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Patient organisations in research
- 1. Impatient Patient Advocacy Ana Mingorance PhD Dracaena Consulting | www.draccon.com @CNSdrughunter “Strategy of patient organisations in driving research from the lab to therapies”
- 3. Dravet syndrome Refractory epilepsy Intellectual disability Behavioral problems Motor/gait problems Sudden death CDKL5 deficiency Refractory epilepsy Intellectual disability Severe motor delay Stereotypies Visual impairment
- 4. 4 Trying to cure a rare disease from the patient side
- 5. Why should patient organizations get involved in research?
- 10. 500 years
- 12. How pharma companies choose a disease
- 13. Feasibility (Science) Business Less profitable Highly profitable Wemightknow howtodoit Notenough knowledge Basic science Overcrowded markets (me-too drugs) Generics Ideal scenario! Fast-movers Opportunistic (e.g. repurposing) How companies choose a disease 13 www.draccon.com
- 14. Feasibility (Science) Business Less profitable Highly profitable Wemightknow howtodoit Notenough knowledge Basic science Overcrowded markets (me-too drugs) Generics Ideal scenario! Fast-movers Opportunistic (e.g. repurposing) How companies choose a disease 14 www.draccon.com
- 15. HOW can patient organizations advance research on their drug disease
- 16. #1 Know where your disease is in the matrix
- 17. Feasibility (Science) Business Less profitable Highly profitable Wemightknow howtodoit Notenough knowledge Rare diseases Rare diseases Rare diseases Rare diseases How companies choose a RARE disease 17 www.draccon.com
- 18. #2 Have a strategy to get into the feasible and profitable box
- 19. Feasibility (Science) Business Less profitable Highly profitable Wemightknow howtodoit Notenough knowledge Rare diseases Rare diseases Rare diseases Rare diseases How companies choose a RARE disease 19 www.draccon.com
- 20. #3 Move your disease higher in the Science axis
- 21. Feasibility (Science) Business Less profitable Highly profitable Wemightknow howtodoit Notenough knowledge Basic science Overcrowded markets (me-too drugs) Generics Ideal scenario! Fast-movers Opportunistic (e.g. repurposing) How companies choose a disease 21 Making your disease much easier to cure
- 22. Rare disease scientific challenges Disease understanding Know what to target Preclinical path Clinical path 22 www.draccon.com
- 23. Moving up your disease in the science axis Only strategic investments Disease education / awareness Diagnosis Cells/tools Animal model generation Animal model validation and distribution Patient registries PROs Outcomes and end-points Broader trial-market collaborations 23 www.draccon.com
- 24. The importance of working together Dravet Syndrome European Federation 2014 – 7 countries 2017 – 15 countries 24 www.draccon.com
- 25. Removing research bottlenecks Over 350 mice shipped Dozens of new laboratories Millions of € into Dravet Made and characterized open-access mouse model 25 www.draccon.com
- 26. Making sure your clinical data is used to advance the field and facilitate drug development Data from almost 300 families 51% is taking a drug Data from almost 600 families Adults no seizure free / burden 2015 2017 26 www.draccon.com
- 27. Creating a community around the disease: Bringing together not only the patient groups Disease-focused forums: • Companies • Academic researchers • Medical doctors • Regulators • Families 27
- 28. #4 Move your disease to the right in the Business axis
- 29. Feasibility (Science) Business Less profitable Highly profitable Wemightknow howtodoit Notenough knowledge Basic science Overcrowded markets (me-too drugs) Generics Ideal scenario! Fast-movers Opportunistic (e.g. repurposing) How companies choose a disease 29 Making your disease more attractive to companies
- 30. 30 Why are orphan indications an attractive market?
- 31. 31 Market saturation Pricing pressures and stricter reimbursement criteria Many “virgin” indications offer low-hanging fruit High clinical unmet need Advances in genetic understanding (disease subgroups) New technologies Drivers of a shift in focus towards orphan www.draccon.com
- 32. 32 A market push and a technology pull
- 33. 33 Companies working on rare diseases have multiple business models
- 34. 34 Different reasons to pursue orphan Market Technology symptom cause Large company with symptomatic drug avoiding competition (opportunistic) Small company with symptomatic drug (business strategy) Large company with disease-targeting drug looking for stepping-stone / de-risk / PoC Large or small company with disease-targeting drug (business strategy) Academic new technology platforms and their spin- offs interestlargeinterestniche www.draccon.com
- 35. 35 So your disease means a different business opportunity for each company!
- 36. Sit down with companies and explain them what your rare disease could mean for them An ideal disease for their technology platform An opportunistic strategy for their approved or shelved compounds (repurposing) An stepping-stone to de-risk their molecule prior to broader indications Part of a cluster of rare diseases that they could pursue with their compound An easy disease for their symptomatic drug (e.g. epilepsy) 36 www.draccon.com
- 37. 37 Time is also important: First, second, and third generations of drugs.
- 38. First symptomatic Superior symptomatic Disease-targeting 38 There are different types of (orphan) treatments Different companies! www.draccon.com
- 39. 39 Your disease represents a different business opportunity for each company and at each stage
- 40. Facilitate the progression of the field from symptomatic to disease-modifying stage Build a large registry that is open and scalable Find more patients (companies need a minimum to get interested) Educate regulators on the disease and best endpoints Facilitate access to research tools and centers/networks Help design more advanced clinical endpoints (data!) 40 www.draccon.com
- 41. Feasibility (Science) Business Less profitable Highly profitable Wemightknow howtodoit Notenough knowledge Rare diseases Rare diseases Rare diseases Rare diseases One goal: getting your field to the feasible and attractive box to get more/better drugs 41 www.draccon.com
- 42. The power of impatient patients: • Have the vision to know where your disease sits in the science/business matrix. • Fill the gaps: build the science and build the business (make it easy and attractive). • Be the glue that comes together to create a joint patient community and that connects all stakeholders. CNSdrughunter http://www.draccon.com/impatient/