Glomerular Filtration and determinants of glomerular filtration .pptx
Neil Dugdale sobi crdn summit 2017
1. Cambridge Rare Disease Network
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 the patient 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
SJIA AUS
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
6. 6
Introduction to Sobi&Co
“Sobi&Co is 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 based on 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 people with 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
10. 2 6
18
20
15
17
6
Trust in the 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
11.
12.
13.
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 talk do 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
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.
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.
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.
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.
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 helpedYet 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.
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.