Hans Scheurer President Myeloma Patients Europe

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

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

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.

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

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

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

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

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

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

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)

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

About myeloma patients: Globally 230000 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

Course of the disease myeloma

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

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

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

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

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

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

Q & A scheurer@mpeurope.org