orphan diseases ie several have indications for treating more than one disease.

Around 2 in 3 orphan products are based on conventional chemical treatments and 1 in 3 involve advanced therapeutic approaches.

The EMA supports SMEs and Academia in orphan drug development through a dedicated contact point, regulatory, administrative and procedural support and engaging with EU bodies and industry stakeholders.

Of the ~2000 SMEs registered with the EMA, 23% are working on orphan medical products.

A video recording will be available after the event on the EMA website.

Raising awareness to combat antimicrobial resistance: European Antibiotic Awareness Day

European Antibiotic Awareness Day EAAD was held on 18 November 2020 with activities taking place during that week. As in previous years, the European Medicines Agency (EMA) actively contributed to raising awareness about Antimicrobial Resistance (AMR) and how to limit it by publishing a set of infocards, developed to reinforce global cooperation to combat AMR.

EAAD is an annual European Union initiative to raise awareness of antimicrobial resistance as an increasing global public health threat and to promote the prudent use of antibiotics.

‘Tackling antimicrobial resistance is a global challenge and a high priority for EMA and the European medicines regulatory network. We all have a role to play in addressing this challenge: regulators, governments, companies through development of new innovative antibiotics, but also healthcare professionals and patients by making sure they prescribe and use antibiotics wisely and responsibly’ said Emer Cooke, EMA’s Executive Director.

To mark EAAD, the EMA launched a social media campaign to highlight the importance of using antibiotics prudently.

A set of info-cards focuses on what patients, healthcare professionals, veterinarians, health-leaders, journalists and the pharmaceutical industry in their respective roles can do on a daily basis to make sure that these important medicines are used wisely.

Antimicrobial resistance occurs when bacteria and other microorganisms change over time and develop resistance to medicines. As a result, antibiotics and other antimicrobial medicines become less effective. This makes infections harder and harder to treat. A prudent use of antibiotics helps slow the process and ensure that treatments continue to be available to treat common and emerging infectious diseases.

For more information on the EMA’s role in the global response to antimicrobial resistance, see Antimicrobial resistance.

You can see examples of the cards below. The full set of these infocards is downloadable on this link. 

 

 

European Medicines Agency to hold a public update on COVID-19 vaccines on 11 December

EMA will organise a public meeting on 11 December 2020 to inform European citizens about the EU regulatory processes for the approval of COVID-19 vaccines and the Agency’s role in their development, evaluation, approval and safety monitoring.

The COVID-19 pandemic is an unprecedented global crisis, with devastating health, social and economic impact. Safe and effective vaccines for COVID-19 are urgently needed to respond to this crisis and reduce its effects.

Together with the European medicines regulatory network, EMA is supporting the development of COVID-19 vaccines and has taken steps to speed up the evaluation processes for these vaccines. The Agency and its partners are doing so while ensuring that the same high regulatory standards for quality, safety and efficacy are being applied to COVID-19 vaccines.

The public meeting will inform citizens about EMA’s role in the pandemic and of EU regulatory procedures. It will also give the opportunity to the public and stakeholder groups to speak and share their needs, expectations and any concerns, that will be considered by EMA and the European medicines regulatory network in the decision-making process.

The EMA will publish the list of speakers who will make an intervention in advance of the event.

The public meeting will be broadcast live and will be open to all citizens. In addition to the selected list of speakers, during the meeting there will be a mailbox open via EMA’s website for the public to make comments.

The agenda and all information related to the event are available on the EMA website event page.

Please check EMA’s dedicated webpage on COVID-19 for the latest updates.