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Background The European Medicine Agency granted marketing approval to 164 orphan medicinal products for rare diseases, among which 28 products intended for the treatment of hereditary metabolic diseases. Taking advantage of its privileged connection with 69 healthcare centres of excellence in this field, MetabERN, the European Reference Network for hereditary metabolic diseases, performed a survey asking health care providers from 18 European countries whether these products are available on the market, reimbursed and therefore accessible for prescription, and actually delivered in their centre. Results Responses received from 52 centres (75%) concerned the design of treatment plans, the access to marketed products, and the barriers to delivery. Treatment options are always discussed with patients, who are often involved in their treatment plan. Most products (26/28) are available in most countries (15/18). Among the 15 broadly accessible products (88.5% of the centres), 9 are delivered to most patients (mean 70.1%), and the others to only few (16.5%). Among the 10 less accessible products (40.2% of the centres), 6 are delivered to many patients (66.7%), and 4 are rarely used (6.3%). Information was missing for 3 products. Delay between prescription and delivery is on average one month. Beside the lack of availability or accessibility, the most frequent reasons for not prescribing a treatment are patients’ clinical status, characteristic, and personal choice. Conclusions Data collected from health care providers in the MetabERN network indicate that two-third of the orphan medicines approved by EMA for the treatment of hereditary metabolic diseases are accessible to treating patients, although often less than one-half of the patients with the relevant conditions actually received the approved product to treat their disease. Thus, in spite of the remarkable achievement of many products, patients concerned by EMA-approved orphan medicinal products have persistent unmet needs, which deserve consideration. The enormous investments made by the companies to develop products, and the high financial burden for the Member States to purchase these products emphasize the importance of a scrupulous appreciation of treatment value involving all stakeholders at early stage of development, before marketing authorization, and during follow up.

Additional file 1: Table S1. Questionnaire addressed to MetabERN centres. Table S2. Responding centres. Table S3. List of the 28 EMA-approved OMPs for HMDs. Table S4. List of MetabERN centres and number of questionnaires completed

Background Cancer has long inspired fear, but the effect of fear is not well understood; it seems both to facilitate and to deter early diagnosis behaviours. To elucidate fear's behavioural effects, we systematically reviewed and synthesised qualitative literature to explore what people fear about cancer. Methods We searched Medline, Embase, PsycInfo, Web of Science, AnthroSource, and Anthrobase for studies on cancer fear in breast, cervical, and colorectal cancer screening and analysed 102 studies from 26 countries using thematic synthesis. Results Fears of cancer emanated from a core view of cancer as a vicious, unpredictable, and indestructible enemy, evoking fears about its proximity, the (lack of) strategies to keep it at bay, the personal and social implications of succumbing, and fear of dying from cancer. Conclusions This view of cancer as ‘an enemy’ reprises the media's ‘war on cancer’ theme and may affect the acceptance of cancer early detection and prevention messages, since cancer's characteristics influenced whether ‘fight’ or ‘flight’ was considered appropriate.

Supplemental Material for Cancer worry frequency vs. intensity and self-reported colorectal cancer screening uptake: A population-based study by Charlotte Vrinten, Sandro Stoffel, Rachael H Dodd, Jo Waller, Yoryos Lyratzopoulos and Christian von Wagner in Journal of Medical Screening

Table S1. Unadjusted and adjusted odds ratios (OR), 95% confidence intervals (95% CI), and significance values for having never been screened (vs. having been screened at least once) by total CASS (cancer stigma) score for cervical (N = 681), breast (N = 326) and colorectal cancer screening (N = 371). (DOCX 14 kb)

Adaptation of items from the Concerns About Recurrence Scale for use in a general population sample. (DOC 86 kb)

Supplemental material for Cancer worries and uptake of breast, cervical, and colorectal cancer screening: A population-based survey in England by Samantha L Quaife, Jo Waller, Christian von Wagner and Charlotte Vrinten in Journal of Medical Screening

Objective: Communication of cancer information is an important element of cancer control, but cancer fear may lead to information avoidance, especially when coping is low. We examined the association between cancer fear and cancer information avoidance, and tested whether this was exacerbated by psychosocial stress. Design: Cross-sectional survey of 1258 population-based adults (58–70 years) in England. Main outcome measures: Cancer fear (intensity and frequency), perceived psychosocial stress and cancer information avoidance. Control variables were age, gender, ethnicity, marital status and education. Results: A quarter (24%) of respondents avoided cancer information. Ordinal logistic regression analyses showed main effects of psychosocial stress (OR = 1.17, 95% CI 1.07–1.29) and cancer fear: cancer information avoidance was lowest in those with no cancer fear (13%), followed by those with moderate (24%; OR = 2.15, 95% CI: 1.49–3.12), and high cancer fear (35%; OR = 3.90, 95% CI: 2.65–5.73). In the adjusted model, the interaction between cancer fear and stress was significant (OR = 1.14, 95% CI 1.004–1.29, p

Multivariate regression analyses by general cancer worry (‘never’ vs ‘at least occasionally’). (DOC 57 kb)

Specific cancer worry items, factor loadings, and Cronbachâ s alpha for each sub scale. (DOC 36 kb)

Briefing document. Pharmacotherapeutic value and benefit/risk aspects of ephedrine, for the indication: add-on therapy for myasthenia gravis. Briefing document as submitted to the National Health Care Institute and the Medicines Evaluation Board for scientific advice. Parts of the document were subsequently published as [29]. (PDF 1102Â kb)