Making a Difference in Rare Diseases: Optimising KOL Identification and Engagement

This report delves into the pivotal role of Key Opinion Leaders (KOLs) in the realm of rare disease drug development. It provides pharmaceutical professionals with a comprehensive analysis of the strategies for identifying and engaging with KOLs, who are essential for offering clinical insights and navigating regulatory landscapes. Gain insights into overcoming the challenges of finding and collaborating with these experts, leveraging tools and approaches to enhance engagement, and optimising the value KOLs bring to treatment pathways. The report also explores best practices for building and maintaining relationships with KOLs, ensuring that their expertise is effectively integrated into the drug development process.

Key Questions Answered:

• 1. Why are rare disease KOLs crucial in drug development?
• 2. What challenges exist in identifying rare disease KOLs?
• 3. How can these challenges in identifying KOLs be overcome?
• 4. What tools and approaches are effective for finding rare disease KOLs?
• 5. What tactics are successful in collaborating with rare disease KOLs?
• 6. How can medical affairs optimise the value brought by rare disease KOLs?

Key Companies:

Partial List of Participating Experts:

• Former Medical Consultant at BridgeBio Pharma, based in Italy
• Former UK country medical director at pharmaceutical companies with rare disease treatments, based in the UK
• European Medical Head at a pharmaceutical company in the top 50 by global sales in 2023, based in the UK
• Former Lead, Global Early-Stage Medical Affairs, Mitochondrial and Rare Disease at Astellas Pharma, based in the UK
• A Senior Manager, Medical Affairs, working at a global biopharmaceutical company based in the US
• A Senior Medical Director at a commercial-stage biopharmaceutical company based in the US
• A Director, Medical Affairs, working in the rare disease area at a biopharmaceutical company based in the US
• A Senior Global Medical Director at a pharmaceutical company in the top 10 by global sales in 2023, based in the US

Methodology

Dossier reports are developed from independent and concise analysis derived from original industry surveys and in-depth interviews with industry thought leaders. Reports analyse in detail significant developments and market trends that pharma executives need to understand if they are to manage the opportunities and challenges that lie ahead. The focus of each report is defined by a review of the secondary literature and any knowledge gaps identified. Based on this initial research, an evidence-based and expert-informed discussion guide is developed to ensure the research answers the most critical questions. Additionally, robust screening criteria are established to ensure that those interviewed are qualified in terms of their experience, knowledge and stature to speak on the topic.

What makes our reports different?

FirstWord Reports, a trusted industry leader with an exclusive focus on the pharmaceutical sector, delivers in-depth, actionable insights for biopharma professionals and decision-makers. Our deep industry knowledge enables us to provide relevant and valuable insights that help you identify emerging trends and navigate complex challenges effectively. Backed by extensive research and independent, impartial insights from leading experts and KOLs, our reports deliver the accuracy and reliability you need. With exclusive access to interviews and data unavailable elsewhere, along with ongoing market monitoring, we give you a comprehensive view of market dynamics. Covering over 40 dynamic disease areas and providing physician intelligence, including KOL insights and quantitative physician surveys, as well as industry expert views on issues in medical affairs, digital health, sales & marketing, market access, and other areas, our reports empower you to make smarter, data-driven decisions and maintain a competitive edge in a fast-moving industry.