Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news.
Commission Creates Roadmap for Evaluation of Pediatric Rare Disease Legislation
The European Commission has created a roadmap for evaluating legislation covering pediatric rare diseases. Officials are embarking on the nearly two-year evaluation to understand why regulations on pediatric and orphan medicines have failed to translate into the hoped-for medical advances.
Following on from the Commission Report on the Paediatric Regulation, officials want to assess whether the two pieces of law have met their objectives. That will entail looking at the strengths and weakness of the legislation, both separately and in combination, in terms of their effect on the development of treatments for serious unmet medical needs and other metrics.
Those metrics include the ways in which the two pieces of legislation have met patients’ needs, their societal consequences and the extent to which they work synergistically.
The Commission will also tackle the incentives and economics of the legal instruments. Officials want to know how drug developers have used the incentives and the financial consequences of these actions, both in general and on a per-stakeholder basis. The stakeholder-specific cost-benefit analyses will cover patients, industry, payers and other groups.
“The evaluation will give a sound evidence base about the functioning of the two legal instruments from a public health and a socioeconomic perspective that will be used to consider the possible need for any future changes,” the Commission wrote.
Officials also plan to factor relevant global developments, particularly in the United States, into their analysis.
A 12-week public consultation is due to start in the third quarter of next year, with a stakeholder meeting organized by the Commission scheduled to follow in the first quarter of 2019. Once the consultation is complete, officials will publish a summary of their findings. The whole process is due to end in the third quarter of 2019.
Rasi: This is the Most Critical Moment for EMA in its Entire 22-year History
Guido Rasi has said the European Medicines Agency (EMA) is facing the most critical moment since it was founded in 1995. The EMA executive director made the comment during a speech in which he detailed the fallout from the Brexit vote and the agency’s forced relocation from London.
Rasi told the European Parliament's Committee on Environment, Public Health and Food Safety (ENVI) Brexit has already “put tremendous strain” on EMA, but the effects have largely been hidden from view. However, with EMA now facing relocation-related staff losses at a time when it has reallocated other employees to the management of the move, Rasi thinks the cracks will start to show eventually.
“We must all be very prepared,” Rasi said. “In future, the impact on the quality, on the efficiency of our activities unfortunately will become visible.”
So far, the secondment of 40 employees to work exclusively on Brexit has forced EMA to reduce its training programs, delay IT upgrades and “severely cut” meetings with stakeholders and partners, Rasi said. This period has been something of a phony war, though. The real difficulties will come now EMA needs to undertake a time-pressured relocation while losing staff who choose to remain in London rather than follow the agency to Amsterdam.
A lot rests on how this process goes. If EMA is put under particular strain, Rasi thinks it may have to postpone or slow down the implementation of the incoming medical device legislation.
EMA’s survey of staff suggests 81% are willing to move to Amsterdam, but Rasi views this as a best case scenario. Losing one-fifth of its staff would strain EMA in the best of circumstances, but the situation is complicated by the fact the agency is yet to learn which employees will stay in London. Without this knowledge, EMA lacks insights into which of its units will face a shortfall of expertise, hindering its ability to make contingency plans.
The situation will become clearer as EMA gets closer to its March 2019 relocation date. Initially, EMA will move into a temporary home in Amsterdam, the identity of which is yet to be finalized. That will give EMA and Dutch authorities time to finish work on EMA’s new permanent location. Rasi said he is “rather optimistic” EMA will have a place to move to when it leaves London, despite what he sees as “an ambitious timetable” for the relocation.
EMA will leave behind a €400 million ($470 million)-plus rent bill on its London office. It is unclear who will pay the bill. The United Kingdom and European Union reached an agreement on the size of the Brexit divorce bill this week, but the relocation fee appears to be outside the scope of its terms.
Asked to name a concession the EU had made to the UK, the EU’s chief negotiator Michel Barnier said it is not “at this stage insisting the UK should repay the removal costs” for EMA and the banking agency, Guardian journalist Jennifer Rankin reported.
Meeting Replay, Rankin Tweet
Hard Brexit Could Cost Big Pharma Companies $135M in Year One: Report
A health economics nonprofit has calculated a hard Brexit could cost companies as much as $135 million. That figure is based on what it will cost a large, US-based drugmaker to perform Brexit-triggered tasks such as the transfer of marketing authorizations and batch release facilities.
The company in the Office of Health Economics’ (OHE) example, the details of which are based on a real business the nonprofit interviewed, has about 22,500 employees in Europe, 10% of whom work in the UK. The UK is a major European market for the company, but most of its European manufacturing footprint is based elsewhere in the region.
OHE estimates the company will spend $28 million transferring its marketing authorizations to an EU group. The other big implementation cost is the creation of an EU-based unit for distributing clinical trial supplies. That is forecast to cost $26 million. OHE predicts the company will spend a further $11 million making packaging changes and $9 million moving its batch testing methods and facilities out of the UK.
Those figures cover implementation activities. The company will also be on the hook for ongoing maintenance costs, most notably the testing of finished products imported into the UK from the rest of Europe. OHE forecasts those tests will cost the company $23 million a year.
The predictions are predicated on the assumption the UK leaves the EU without agreements on trade or mutual recognition. OHE also runs the numbers for three other scenarios, under which the company's year-one Brexit bill is predicted to range from a negligible amount up to $97 million.
Some of these costs, such as those tied to the transfer of marketing authorizations, are unique to large organizations. OHE’s other case studies show smaller businesses will face expenditures, too, and that these do not necessarily scale with company size.
“The implementation cost of creating batch release facilities and transfer batch testing methods for a standalone regulatory country has been estimated to be around $20 million regardless of the size of the company,” OHE wrote.
OHE Report, Technical Annex
EMA has adopted its first guidance on the use of monoclonal antibodies in animals. The guidance is a question and answer document. Press Release, Final Q&A