As leiomyosarcoma patients are challenged by the development of metastatic disease, effective systemic therapies are the cornerstone of outcome. However, the overall activity of the currently available conventional systemic treatments and the prognosis of patients with advanced or metastatic disease are still poor, making the treatment of this patient group challenging. Therefore, in a joint effort together with patient networks and organizations, namely Sarcoma Patients EuroNet (SPAEN), the international network of sarcoma patients organizations, and the National LeioMyoSarcoma Foundation (NLMSF) in the United States, we aim to summarize state-of-the-art treatments for leiomyosarcoma patients in order to identify knowledge gaps and current unmet needs, thereby guiding the community to design innovative clinical trials and basic research and close these research gaps. This position paper arose from a leiomyosarcoma research meeting in October 2020 hosted by the NLMSF and SPAEN.

Background The MHRA Innovative Licensing Access Pathway (ILAP) is a project designed to provide new medicines with a quicker route through the regulatory system and thus to patients. Public and patient involvement in ILAP was established by MHRA through a Public and Patient Reference Group (PPRG). This was done prior to publication of its PPI Strategy and ILAP is included in that Strategy. What Happened The PPRG was established within the ILAP programme to advise on patient engagement within the ILAP process. Some members also volunteered to attend meetings of the Steering Group from mid-2021 and took part in the review of pharmaceutical company applications for entry to ILAP. Over the course of two years, as the ILAP programme was extended, the number of the involved members fell, while experience grew and the input of those remaining became more valued. At the same time the MHRA was developing its PPI strategy and looking for ways to address the challenge of involvement across the considerable breadth of its activities. Conclusion Public and patient involvement in the ILAP project has been positive, facilitated by MHRA staff and their colleagues from ILAP partner organisations. Patients’ knowledge and experience from their previous involvement in medicines development and other research has contributed to the ILAP process. It has clearly demonstrated that experienced patient advocates can address issues which lie outside their personal ‘lived experience’ and which may also be innovative and scientifically challenging. However, the corporate communication with PPRG members has been variable. Members of the group were not consulted when MHRA reviewed the ILAP programme or included in discussions about the future of patient involvement in ILAP or later successor programmes. Decisions on other factors, such as whether patient advocates should be paid for their time, as is the case in other agencies, have been deferred.