Sunday

27th May 2018

Feature

Life-saving drug hits eurocrat wall - a Kafkaesque tale

  • EU commission headquarters: a court decision on the case is due later this year (Photo: ec.europa.eu)

A French medical laboratory and the European Commission are currently locked in a furious legal battle - at stake is whether a handful of EU civil servants, through use of byzantine rules, can defy medical opinion and keep a life-saving drug off the market.

The battle has been going on for three years.

Dear EUobserver reader

Subscribe now for unrestricted access to EUobserver.

Sign up for 30 days' free trial, no obligation. Full subscription only 15 € / month or 150 € / year.

  1. Unlimited access on desktop and mobile
  2. All premium articles, analysis, commentary and investigations
  3. EUobserver archives

EUobserver is the only independent news media covering EU affairs in Brussels and all 28 member states.

♡ We value your support.

If you already have an account click here to login.

The eventual court judgement will directly affect patients with a very rare genetic disease. More broadly, it will define the boundaries within which commission officials can play around with internal rules to achieve a result they alone want.

The dispute concerns whether to give a drug called Orphacol an EU-wide market authorisation.

The drug is used to treat two forms of liver disease - 3 Beta HSD deficiency and Delta 4-3-OxoR deficiency - so rare that they do not have colloquial names. There are an estimated 90 patients at most in Europe. Ophacol's positive effects are undisputed.

"Patients basically go from being fatally ill with the only therapeutic option being a liver transplant to being completely normal human beings with no signs of the disease," says Andreas Vogel, regulatory director at CTRS, the small French laboratory that made the EU market application.

The story goes back 20 years. In 1993 it was discovered that cholic acid, a type of bile acid produced by the liver, helped to combat the disease. Without treatment the condition is fatal. In 2007, Paris's public hospital system, which discovered and subsequently managed the medicine, decided to hand over the rights to the CTRS lab.

CTRS, run by Antoine Ferry, gave the medicine the name Orphacol and obtained a special licence from the French medical agency. Orphacol is now being used to treat 22 patients in France - 17 French, two Swiss, one Italian and one Portuguese.

In 2009, Ferry decided to apply for EU-wide market authorisation. Just over a year later, the European Medicines Agency (EMA) in London voted unanimously in favour of the idea.

Power to actually authorise a product for the EU market lies with the European Commission. It is normally easier to get regulatory approval for a so-called orphan drug, which treats rare conditions. And it was among the rarest diseases ever dealt with by the EMA.

The legal odyssey

Yet here is where CTRS' as-of-yet fruitless odyssey began.

To date the only result Ferry and Vogel have to show for their efforts is an impressive grasp of comitology - how legal decisions are made at committee level in the European Union, a process reminiscent of the surreal stories of Czech writer Franz Kafka.

The European Commission, and specifically the human-use drugs unit in the commission's health directorate, asked the EMA to better justify its positive stance. The agency in 2011 confirmed its original opinion and emphasised that Orphacol is of distinct public interest.

A commission decision should have come around two months later. But the commission was still not happy.

The French lab lacked clinical trial-based evidence, it said. And, continuing to cite chapter and verse of the rules, it argued the company was therefore not entitled to apply for market authorisation under exceptional services.

Clinical evidence, says CTRS, is impossible given the degenerative nature of the disease.

"A controlled clinical trial would have meant taking some people off the medicine." says Vogel. "The alternative - comparing a new patient before and after treatment - is not feasible because there are so few patients. In December 2012 we identified the first new patient in France since 2007. So you can't plan for a clinical study."

Legal uncertainty

In July 2011, the commission decided to reject the authorisation.

Member states' experts disagreed and voted down the commission decision. But the commission was not deterred. It appealed the decision. The appeal committee, again with member states' experts, rejected the commission's position once again.

With the second rejection, the whole process moved into uncertain legal territory.

The rules are unclear at this stage, saying only that the commission cannot adopt its original decision - the rejection of market authorisation of Orphacol.

For a while nothing happened. The French laboratory pressed for a decision. But the then health commissioner John Dalli wrote back to say that the commission will neither approve or disprove market authorisation, creating an unhelpful status quo.

Ferry then decided, in January 2012, to bring the commission to court for failure to act.

A hearing was due in April but at that point the commission sent a third proposal for refusal to member states.

This time, the meeting of member state experts fell on a public holiday. Some member states were missing and the required qualified majority needed to reject the commission's latest rejection was not reached.

Finally the commission had the result it wanted - it rapidly adopted a decision to reject market authorisation for Orphacol.

The highly unusual manoeuvre - coming in the midst of legal proceedings at the EU court in Luxembourg - also meant that the failure-to-act premise of Ferry's case collapsed. The judges, irritated by the commission's antics, ordered the Brussels bureaucracy to pay for CTRS' costs.

Member states alarmed

The French laboratory is now in the process of taking the commission to court again.

This time they have the support of a handful of member states, alarmed at what they see a precedent-setting case for abuse of power by EU civil servants. They want legal clarity.

"We are taking the commission to court on the merits of the case and on the comitology procedure," says CTRS' Antoine Ferry. "We want to know if it is possible to reintroduce a draft decision as often as the commission wants until it gets the decision it wants."

For its part, the commission argues that it cannot loosely interpret market authorisation rules for one product as this would set an example for the future.

"This case is not about a formalistic interpretation of the law disconnected from public health," says Frederic Vincent, the commission's health spokesperson.

"It is the first time that such an application is ever submitted and the commission considers that lowering the requirements is not in the interest of public health."

A court decision is due later this year.

MEPs back law favouring child medicines

The European Parliament on Wednesday voted in favour of a new regulation on medicine, obliging pharmaceutical companies producing a drug for adults in the EU to test whether it can also be used for children.

Feature

Resetting the gender balance through football

Many sports, like football, have been so heavily male-dominated at every level that women and girls have battled against poor odds to be treated as equals in the game. FIFA aims to change that.

EU takes step closer to 'posted workers' deal

Negotiators from the member states, EU Parliament and Commission reached a 'common understanding' to guarantee equal pay for equal work in the EU. They hope to reach a final agreement in June.

Opinion

Paying a high cost: EU's role in Spain's painful health cuts

The EU should either conduct, or ask states to conduct, human rights impact assessments of country-specific recommendations to ensure that the implementation of austerity measures does not result in reduced human rights protections.

News in Brief

  1. Italy set to pick eurosceptic finance minister
  2. UK foreign minister fooled by Russian pranksters
  3. Rajoy ally gets 33 years in jail for corruption
  4. Close race as polls open in Irish abortion referendum
  5. Gazprom accepts EU conditions on gas supplies
  6. Facebook tells MEPs: non-users are not profiled
  7. Commission proposes ending France deficit procedure
  8. UK households hit with Brexit income loss

Stakeholders' Highlights

  1. Counter BalanceEuropean Ombudsman requests more lending transparency from European Investment Bank
  2. Nordic Council of MinistersOECD Report: Gender Equality Boosts GDP Growth in Nordic Region
  3. Centre Maurits Coppieters“Peace and reconciliation is a process that takes decades” Dr. Anthony Soares on #Brexit and Northern Ireland
  4. Mission of China to the EUMEPs Positive on China’s New Measures of Opening Up
  5. Macedonian Human Rights MovementOld White Men are Destroying Macedonia by Romanticizing Greece
  6. Counter BalanceControversial EIB-Backed Project Under Fire at European Parliament
  7. Nordic Council of MinistersIncome Inequality Increasing in Nordic Countries
  8. European Jewish CongressEU Leaders to Cease Contact with Mahmoud Abbas Until He Apologizes for Antisemitic Comments
  9. International Partnership for Human RightsAnnual Report celebrates organization’s tenth anniversary
  10. Nordic Council of MinistersNordic Cooperation Needed on Green Exports and Funding
  11. Mission of China to the EUPremier Li Confirms China Will Continue to Open Up
  12. European Jewish CongressCalls on Brussels University to Revoke Decision to Honour Ken Loach

Stakeholders' Highlights

  1. Sustainable Energy Week 2018"Lead the Clean Energy Transition"- Register and Join Us in Brussels from 5 to 7 May
  2. EU Green Week 2018Green Cities for a Greener Future. Join the Debate in Brussels from 22 to 24 May
  3. Nordic Council of Ministers12 Recommendations for Nordic Leadership on Climate and Environment
  4. Macedonian Human Rights MovementOxford Professor Calls for an End to the Anti-Macedonian Name Negotiations
  5. ACCAPeople Who Speak-Up Should Feel Safe to Do So
  6. Mission of China to the EUProgress on China-EU Cooperation
  7. Nordic Council of MinistersWorld's Energy Ministers to Meet in Oresund in May to Discuss Green Energy
  8. ILGA EuropeParabéns! Portugal Votes to Respect the Rights of Trans and Intersex People
  9. Mission of China to the EUJobs, Energy, Steel: Government Work Report Sets China's Targets
  10. European Jewish CongressKantor Center Annual Report on Antisemitism Worldwide - The Year the Mask Came Off
  11. UNICEFCalls for the Protection of Children in the Gaza Strip
  12. Mission of China to the EUForeign Minister Wang Yi Highlights Importance of China-EU Relations

Stakeholders' Highlights

  1. Nordic Council of MinistersImmigration and Integration in the Nordic Region - Getting the Facts Straight
  2. Macedonian Human Rights MovementMacedonians in Bulgaria Demand to End the Anti-Macedonian Name Negotiations
  3. Counter BalanceThe EIB Needs to Lead by Example on Tax Justice
  4. ILGA EuropeTrans People in Sweden to be Paid Compensation for Forced Sterilisation
  5. International Partnership for Human RightsThe Danger of Standing Up for Justice and Rights in Central Asia
  6. Mission of China to the EUChina and EU Must Work Together to Promote Global Steel Sector
  7. Swedish EnterprisesEU Tax Proposal on Digital Services Causes Concern for Small Exporting Economies
  8. European Jewish CongressCondemns the Horrific Murder of Holocaust Survivor Mireille Knoll in Paris
  9. Mission of China to the EUAn Open China Will Foster a World-Class Business Environment
  10. ECR GroupAn Opportunity to Help Shape a Better Future for Europe
  11. Counter BalanceControversial Turkish Azerbaijani Gas Pipeline Gets Major EU Loan
  12. World VisionSyria’s Children ‘At Risk of Never Fully Recovering', New Study Finds