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Neil Dugdale sobi crdn summit 2017
This document summarizes a presentation given by Neil Dugdale of Sobi (Swedish Orphan Biovitrum) at the 2017 Cambridge Rare Disease Network Summit. The presentation discusses Sobi's work in rare diseases, including developing orphan drugs, partnering with patient advocacy groups, and donating factor therapy to expand treatment access for hemophilia in developing countries. Sobi aims to pioneer new approaches to rare disease management through multi-stakeholder engagement and community co-creation.
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Neil Dugdale sobi crdn summit 2017
- 1. Cambridge Rare DiseaseNetwork 2017 Summit Neil Dugdale BSc (Hons) DipM MCIM General Manager United Kingdom and Republic of Ireland NP-2981 Date of preparation: October 2017
- 2. 2 Guided by thepatient journey: Many different “customers” along the way Symptoms Treatment Data Capture Outcomes Reporting Ongoing Mgt. Monitoring Awareness Testing Health System Access Label Updates Diagnosis + Staging Revised Treatment Guidelines Physicians Publications Identification of Unmet Medical Need Diagnosis + Treatment Outcomes DevelopmentDisease Management Optimizing Care & Advancing the Science Centres of Expertise
- 3. Developing orphan drugs:a case study in HT-1 1982 Bottle Brush plant 1989 Prof. Sven Lindstedt 1993 2005 2002 4 yr. survival 29% vs 93% 1992 Patient voice inspires our commitments 2016 International filings / oral suspension / 20 mg 2012 Pioneering Corporate-Academic-Public Collaboration Our Legacy in Rare Diseases 3
- 4. 2020 (est) Gout 2019 (est) Stills 2015 SJIAAUS 2012 Paediatric Investigation Plan Approval (EU) Filing US (NOMID) Filing EU (CAPS) FDA Approval NOMID Developing orphan drugs: a Sobi case study 4 2010 A letter from a physician Orphan Drug Designation US FDA pre-meetings 2011 Scientific Advice EU 2013 EC Approval CAPS 2002 RA 2000 2005 2010 2015 2020
- 5. Co-Creating with Patientson primary packaging & administration creates a better result 5
- 6. 6 Introduction to Sobi&Co “Sobi&Cois a multi-stakeholder engagement programme to obtain real- world insights, leading to the co-creation of ideas and solutions with the haemophilia community that will address patient/carer unmet needs” Sobi´s Vision: A community where people with haemophilia are free to make real choices in their treatment and the way their condition is managed Sobi´s Mission: To re-join the haemophilia community with fresh eyes and as a result develop an innovative approach to haemophilia
- 7. 7 Key themes basedon the identified unmet needs Transitioning Taking responsibility Physio & physical activity Psychosocial support • Psychosocial support via a variety of channels is vital, enabling the parent to ‘let go’ and children and adolescents to gain autonomy • Effects of a good support structure will promote an improved QoL and normalisation • Improved patient understanding of disease and life implications • Patient empowerment to support improved self- efficacy and expectations of care and therapy • Emotional and educational support for the family, for better adherence and overall life outcomes • Across the entire lifespan of the patient • The positive impact in a multitude of life aspects: quality of life, normalisation, social interaction, health and fitness • Across the entire lifespan of the patient and throughout all interventions and educational materials
- 8. • All peoplewith haemophilia should receive the treatment they need, regardless of where they live. That’s why, together with Bioverativ, we are donating up to 1 billion IUs of factor therapy over 10 years. • We hope to help close the treatment gap in the developing world, where the vast majority of people with haemophilia have inadequate care or no treatment at all. • This unprecedented donation, which includes providing 500 million units of factor over five years to the World Federation of Haemophilia Humanitarian Aid Program, has already doubled the percentage of children receiving treatment. • The largest single donation of its kind has resulted in the expansion of the World Federation of Haemophilia Humanitarian Aid Program and led to a paradigm shift in how people in developing countries are being treated. 8 World Federation of Haemophilia
- 9. 0% 10% 20% 30% 40% 50% 60% 70% 80% Technology Automotive BankingPharmaceuticals % of trust in2015 78% 71% 53% 61% 78% 71% 53% 61% Trust In technology based Industries in 2015; ABPI data
- 10. 2 6 18 20 15 17 6 Trust inthe Pharmaceutical Industry 0=very untrustworthy /10=very trustworthy 10 9 8 7 6 5 4 3 2 1 HCPs 8 16 2116 16 4 Favourability towards the Pharmaceutical Industry 0=very unfavourable / 10=very favourable 10 9 8 7 6 5 4 3 2 1 HCPs • 33% of HCPs score 5 or lower on favourability on the Pharmaceutical Industry 2 3 12 12 2210 6 Trust in the Pharmaceutical Industry 0=very untrustworthy /10=very trustworthy 10 9 8 7 6 5 4 3 2 1 Medical Students 4 13 12 21 18 10 Favourability towards the Pharmaceutical Industry 0=very unfavourable / 10=very favourable 10 9 8 7 6 5 4 3 2 1 Medical Students Pharmaceutical Industry ABPI data 2015 Healthcare Professionals and Medical Students • 43% of Medical Students score 5 or lower on trust for the Pharmaceutical Industry • 39% of HCPs score 5 or lower on trust for the Pharmaceutical Industry • 46% of Medical students score 5 or lower on favourability on the Pharmaceutical Industry
- 14. The Sobi Community:Why we are here 14 Our Mission To develop and deliver innovative therapies and services to improve the lives of patients Our Vision We are inspired to pioneer a world in which rare disease patients are diagnosed at birth, receive effective and sustainable therapy, and go on to live full and healthy lives
- 15. •After this talkdo you feel that Sobi is listening and is making a positive difference to rare disease? 15 My question NP-2981 Date of preparation: October 2017
Editor's Notes
- #3 The model on the left is something we are all familiar with and it’s wonderful that for some rare diseases there is awareness, tests, diagnosis and treatments – but still only 5% of rare diseases have a treatment and of course, there is so much more to achieve. Collaborative approaches have always been vital in the field of rare diseases. Patients are rare and expertise is rare. There are many decision points on the road to real patient access and evidence is needed to inform each decision. The ability to listen to the needs of the patient and their families and be guided by this in our interactions with different decision-makers at all stages is critical. Different stakeholders have different, albeit overlapping, needs in order to be able to take the best decisions that they are responsible for taking. Each opportunity is a chance for us to listen, to take on board what we are hearing and to constantly refine and fine-tune our approaches to secure that we are meeting the real needs as expressed by the communities. We believe that this is a way to secure that we are patient centric.
- #4 The compound that has gone on to be an effective treatment for Hereditary Tyrosinaemia Type 1 (HT1) was originally a herbicide owned by ICI. It was discovered to have an action in the pathway of HT1 and was developed and authorised as an orphan drug in 2005. In this disease area we are all delighted that survival at 4 years of age has risen from 29% to 93%. And for the first time these patients now grow up, become teenagers, fall in love and also become parents. And the journey with the patients’ community does not stop there. As our patients survive and grow, their needs change. Support for the diet, support for the growing teenagers. Further to feedback from the community and encouragement from the regulatory authorities Sobi went on to develop and request marketing authorisation for a new liquid formulation to facilitate accurate dosing for young children. At the same time we added a higher strength capsule to support easy dosing for our older patients Another rare disease community reached out to us when the compound was discovered to have a potential impact on another rare disease – Alkaptonuria (AKU). The AKU Society set up a patient-led research consortium to conduct a full clinical trial and development programme for nitisinone in AKU. Sobi is participating in that patient-led programme and the clinical trials are in their latest phases. The AKU Society could request Marketing Authorisation in the coming years. These stories are driven by the patients – a long-term story and a long-term commitment where Sobi collaborates to deliver what the patients want and need.
- #5 Again, a journey driven by the patients – a long-term story and a long-term commitment where Sobi collaborates to deliver what the patients want and need. It is a “pull” from the community that drives what we do, it is not a company “pushing” our own agenda. Our product was originally authorised for the treatment of Rheumatoid Arthritis. Because of its mechanism of action , it was discovered by treating physicians to have a particular value in treating certain paediatric inflammatory conditions. Physicians were using it, for example, to treat a rare, painful inflammatory condition called CAPS. But off-label use is not ideal – patients suffering from rare conditions deserve to have authorised treatments where possible. Sobi got a cry for help from the NIH in US who kindly shared their patient data so we by collaboration could submit and work through a marketing authorisation for the treatment of CAPS. We continue to work with the community to understand where we should be focussing our research and development efforts and, as a result of this, we have initiated 2 more development programmes for the compound that we hope will contribute to better treatment.
- #6 Working with the guidance of the patients and the community can happen at every stage. Engaging with the community can lead to all kinds of outcomes at every stage of development. For example, Sobi heard that packaging and dosing syringes for haemophilia are not always designed to meet the needs of people with haemophilia, being sometimes bulky and with syringes that are difficult to manage negatively affecting compliance particularly in children and adults with impaired joint mobility, often a result of the condition. Sobi initiated a dialogue with more than 100 individuals, including haemophilia organisations, carers and healthcare professionals to explore whether more user-friendly packaging and administration could make life easier for people with haemophilia. The insights allowed us to create new designs for the packaging and syringes which were shared with the haemophilia community to explore if these designs would better meet the needs of people with haemophilia The end result was something that has been designed with the community that it is intended for. And feedback from users suggested that this is aa major improvement for people with haemophilia. Through genuine co-creation with people with haemophilia and the community, we believe that Sobi has gathered new insights into how packaging can be developed resulting in real improvements for people with haemophilia.
- #9 Patient needs do not stop at borders. The lack of access to effective diagnosis, treatment and care is as real in developing countries and is an urgent and important public health challenge. In the field of Haemophilia, the World Federation of Hemophilia (WFH) is leading the effort to change this lack of access in developing countries with their Humanitarian Aid Programme, because the cost of products to treat is prohibitively expensive for the majority of those affected with a bleeding disorder The WFH has been running this programme successfully by the since 1996, providing clotting factors to developing countries across the world and supporting the development of specialised treatment centres. 322,000,000 IU’s have been distributed in 90 countries and over 100,000 people have been helped Yet the WFH highlighted the need to move deeper and further into providing prospective and predictable supply and called upon the industry to help them respond to this need. Because they want to go further to provide consistent and predictable access to treatment for all. Having understood the global need in haemophilia, based on listening to the needs of the community, Sobi and Biogen, now Bioverativ, built this into the manufacturing capability and have committed to provide 1 Billion Units of commercially manufactured clotting factors over the next 10 years. This has enabled the WFH in many countries to think differently about how they are able to provide the treatment and we hope that this will also encourage other clotting factor manufacturers to participate in the programme in a similar way, because it can revolutionise the way haemophilia diagnosis, treatment and care is delivered and made available. This progress is significant but we must remember the children who cant be helped by this initiative because they died in infancy from complications due to bleeding, who died before they were even diagnosed. This happening in all countries in many rare diseases which is why all people’s efforts are so crucial in this special field.
- #15 I hope that this talk has given you a small insight into Sobi and the above sums up our company philosophy and almost concludes this presentation.