Delays in diagnosis and inequalities in access to treatment across the EU are among the main challenges in the fight against multiple myeloma, a rare and incurable form of blood cancer.
Every year around 50,000 people in Europe are diagnosed with multiple myeloma, a rare cancer of the plasma cells in the bone marrow.
Plasma cells are responsible for producing antibodies to recognize and fight germs, such as viruses and bacteria. In multiple myeloma, however, the division of plasma cells gets out of control and the immature ones begin to multiply and fill the bone marrow, becoming cancerous.
Multiple myeloma, also known as Kahler’s disease, has no effective cure: the disease develops resistance to almost all treatment approaches.
This aspect distinguishes the path of the disease and its pathology with a slow progression and relapses, which further reduce the quality of life of patients.
The recent attention of the European Commission towards the cancer it culminated in the “Europe’s Beating Cancer” plan, which includes 10 initiatives that address the whole path of the disease with a “people-centred” approach.
One of the main features of the plan is the heavy reliance on what has been described in the plan as “a whole-of-society effort following the ‘Health in all policies’ approach”.
“Cancer treatment is no longer the responsibility of the healthcare sector alone. It requires engagement and purchase by a broad range of sectors and stakeholders,” reads the Plan itself.
“Europe is making a huge effort to try and increase myeloma education, training and awareness,” said Charlotte Pawlyn, consultant haematologist at the Royal Marsden in London, at an event to mark the first European Myeloma Day .
“But I think we also need to be aware that it remains a rare disease,” he added, referring to the importance of other broader approaches that go beyond relying on doctors alone.
Early diagnosis and access: the main problems
The unmet needs of myeloma patients vary in different European countries, Kate Morgan, co-CEO of Myeloma Patients Europe (MPE) explained to EURACTIV.
“However, early detection and inequalities in access to myeloma treatment are two elements that we identify as major and general problems,” he added.
A report prepared by the MPE showed that the diagnosis of myeloma can take more than five months and more than four medical consultations with more than three different specialists.
It is common for many myeloma patients to initially be misdiagnosed with more common conditions before finally being referred to a hematology department and having the diagnosis confirmed.
Myeloma plasma cells present in the bone marrow cause symptoms ranging from anemia, back pain, fatigue, kidney and bone damage.
“The challenge for primary care physicians and specialists is that these are very common and non-specific symptoms, so they don’t always indicate myeloma,” said hematologist Pawlyn.
A late diagnosis affects patients’ ability to carry on with their daily, work and home lives, as they suffer more pain and are at risk of more bone damage.
“The patients [con diagnosi tardiva] they may be increasingly fragile, which may impact how we administer treatment or the doses of treatment we can use to treat myeloma,” Pawlyn added.
Inequalities in access to myeloma treatment are also a major issue for most European countries, especially Central and Eastern Europe and some non-EU Balkan countries.
“But even Northern and Western European countries have problems accessing innovative treatments such as CAR-T therapies in myeloma and other therapies in a heavily pre-treated setting,” said Morgan of the MEP.
The representative of the myeloma patients’ association recalled that another problem in the heavily pre-treated context is the absence of Phase III clinical trials with comparative data, resulting in “uncertainty” for reimbursement agencies as to whether to allocate funds.
“Only 6% of the 3,229 myeloma studies conducted globally included patients from Central and Eastern European countries over the past 20 years, and this is something we need to understand and address,” he concluded.
The role of innovation
Research and innovation are the starting point for a new approach to cancer treatment in the EU, underlined Health Commissioner Stella Kyriakides, on the occasion of the launch of the Cancer Knowledge Center, the first action carried out under the Cancer Plan.
This is especially true for myeloma, where innovation is a crucial component, with an expansion in the number and range of new myeloma treatments available in recent years.
The European Medicines Agency (EMA) recognized the need for new drugs to treat myeloma, as patients already treated with the three main classes of drugs for the treatment of this blood cancer – immunomodulatory agents, proteasome inhibitors and monoclonal antibodies – no longer respond to them products.
In disease management, the combination of two or three therapeutic effects of different types of drugs is commonly used.
Compared to the monotherapy approach, the combination of two or more therapeutic agents potentially reduces drug resistance while providing anticancer therapeutic benefits, such as reductions in tumor growth and cancer stem cell populations.
“Despite the significant value that combination therapies bring to cancer care, there are several challenges to their access in Europe,” a representative of the EU pharmaceutical manufacturers’ association (EFPIA) told EURACTIV.
The current case-by-case regulatory approach to new oncology combinations is “unsustainable,” the representative said, adding that it denies patients access to important new treatments.
For the pharmaceutical industry, the way forward should be a collaborative approach that takes into account the views of politicians, consumers, authorities, doctors, patients and manufacturers to address the problems of access to combination therapies.
Read the original article Who.