Remco de Vrueh. Driving partnerships from idea to success

Transcription

1 Remco de Vrueh Driving partnerships from idea to success Rare Disease Symposium: the rare disease patient in 2030

2 Outline 2 Current status Orphan drug development Public-Private Partnerships (PPPs) Lygature - case study: from idea to success Partnerships in the area of rare diseases

3 EU and Dutch Orphan Drug (OD) pipeline Fully adopted by biopharmaceutical sector 3 Dutch orphan designations EU orphan designations No of Dutch companies Two Dutch ODs approved: Ruconest (2001 > 2010) Glybera (2004 > 2012)

4 Orphan products From exception to mainstream FDA approvals New Chemical Entities Orphan Drugs Kaitin KI, Manocchia M. The new drug approvals of 1993, 1994, and 1995: trends in drug development. American journal of therapeutics Jan 1;4(1): Owens J Drug Approvals: Finding the Niche. Nat Rev Drug Discov. 2007;6(2):

5 Origin of new medicines in the EU ( ) SMEs: Engine behind OD development Large: Crossing the finish line Originator % of OD % of non-od Large/Intermediate-sized SME 61 18,5 Academic/public bodies /partnerships 17 26, Large Intermediate-sized SME Academic/public bodies/ppps Originator MA holder Private-Private collaboration Lincker H et al. Regulatory watch: where do new medicines originate from in the EU?. Nature Reviews Drug Discovery Feb 1;13(2):92-3.

6 6 "The way forward in multidisciplinary research is to engage in predictive, personalized, pre-emptive and participatory medicine. For the creation of the optimal innovation climate that would allow for such a strategy, public-private partnerships have been proposed. FORMER NIH DIRECTOR ELIAS ZERHOUNI

7 University & industry have been partnering for decades 7 Informal engagement Formal engagement Teaching & training Policy initiatives, like US Bayh-Dole Act (1980) Commercialisation Patenting Licensing Academic entrepreneurship Collaborative Research Contract research Consulting Publishing Conferencing Networking Facility sharing Adapted from Kutvonen A, et al. University-industry collaboration and knowledge transfer in the open innovation framework. In University-Industry Interaction Conference: Challenges and Solutions for Fostering Entrepreneurial Universities and Collaborative Innovation 2013 (pp ).

8 Multi-stakeholder PPPs on the rise Source No of PPPs/year 8 54 Lim (2014) No of PPP scientific articles/year 7 44 De Vrueh et al. submitted M. D. Lim, Consortium sandbox: Building and sharing resources. Sci. Transl. Med. 6, 242cm6 (2014). Patient organizations Biotech companies Academic groups Medtech companies Charities Pharma companies Government NGO s Regulatory bodies So is the diversity of partners

9 Lygature drives partnerships from idea to success 9 Building partnerships Idea generation Contract negotiation Launching partnership Program management Creating sustainability Medical solution

10 Open innovation = sharing Shared infrastructure Shared resources Shared interests Shared responsibility PPP example TraIT IMI - European Lead Factory IMI - Adapt-Smart Pediatric praziquantel consortium Aim Deliver an open access IT infrastructure for translational research Deliver innovative drug discovery starting points through a collaborative public-private effort Facilitate and accelerate the availability of adaptive pathways Develop and provide a formulation for treating schistosomiasis in preschool-aged children Key facts & results > 2700 users > 200 running projects 98 screening programs >3500 hits Agenda setting in regulatory science Payers involved Two formulations in development Precompetitive Product development

11 11 Product development for young children Corporate 1 april Presentation 2017

12 Schistosomiasis Global disease burden Courtesy of Jennifer Keiser, Swiss TPH Neglected High medical need WHO: Status of schistosomiasis endemic countries 2012: last accessed 7 April 2014

13 The Pediatric Praziquantel Consortium Non-profit R&D consortium, established in 2012, to develop and make accessible an orally disintegrating tablet (ODT) formulation for treatment of preschool-aged children with schistosomiasis. International Expert Panel (World Health Organization) as observer Leadership Commitment Building trust Agreements/Governance ( Announced today: US$ 4,9M in support of Phase III program 13

14 Part of the solution Praziquantel (PZQ) An oral anti-helminthic drug: current gold standard of treatment (>6 years of age) Developed in the 1970s, proven to be safe and efficacious Current drug is a racemic mixture of levopraziquantel (L-PZQ) and dextropraziquantel (D-PZQ) Not approved or suitable for very young children (3 months - 6 years) Drug repurposing

15 Results New PZQ tablet formulations Orally Disintegrating Improved taste Standard processes Cesol 600 mg Current commercial tablet PZQ new ODT 150 mg tablet Not an NCE, still true innovation

16 Status Clinical program Resource-limited settings: Dealing with uncertainties Planned Phase III confirmatory trials (TbD) Ongoing Phase II PK/PD dose finding Study (Ivory Coast) Completed Taste Study of the new ODTs in African children (Tanzania) Completed Two Phase I Bioavailability studies (South Africa) 16

17 Keys for success Communication & stakeholder engagement Consortium Team and Board representatives together with African regulatory experts in Geneva (2014) Overcoming clinical, regulatory and other challenges with external support

18 Access Unchartered territory Making a sustainable business case Embrey, M. Managing Access to Medicines and Health Technologies. Manag. Sci. Heal. (2012)

19 Partnership opportunities for Rare diseases 19 Clinical trial design Use of real world data Registries Health Technology Assessment methodology Regulations Joint development programs Stuurgroep Weesgeneesmiddelen

20 To find solutions for and with patients 24

21 21 Lygature takes its name from the ligatures used to bring components together, in typography, music, and medicine. events.lygature.org