1. 1
FUNDING FROM PATIENTS ASSOCIATIONS
• Patients associations & industry?
• Patients organizations have a very strong interest in the
development of drugs for unmet medical needs
• Patients organizations: « mum & dad » initiatives or
prominent associations
• Funding from these organizations: non-dilutive and/or
dilutive (grants, convertible loans, equity)
• Hundreds of fundings observed (US.. and EU)

2. 2
FUNDING FROM PATIENTS ASSOCIATIONS
• Company: Trophos (FR)
• Patients association: AFM-Téléthon (FR)
• R&D programs: ALS and SMA
• Dilutive funding: 3.6M€
• Non-dilutive funding: 19M€+
• Acquired by Roche (2015): 470M€
• AFM-Téléthon gets 43M€ (8M€ upfront + 35M€ milestones)
• 2X

3. 3
FUNDING FROM PATIENTS ASSOCIATIONS
• Company: Aurora Biosciences/Vertex Pharmaceuticals (US)
• Patients association: Cystic Fibrosis Foundation (US)
• R&D program: Cystic Fibrosis
• Dilutive funding: -$
• Non-dilutive funding: 150M$ (royalty-based grants)
• Cystic Fibrosis Foundation’s rights sold to Royalty Pharma (2015)
• For a cash payment of.. 3.2B$!
• 22X!

4. 4
FUNDING FROM PATIENTS ASSOCIATIONS
• Benefit: access to funding/non-dilutive funding from non-financial
organizations (different rationale)
• Benefit: cost of money
• Benefit: not only money, but support from patients (enrollment in
clinical trials, discussions with regulation agencies and payors)
• Challenge: identify relevant associations
• Challenge: industry vs. patients organizations cultures
• Challenge: set up syndications (few associations have deep
pockets)

5. 5
FUNDING FROM PATIENTS ASSOCIATIONS
• Patients associations fund drugs, diagnostics & devices
• Patients associations should be included in every funding
campaign, particularly in rare diseases, but not only
• Contact: Christian Girard, Consultant
christian@orphandrugsindustry.com