Management of patients with rare adult solid cancers: objectives and evaluation of European reference networks (ERN) EURACAN.

About 500,000 patients with rare adult solid cancers (RASC) are diagnosed yearly in Europe. Delays and unequal quality of management impact negatively their survival. Since 2017, European reference networks (ERN) aim to improve the quality of care of patients with rare disease. The steering committee of EURACAN, including physicians, researchers and patients review here the previous actions, present objectives of the ERN EURACAN dedicated to RASC. EURACAN promoted management in reference centres, and equal implementation of excellence and innovation in Europe and developed 22 clinical practice guidelines (CPGs). Additionally, fourteen information brochures translated in 24 EU languages were developed in collaboration with patient advocacy groups (ePAGs) and seventeen training session were organized. Nevertheless, connections to national networks in the 26 participating countries (106 centres), simplification of cross-border healthcare, international multidisciplinary tumour boards, registries and monitoring of the quality of care are still required. In this Health Policy, evaluation criteria of the performances of the network and of health care providers are proposed.

Views of European Drug Development Stakeholders on Treatment Optimization and Its Potential for Use in Decision-Making.

BACKGROUND: The current drug development paradigm has been criticized for being too drug-centered and for not adequately focusing on the patients who will eventually be administered the therapeutic interventions it generates. The drug-driven nature of the present framework has led to the emergence of a research gap between the pre-approval development of anticancer medicines and their post-registration use in real-life clinical practice. This gap could potentially be bridged by transitioning toward a patient-centered paradigm that places a strong emphasis on treatment optimization, which strives to optimize the way health technologies are applied in a real-world environment. However, questions remain concerning the ideal features of treatment optimization studies and their acceptability among key stakeholders. OBJECTIVES: The aim of this study was to explore the views of key stakeholders in the drug development process regarding the concept of treatment optimization. METHODS: Semi-structured interviews were conducted between December 2018 and May 2019 with 26 participants across ten EU Member States and six different stakeholder groups, including academic clinicians as well as representatives of patient organizations, regulatory authorities, health technology assessment agencies, payers, and industry. RESULTS: Based on the input of the experts interviewed, clarification was obtained regarding the optimal features of treatment optimization studies in terms of their conduct, funding, timing, design, and setting. Moreover, a number of opportunities and challenges of undertaking such trials were identified. Inter-stakeholder discussion during their design was seen as desirable. There was also broad support among the participants for regulatory measures to facilitate treatment optimization, although there was no agreement on the optimal scale and nature of these initiatives. Furthermore, the interviewees believed that the evidence strength of well-designed treatment optimization studies performed according to rigorous quality standards is greater than or at least equal to that of classical clinical trials. In addition, there was a strong consensus that the results of treatment optimization studies should be taken into account during the decision-making of regulators, payers, and/or clinicians. CONCLUSIONS: Stakeholders involved in drug development consider treatment optimization studies to be valuable tools to address current evidence gaps and support their implementation into the existing research framework.

Towards personalized medicine in the EU: what is needed to facilitate the complex international clinical research?

Cancer is a major health problem in developed countries. Despite important progress, cancer remains a fatal disease. In Europe, cancer care is becoming less and less sustainable because it is not cost effective. Better treatments should be made available to patients. The biology of the disease should be known. Drug development should be based on methodologically robust clinical trials testing drugs selected on the grounds of convincing preclinical evidence. Personalized medicine will be established only if sophisticated clinical research is conducted, maximizing the use of recent technologies. Modern clinical research will request a strong partnership between industry and academics, with close contact with regulators and payers. European legal framework should be streamlined and simplified in order to become researcher friendly.

Laetrile treatment for cancer.

BACKGROUND: Laetrile is the name for a semi-synthetic compound which is chemically related to amygdalin, a cyanogenic glycoside from the kernels of apricots and various other species of the genus Prunus. Laetrile and amygdalin are promoted under various names for the treatment of cancer although there is no evidence for its efficacy. Due to possible cyanide poisoning, laetrile can be dangerous. OBJECTIVES: To assess the alleged anti-cancer effect and possible adverse effects of laetrile and amygdalin. SEARCH METHODS: We searched the following databases: CENTRAL (2011, Issue 1); MEDLINE (1951-2011); EMBASE (1980-2011); AMED; Scirus; CancerLit; CINAHL (all from 1982-2011); CAMbase (from 1998-2011); the MetaRegister; the National Research Register; and our own files. We examined reference lists of included studies and review articles and we contacted experts in the field for knowledge of additional studies. We did not impose any restrictions of timer or language. SELECTION CRITERIA: Randomized controlled trials (RCTs) and quasi-RCTs. DATA COLLECTION AND ANALYSIS: We searched eight databases and two registers for studies testing laetrile or amygdalin for the treatment of cancer. Two review authors screened and assessed articles for inclusion criteria. MAIN RESULTS: We located over 200 references, 63 were evaluated in the original review and an additional 6 in this update. However, we did not identify any studies that met our inclusion criteria. AUTHORS' CONCLUSIONS: The claims that laetrile or amygdalin have beneficial effects for cancer patients are not currently supported by sound clinical data. There is a considerable risk of serious adverse effects from cyanide poisoning after laetrile or amygdalin, especially after oral ingestion. The risk-benefit balance of laetrile or amygdalin as a treatment for cancer is therefore unambiguously negative.

Laetrile for cancer: a systematic review of the clinical evidence.

BACKGROUND: Many cancer patients treated with conventional therapies also try 'alternative' cancer treatments. Laetrile is one such 'alternative' that is claimed to be effective by many alternative therapists. Laetrile is also sometimes referred to as amygdalin, although the two are not the same. OBJECTIVE: The aim of this review is to summarize all types of clinical data related to the effectiveness or safety of laetrile interventions as a treatment of any type of cancer. MATERIALS AND METHODS: All types of clinical studies containing original clinical data of laetrile interventions were included. We searched the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE (from 1951), EMBASE (from 1980), Allied and Complementary Medicine (AMED), Scirus, CancerLit, Cumulative Index to Nursing and Allied Health (CINAHL; all from 1982), CAMbase (from 1998), the MetaRegister, the National Research Register, and our own files. For reports on the safety of laetrile, we also searched the Uppsala database. No language restrictions were imposed. RESULTS: Thirty six reports met our inclusion criteria. No controlled clinical trials were found. Three articles were nonconsecutive case series, 2 were consecutive case series, 6 were best case series, and 25 were case reports. None of these publications proved the effectiveness of laetrile. CONCLUSION: Therefore, the claim that laetrile has beneficial effects for cancer patients is not supported by sound clinical data.

Methodological quality of patient-reported outcome research was low in complementary and alternative medicine in oncology.

OBJECTIVE: To evaluate the methodological robustness of patient-reported outcomes (PROs) evaluation in complementary and alternative medicine (CAM) randomized controlled trials (RCTs) in oncology. STUDY DESIGN AND SETTING: CAM RCTs with a PRO endpoint were retrieved from a number of electronic databases. CAM interventions were defined according to the five major categories of the National Center for Complementary and Alternative Medicine. The "Minimum Standard Checklist for Evaluating HRQOL Outcomes in Cancer Clinical Trials" was used to assess the quality of the PRO reporting in these trials. RESULTS: Forty-four RCTs enrolling 4,912 patients were identified: six studies involved alternative medical systems, 14 involved mind body interventions, 15 dealt with biologically-based therapies, seven involved manipulative and body-based methods, and two energy therapies. Eighty-nine percent of studies used a PRO as a primary endpoint and 59% documented PRO missing data. Although 84% of the studies used a validated PRO questionnaire, only 37% stated an a priori hypothesis and 20% addressed clinical significance of the outcomes. Overall, 64% of the studies analyzed exhibited a number of methodological drawbacks. CONCLUSIONS: To facilitate the interpretation of results from such CAM RCTs, investigators are encouraged to pay greater attention to key methodological issues as identified in this study.

The role of complementary and alternative medicine in the management of early breast cancer: recommendations of the European Society of Mastology (EUSOMA).

Patients diagnosed with breast cancer have many needs that for a start include the expectation of cure. Where cure is unlikely there is always a place for hope and spiritual support. Furthermore whether dealing with the early stages or with the advanced disease patients require symptomatic control that encompasses pain relief, control of nausea and vomiting and psychological distress. To achieve all of these goals there is a need that goes beyond the role of scientific medicine. This position papers describes the guidelines for the use of complimentary and alternative medicine (CAM) developed by a workshop on behalf of the European Society of Mastology (EUSOMA).

Role of complementary and alternative medicine in the care of patients with breast cancer: report of the European Society of Mastology (EUSOMA) Workshop, Florence, Italy, December 2004.

The aim of the European Society of Mastology (EUSOMA) Workshop, Florence, Italy, December 2004, was to produce guidelines on the use of complementary and alternative medicine (CAM) for breast cancer. The widespread use of CAM has to be acknowledged and the reasons for this understood. Deficiencies in the practice of conventional medicine that lead to the adoption of CAM need to be addressed. At the same time, CAM use for breast cancer should be quality controlled, avoiding double standards in evaluation, whilst recognising the problems of CAM research. These guidelines, which appear in an accompanying paper, aim to help oncologists and cancer patients alike.