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Overcoming real-world challenges to maximise the benefit of treatment for patients with multiple myeloma

Published on 02/10/17 at 10:32am

Dr Antonio Palumbo, Distinguished Research Fellow Oncology, Takeda Pharmaceuticals addresses hwo far we’ve come in treating multiple myeloma, and how far we still have to go.

When I first started practicing as a haematologist more than twenty-five years ago, I had only a handful of treatment options to offer my patients with multiple myeloma, a rare and devastating blood cancer. The therapies that were available, including traditional chemotherapy and corticosteroids, produced limited increases in survival. Since then, however, dramatic advances occurred in the treatment of this relapsing and incurable disease, changing what it means to be diagnosed with multiple myeloma.

Novel agents introduced during the last decade, including proteasome inhibitors and immunomodulatory drugs, contributed to doubling survival rates since the 1990s, when cytotoxic chemotherapy was the primary form of treatment. For example, the five-year relative survival rate has increased from 29% for patients diagnosed between 1990 and 1992 to approximately 50% for those diagnosed between 2006 and 2012. For instance, when considering the effectiveness of proteasome inhibitors specifically, improvements have been observed in depth of response among elderly patients as induction therapy, as well as significant improvements in response, and both progression-free and overall survival following stem cell transplantation.

However, the arrival of additional therapeutic options has not eliminated all the challenges patients with multiple myeloma face along their treatment journey. Many patients still encounter issues with drug tolerability, logistical or geographic problems that can lead to discontinuation of treatment. Healthcare providers routinely make compromises, such as reducing doses, interrupting treatment, or terminating treatment, all of which may undermine the goal of longer-duration therapy, which has been associated with improved survival.

It is imperative that healthcare providers who treat patients with multiple myeloma not only acknowledge, but also fully understand these barriers to accessing effective, practical and sustainable therapies with good tolerability that support long-term disease control and improved quality of life. Recognising the potential benefit of shared decision-making with patients is also essential. By taking steps to partner with our patients and their caregivers, we can help them overcome the challenges that stand in the way of achieving the best possible outcomes. The following are among the most common barriers patients face.

Barriers that impede the optimal treatment of multiple myeloma

Tolerability issues

Continuous treatment is one strategy that has the potential to extend the overall survival of patients with multiple myeloma. It involves a patient continuing treatment as opposed to stopping treatment once the disease goes into remission. While evidence from recent studies show the potential benefits to maintaining or deepening treatment response by staying on treatment, in practice this is challenging due to tolerability issues. These issues include the development of late toxicities, raising questions around the optimal duration of therapy and selection of an agent that will provide an adequate tolerability essential for chronic administration.

Additionally, it is difficult to study the use of continuous therapy in the real-world. In every day practice, treatment duration tends to be shorter than those reported in clinical trials. A considerable proportion of patients, such as those with comorbidities and those who are older, are usually excluded from clinical trials based on incompatibility with current inclusion and exclusion criteria. Given these considerations, lower risk of drug discontinuation and dose reduction enabled by better treatment tolerability may translate to improved outcomes. 

Logistical and geographic barriers

Though commonly used in the treatment of multiple myeloma, therapies requiring administration at a hospital, clinic or physician’s office may create logistical obstacles. Some patients may struggle with transportation to and from the treatment site. Patients who feel the effects of these challenges acutely may decide to discontinue treatment.

Patients living outside major cities also face challenges in accessing care. Since medical schools and teaching hospitals are in large cities, there are not as many physicians in rural areas and people living outside of major cities can experience delays in diagnosis and treatment. This problem is persistent around the world, and in many countries oncology care is concentrated in urban areas. As a result, there are many areas where patients must travel great distances to receive therapy. For instance, in the United States, only 3% of medical oncologists practice in rural areas and more than 70% of counties across the country do not have medical oncologists.

Further, geographic circumstances may also dictate the availability of new multiple myeloma treatments. Seventeen of the 49 new oncology medicines approved between 2010 and 2014 are available in ten or fewer countries. For example, patients in some countries within the European Union are sometimes unable to access the latest treatment options approved by the European Medicines Agency (EMA). This is in part due to the different processes required of manufacturers for filing and registering new medicines in each country – a process that ranges in complexity and duration the world over. Additionally, where a patient resides may also impact their ability to access optimal care, such as a leading multiple myeloma expert familiar with the latest treatment.

Solutions for helping patients achieve the best possible outcomes

The good news for patients is that the multiple myeloma community, from researchers to physicians to advocates, has made a concerted effort to address many of the issues that hinder multiple myeloma treatment. The most effective ways to address these barriers and improve patient outcomes include:

  • Putting patients’ priorities first by listening to their needs and taking action to support shared decision-making with patients and caregivers
  • Working together to identify professional education gaps and ensure that all members of a patient’s care team are utilising continuing education resources to learn about the latest multiple myeloma treatments and how they are best used in clinical practice
  • Helping raise awareness about multiple myeloma among all members of the health care community so that health care professionals, specifically those who treat elderly patients regularly, can better recognise symptoms of the disease leading to a more timely diagnosis
  • Supporting the development and use of technologies that can help bring the right care to the right patient, such as telemedicine
  • Sharing resources developed by advocacy organisations to help educate patients about their disease and available treatment options
  • Partnering with professional societies and health authorities to encourage updates to treatment guidelines to include newer, more convenient therapies

Despite significant advances in treatment that have vastly improved outcomes for patients, multiple myeloma remains an incurable disease with considerable burdens for both patients and caregivers. If we commit ourselves to understanding the issues patients face, we can work together toward the goal of attaining long-term disease control and improved quality of life.

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