Michael Skynner - 'Aunando esfuerzos para el futuro de la investigación en enfermedades poco frecuentes'
•
1 like•680 views
'Aunando esfuerzos para el futuro de la investigación en enfermedades poco frecuentes'. Éste fue el lema de la jornada que tuvo lugar el 27 de abril de 2015, en la Fundación Ramón Areces. Organizada en colaboración con la Fundación Pfizer y el Instituto de Salud Carlos III, reunió a algunos de los principales expertos en Enfermedades raras para analizar el estado de la investigación.
Recommended
Recommended
More Related Content
What's hot
What's hot (20)
Viewers also liked
Viewers also liked (18)
Similar to Michael Skynner - 'Aunando esfuerzos para el futuro de la investigación en enfermedades poco frecuentes'
Similar to Michael Skynner - 'Aunando esfuerzos para el futuro de la investigación en enfermedades poco frecuentes' (20)
More from Fundación Ramón Areces
More from Fundación Ramón Areces (20)
Recently uploaded
Recently uploaded (20)
Michael Skynner - 'Aunando esfuerzos para el futuro de la investigación en enfermedades poco frecuentes'
- 1. Pfizer confidential; Not for distribution Importance of Collaboration in Rare Disease Research Michael Skynner (Head Rare Disease Alliances) 27th April 2015
- 2. Confidential – Internal Use Only RDRU formed in 2010 - Collaborative approach through connections with patient groups, community, academia RDRU formed in 2010 - Collaborative approach through connections with patient groups, community, academia Close-knit end to end integration from research to commercialization helps to foster a robust pipeline Close-knit end to end integration from research to commercialization helps to foster a robust pipeline Pfizer - Positioned to make a difference for patients │ 2 * Orphan designation in the EU and/or U.S. Serving thousands of patients with a portfolio of 22 products approved to treat rare diseases worldwide* Serving thousands of patients with a portfolio of 22 products approved to treat rare diseases worldwide*
- 3. Confidential – Internal Use Only Pfizer’s research focused on areas of health needs │ 3 Hematologic Disease Internal Focus Neuromuscular Disease Pulmonary Disease Collaborative Focus Cystic Fibrosis Duchenne Muscular Dystrophy Haemophilia Sickle Cell Disease
- 4. Pfizer confidential; Not for distribution Collaboration is key in rare disease
- 5. Pfizer confidential; Not for distribution What are the challenges? Access to breaking science New model for DD Disease diversity Expertise
- 6. Confidential – Internal Use Only UK is a major centre for rare disease research Bio-cluster larger than Boston 4/6 universities in the world’s top 10 $1bn grant funding for rare disease ($400m 42% total Wellcome Trust funding) ~20m patient catchment Location of majority of tertiary rare disease referral centres ~60% of UK’s rare disease publications Collaboration, where should we start? Pfizer Rare Disease Consortium 6 www.rarediseaseconsortium.co.uk
- 7. Confidential – Internal Use Only Master agreement signed: Dec 2013 Operating model: Joint research team (co-chair) Pfizer funded post-docs Access - Pfizer talent, tools, technology Principles: Complimentary skill sets Shared risk / rewarding success “Making a Medicine” Working Framework: Research Plan Option, Milestones and Royalties IP terms RDC - Model for interaction Pfizer Rare Disease Consortium 7 www.rarediseaseconsortium.co.uk
- 8. Confidential – Internal Use Only Expansion of RDC in UK…and beyond? 8 QS 2013 Rankings
- 9. Pfizer Confidential – Do Not Distribute – Discussion Purposes Only Science Call Themed science areas Access academic creativity & novel science Road show One to one meetings Honest feedback, easy to have the conversation Principle investigator/Consortium Name & Title Institute/Sub-division/Address Contact details (email/phone) Other investigators and their affiliations Disease Background Disease Name & Genetic Cause (if know) Therapeutic Area (Call Area) Incidence /prevalence/geographical bias Overview of disease, current treatments and availability of clinical scales or pharmacodynamic endpoints List clinical research centres / patient groups / charities Proposal Scientific proposal: include any available supporting pre-clinical or clinical data also highlight the major scientific risks and expertise gaps (maximum 500 words, or one page) Requirement from Pfizer: approximate the skills/resources/ budget/timelines requested (NB this is non-binding and will be accurately reconciled if application is accepted) Brief overview of your laboratory and its capabilities relevant to this application Identify any access to patient material Other relevant information Intellectual Property relevant to this application Identify any Conflicts of Interest List a maximum of three relevant publications (NB To be completed by Pfizer) Resources required/timings Cost/Location/Sourcing Reviewer comments and actions Two page Expression of interest Simple rapid non-confidential preliminary application Business plan Full proposal – research agreement Confidential, goal driven science, flexibility to incorporate 3rd parties RDC process and principles
- 10. Pfizer Confidential – Do Not Distribute – Discussion Purposes Only What have we achieved? Class of 2014 First call Neuromuscular and Haematology (>400 applications) 10 www.rarediseaseconsortium.co.uk
- 11. Confidential – Internal Use Only Collaborating across Rare Disease Ecosystem │ 11 New Drugs to Patients in Need New Drugs to Patients in Need Biotech – Selected Examples Academia Genetics Medicine Institute Patient Advocacy & Foundations
Editor's Notes
- Morning and thank you Talk about the importance of collaboration in Rare Disease research Convince you of three things today That rare disease research requires a different paradigm That the paradigm required is collaboration And that that collaborative paradigm can work to mutual benefit
- Before I do I want to say a few words about Pfizer rare diseases People don’t automatically think about Pfizer as a RD company, but has a proud history and a portfolio of 22 approved medicines, about 50% in non-oncology indications and in 2013 was ~2 in the world by sales. >4 years ago this effort was enhanced by formation of a dedicated research unit to bring forward new medicines in this area Unit ~60 biologists, initially based initially in Boston, was formed to punch above their modest numbers by crystalysing and evolving a highly networked and collaborative model for DD. Not only that but to also develop close links internally with the commercial teams in the business units involved in bringing medicines to market to ensure end to end integration and rapid delivery of new medicines
- Heard a little about the complexity of rare disease and so initially we have focused our research efforts in three main areas Haematology Many will be familiar with our existing franchise of factor replacement products (8&9 for Haem A and B) But also with a n eye to the future the deal signed in Dec last year with Spark to develop future therapies GT Haem B But also a growing portfolio of developmental drugs for SCD, which is a dreadfully underserved disease with the last new medicine HU being developed in in 1869 Neuromuscular Main focus in on DMD and at end of last year unit proud initiated our first phase 2 clinical trial in this disease with an anti myostatin antibody, negative regulator of muscle growth Third area is Pulmonary, specifically in CF where we have a significant research effort. Where as Haem and NMD are examples of disease areas where we have internal efforts CF is an example of an area where we have chosen to partner, in this case with the CFF. So what does that mean, well firstly at face value CFF provide the finance to fund the work…so money But collaboration brings far more than that as at quarterly JSCs gain access to advice and guidance driven from detailed understanding of patient needs breaking science key researchers in CF field
- So to pick up on some of the themes from the film, what are the challenges in RD Probably the major challenge working in the RD space comes from the huge diversity that exists across the 7,000 orphan conditions diversity can come in many forms: from age of onset (some devastating diseases of infants like, some like HD impacting adult patient population) different frequencies of disease (from 1 in the world to 500K) importantly, each with a different underlying cause and different pathophysiology and clinical course The consequence of this disease diversity is that we can’t expect to be experts in house to all the different diseases. It simply isn’t efficient or cost effective to employ expert in all these diseases, so how can we operate efficiently in this area Much more efficient strategy is to Need to identify the expertise externally, where ever that may be in the globe, and engage with those physician scientists (as these are the people who know the disease, these are the people who see the patients and critically they understand the unmet medical need and what the patient needs a therapy to deliver) Having identified these key experts we then need to access breaking science to address that unmet medical need here i have shown logos for two high profile science journals Historically this would be the traditional point of entry for pharma companies to start DD programs However (as we have already heard) there is a need to accelerate the rate of RD drug discovery the way to do this is to engage with academia earlier, at the point of idea inception not idea publication as this allow access to novel science at its earliest point, will save years from the development timeline and provide exclusivity on targets to reduce competition Finally the last challenge is to build the networks that are crucial to effectively tackle these rare diseases. that was so well articulated in the animation to help deliver these future medicines
- So our solution to the challenge is RDC. This is an agreement with GMEC (Global Medical Excellence Cluster) which is a consortium of the major leading universities in South of England (list) It was attractive to access these universities because (read)
- At the heart of the Agreement is the ability to access breaking and novel science in these institutions in RD space. Having done so we uniquely place that investigator at the centre of what we do and wrap a team around them Access to three Ts etc
- Whilst GMEC are important founding members, the RDC was always designed with expansion in mind and in the last 18 months we have expanded to ~19 UK universities and have initial inroads outside the UK into the US and beyond. These cover the highest quality institutions in the UK as shown by QS university rankings from 2013.
- How does RDC operate, essentially a vehicle to allow us to crowd source solutions to problems and to identify novel science to invest in built on 3 principles scientific engagement personal interaction rapid and honest feedback
- RDC isn’t just talk, we have delivered numerous funded and now ongoing scientific projects We were staggered when we conducted our first call at the level of interest and desire to collaborate But its not just a numbers game, what can RDC do differently? So its as much about the journey as the destination…..now I’d like to highlight 2 unexpected benefits of a collaborative model with 2 vignettes from our first call.
- Finally, where does collaboration take us and why are we doing DD this way Well we collaborate with many different groups and in many different ways….. but fundamentally the purpose is singular To bring new drugs to patients that need them and collaboration in, my view is the only way to do this effectively thankyou