1. Hans Scheurer
President Myeloma Patients Europe

2. Disclosure
I have nothing to disclose, I declare that I have no relevant or material financial interests that relate to the ATTR program.

3. How patients of a rare disease can organise themselves
The case of..

4. Myeloma Patients Europe (MPE)
MPE is an umbrella organisation of multiple myeloma patient organisations across Europe
Growing organisation - 40 members in over 28 countries across Europe (incl Israel, Armenia and Turkey)
Myeloma is a rare cancer of the plasma cells in the bone marrow. In Europe, approximately 39,000 patients are diagnosed with myeloma each year
Because of the strong link between Myeloma and AL Amyloidose, we have amended our constitution to include coverage of patients with AL amyloidosis.

5. How and why MPE was formed
Knew the importance of pan-European networks
Amalgamation between two membership organisations in 2011 – European Myeloma Platform and Myeloma Euronet
Both past organisations had been successful but efforts dissipated and sometimes calling for different things
However it was agreed that the support, education and advocacy needs of myeloma patients and their families across Europe were better served by one organisation, coordinated with a small team of staff
Outline the challenges associated with amalgamating two organisations and how we overcome them through effective collaboration and the derivation of clear strategic goals that all organisations believed in

6. Strategic goals Build a professional and sustainable organisation
Facilitate development, capacity and sustainability of members
Encourage research and the development of new effective treatment
Ensure the timely adoption of research outcomes or results and access to state of the art diagnostic tools and treatment
Encourage healthcare professionals to provide an excellent standard of care to patients and their families
Develop an evidence base for the needs and wants of patients and ensure access to high-quality information and psycho-social support

7. Association structure of MPE
MPE members
MPE Board
Board committees
MPE staff
CEO
staff members
MPE Constitution

8. Association structure of MPE
MPE structure
MPE members
MPE Board
Board committees
MPE staff
CEO
staff members
MPE Constitution
MPE Strategic plan

9. Association structure of MPE
MPE structure
MPE members
MPE Board
Boardcommittees
MPE staff
CEO
staff members
MPE Constitution
MPE Strategic plan

10. Strategic goals Build a professional and sustainable organisation
Facilitate development, capacity and sustainability of members
Encourage research and the development of new effective treatment
Ensure the timely adoption of research outcomes or results and access to state of the art diagnostic tools and treatment
Encourage healthcare professionals to provide an excellent standard of care to patients and their families
Develop an evidence base for the needs and wants of patients and ensure access to high-quality information and psycho-social support

11. Examples of MPE programmes & projects
Upskill national patient organisations to identify and develop strategies to overcome challenges themselves
Atlas of Access
Advocacy Development Programme
Annual Masterclass
Patient information guides on myeloma and AL Amyloidosis
Policy/strategy plan
Clinical trial access
Medicines pricing
Working with industry
Working with regulators (EMA, HTA..)
Programmes with partners
Harmony (51 partners, ‘big data’)
Eurobloodnet (European Reference Network)

13. Strategic goals Build a professional and sustainable organisation
Facilitate development, capacity and sustainability of members
Encourage research and the development of new effective treatment
Ensure the timely adoption of research outcomes or results and access to state of the art diagnostic tools and treatment
Encourage healthcare professionals to provide an excellent standard of care to patients and their families
Develop an evidence base for the needs and wants of patients and ensure access to high-quality information and psycho-social support

14. About myeloma patients:
Globally people living with multiple myeloma
1% of all cancers, 2% of all cancer deaths
95% of patients diagnosed in advanced stages of disease
Most frequently diagnosed in patients 65-74
10% of the MM patients develops AL Amyloidose
1990: average survival 2-3 years, present 7–10 years
Most patients respond to initial therapy, but nearly all patients eventually relapse
Disease burden increases with each relapse
Patients ability to achieve and sustain a meaningful response declines with each relapse due to acquire drug resistance and disease biology
Relapsed patients may have worsened health status as a result of disease progression, co-morbidities

15. Course of the disease myeloma

16. Strategic goals Build a professional and sustainable organisation
Facilitate development, capacity and sustainability of members
Encourage research and the development of new effective treatment
Ensure the timely adoption of research outcomes or results and access to state of the art diagnostic tools and treatment
Encourage healthcare professionals to provide an excellent standard of care to patients and their families
Develop an evidence base for the needs and wants of patients and ensure access to high-quality information and psycho-social support

17. Bring your pizza Patient experiences Patient preferences
Quality of life
Burden of the disease
Unmet needs
perspectives

18. Challenges & some lessons learned
Differences in capacity, ability and opinion across Europe. Important to understand and take account of these differences
Differences in agendas between patient organisations and different understanding of where the problems lie
Big divergence in standards of treatment and care and clinical trials. Important to focus on cross-cutting issues that can help all countries (such as initiatives on clinical trials etc.) and on designing ways of giving local organisations the tools to influence decision-making themselves
It is impossible to achieve everything, so focus on meaningful and achievable goals

19. Interest MPE in AL Amyloidose
Clear link between AL amyloidosis and myeloma
For the first time we are seeing AL amyloidosis specific medicines being brought to market
Good knowledge of regulatory issues and challenges in Europe, which we can apply directly to the experience of AL amyloidosis
Can collaborate with existing AL amyloidosis charities on coordinated patient advocacy at both European and national level
MPE can apply and provide knowledge, infrastructure and broker collaboration across a range of stakeholders

20. Plans MPE for AL Amyloidose
AL amyloidosis is a very important area for MPE
AL amyloidosis groups are associated members of the group. Invited them and they attended our annual Masterclass, which we found very beneficial
Plans to better understand and develop our knowledge of AL amyloidosis groups in Europe and to aid collaboration on a wide range of issues where relevant and where we can add value
In the short term we are looking at the potential information we can provide on new treatments coming to market in AL amyloidosis
In the longer-term, we are also aware of new medicines and regulatory challenges coming down the line for AL amyloidosis drugs (we are looking at one of the first products licensed products in myeloma). Where helpful, we plan to understand these issues and work collaboratively to try and overcome them
We are very keen to collaborate with amyloidosis groups in Europe and to support ongoing work – rather than reinvent the wheel

21. Ideas from MPE to ATTR
Prioritise effectively to establish where you can add value as an network
Ensure you understand the issues and challenges that you need to overcome
It is about small incremental steps – it isn’t going to develop overnight
Map out and engage with other stakeholders (such as industry and clinicians) – they can provide support too and help you understand issues and challenge
We have been through the process of developing a network, hope some of the insights we share will help with this – however, happy to provide further information where possible

22. Q & A