Drug Evaluation: Expert Opinion on Orphan Drugs
Scope: Drug Evaluations will present an evaluated overview of the clinical experience with the biologic compound. Although discussion should encompass basic pharmacology and pharmacokinetics, the primary focus of the review should be the clinical efficacy of the compound. For launched compounds the information should be limited to approved indications and avoid off-label discussion.
Peer review: All articles are subject to peer review by 2-3 independent reviewers. As the Drug Evaluations include in-depth analysis of a single compound, the manuscript will also be sent to the company responsible for marketing or developing the drug to check the accuracy of the data. This is for a technical check only and the final editorial decision is based entirely on the recommendation of the independent peer review. Comments remain confidential and are shared only with the corresponding author or submitting party.
Word limit: The word limit for Drug Evaluations is 3,000 words (not including tables, figures and references).
Every article must contain:
Title: Should be concise but informative, and contain no brand names. Titles should be impartial and non-promotional.
Authors’ names and addresses: Including address, academic qualifications and job titles of all authors, as well as telephone number, fax number and email address of the author for correspondence on a separate cover sheet as the peer reviewers will be blinded to the authors’ identity. Please note that only the address of the first author of the article will appear on Medline/PubMed, not necessarily the corresponding author.
Structured abstract (maximum 200 words): The aim of the abstract is to draw in the interested reader and provide an accurate reflection of the content of the paper. We therefore request the following structure is followed for full-length review articles:
- Introduction: Authors are required to describe the significance of the topic under discussion.
- Areas covered: Authors are required to describe the research discussed and the literature search undertaken.
- Expert opinion: Authors are required to summarise briefly their Expert opinion section.
References must not be included in the abstract.
Keywords: A brief list of keywords, in alphabetical order, is required to assist indexers in cross-referencing. The keywords will encompass the therapeutic area, mechanism(s) of action, key compounds etc.
Introduction: Incorporating basic information on disease incidence and prevalence, unmet medical need and present treatment guidelines (highlighting regional variations where appropriate).
Body of the article:
- Overview of the market:
- What are the unmet needs of currently available therapies?
- Which competitor compounds/classes of compounds are in the clinic/late development?
- Why the indication is considered rare, information on epidemiology and prevalence.
- Introduction to the compound including history of orphan drug designation: Chemistry; Pharmacodynamics; Pharmacokinetics and metabolism.
- Clinical efficacy: (Phase I studies); Phase II studies; Phase III studies; Post-marketing surveillance.
- Safety and tolerability
- Regulatory affairs
- Conclusion: An analysis of the data presented in the review.
Expert opinion: To distinguish reviews published in the Expert Opinion journals, authors must provide an additional section entitled Expert opinion. This section affords authors the opportunity to go beyond the conclusion and provide their interpretation of the data presented in the article. Authors should answer the following:
- What, if any, improvement does the drug hold over other therapies?
- In developing this drug, what are the key lessons for R&D scientists in this field? Could these be applied to other rare diseases/niche indications?
- What, if any, impact is this drug likely to have on current treatment strategies?
- How likely are physicians to prescribe the drug?
- What data is still needed?
- Where is drug likely to be in five years’ time?
Please note that ‘opinions’ are encouraged in the Expert opinion section, and as such, referees are asked to keep this in mind when peer reviewing the manuscript. However, authors are requested to focus their discussion on approved uses of the drug.
Drug summary box: To provide the reader with a visual summary of the paper, each paper should include a Drug summary box including basic data on the drug as follows:
If you are unable to provide this information, editorial support will be provided.
References: A maximum of 100 references is suggested. Ensure that all key work relevant to the topic under discussion is cited in the text and listed in the bibliography. 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.
Annotated bibliography: Important references should be highlighted with a one/two star system and brief annotations should be given (see the journal’s Instructions for Authors page for examples and for a more detailed description of our referencing style).
Figures and tables: Up to five figures and five tables are permitted. For further information on tables and figures, please see our formatting guide.