If adopted, the legislation will bring in a number of new measures designed to ensure that medicines used in children are properly tested. As part of the overall market authorization process for a new medicine or a line extension, pharma companies will have to supply clinical trials results in children. A waiver can be granted if it's a medicine that's clearly not intended for pediatric use, or a deferral if it is deemed safer to get more data in adults first (and doing pediatric testing would delay adult approval).
In return, the company will receive a six-month extension to the drug's supplementary protection certificate (SPC); commercial rights on orphan drugs for kids will be extended from 10 to 12 years.The EU will also provide funding for the Medicines Investigation for the Children of Europe research program, providing cash for non-commercial scientific institutions to carry out investigations. A network of researchers and centers will be set up, under the supervision of the European Medicines Agency, to prevent duplication of efforts.
There is also a proposal to set up a pediatric committee to oversee the R&D system for children's medicines. The proposal allows for penalties to be imposed on companies if they do not comply with the requirements for pediatric testing.
The impact of the regulation will be assessed six years after it goes into effect, to see if it has indeed improved the availability of properly tested children's medicines. This will include an investigation into whether industry's rewards are in line with the investments they have made in the testing process.
Some of the discussion within the Environment Committee was about whether the duration of additional protection should be made variable. Suggestions included that the length of time be proportional to the profits earned, making it longer in some cases and shorter in others. However, the overall feeling was that this would introduce insurmountable difficulties in implementation. The simpler original proposal of a standard six months was agreed on despite its initial rejection.
Perhaps the most important—and most argued—part of the proposed legislation is the extension of exclusivity. "The [fixed] six months [extension] follows the principle of patent law, giving a predictable return on investment," said Peter Arlett of the European Commission's pharmaceuticals unit. "It is simple to operate. The proposed amendments had not been thought through in a technical way, and would not work."
Currently, the patent and SPC give up to 15 years of protection for a medicine. The European Commission estimates that the extra six months would increase profits by between €800,000 ($990,358) and €9 ($11.2) million, compared with the cost of a clinical trial, which can be as much as €4 ($4.9) million.
The European pharma industry trade association EFPIA has broadly welcomed the agreement, and is particularly pleased that the straight six-month extension has been imposed, as is the case in the United States.
"It is very important to get the pediatric legislation through [Parliament] with the six-month patent extension," said Franz Humer, EFPIA president and CEO of Roche. "If that does not happen—five years after the US—it will send a [negative] signal to industry."
The generics manufacturers, on the other hand, claim the move will cost the EU healthcare system as much as €2 ($2.4) billion a year. Of particular concern to generic manufacturers is the Environment Committee's decision to reduce the period between the end of an SPC and a company's application for an extension.
"This creates a commercially impossible situation for manufacturers who have less expensive generic products under development for marketing immediately upon SPC expiry," said a spokesperson for the European Generics Association.
However, if the full Parliament agrees on the proposal, it will go a long way towards ensuring that the medicines used in children have been tested appropriately.
"The motivation for this proposal is the improvement of child health in Europe," Arlett said. "Continued support from industry is essential."
Sarah Houlton, PhD, is Pharmaceutical Executive's global correspondent. She can be reached at [email protected]