What is it?

The leader’s forum for the Orphan Drugs space, where executives who are driving forward the orphan drugs industry gather to shape the future of rare disease drug development, commercialization and patient engagement.

This is a 40-person Chatham House Rule, formatted forum in which industry leaders discuss and shape the future of their sector behind closed doors. They are hollow-square formats and not your standard one-dimensional meeting format meaning all executives engage in meaningful strategic and sector debate and discussion. The forums will address the key industry challenges head on, providing genuine outcomes for the executives and the sector as a whole.

The 40 seats will be allocated with no fewer than 36 executives and KOLs, with the remaining seats taken by  Forum Co-Hosts that may include 1 Pharma, 1 Global Advisory, 1 Law Firm, 1 CRO and / or 1 Investor. The GEO breakdown will roughly be 40 US, 50% UK and EU and 10% RoW and as we are focused on  diversity, we are looking to ensure that at least 30% of the attendees are women Executives.

who is it for? 

CEO, CFOs, CBOs and executives of companies developing solutions to treat rare disease around the world . It is not for scientists, researchers and technical job titles in the orphan drugs space.

the case studies 

CEOs and/or event partners put forward case studies – either success stories or current challenges and use the group as “consultants”. Each case study follows this format:

Headline – short and enticing
Nutshell – a concise statement of the issue
Background – the players, the issue, what was at stake, etc
What Was Tried? - what worked and what didn’t
Questions - clarifying questions from the group.
Peer Consultation
Review - what are you taking back with you? what will you try?

the general discussion

All the attendees in the room will be asked: “If you could have the answer to one question, that would fundamentally help change the way your business runs, what would it be?"

The questions will be scored– the highest score going to the question asked by the most people, and that becoming the focus of the discussion.

General notes:

  • Time will be managed carefully, with plenty of time for discussion
  • Each speaker will be asked to communicate a clear message & solicit specific feedback
  • The moderator will be briefed to guide the discussion to touch on all participants' concerns

Executives can explore in this session:

The Industry and Business Agenda
Building successful clinical development and research teams accommodating various product development stages to advance to market approval, nurturing and growing orphan drugs businesses whilst retaining the patient centric approach, reimbursement in rare diseases

The R&D Agenda
Building successful clinical development and research teams to accommodate various product development stages to advance to market approval

The Investment Agenda
Creating shareholder value in orphan drug companies, overcoming investment challenges for early and growing private companies

The Future Agenda
Ensuring global collaborative drug discovery and innovative therapies for known and new rare diseases


Are you interested in a seat at the table and would like to be considered for an invitation to attend the Orphan Drugs Forum?