Despite the dramatic number of unmet needs that still exist across many tumor types, we have made significant progress in treating some cancers. Since the early 2000s, novel treatment options have fundamentally changed survival rates in chronic myeloid leukemia, metastatic melanoma, HER2+ breast cancer, chronic lymphocytic leukemia and many others. These advancements mark an important and growing trend in the cancer treatment landscape: a shift from acute to chronic treatments. 

As a case study, let’s examine multiple myeloma. It’s fair to say that myeloma treatment has largely shifted from an acute to a chronic treatment mindset (although options for high-risk patients are still desperately needed). ZS estimates that median overall survival will increase from 30 months in 2001 to around 72 months by 2022—a 140% increase. These gains are largely driven by the introduction of novel drug therapies, which will continue to transform options for patients suffering from this disease. Several studies recently published by sources such as the National Comprehensive Cancer Network, the Leukemia and Lymphoma Society and more on survival in multiple myeloma appear to have similar findings.

The shift to chronic care in multiple myeloma raises some important questions: 

  1. What will the myeloma-prevalent population look like in 2022? Certainly, it will be considerably larger than today, but it will also be significantly older, with more co-morbid disorders and more exposure to treatment. With so many treatment options available, patient lifestyle and quality-of-life concerns will increasingly play a role in treatment selection. To be successful in this new landscape, manufacturers must design today’s trials with tomorrow’s patients in mind. This means developing real world inclusion/exclusion criteria and picking comparators that reflect new standards of care. It’ll also require the thoughtful inclusion of supportive services, for example in the form of nursing monitoring, geriatric services or innovative technology adherence applications as part of the actual trial design.
  2. Is overall survival still an achievable endpoint for early-line trials? When half of the patients contending with a disease are living six-plus years, this becomes an increasingly daunting challenge. Are today’s first-line therapies “as good as it gets” in myeloma and similar diseases? While the improvement in outcomes has been impressive, we’re still a long way from a cure. The industry and payers/health technology associations (HTAs) globally must collaborate to tackle the challenges (both ethical and financial) that this presents. This means identifying and targeting high-risk patients in the front-line setting, deploying pan-cancer genomic testing to identify which patients receive the greatest benefit, and, most importantly, deprioritizing trials that are likely to result in only middling benefits.
  3. Are treat-to-progression (TTP) regimens the right approach in a chronic disease landscape? Here, both medical and financial toxicities compound quickly. Even the best targeted therapies have off-target effects, and with the recent focus on cancer immunotherapy, the stakes are even higher. On the medical side, the long-term effect of prolonged exposure to anti-cancer therapeutics remains embarrassingly under-researched. Financially, the burden on patients and health systems has been well documented. Certainly, there are cases where TTP regimens forestall or even eliminate the need for more costly medical procedures. However, many people believe that the long-term costs are unsustainable. Again, collaboration between the pharmaceutical industry and payers/HTAs globally must prevail. We must accelerate efforts to implement innovative reimbursement models like indication-based pricing and pay for performance.

Leading companies have already started tackling these questions head-on, and we think that the questions deserve broader industry consideration. To stay ahead of the pack, biopharmaceutical manufacturers must proactively create product development strategies for tomorrow’s patient populations rather than for today’s. The first step is to recognize the shift from acute to chronic treatment and its implications. 


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Topics: oncology, Malik Kaman, chronic, acute, multiple myeloma