PhRMA - Pharmaceutical Research and Manufacturers of America

01/15/2021 | News release | Distributed by Public on 01/16/2021 17:13

Research shows gene therapies can offer cost savings potential of up to $1.8M over five years for patients with certain blood diseases

A recent analysis by Health Advances looks at two particularly burdensome hematological (or blood) diseases - beta thalassemia and hemophilia A - and finds that potential gene therapies in the pipeline could reduce per patient costs by as much as 41% and 90%, respectively, over five years. Research-based knowledge gained in recent years about the genetic foundation for these diseases has facilitated the pursuit of several promising gene therapy approaches.

Health Advances examined several potential gene therapies in late-stage development, based on initial clinical data. The analysis sought to estimate the direct medical costs and indirect costs that could be expected to decline with the use of these medicines. The findings highlight the tremendous cost-saving potential of not only these medicines but the many others that are being researched to treat a range of diseases and conditions. The pace of research and development in both cell and gene therapy is increasing rapidly, with nearly 400 potential therapies in the pipeline - including several that address hematological conditions.

Gene therapies specifically use genetic material, or DNA, to modify a patient's cells for the treatment of an inherited disorder or acquired disease. Because these therapies enable a person's own body to address the root cause of disease, they offer the potential for a dramatic reduction in direct medical costs associated with the previous standard of care and indirect costs resulting from missed work or reduced productivity for caregivers and patients:

  • The current standard of care for hemophilia A requires lifelong treatment involving regular administration of factor replacement therapy, often multiple times a week.

  • Beta thalassemia requires regular blood transfusions starting from birth along with administration of therapies to manage treatment-related complications.

  • Both diseases may also result in significant ER visits and repeat hospitalizations. As a result of this tremendous disease burden, hemophilia A and beta thalassemia are associated with substantial direct and indirect costs.

Depending on disease severity, the Health Advances analysis indicates that direct medical costs and indirect costs associated with hemophilia A can range between $441,000 and $1.94 million per patient over a five-year period. Through reducing bleeding rates and thus the need for factor replacement therapy, potential gene therapies could reduce per patient direct and indirect costs by 63% to 90% over that same time period for patients with hemophilia A. For example, average costs over five years associated with an individual hemophilia A patient with severe disease could be reduced by as much as $1.8 million. These savings include $1.7 million in avoided medical costs and $101,000 in indirect costs associated with missed work or reduced productivity.

Similarly, beta thalassemia is associated with substantial economic burden totaling $766,000 in direct medical costs and indirect costs per patient over a five-year period. Potential gene therapies could help reduce or eliminate the need for blood transfusions, which over that same time period could reduce per patient direct and indirect costs by 41%. As detailed below, average costs over five years associated with an individual beta thalassemia patient could be reduced by as much as $325,000.

Gene therapies such as these harness the latest scientific advances and have the potential to transform the trajectory and even curing some of the most complex and devastating diseases facing patients today. These therapies could reduce years of pain and suffering for patients and produce incredible savings for our health care system.

Unfortunately, our coverage and payment system has not kept pace with these exciting biomedical innovations. By moving toward a value-driven health care system and advancing innovative approaches to payment and coverage, we can make potentially transformative gene therapies more accessible and affordable. For example, innovative contracts and flexible payment arrangements can increase access for patients. These flexible payment arrangements include outcomes-based contracts that link payment to patient health or other outcomes as well as alternative financing arrangements, like pay-over-time and subscription models. We should prioritize removing barriers to these types of innovative, market-based solutions so we can help provide patients with access to these potentially transformative medicines.

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