The EHE Group are delighted to report that a paper addressing the important issue of patient access to the drug sirolimus (one of a class of drugs known as mTOR inhibitors) has been published in Frontiers in Oncology, a prestigious, peer-reviewed scientific journal, on 26th February. This paper, entitled “The patient perspective on sirolimus for epithelioid hemangioendothelioma (EHE): results of a community survey highlighting the importance of equitable access to treatments” presented the results of a survey of the global EHE patient community, conducted in 2023, about their access to, and results of treatment with, sirolimus.
The survey, coordinated by Denise Robinson, Research Direct of The EHE Foundation, and Hugh Leonard, Chair of Trustees of The EHE Rare Cancer Charity UK, was undertaken to support the ongoing application to the European Medicines Agency (EMA) for a label extension of sirolimus (also called rapamycin and marketed by Pfizer under the tradename Rapamune®) to include the treatment of EHE. While sirolimus has been globally approved and widely used for many years, mainly as an immunosuppressant, it can still only be prescribed to EHE patients ‘off label’ as it has not been formally approved for the treatment of EHE. This means sirolimus is only available to patients who access the drug off-label, resulting in inequality of access to this important treatment.
There is a growing body of evidence, including the experience of our patient community, that sirolimus is now the leading front-line drug for the systemic treatment of EHE. In 2021 the ESMO paper “Epithelioid hemangioendothelioma, an ultra-rare cancer: a consensus paper from the community of experts” recognised, when comparing different drugs used to treat EHE, that “the highest clinical activity has been reported for mTOR inhibitors” and confirmed that “the panel [of experts] agrees that these [mTOR inhibitors] represent the preferred treatment options for patients with advanced and moderately progressive disease”.
However, because sirolimus is not formally approved for EHE and can only be prescribed off-label, many patients cannot access it if their doctor, or hospital, or national health system won’t allow off-label use. Even if they can access off-label prescription, the drug may need to be self-funded if it is not funded by their health system, resulting in sirolimus being out of reach for many patients. This creates a huge inequality in the access to the drug and is why we are determined to secure regulatory approval for a label extension.
The results of the sirolimus survey, that 130 of the EHE patient community participated in, are an excellent example of why patient data is so important. The data supports the position expressed in the ESMO paper described above, namely that mTOR inhibitors like sirolimus are being successfully used in the treatment of EHE. It also shows that these drugs are not equally accessible for all patients due to only being available off-label.
Both Hugh and Denise want to thank the other authors of the paper, Giacomo Giulio Baldi, MD, Pan Pantziarka, PhD, Silvia Stacchiotti, MD, Professor Robin Jones and William Tap, MD for their excellent hard work, guidance, and commitment in helping to produce the paper. Huge gratitude is also extended to The EHE Rare Cancer Foundation Australia, EHE Canada, and EHE Italia Associazione Non Solo Laura ODV, all of whom were instrumental in mobilising support and participation of EHE patients. But the biggest thanks, without doubt, goes to each and every one of the 130 EHE patients who took part in the survey. Without their fantastic support, there would have been no data to work with.
If you wish to read the paper, it can be found at:
https://www.frontiersin.org/journals/oncology/articles/10.3389/fonc.2024.1367237/full