Original Research: Expert Opinion on Orphan Drugs
A guide for authors submitting to the Expert Collection
Contributions are welcomed in the form of original research papers reporting the results of early or late stage clinical investigations or HE/OR data on orphan drugs and studies in the treatment of rare diseases.
Expert Opinion will only consider work that has not been previously published in full. Abstract, poster or oral presentations do not constitute prior publication, but should be mentioned in the covering letter and details included as a footnote on the manuscript title page.
All manuscripts must be accompanied by a covering letter and an electronic version of the Disclosure Form signed by the principal author(s). The covering letter should include all of the following information.
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The name and contact details (telephone, fax, postal and email addresses) of the corresponding author who will deal with the comments from reviewers and approve final proofs.
A statement that the contribution represents original work that has not been previously published or simultaneously submitted for publication elsewhere.
A statement that the manuscript has been read and approved by all the authors, and that all the conditions as previously stated by the ICMJE have been met.
The body providing explicit ethical approval of the work reported should also be stated.
A statement of financial or other relationships of a declarable nature (i.e., commercial associations that might lead to a conflict of interest), including disclosure of sources of support in the form of sponsorship, grants, materials (drugs) or equipment, and editorial or manuscript support. In case there are no disclosures of a declarable nature, or acknowledgements for financial or editorial support, this should be clearly stated.
All complete submissions will be acknowledged by the Editorial Office upon receipt. Please note that manuscripts without a covering letter or a Disclosure Form will not be considered to be ‘complete’ submissions.
Manuscripts submitted should be in English, using US spellings, and written for an international, general medicine readership. Where national or regional issues are discussed, the international context should also be considered. When a licensed drug or device is being discussed outside its licensed indication, this must be made clear to the reader.
Manuscripts should be double-spaced with numbered pages. Keep the layout as simple as possible – it will be set to house style during the production process.
Every article must contain
All articles should have a concise, informative title that contains no brand names. In addition to the title itself, the title page should also have the name(s) and initials of all the authors and their institutional affiliation(s). The name of the corresponding author and their mailing address should be given in full, including email, telephone and fax numbers. Should any of the material contained in the paper have been previously presented at a meeting, the full name, location and inclusive dates of the meeting should also appear on the title page. Authors are to avoid using Trade names in their titles, and are encouraged to have non-promotional titles for their articles.
The second page should contain a brief, structured abstract of the paper (no more than 200 words) summarizing the main facts, findings and principal conclusions. Suggested headings:
Background (including the reason for the study).
Research design and methods (including study population and setting, study blinding, comparators, dosage, treatment regimens and durations, efficacy and safety issues); Main outcome measures.
Results (both efficacy results and adverse events should be given).
Conclusions (qualified by any key limitations). No references are to be cited in the Abstract. Please bear in mind that the Abstract needs to accurately reflect the content of the article and that the aim of the Abstract is to draw in the interested reader.
Trial Registration (if a clinical trial): e.g., The trial is registered at ClinicalTrials.gov (CT.gov identifier: NCT02263066).
This should state the clinical relevance and background to the study, its rationale and purpose.
4–6 keywords, listed in alphabetical order, are required to assist indexers in cross-referencing. The keywords will encompass the therapeutic area, mechanism(s) of action, key compounds and so on.
Body of the article
This should include the following sections: introduction; patients and methods; results; discussion; conclusions.
Patients and methods
This should contain details of the study population and setting, subject selection (inclusion/exclusion criteria), methods of randomization and blinding, and efficacy and safety measures. The study design and statistical methodology should be described, with justification for the choice of analysis and sample size given; CONSORT guidelines should be considered where appropriate. All materials should be identified precisely, with drugs referred to by their generic names (proprietary names, if required, should be given in parentheses along with the company name and country of the manufacturer), and with dose and routes of administration. The ethical approval procedure followed and the name of the ethics committee should be stated. Indicate how adverse events were determined (and by whom) and indicate if/how compliance was measured. (Authors are reminded to ensure that they follow the ICMJE requirements when dealing with privacy and informed consent from patients – see Section II.E.1.of the ICMJE requirements document). Trial registration numbers with full details should be in included.
Use should be made of tables and figures to help with the clear presentation of results data. The sample size of each data point should be shown, with p-values and confidence intervals quoted for significant findings. Any data not included in the analysis (including patients withdrawn from the study) should be detailed. Details of data on efficacy and adverse events should be provided in a balanced fashion.
This will essentially be a discussion of the results and experimental data collected. The section should include implications of the findings and their limitations, with reference to all other relevant studies and the possibilities these suggest for future research. In addition, the discussion affords authors the opportunity to discuss the developments that are likely to be important in the future and the avenues of research likely to become exciting as further studies yield more detailed results.
This section must summarize the paper, with a concise statement of the clinical implications of the study results. Ensure that extrapolations are reasonable and that conclusions are justified by the data presented. Indicate if the study design can be generalized to a broader patient population.
Acknowledgements: This must include any declaration of interest by the authors, including grants, fellowships or any commercial assistance or financial sponsorship received. It should also list any affiliation(s), organization(s) or entity(ies) that are relevant to the work reported. Some or all of this information may be published at the discretion of the Editor. Any contributions to the research, data analysis or assistance in manuscript preparation must also be acknowledged in this section. Finally, if any trial registration information and/or a trial registration number are available for the study, this should also be mentioned in the acknowledgements section.
Author contributions: Authors should briefly highlight which author(s) were involved in the conception and design, or analysis and interpretation of the data; the drafting of the paper or revising it critically for intellectual content; and the final approval of the version to be published; and that all authors agree to be accountable for all aspects of the work.
References: Full references to relevant material cited in the text should be provided. References should be comprehensive, accurate and up-to-date: wherever possible, please use primary references, and as far as possible, avoid the use of unpublished references (such as ‘Data on file’ or ‘Poster’). Reference to unpublished data should be kept to a minimum and authors must obtain a signed letter of permission from cited persons to use unpublished results or personal communications in the manuscript.