Welcome one and all – this week’s first update from the European Alliance for Personalised Medicine (EAPM) brings you more news of the EU’s orphan drug legislation, which the European Commission has produced a 400-page review of, for those of you looking for distraction on your jolly hols, please see the following link, writes EAPM Executive Director Dens Horgan.
Orphan process
The EAPM has already published an article on the progress made on the Orphan Drug Regulation, take a look here.
The EU Orphan Regulation has been in place for 19 years – concerning the stock of available orphan medicines and the level of investment in R&D relevant to rare diseases, and since the Regulation went into effect, the level of activity to develop new treatments for rare diseases has markedly increased. By the end of 2017, 142 orphan medicines had been authorised. These cover a wide range of conditions and indications, including many very rare diseases. Based on estimates of disease prevalence and the population size in the EU, it is estimated that a maximum of 6.3 million people stand to benefit from these treatments.
In reality, though, orphan medicines target sub-sets of such patients, based on factors such as disease stage and severity, age, or presence of certain gene mutations. Therefore, the actual treatment population of patients at any time is only a portion of this.
A growing proportion of Europe’s pharmaceutical and biotechnology companies have become involved with rare disease research and the development of orphan medicines. The European Medicines Agency (EMA) has processed the applications of around 1,000 sponsors in the past 19 years. The public sector’s annual expenditure on rare disease research has increased across the period, from several tens of millions of euro in the late 1990s to several hundred millions of euro from 2018.
Coronavirus spike – new restrictions needed, says EU
Countries across Europe are introducing fresh restrictions to curb the spread of the coronavirus, while UK Chancellor Rishi Sunak has hinted that more destinations could be added to the UK’s quarantine list. Norwegians have been urged to avoid travel abroad, even to places with few Covid-19 cases, to prevent a resurgence. Norway added France and Switzerland to the list of countries from which arrivals must self-isolate due to rising infections – France recorded 1,604 cases, marking the first time since April that it saw more than 1,600 new infections on two consecutive days.
Meanwhile, Malta is banning mass gatherings and making face masks compulsory in public spaces; in parts of Marseille, France’s second largest city, face coverings will be mandatory outdoors and in Scotland, where Aberdeen is in local lockdown, pubs will be required to collect customers’ contact details. There are fears that both Malta (33 cases per 100,000) and France (23.4 per 100,000) could be added to the UK’s “red list”, following the addition of Belgium, the Bahamas and Andorra last Thursday (6 August).
The UK government will not hesitate to impose measures for more countries when required, according to Mr Sunak. And the EU’s infectious disease agency has warned that the bulk of the recent increase is attributable to relaxing containment measures. “There is a true resurgence in cases in several countries as a result of physical distancing measures being relaxed,” the agency said, recommending that countries reinstall or reinforce various measures in a “phased, step-wise and sustainable approach,” said Mike Catchpole, the European Centre for Disease Prevention and Control (ECDC)’s chief scientist.
Germany and France say ‘No’ to Trump on WHO overhaul
France and Germany have quit talks on reforming the World Health Organization in frustration at attempts by the United States to lead the negotiations, despite its decision to leave the WHO, three officials told Reuters. The move is a setback for President Donald Trump as Washington, which holds the rotating chair of the G7, had hoped to issue a common road map for a sweeping overhaul of the WHO in September, two months before the US presidential election.
The United States gave the WHO a year’s notice in July that it is leaving the UN agency – which was created to improve health globally – after Trump accused it of being too close to China and having mishandled the coronavirus pandemic. The WHO has dismissed his accusations. European governments have also criticized the WHO but do not go as far as the United States in their criticism, and the decision by Paris and Berlin to leave the talks follows tensions over what they say are Washington’s attempts to dominate the negotiations. “Nobody wants to be dragged into a reform process and getting an outline for it from a country which itself just left the WHO,” a senior European official involved in the talks said.
Commissioner Kyriakides: Coronavirus vaccine could be ready soon
A coronavirus vaccine could be ready to roll out this year or early next year, according to the EU’s health commissioner. Stella Kyriakides told German newspaper Handelsbatt: “Although making predictions is risky at this point, we have good indications that the first vaccine will be available toward the end of this year or beginning of next year.” The interview comes as dozens of research teams around the world race to try and develop a vaccine. But scientists have not yet established whether it will even be possible to create an effective vaccine against the coronavirus.
UK orders recall of coronavirus testing kits over safety standards
Randox Laboratories, a Northern Ireland-based medical technology company, has been instructed by the UK’s medicines regulator to recall up to 741,000 coronavirus test kits from the national test and trace programme as a precautionary measure. The government had on 15 July instructed the programme, run by the NHS, to stop using the kits, citing concerns that they may not meet required safety standards. “The Medicines and Healthcare products Regulatory Agency has instructed Randox to recall all Randox test kits from NHS Test and Trace testing settings,” the Department of Health and Social Care said in a statement on Friday (10 August).
UK deal to send corona vaccination doses to vulnerable countries
The UK government has signed deals for 100 million doses of promising coronavirus vaccines that are being developed for coronavirus ‘vulnerable’ countries. The vaccines are being researched by an alliance between the pharmaceutical companies BioNtech and Pfizer as well as the firmValneva. The new deal is on top of 100 million doses of the Oxford University vaccine being developed by AstraZeneca. However, it is still uncertain which of the experimental vaccines may work. A vaccine is widely seen as the best chance of getting everyone’s lives back to normal. Research is taking place at an unprecedented scale – the world became aware of coronavirus at the beginning of 2020, but already more than 20 vaccines are in clinical trials. Some can provoke an immune response, but none has yet been proven to protect against infection.
And that is all for now, EAPM hopes you are enjoying your holidays, wherever you are.
