The European Medicines Agency: A Modern Robin Hood?

Social injustice is increasingly discussed by sociology, gender studies, statistics, and other academic institutions. Economic and social inequalities also affect our health.^1-3

Credit: toeytoey - stock.adobe.com

You do not need to have studied medicine, pharmacology, or social sciences to know that a well-off person can afford better housing, clothing, nutrition, access to health care, and more. On the other hand, the higher your income, the higher the danger that “specialists” will talk you into expensive check-ups or treatments for existing or nonexisting health challenges. A balance and careful consideration of treatment options is always required.

Is our world really full of injustice and disadvantage? An important, but also complex question. Social injustice is as old as human civilization. In the Stone Age, there was little injustice because everyone was equally poor and most people were healthy. Those who got sick died quickly.

Many diseases of civilization did not yet exist, such as colonic cancer, high blood pressure, or obesity. Were people living during the Stone Age happier than we are today? That is another question—and we cannot go back.

Also, complaints about and recipes against injustice go back millennia. All higher religions based on written texts demand mercy, charity, and sharing with the poor. Also frequent are the sagas and fables of those who fight against injustice.

Robin Hood is a legendary heroic person who originated in English folklore. As a good swordsman and archer, he and his gang of noble-minded guerrilleros robbed the rich and gave to the poor.⁴ Who would not wish to be a modern-day form of Robin Hood, tackling and righting social injustices?

When politicians complain about injustices, they want to be elected.Scientists accusing social injustices work on their career. A sociologist who excels in complaints may get a tenured professorship at a university with a name in social injustice studies. Others have to make ends meet as journalists, bloggers, taxi or Uber drivers.

Criticism is the lifeblood of a living free society. Countries such as Russia or China officially know neither poverty nor inequality and anyone who dares to say such a thing could go to jail or disappear. On the other hand, we have to watch out for hidden interests in all critical voices.

Drug development and pediatrics experienced a tremendous upswing in the 20th century. Child mortality decreased and many rare diseases can now be cured or at least managed. Without the development of new vaccines, drugs, and devices, this progress would not have been possible.

In this surge of progress, researchers claim to have discovered a new injustice: children as a neglected population because many drugs were traditionally not approved for children.⁵ Pediatric studies were identified as a way to rectify this injustice. Because these were not interesting enough for the greedy pharmaceutical industry, ways to compensate it for the costs must be found, or they must be forced.

The United States started with the first, the European Union (EU) predominantly with the second option. Without a pediatric investigation plan (PIP) approved by the European Medicines Agency (EMA), the EMA will not even consider marketing approval applications fornew drugs.⁶ 

Children as a neglected population originated with the term “therapeutic orphans” in 1968,⁷ a claim that led in 1997 to US legislation that rewarded pediatric studies with patent extensions. The EU enacted an even more ambitious legislation.⁶

Its preamble outlines that the forces of the market have been unable to provide for the necessary pediatric studies—the pharmaceutical industry has refused to perform them. Therefore, the state, representated here by the regulatory authorities, must step in and make sure that industry commits to these studies to increase social justice and child health care.⁸

When physicians running a pediatric ward need new drugs or devices, they discuss and publish it. Also, parents with sick children have become more active. Whatever is developed to improve child health care must overcome many hurdles.

The mechanism for how children’s, pediatricians‘ and parents‘ needs are transformed into goods and offers of services is called "market." The EU pediatric regulation states that the forces of the market failed to provide the necessary studies for children's drugs and that the state must intervene.⁶

Is that true? US and EU pediatric legislation have several parts. For decades, many medicines were not available in formulations that were suitable for babies and small children, but only as tablets. Hospital pharmacists had to crush them and turn to reconstitute them as liquids.

This part of pediatric legislation makes the most sense, but the bulk of drug development costs are clinical trials. Here the EMA, with its PIPs, requires a complete repetition of the E&S studies on "children," defined as under 18 years of age, regardless of whether these young people are still physically children or not. Is there a medical need for these studies? Definitely not for adolescents.⁹ They are massively exaggerated for school-age children.

Children need correct dose recommendations, not large, international, randomized double-blind placebo-controlled trials. The seemingly noble fairy tale of the good EMA Robin Hood-like authorities who force the rich industry to do something good for children is just a fairy tale.

Bureaucrats would like to dictate many things that the market can regulate much better on its own. History has shown the danger of giving bureaucrats too much power. The EMA is now terrorizing the research-based life science industry for more than 15 years. This is something that on closer inspection are not studies that advance child health care, but studies in administratively-defined minors that are justified by pseudo-scientific claims, yet in most cases, have no clinical value and often even harm young patients.^10-12

For a robber to be portrayed as noble, the robbed individuals and institutions must be characterized as evil, which has been done extensively. The pharmaceutical industry is described as evil, greedy, and comparable to organized crime.^13-15

A 21st century division of labor: noble scientists justify the need for pediatric studies. Noble regulatory officers force the greedy industry to organize and pay these studies.

At present, the main obstacle to these studies is that there are not enough children worldwide for all the research specifically requested by the EMA.¹⁶ And that parents are increasingly asking themselves whether these studies are even necessary.^10-12 Institutional review boards and ethics committees have not yet started to systematically turn down submission for questionable studies in minors.¹⁷

The world is becoming more complex. Children with cancer used to just die, whereas today, we can cure many but not all of these diseases. The same is true with many rare diseases.

As the skills of medicine advance, mothballed myths and fairy tales about the alleged fight for more social justice for neglected children allow pointless studies.⁵ Today’s tobacco industry sells smoking as cool and as a sign of protest against mainstream conformity. In the past, the tobacco industry successfully used physicians as role models for advertising cigarettes, which thankfully no longer works.^18,19

But the EMA can still sell itself as noble, claiming it is enforcing "better medicines for children."²⁰ Whether we call this self-illusion, euphemism, or lying is a matter of taste. What the EMA’s PIP-enforced studies achieve is additional labels for minors, even if these are bodily already mature. In its 10-year report, the EMA claims to have made medicines “available” for children.²¹

Many anticancer drugs were available before 2007 and were used to save hundreds of thousands of children with cancer. Two EMA officers criticized that anticancer treatments “only” worked, but did not result in applications for marketing authorization to the authorities.²² A revealing statement—for them, drug approval is more important than improving health care and saving lives.

The “therapeutic orphans” were a good story as long as nobody looked deeper. The EMA's feeling as the noble enforcer of good studies is a self-illusion. Noble robbers such as Robin Hood exist only in heroic sagas.

Robin Hood was a good archer and swordsman. The EMA’s weapon is blocking the approval of new medicines. Calling this “Better Medicines for Children”²⁰ is cynical. The FDA and other national regulatory agencies should take a critical look at their close collaboration with the EMA.

Regulatory officers characterize “pediatric drug development” (PDD) as a worldwide ecosystem.²³ Ecosystems have inflows and outflows. The PDD ecosystem’s financial feeder is the life science industry, albeit involuntarily. Its beneficiaries are "pediatric" researchers and authorities who sell themselves as child-friendly, but not young patients. 

About the Authors

Earl B Ettienne, BScPharm, MBA, LPD, Assistant Dean of Graduate Programs and Industrial Partnerships, Howard University, College of Pharmacy, Washington, DC; and Klaus Rose, MD, MS, klausrose Consulting, Riehen (BS), Switzerland.

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