Multiple myeloma patients face late diagnosis, unequal access to therapies

Delayed diagnosis and inequalities in access to treatments across the EU are among the main challenges in fighting multiple myeloma, an incurable and rare form of blood cancer.

Roughly 50,000 people in Europe are diagnosed yearly with multiple myeloma – a rare cancer of plasma cells found in the bone marrow.

Plasma cells are tasked with making antibodies to recognise and fight germs, such as viruses or bacteria. With multiple myeloma, however, the division of plasma cells is out of control and immature plasma cells start multiplying and filling up the bone marrow, becoming cancerous.

Multiple myeloma, also known as Kahler’s disease, does not have any effective cure – the disease develops resistance to almost all therapeutic approaches.

This aspect distinguishes the disease’s pathway and its pathology with a slow progression and recurrences, further reducing the quality of life of patients.

The recent focus on cancer by the European Commission culminated in its flagship Europe’s Beating Cancer Plan, which featured 10 flagship initiatives that address the entire disease pathway in a ‘people-centred’ approach.

One of the main features of the plan is the strong reliance on what has been described in the Plan as “a whole-of-society effort that follows the ‘Health in All Policies’ approach”.

“Cancer care is no longer the responsibility of the health sector alone. It requires engagement and buying from a wide range of sectors and stakeholders,” the Plan itself reads.

“Europe is putting lots of effort into trying to increase education, increased training, and increase awareness of myeloma,” said Charlotte Pawlyn, haematologist consultant at the Royal Marsden in London, in an event on the occasion of the first European Myeloma Day.

“But I think we also have to be aware that it remains a rare disease,” she added referring to the importance of other broader approaches that go beyond reliance on just doctors.

Early diagnosis and access: The main problems

There are varying unmet needs of myeloma patients across European countries, Kate Morgan, co-CEO of Myeloma Patients Europe (MPE) explained to EURACTIV.

“However, early diagnosis and inequalities in access to myeloma treatment are two elements we identify as main, overarching problems,” she added.

A report drafted by MPE showed that myeloma diagnosis can take over five months and require more than four medical consultations with more than three different specialists.

It is common that many myeloma patients have been initially misdiagnosed with more common conditions before finally being referred to a haematology department and having a diagnosis confirmed.

Myeloma plasma cell in the bone marrow leads to symptoms that range from anaemia, back pain, and fatigue to kidney and bone damage.

“The challenge for general practitioners and specialists is that these are very common and non-specific symptoms – so they don’t always mean myeloma,” said the haematologist Pawlyn.

A late diagnosis affects patients’ ability to carry on with their everyday work and home life since they suffer from more pain and risk more bone damage.

“Patients [with late diagnosis] might be increasingly frail which can affect how we deliver treatment or the doses of the treatment we can use to treat myeloma,” Pawlyn added.

Inequalities in access to myeloma treatment are also an important issue for most of the European countries impacting mainly Central and Eastern Europe – and some non-EU Balkan countries in particular.

“But countries from Northern and Western Europe are also experiencing issues to get access to innovative treatments such as CAR-T therapies in myeloma and other treatments in the heavily pre-treated setting,” said MPE’s Morgan

The representative of the myeloma patients’ association mentioned that another issue in the heavily pre-treated setting is the absence of Phase III clinical trials with comparative data, resulting in a lot of ‘uncertainty’ for reimbursement bodies on whether they should allocate funding.

“Only 6% of the 3,229 worldwide myeloma trials included patients from Central and Eastern Europe countries over the past 20 years, which is something we need to understand and address,” she concluded.

The role of innovation

Research and innovation represent the starting point for a new approach to cancer care in the EU, Health Commissioner Stella Kyriakides pointed out at the launch of the Knowledge Centre on Cancer, the first flagship action delivered under the Cancer Plan.

This is particularly true for myeloma, where innovation is a crucial component with an expansion in the number and the range of new treatments for myeloma which become available in recent years.

The EU’s medicines agency (EMA) acknowledged that new medicines for treating myeloma are needed, as patients who have already been treated with three major classes of drugs to treat this blood cancer – immunomodulatory agents, proteasome inhibitors, and monoclonal antibodies – no longer respond to these products.

A combination of two or three different treatment effects from different types of drugs is commonly used in managing the disease.

Compared to the mono-therapy approach, combining two or more therapeutic agents potentially reduces drug resistance while also providing therapeutic anti-cancer benefits, such as reducing tumour growth and cancer stem cell populations.

“Despite the significant value combination therapies bring to cancer care, there are several challenges to their access in Europe,” a representative from the EU’s pharmaceutical manufacturer association (EFPIA) told EURACTIV.

The current regulatory case-by-case approach to novel oncology combinations is ‘unsustainable’, the representative said, adding that it denies patients access to important new treatments.

For the pharma industry, the way forward should be found in a collaborative approach that takes into account the views of policymakers, payers, authorities, doctors, patients, and manufacturers to address problems in access to combination therapies.