The Daily Mail print edition today (22 December) runs a piece headlined “Children denied cancer drugs due to EU delays.”
There is no doubt that the issue of extending drug trials to children is a real one and that campaigners have written to Commission President Jean-Claude Juncker urging further strengthening of EU rules requiring drug companies to extend trials to children. The Commission is, of course, continuously listening to stakeholders and is determined to do everything possible to help give patients access to safe and effective drugs.
The Daily Mail is quite right to pay attention to this matter and to report the views of campaigners, so this is not a typical Euromyth entry refuting entirely or largely untrue and misleading stories.
However, the Mail does not make clear that these EU rules are already the strongest in the world and have led to significant improvements compared to the previous situation. Neither does the piece illustrate that this is a complex medical, ethical and practical issue with no easy solution. Just to highlight one of those aspects, as one reader responds to the Mail Online: “Imagine if a child died as a result of a clinical trials…”
As the Mail does, to its credit, recognise, this is a cross-border and global issue, and the UK will, like any other single country, not be in a position post-Brexit to compel drug companies to act differently, simply by acting alone.
Here is a statement we sent to the newspaper, now partially reflected in the online piece.
“EU rules certainly do not prevent pharmaceutical companies from carrying out research into using new drugs to treat children. The rules require companies to do so unless they can justify otherwise. This has been a major factor in the recent increase in the number of new medicines being authorised for children: 260 have been given the go-ahead in the last ten years.
This is a highly complex area, where regulators – not just in the EU, where rules are already stronger than other jurisdictions – must proceed with caution in order to maximise the availability of drugs for children while avoiding harming patients, discouraging research, or inducing companies to move it to other jurisdictions where there are fewer requirements.
Together with the European Medicines Agency (EMA), the Commission is acting to streamline how the Paediatric Regulation works. This includes analysing the use of deferrals, looking at ways to ensure speedier completion of PIPs, reviewing guidelines for handling PIP applications and stepping up efforts to foster international cooperation and harmonisation of rules. Together with the EMA, the Commission intends to bring together all stakeholders, including patients’ organisations, to establish an open and transparent dialogue on paediatric needs.
Before proposing regulatory changes to further improve the situation – those would need to be agreed by Member States and the European Parliament – the Commission needs to further scrutinise, along with regulators and stakeholders, how best to combine the effects of the Paediatric Regulation with the Orphans Regulation (covering rare diseases) in order to benefit as many patients as possible.
The EU Paediatric Regulation came into force in 2007 – it was the first ever in the world to force companies to engage in a specific type of medical research. The Commission’s report on the ten years of implementing the Regulation shows that over 260 new medicines for use with children have been authorised. Over 1000 Paediatric Investigation Plans (PIPs) – the first step in developing medicines for children – have been agreed. There is a clear upward trend in the number of completed PIPs.
PIPs may be waived under specific circumstances to avoid unnecessary, or even unethical research. For example, if a treatment is likely to be ineffective or unsafe or will not provide a significant therapeutic benefit over other existing treatments.
The European Medicines Agency reviewed the waiver decision in 2015 and limited its scope, so those companies that want a waiver must justify it to the Paediatric Committee. This forces companies to contact the committee at an early stage of development, to discuss the paediatric potential and seek certainty about requirements. The three year transitional period is not over so the effects of the class waiver review cannot yet be adequately evaluated.
The Commission report recognises that the least progress is indeed being made in diseases that are biologically different in adults and children, or which only affect children. This is often the case with rare diseases, including childhood cancers.
Complementing the Paediatric Regulation, “Orphans” legislation aims to increase the treatment options available for rare diseases, including rare cancers in children. It sets out other incentives such as a 10 year market exclusivity or fee waivers for the regulatory procedure.”
This issue has been covered previously on this site here.