Unity is strength ! Today, it is time to find a new model for financing therapeutic innovation for children and adolescents with cancer. In an article published in Le Quotidien du Médecin and Economie Matin, our President, Patricia Blanc, pleads in favor of major collective action, an association between solidarity and innovation to go even further.
UNION, THE BEST rampart AGAINST CHILDREN’S CANCERS
Every year, 2,500 children are diagnosed with cancer in France. Beyond the pain and after the shock of the announcement, families and their children embark on what could be called the fight of a lifetime. It is crucial that this battle is not lost in advance, especially for the 500 children whose only hopes rest on advances in research. This is why we are calling for collective action, the union of solidarity and innovation to counter the predictions. Today, the union of all forces constitutes the best defense against pediatric cancers, involving manufacturers, public authorities, research centers of excellence, associations, and of course, the general public. Together, let’s break down unnecessary barriers in drug development and form a united front against disease.
10 YEARS OF VICTORIES WHICH SHOULD SERVE AS EXAMPLES
The last ten years of research and progress have demonstrated that hope is very present, and even growing. Thanks to the mobilization of associations, their actions and the generosity of their donors, increased resources have been allocated to research. In a decade, the sequencing of molecular factors linked to high-risk and relapsed pediatric cancers has been generalized. This advance has refined our understanding of childhood tumors, taking a crucial step toward precision medicine that specifically targets tumor weaknesses. However, 70% of the therapeutic targets revealed by these genetic studies remain without effective treatment to neutralize them, thus losing a real chance of curing the most serious pediatric cancers. Our priority must therefore be to respond to this urgent need, especially since the first results of precision medicine raise high hopes. Recent research has shown the remarkable effectiveness of larotrectinib, a drug in syrup form, targeting a genetic anomaly frequently present in childhood fibrosarcoma and which attacks tumor cells with unprecedented efficiency.
In addition, the deployment of this precision medicine would meet another need: 2/3 of treated children suffer significant side effects and after-effects. It is our duty to ensure that cancer is also a distant memory for cured children, so that they can grow up without the pain, cognitive or emotional problems, heart problems or deafness caused by treatments. We are convinced that the deployment of precision molecular medicine and other innovative therapies will allow children to suffer less from these after-effects.
OUR STRENGTH LIES IN UNION
The issues are clear. We must continue to innovate, progress and fund the most promising research programs, but we must do it together. The ability of doctors and researchers to bring together skills and expertise is also crucial, as demonstrated by the SACHA project. This study brings together a large number of pediatric oncologists in France and internationally, as well as authorities such as the ANSM or the HAS, and pharmaceutical companies. It aims at bringing together data from patients who have received innovative and experimental medicines, prescribed outside of a clinical trial or marketing authorization. Unlike other pathologies, the toxicity and effectiveness data of these drugs, generally prescribed on a compassionate basis, were neither collected nor analyzed. Thanks to the participation of more than 650 children and their families, this initiative now makes it possible to formulate several therapeutic recommendations for all pediatric oncologists and to secure access to these treatments.
This collective vision of research in pediatric oncology, encouraged by associations and desired by families, is decisive in this race against time where every second counts. It is our duty to amplify the synergies on the solid and united foundation that we have succeeded in building. Members of the large research community, public institutions, pharmaceutical and biotechnology companies, associations and civil society, let’s come together, break down barriers and work together to meet the needs of children.
LET’S RESPOND COLLECTIVELY TO THE LACK OF EFFECTIVE TREATMENTS FOR CHILDREN’S CANCERS
It is time to make cancer a real health priority also for children. Over the past 15 years, only 16 anti-cancer drugs have been authorized in Europe for children compared to more than 150 for adults. This is clearly insufficient, especially since most of these molecules are prescribed for cancers with favorable prognoses. A substantial effort is therefore necessary to meet this need for targeted therapies, highlighted by each genetic analysis of each patient’s tumors.
We are aware that the prospects for economic gains are limited for many pharmaceutical companies, that clinical studies leading to the validation of new therapies are long and costly, and that regulations are not sufficiently adapted to the needs of professionals in terms of sharing of data and financing of innovation, despite a favorable scientific and technological context.
We therefore propose to strengthen proximity between stakeholders to encourage exchanges and collaboration, and accelerate the processes of discovery and development of new therapies. We call for the implementation of shared actions and encourage the free circulation of ideas, knowledge, skills and resources. Let us establish together a roadmap enabling the efficient development of new drugs, introduce the regulatory changes necessary for data sharing and encourage the creation of new dedicated companies, implement new methods of financing innovation and take advantage of everything that research produces for the common good.
Let’s make pediatric oncology research an example of collective action in health, so that tomorrow will be a world with children without cancer.
Patricia Blanc
Founding President of Imagine for Margo – Children without Cancer
