The case in court – the interlocutory proceedings-
Full text of the verdict (in Dutch, English translation available upon request)
Reactions by law experts and others in NRC.nl (in Dutch, English translation available upon request)
On the 7th of September 2011 Genzyme (which had already been taken over by Sanofi in February 2011) announced to have relinquished the European rights to the use of Ataluren to PTC. Because of this take-over the pledge to start a new study before the end of 2011, made in court by Genzyme, was not honoured.
Immediately after the court verdict our lawyer, in consultation with the Verhoek family, made all the preparations to bring an appeal. For this appeal a medical certificate was required and another request for compassionate supply had to be filed, this time to be directed at PTC. Before the interlocutory proceedings the neurologist in attendance had not issued the medical certificate in spite of the fact that earlier he had issued a written a pledge to this effect. Therefore the Verhoek boys had to find a new specialist in attendance in order to obtain such a medical certificate. This specialist was found in another University Medical Centre. By mid December the hospital pharmacist filed a request for ‘compassionate supply’ with PTC. On the 17th of February 2012 PTC announced that they were only willing to supply the drug within a study context and that the study would be initiated as soon as possible.
Meanwhile on the 7th of November PTC’s lawyer, mr Meulenbelt had conveyed this message to mr Peter Burgers: PTC is attending to the last details in co-operation with the European study centres… because not all the medical-ethical committees have given their approval yet (among which the University Hospital Leuven, with Dr Goemans, to whom the Verhoek boys have been assigned)…. This will be an open-label study with all the patients who participated in the previous study’. Obviously he could not provide any guarantees for the exact moment when the study was to be initiated.
On the 9th of November Dr Goemans confirmed this announcement to the Verhoek family.
Because of these positive announcements the appeal was withdrawn, but since no certainty could be given as to the date on which the study would be started, the efforts to obtain compassionate supply were continued.
By the end of December 2011 the hospital at Leuven received the documents and contracts concerning the follow-up study from PTC for assessment by the lawyers and the local medical-ethical committees. On the 9th of February 2012 a notification from PTC was received through DPPN (Duchenne Parent Project Netherlands) that it was to be expected that ‘… most study sites would open between March and June’.
Unfortunately it was not until the 16th of May 2012 when the approval by the Leuven Medical Ethical Committee was granted.
On the 25th of May the Verhoek boys could start with the drug Ataluren, at present for a trial period of approximately 48 weeks.
Comment by Cinderella
In view of all of this we conclude that the legal intervention initiated by Cinderella has not resulted in the intended objective, contrary to expectations. It cannot be excluded that the proceedings and the campaigns have induced the parties concerned to do everything possible to start the follow-up study at the earliest possible date. For the family it was of great significance that so many people made such an effort in the interest of their children. The media coverage showed a great deal of sympathy and support thanks to the excellent advice given by the public relations agency 2Twintig in Rotterdam that offered its services for this purpose free of charge. This case has also provided important new insights that are useful not only to Cinderella but to patients and to health professionals as well.
These insights include:
# The duty judge has ruled that the Helsinki declaration is only binding for members of the medical profession.
# Pharmaceutical companies can back out of the obligations pertaining to the rights of subjects that were laid down in the Helsinki declaration without any sanctions whatsoever. Physicians in attendance, medical-ethical committees and hospital boards should see to it that these rights are laid down in their patient agreements.
Patient associations should draw their members’ attention to this aspect of participation in clinical trials.
# Contrary to Genzyme’s claims the Dutch Ministry of Health, Welfare and Sport has approved the import and supply of Ataluren when there is a medical certificate to this effect.
# The Dutch specialist who treated these patients refused to sign the medical certificate that was required for the interlocutory proceedings, contrary to an earlier pledge to this effect made to these patients. Physicians of another University Medical Centre proved willing to take over the patients and saw to it that a medical certificate was issued that was also required for a higher appeal if there was to be such an appeal.
# With respect to the patients and to UPPMD Genzyme pretended to the judge to be involved in preparations for the follow-up study even at the time of the interlocutory proceedings, while in reality negotiations were going on between Sanofi (that had taken over Genzyme in February 2011) and PTC; this resulted in the termination of Genzyme’s participation in the development of Ataluren.
# The medical-ethical committee of the Leuven University Hospital needed over 4 months to process the study application while they were fully familiar with the subject through the previous study.
# Although Ataluren is available in bulk, the necessary analyses for purity and the formulation by a contract company proved to encounter some problems. The greatest obstacle was the fear of a conflict with Genzyme/PTC about the patent rights. Cinderella is of the opinion that these rights are not at issue as long as no trade is being conducted, but still the chemical company concerned did not want to risk a confrontation.