Nothing About You Without You

Monday, 18 February 2019

Contributed by Camilla Krogh Lauritzen, Chief Patient Officer, LEO Pharma 


28 February is Rare Disease Day. Hardly any surprise to anyone who is committed to changing the status quo for the millions of people affected by one or more of the 7000+ rare diseases. But then again, it may be news for many to hear how we actually work to change the status quo. To mark Rare Disease Day 2019, Camilla Krogh Lauritzen Chief Patient Officer LEO Pharma, explores the LEO Pharma Research and Design partnership (R&D) between experts and disease experience experts.

Disease experience experts (aka ‘patients’) are increasingly involved in medicines R&D, advising on, for example, the design of clinical trials and related strategies. Much so, as reflected in the global patient movement mantra ‘Nothing about us without us’ a mantra that IAPO backs. People afflicted by diseases - or have loved ones who are - want to be involved from the very start; at the level of setting the research priorities.

To that end, LEO Pharma hosted the Game Changer Summit in August 2018 to understand what ‘great’ looks like – and how we get there. We invited ten global and regional patient leaders, including IAPO’s CEO, Kawaldip Sehmi, to share their vision, perspectives and advice. Click here to see a short film on what some of the Summit attendees had to say.

While collaborating with key patient opinion leaders and their communities at the very early stages of medicines R&D is not exactly ‘business as usual’ for pharma, the expectation and ask from the patient leaders at the Game Changer Summit was crystal clear: Engage us and our communities from the get-go! 
On that day in August, LEO Pharma’s rare disease commitment ‘Nothing About You Without You’ was set in stone. 

So, what does this commitment entail? It entails that insights and advice from disease experience experts are an integral part of all key decision-making in the rare disease R&D efforts at LEO Pharma (Fig 1).

We would like to share one example regarding a major business decision to enter a R&D business-to-business partnership. In the spring of 2018, LEO Pharma was looking into partnering with PellePharm, a San Francisco-based biotech company, as we believed that by joining forces, we would be able to drive a transformative change for people affected by Gorlin Syndrome. The latter is a rare disease that is caused by a mutation in the tumor suppressor gene PTCH1. One of the most feared and burdensome consequences, seen from a patient perspective, is the repeated development of basal cell carcinomas, i.e. skin cancers.

Having consulted scientific and medical experts, as per ‘business as usual’, to confirm the scientific and medical rationale related to the molecule at the center of the partnership in spe, LEO Pharma turned to the patient community and asked for their advice. We did so, in order to understand to what extent people with Gorlin Syndrome would see the implied potential future solution, as one that would be value-adding.

Needless to say, neither any of the specifics related to the actual molecule, nor the name of the partner in spe were disclosed to the Gorlin disease experience experts at that time; rather we discussed the principles around what a great solution would look like, as seen from a patient perspective. Fortunately, there was a match between this and the profile of the solution that we were in discussion with PellePharm to partner on.

Upon having entered the partnership with PellePharm in November 2018, our CEO, Gitte Aabo, invited some of the disease experience experts that had served as key advisors to join us in Copenhagen, so that she could share the news with them directly; they became the first external parties to know that the partnership with PellePharm was now a reality. We captured this in a small film that will be released on World Rare Disease Day. With this film we wish to highlight how LEO Pharma’s dedication to dermatology goes beyond the skin!