Interview with Prof. Phillip D.K. Lee: Exploring the Frontiers of Pediatric Endocrinology and Scientific Publishing

On February 20, 2025, the editorial team of Rare Disease and Orphan Drugs Journal (RDODJ) have the honor of interviewing Prof. Phillip D.K. Lee, a distinguished expert in Pediatric Endocrinology. The conversation focused on Prof. Lee's extensive career, his contributions to the field, and his perspectives on current research trends.

Throughout the interview, we explore Prof. Lee's motivations for dedicating his career to Pediatric Endocrinology, the challenges faced in this domain, and the exciting advancements shaping its future. Additionally, Prof. Lee shares insights into high-quality scientific publishing, reflecting on his role as an Associate Editor of RDODJ. We also discuss his thoughts on fostering a journal with international influence and the critical factors that define impactful scientific manuscripts.

Below, Prof. Lee responds to specific questions in this exclusive interview:

Q1: You have been working in Pediatric Endocrinology for many years. What motivates you to keep working in this field?

A: My choice of pediatric endocrinology as a career subspeciality was partially due to my interest in the physiology and pathophysiology of human growth and development. Over many years, I have acquired and continue to acquire knowledge and experience related to disorders of childhood growth and development, as well as other endocrine conditions including diabetes mellitus. Applying this knowledge to patient care while also learning from their conditions has been a primary motivator for my ongoing clinical activities.

Q2: Could you please share about your team's recent research progress?

A: Earlier in my career, I led very active laboratory and clinical research and development teams in both academic and industry settings (i.e., diagnostics and pharmaceutical industry). In my current position, my primary focus has been on clinical care and education.

Q3: Were there any challenges during the research on Pediatric Endocrinology?

A: Physician-conducted academic research has become increasingly difficult due to a variety of factors, including regulatory requirements (some of which are clearly necessary), limited funding, and an emphasis on patient care-generated revenue. These limitations vary by country, region, and institution. Unfortunately, many academic clinical departments have placed less emphasis on research. Physician researchers play a vital role in bridging clinical practice with basic science.

Q4: With your expertise in Pediatric Endocrinology, could you discuss the current research hotspots and the field’s prospects?

A: Two areas are particularly interesting to me: First, the rapidly expanding connection between genetics and endocrinology, with tremendous potential for newborn screening based on genetic testing, diagnostic testing in the clinics, disease risk modeling, and therapeutics. Second, feedback-regulated medical treatments for endocrine disorders, an area still largely unexplored. The closest current example is the treatment of Type 1 diabetes mellitus with semi-closed loop external pump-monitor systems. In the future, treatments might be designed to harness endogenous endocrine physiology, such as ACTH-regulated cortisol delivery for primary adrenal insufficiency and TSH-regulated thyroid hormone delivery for primary hypothyroidism.

Q5: As a scholar with exceptionally high standards for manuscript quality, especially when reviewing articles for the PWS Special Issue you are leading, what do you think are the most important factors that define a high-quality article, whether it is a review or original research?

A: There are many elements that go into writing a defensible and meaningful manuscript. Here are a few elements that I consider in my reviews.
•Purpose: When planning the manuscript, authors should start with a defined purpose that addresses an unmet need, unanswered question, or current interesting issue. The purpose should be clearly stated in the Introduction. For research reports, there should be a hypothesis statement and clearly defined endpoints.
•Structure and Focus: The manuscript should be well-structured, organized, and focused on the selected purpose (or hypothesis/endpoints), avoiding tangential or extraneous details.
•Study Design: In research reports, the study design should be relevant to the hypothesis, clearly described, and statistically supported (when applicable).
• Literature Review: The manuscript should properly describe and reference relevant existing literature, emphasizing primary sources rather than review articles.
•Discussion: For research reports, the discussion should avoid over-interpretation of the data. Some conjecture based on data is expected, but there is always the temptation to go too far. This can detract from the meaning of the report even if the authors intended the opposite.
•Readability: The manuscript should be clear and readable. This includes correct grammar and spelling, well-constructed tables and figures that support the text, avoiding redundancy and internal contradictions.
•Objectivity: Avoidance of undue bias in all aspects of the manuscript is essential.

Q6: As a new Associate Editor of RDODJ, we are honored to work with you. Do you have any suggestions for the future development of RDODJ? What do you think are the most critical factors in building a journal with international influence?

A: As with any medical science journal, RDODJ should commit to being a forum for high-quality topical manuscripts that attract readers and, when appropriate, encourage contributions from them. For any given topic, there should be a balanced mix of invited reviews, perspective and opinion pieces, and original research, guided by an editor experienced in the field. The journal itself should maintain a high level of quality and integrity, safeguarding against becoming a “publication mill”. Distribution and publication costs should be controlled and competitive with similar publications. The journal or its parent organization could also consider hosting symposia or contributing in other ways to international meetings that address rare disorders and offer support for selected organizations working on rare disorders.

Editor: Yuna Ding
Language Editor: Catherine Yang
Production Editor: Ting Xu
Respectfully submitted by the Editorial Office of Rare Disease and Orphan Drugs Journal