Myeloma is a rare, relapsing and remitting cancer of the plasma cells found in bone marrow. Every year 4,800 people in the UK are diagnosed with myeloma. There is currently no cure; and treatment options are focused on managing and extending the lives of patients.

Advances in the treatment over the last decade or so have resulted in survival for patients improving at a faster rate than almost any other cancer in the UK. However, overall survival remains poor and almost every myeloma patient will die of the cancer and/or its complications.

Whilst there has been innovation in research into the genetics and biology of myeloma, and there are a number of novel new treatments coming down the line, without collaboration and streamlining in drug discovery and development and earlier engagement with regulators, HTA bodies and payers, patients may not fully benefit from this innovation.


Delivering effective treatment and improved care


Treatment of myeloma consists of a combinations of anti-myeloma drugs and high-dose therapy and stem cell transplantation in eligible patients. Treatment is largely dictated by national guidance rather than by the informed choice of doctors and patients meaning that all patients get the same treatment irrespective of individual disease, clinical or personal factors.

However, in addition to being relapsing and remitting, myeloma is not one cancer but is made up of several different types ranging from high risk with a poor prognosis to low risk with a more favourable prognosis. What is more, we know that evidence suggests that the majority of treatments do not work in every patient and this therefore has negative impact on survival and quality of life as well as the emotional state of patients and their families.

If we are to overcome this, and truly realise the benefit of innovation in the pipeline, we need to think differently about how research is done in order to generate the evidence needed to ensure that patients gets the right treatment at the right time. Significant investment is therefore needed in stratified medicine and in the design of clinical development programmes.

To ensure the process is robust, it must be informed by genetic subtypes and real-world needs of patients, healthcare professionals and payers. There is also a requirement for more sophisticated platforms for translating evidence into clinical practice and treatment algorithms that better reflect the heterogeneity of myeloma. If this can be achieved, it will make the identification and prioritisation of pipeline treatments easier and speed up the development journey.


Accessing treatment


There are a number of promising new treatments coming down the line, from immunotherapy drugs like daratumumab and elotuzumab, and several new drugs that are in the later phases of development such as Kyprolis® (carfilzomib) and ixazomib which will hopefully gain marketing authorisation soon.

However, there are likely to be challenges in securing access to these drugs for patients.

For far too long clinical trial programmes have been developing trials based solely on safety and efficacy to get a licence – this is not compatible with the data and information of the UK’s health technology assessment (HTA) bodies and payers.

Factor into this rapidly escalating cost of cancer drugs, the down pressure of global markets and pricing and the inflexibility in the UK to pricing, you can see why patients have concerns about getting access to the treatments they need.


A collective force driving change


As a patient-driven organisation, Myeloma UK focuses on driving developments in research and access to new treatments that benefit myeloma patients. Unfortunately, silo working and thinking among stakeholder groups in the research, development and drug access spaces is common place. Many of these groups are driven by vested interests and operate in a system where there is disconnect between rewards and incentives, and an absence of aligning common goals.

It is imperative that researchers from sectors such as academia, Government, industry, non-profit and clinical care find a common goal with a strategic focus and outcome drivers that can be used by each stakeholder to benefit patients.

Myeloma UK recognised that we are perfectly placed to drive forward this change and to be an honest broker in bringing different stakeholders together to work towards common goals.

Over the last year we have been implementing a business model that delivers research platforms and collaborative environments to bring stakeholders round the table to work together. Our work has included setting up a national translational research network, a national bio-bank and Early Phase Clinical Trial Network which has over 35 hospitals taking part.

Increasingly we are being seen as a catalyst with the business model that has moved rapidly from a primary emphasis on grant funding, to a driving force that is advancing scientific development and leading cutting edge patient-centred research.