#EAPM – Time for change concerning rare diseases 



For those who have not yet departed for very well-earned summer hols, welcome to the week’s first update from the European Alliance for Personalised Medicine (EAPM), and we hope you had an enjoyable weekend – following on from the first virtual EAPM Global Conference, which, despite all associated problems of COVID-19 still managed to attract more than 480 delegates on 14 July, and for which a report will be available later this week, EAPM has also had an important multistakeholder authorship academic article published, writes EAPM Executive Director Denis Horgan. 

The title is: Time for change? The why, what and how of promoting innovation to tackle rare diseases – Is it time to update the EU’s Orphan Regulation? And if so, what should be changed?

The link to the full academic article is here

Orphan drugs need harmonization

As far as the article’s themes are concerned, it highlights the fact that, with global developments in the health-care arena, more should be done to align EU and other big markets’ regulatory practices for rare disease patients. Notwithstanding efforts and co-operation between the US and EU aimed to harmonize their strategic plans in the field of orphan drugs, regulatory criteria and procedures to gain the designation, terms and classifications should be still harmonized. Overall, the article argues, there is a need for joined-up regulatory process coordination, and better integration of regulatory pathways and better integration of regulatory systems, such as sci- entific tools and methods to generate evidence, would be helpful. 

EU summit deadlocked

And, if you are unhappy with the speed at which Monday has come round, spare a thought for the EU ministers and leaders who spent the past three days wrangling over the EU’s long-term budget and organizing an economic recovery fund. 

They went at it to the early hours of this morning (20 July), until just after 5h30, in fact, when European Council President Charles Michel told all parties that he will put forward a proposal for a recovery fund that includes €390 billion in grants. Leaders are set to reconvene at 16h this afternoon (20 July), so here’s hoping – European Central Bank President Christine Lagarde had this to say: “It’s better to agree on an ambitious facility along these lines, even if it takes a bit more time. I would hope that the leaders agree on something that’s ambitious rather than fast,” she told Reuters.

Parliament at work on youth unemployment

In light of the COVID-19 pandemic, the European Parliament published a press release on 15 July, tying into the new initiative proposed by the European Commission, consisting of a reinforced Youth Guarantee, improved vocational education and training, renewed impetus for apprenticeships and additional measures to support youth employment. Prior to the pandemic, EU youth unemployment (15-24) was 14.9%, down from its peak of 24.4% in 2013. By April 2020, it had risen to 15.7%.

Reshuffle planned to deal with economic recovery

European Commission President Ursula von der Leyen has plans for a reshuffle in the secretariat general so the Commission can more effectively deal with the economic recovery from the coronavirus crisis: She will set up a new recovery task force in the Berlaymont’s power centre.

EMA boss says drugs regulators ‘closer together’

Speaking to POLITICO, European Medicines Agency (EMA) chief Guido Rasi says that the competition among governments to secure access to medicines and future vaccines has brought drugs regulators them closer together. 

Rasi is responsible for recommending drug and vaccine approvals for all 27 EU members, and he said that a unified front among global regulators on the data necessary to approve new medicines or vaccines for the novel coronavirus will help them withstand political pressure at home, as governments compete to be the first to offer their citizens vaccines or treatments.

If everybody else has this position, it’s easier for one to stand and to explain that it’s not [only the individual agency’s position], it’s a global view,” he said. “In that perspective it’s giving a lot of strength to each one of us.”

Rasi also chairs the International Coalition of Medicines Regulatory Authorities (ICMRA), a voluntary cooperation between 28 drug agencies around the world, including the US Food and Drug Administration (FDA), China’s National Medical Products Administration and Brazil’s National Health Surveillance Agency.

Coronavirus mutations – more infectious?

The coronavirus that is now threatening the world is subtly different from the one that first emerged in China, according to BBC reports.  Sars-Cov-2, the official name of the virus that causes the disease Covid-19, and continues to blaze a path of destruction across the globe, is mutating.  But, while scientists have spotted thousands of mutations, or changes to the virus’s genetic material, only one has so far been singled out as possibly altering its behaviour.  

The crucial questions about this mutation are: does this make the virus more infectious – or lethal – in humans? And could it pose a threat to the success of a future vaccine? This coronavirus is actually changing very slowly compared with a virus-like flu. With relatively low levels of natural immunity in the population, no vaccine and few effective treatments, there’s no pressure on it to adapt. So far, it’s doing a good job of keeping itself in circulation as it is.  

The notable mutation – named D614G and situated within the protein making up the virus’s “spike” it uses to break into our cells – appeared sometime after the initial Wuhan outbreak, probably in Italy. It is now seen in as many as 97% of samples around the world.

European Health Union

COVID-19 has highlighted the need to give the EU a far stronger role in the area of health, MEPs say concerning the EU’s future public health strategy. In a resolution adopted on Friday (17 July) the European Parliament set out the principles of the EU’s future public health strategy post-COVID-19, underlining the need to draw the right lessons from the COVID-19 crisis and engage in far stronger co-operation in the area of health to create a European Health Union. 

This should include common minimum standards for quality health care, based on urgently needed stress tests of member states’ healthcare systems to identify weaknesses and verify that they are prepared for a possible resurgence of COVID-19. The resolution calls for a European Health Response Mechanism to be created rapidly to respond to all types of health crises through better coordination and management of the strategic reserve of medicines and medical equipment.  

The upcoming EU pharmaceutical strategy must contain measures to make essential medicines more immediately available in Europe, say MEPs. Diversified supply chains also need to be put in place to guarantee affordable access at all times. 

The new dedicated €9.4 billion EU4Health Programme is strongly welcomed and MEPs believe that long-term investments and commitments are needed. 

They request the establishment of a dedicated EU fund to improve hospital infrastructure and health services.  On top of that, the European health agencies ECDC and EMA, as well as joint health research, must be strengthened.

And that is everything for your week’s first update – do keep a lookout for the EAPM Global Conference report, which will be available later in the week, and stay safe until then. 

Here once more is the link to the full article, entitled again: Time for change? The why, what and how of promoting innovation to tackle rare diseases – Is it time to update the EU’s Orphan Regulation? And if so, what should be changed?’ 



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