PRIME-ROSE (Precision Cancer Medicine Repurposing System Using Pragmatic Clinical Trials) is a Horizon Europe Mission on Cancer project with 24 partners from altogether 18 European countries. Moreover, PRIME-ROSE is part of the Cancer Mission cluster of projects on Diagnosis and Treatment.
Funded by the European Union
PRIME-ROSE (Precision Cancer Medicine Repurposing System Using Pragmatic Clinical Trials) is funded by the EU Cancer Mission and will run over five years (2023 – 2028). The consortium consists of altogether 24 partners.
Millions of people in the EU are affected by cancer, and cancer is one of the leading causes of death and morbidity in Europe. PRIME-ROSE shares the ambition of the EU Cancer Mission to save and improve 3 million lives by 2030, and fully endorses the need for sustainable and affordable access to new and innovative drugs for cancer patients in Europe.
Precision cancer medicine is an approach that depends on access to adequate molecular diagnostics and drugs to have impact and move towards implementation in the national healthcare systems.
Today there is inequality in access to precision cancer medicine (PCM) between and within EU countries, and while the promise of PCM is clear, implementation remains a challenge. This in particular affects cancer patients with the poorest prognosis who have exhausted all lines of standard of care treatment, rare tumors, those with tumours carrying rare mutations and patients with carcinoma of unknown primary.
The PRIME-ROSE vision is access to affordable precision cancer medicine that prolongs life at the best quality possible for all cancer patients.
The PRIME-ROSE consortium composition reflects the unique approach taken by the community that has assembled around the shared interest of providing cancer patients who have exhausted other treatment options with access to precision medicine diagnostics and treatments.
PRIME-ROSE builds on a bottom-up, clinician-initiated family of precision cancer medicine clinical trials, which have been particularly successful in bringing up inclusion rates to offer additional lines of treatment and in providing patient benefit. These trials share the pragmatic clinical trial design of the original Dutch DRUP trial, with broad inclusion criteria and a limited set of endpoints. However, the trials are still anchored into a national context and are funded independently. The result is a distributed DRUP-like clinical trial network that addresses local priorities while collaborating internationally for scale and impact. PRIME-ROSE is building on and collaborating closely with the related EU project PCM4EU.
PRIME-ROSE is treatment-oriented whereas PCM4EU focuses on deployment of novel PCM diagnostic tools. Altogether, this EU-wide precision cancer medicine deployment will address key scientific and methodological questions, leading to accelerated and improved access to new and effective cancer treatments. The ongoing effort is already leading to harmonization, standardization and pragmatic consensus.
Increase access to optimised and affordable treatment interventions that prolong life and increase cancer patient’s quality of life
Generate clinical evidence and evaluate effectiveness in PCM-relevant and synthetically randomised evidence-based treatment interventions that improve outcomes in real life for routine cancer care
Work together with regulators, policymakers, payers, healthcare providers and patient advocacy groups to implement evidence-based PCM in routine practice and address inequalities in access
Enable cross-border data sharing
Build synthetic randomised trials
Design and conduct pragmatic clinical trials
Provide the necessary data for implementation
Involve patients in a consistent and meaningful manner
Focus on multi-stakeholder collaboration
Share knowledge and provide education and training
PRIME-ROSE and the DRUP-like trial community effectively work according to the principles described as typical for a Living Lab:
Academically initiated real-word trials treating patients in a clinical setting and in collaboration between research institutions, clinicians, the pharmaceutical and diagnostic industry, payers and civil society.
The trial design is innovative regarding how it includes pragmatic outcome-based performance agreements between the pharmaceutical industry and the national insurance systems.
The healthcare system has multiple ‘users,’ with patients as the ultimate beneficiary.
DRUP trials are all multisided, iterative agreements that are refined over time based on learnings and new insights.
The DRUP community includes all partners of a classic penta-helix set-up, with DRUP-like consortia assuming the role of the convening actors.
Research is an active part of the setup and facilitates the multidisciplinary nature of the process.
In DRUP-like trials, research spans the full spectrum of translational, medical, implementation, innovation and health-economic research, as well as methodological developments such as the use of real-world evidence in decision-making.
Various collaborations in physical and virtual space to create desired outcomes
The various parties within the DRUP ecosystem collaborate in variable set-ups and initiatives that can take place at local, national or European level.
The different trial set-ups are linked by a Memorandum of Understanding, but remain independently governed.
This project and the DRUP-like clinical trials in general provide significant scientific, societal and economic impact by providing a pragmatic means to leverage the potential of precision medicine in cancer. By combining broad access to novel precision diagnostics and medicines for cancer patients with poor prognosis with measures ensuring affordability and continuous learning, the trials benefit both current and future patients.
We believe PRIME-ROSE outcomes will have both medium- and long-term impact on several stakeholder groups:
Initiation of stage III expansion cohorts in the ongoing DRUP-like clinical trials (DLCTs) will provide immediate access to PCM for cancer patients with poor prognosis in the countries of the consortium partners.
DRUP-like clinical trials are expected to launch in additional countries, continuously increasing the access opportunities for cancer patients in Europe.
PRIME-ROSE will analyse and address the specific challenges for medium-income countries to facilitate that in the medium- to long-term, PCM becomes available to all cancer patients in the EU region, not just those in high-income countries.
The iterative design of the trial set-up further ensures that diagnostics and therapies are continuously optimised and that cancer patients receive the best possible care at any moment in time.
PRIME-ROSE will provide clinicians involved in the project with additional treatment options for those of their patients who have exhausted all lines of therapy.
Limited exclusion criteria translate to options independent of the cancer type in addition to including patients with cancers of unknown primary and those for co-morbidities who are often excluded from commercial trials.
By continuously contributing to the generation of clinical evidence, clinicians will ensure ‘learning from every patient treated’.
The cross-country collaboration provided by PRIME-ROSE gives clinicians access to evidence that otherwise could not have been generated at a national level. This will give the clinicians confidence to deploy treatments proven to improve patient outcomes.
PRIME-ROSE serves as a case study for national and regional healthcare providers on how to pragmatically implement PCM in their respective country / region.
Healthcare providers will also be able to appreciate the differences in implementation in a range of healthcare systems, including middle-income countries.
With the pragmatic risk-sharing feature of the DRUP-like clinical trials, pharmaceutical companies provide therapies free of charge for the first 16 weeks of treatment.
If the patient derives benefit at 16 weeks, the healthcare system may then, if adopting the model, reimburses a treatment that has demonstrated efficacy for the individual patient.
Healthcare providers can be confident to only reimburse beneficial therapies. In the long term, we anticipate the model to evolve into a general, broadly available introductory mechanism that will become part of everyday oncological practice and independent of clinical trials.
Policy makers and authorities will be able to base their decisions on independently and continuously generated evidence in their respective area as well as the resulting outcomes.
This will ensure cost-effectiveness and with that contribute to the affordability and overall sustainability in healthcare.
In the medium- to long-term this could also lead to the refinement and optimisation of the indications for which therapies are used, increasing the value for patients and society at large.
In the long-term, PRIME-ROSE and the DRUP-network will provide considerable societal impact as they contribute to the transformation of cancer care by providing a pragmatic model for the broad, equitable and sustainable implementation of PCM also outside the clinical trial setting.
Truly equitable access to molecular diagnostics and medicines may prevent a two-tiered system of those who can afford private healthcare versus those who cannot and by this contribute to the SDG of Universal Healthcare.
PRIME-ROSE is also expected to accelerate innovation as every single patient contributes to the evidence-generation process for the benefit of society as a whole.
Sustainably improve access to PCM treatments in oncology throughout the EU and beyond by providing a pragmatic model for PCM introduction
Facilitate PCM implementation through a flexible model that accommodates different socioeconomic realities and reimbursement systems
Accelerate the usability of molecular profiling and by consequence, up-take of PCM
Improve patient and citizen involvement in PCM
Systematically generate real-world Quality of Life data and safety evaluations of therapies
Provide policy makers and healthcare authorities at regional, national and European level with evidence for decision-making
Benefit European society at multiple levels: patients, physicians, payers and the private sector
Contribute to Europe’s Beating Cancer Plan, the Cancer Mission and the European Health Data Space
