Real-world evidence research in metastatic colorectal cancer: raising awareness of the need for patient contributions.

Real-world evidence (RWE) research helps determine whether outcomes observed in clinical trials happen in real-life clinical practice. RWE research may help patients receive more appropriate treatment, closer to their needs and wishes. RWE for metastatic colorectal cancer is currently limited. The PROMETCO RWE study is an important example of an ongoing initiative that focuses on patient-reported outcomes in metastatic colorectal cancer. Patients play an active role throughout the RWE research process, including study design, participation and results dissemination. This involvement can encourage greater patient empowerment through active engagement, potentially resulting in various benefits that can lead to improved clinical outcomes. Greater patient engagement can increase involvement in RWE, helping more patients to access the benefits of RWE research. Clinical Trial Registration: NCT03935763 (ClinicalTrials.gov).

Real-world evidence (RWE) research provides information that is essential to improving medical treatment. When it comes to metastatic colorectal cancer ­ cancer that has spread to other parts of the body ­ only a few RWE studies have been conducted. RWE studies, such as the ongoing PROMETCO study in patients with metastatic colorectal cancer, differ from clinical trials in that they include a wider range of people with fewer restrictions on type of treatments received. They can also place more attention to the patients' own opinions. By joining RWE studies, patients are likely to become more interested in their disease and take a more active role in their treatment. In the end, this can help to improve their quality of life and possibly improve the outcomes of their treatment. Doctors need to work in partnership with patients to increase participation in RWE studies.

Meeting the Need for a Discussion of Unmet Medical Need.

As Europe and the world continue to battle against COVID, the customary complacency of society over future threats is clearly on display. Just 30 months ago, such a massive disruption to global lives, livelihoods and quality of life seemed unimaginable. Some remedial European Union action is now emerging, and more is proposed, including in relation to tackling "unmet medical need" (UMN). This initiative-directing attention to the future of treating disease and contemplating incentives to stimulate research and development-is welcome in principle. But the current approach being considered by EU officials merits further discussion, because it may prove counter-productive, impeding rather than promoting innovation. This paper aims to feed into these ongoing policy discussions, and rather than presenting research in the classical sense, it discusses the key elements from a multistakeholder perspective. Its central concern is over the risk that the envisaged support will fail to generate valuable new treatments if the legislation is phrased in a rigidly linear manner that does not reflect the serpentine realities of the innovation process, or if the definition placed on unmet medical need is too restrictive. It cautions that such an approach presumes that "unmet need" can be precisely and comprehensively defined in advance on the basis of the past. It cautions that such an approach can reinforce the comfortable delusion that the future is totally predictable-the delusion that left the world as easy prey to COVID. Instead, the paper urges reflection on how the legislation that will shortly enter the pipeline can be phrased so as to allow for the flourishing of a culture capable of rapid adaptation to the unexpected.

Time for Change? The Why, What and How of Promoting Innovation to Tackle Rare Diseases - Is It Time to Update the EU's Orphan Regulation? And if so, What Should be Changed?

Since developments are global in the healthcare arena, more should be done to align EU and other big markets' regulatory practices for rare disease patients. Notwithstanding efforts and cooperation between the US and EU aimed to harmonize their strategic plans in the field of orphan drugs, regulatory criteria and procedures to gain the designation, terms and classifications should be still harmonised. Aligning the criteria of prevalence and support to orphan medicines in the various jurisdictions internationally, would facilitate patient recruitment eventually at global level, so as to gain the data and the biological insights required to identify biomarkers and appropriate endpoints needed for progressing clinical development. A conducive regulatory environment can further support the development of medicines to treat rare diseases. Overall there is a need for joined-up regulatory process coordination. Better integration of regulatory pathways and better integration of regulatory systems, such as scientific tools and methods to generate evidence, would be helpful. There is a need to revise and agree the current frameworks to be improved which will take into account the considerations and challenges to diagnose and treat different rare diseases and improve quality of life. Deliberative processes with multi-stakeholders' involvement for reimbursement should be considered. This paper explores the successes and limitation of both the regulation and its implementation mechanisms in the current regulatory context, and suggests some improvements that could maximise its benefits and boost rare disease research even further.

A remarkable insight into the cancer journey of Barbara Moss.

Barbara Moss speaks to Roshaine Wijayatunga, Senior Editor Oncology at the 2018 European Society for Medical Oncology World Congress on Gastrointestinal Cancer (ESMO GI; Barcelona). Barbara Moss is a Patient Ambassador for EuropaColon and Bowel Cancer UK. At the age of 52, in 2006, Barbara was diagnosed with stage IV colorectal cancer of the colon, spread to the liver and was told she had 3-5 months to live! Barbara is now an active campaigner and an active member of several organizations in the UK and Europe. Her main wish is for patients to be able to access the medicine that they need, to have choices clearly explained and to be treated personally.

Involving patients in a multidisciplinary European consensus process and in the development of a 'patient summary of the consensus document for colon and rectal cancer care'.

CONTEXT: High-quality cancer care should be accessible for patients and healthcare professionals. Involvement of patients as partners in guideline formation and consensus processes is still rarely found. EURECCA, short for European Registration of Cancer Care, is the platform to improve outcomes of cancer care by reducing variation in the diagnostic and treatment process. EURECCA acknowledges the important role of patients in implementation of consensus information in clinical practice. OBJECTIVE: The aim of this article is to describe the process of involving patients in the consensus process and in developing the patient summary of the consensus for colon and rectal cancer care. METHODS: The Delphi method for achieving consensus was used. Three online voting rounds and one tele-voting round were offered to an expert panel of oncology professionals and patient representatives. At four different stages, patients and/or patient representatives were involved in the process: (1) during the consensus process, (2) lecturing about the role of the patient, (3) development of the patient summary, and (4) testing the patient summary. RESULTS: Representatives were invited to the voting and commenting rounds of this process and given an equal vote. Although patients were not consulted during the planning stages of this process, patient involvement increased following the panel's discussion of the implementation of the consensus among the patient population. After the consensus meeting, the patient summary was written by patient representatives, oncologists and nurses. A selection of proactive patients reviewed the draft patient summary; responses were positive and several patient-reported outcomes were added. Questionnaires to evaluate the use and implementation of the patient summary in daily practice are currently being developed and tested. Patient consultation will be needed in future planning for selection of topics. DISCUSSION: The present study may function as a model for future consensus processes to involve patients at different stages and to implement both patient and healthcare professional versions in daily practice.