Aplasia Treatment Market Trends

Growth Drivers 

• Healthcare quality improvement initiatives: Agencies within the government are emphasizing early intervention strategies for rare diseases. For instance, the U.S. Food and Drug Administration (FDA) has launched initiatives to accelerate advancements of gene therapies for rare diseases. Additionally, in 2023, the federal government allocated around USD 830 billion for Medicare. The extensive amount of government funding available through Medicare indicates that the government is willing to fund treatments for conditions ranging from simply chronic to rare diseases.

• Patient pool and disease prevalence: Over 25 million Americans are affected by rare diseases. This includes a significant number of conditions, including aplasia. The large patient population, representing a significant number of patients, allows for patients to access reasonably effective treatment plans, which ultimately leads to pressure to develop treatment options in aplasia.

• Technological advances in treatment development: The FDA's progress toward utilizing artificial intelligence and machine learning in medical product development is meant to increase the productivity and efficacy in developing new treatments.  Such innovations can expedite the time frame for creating new therapies, including for rare diseases like aplasia. The collaboration between the FDA, NIH, and private organizations is an attempt to try and reach more effective gene therapies for rare diseases.  Collaborations are important in unifying resources and experts together to help advance usual and unusual treatment options such as aplasia.

Challenges

• High upfront expenses of gene therapies: Gene therapies, often prescribed to treat aplasias, are associated with high upfront expenses. Consequently, this poses a significant challenge for Medicaid programs with capped budgets to provide access to therapies for all eligible patients.

• Inconsistent medicaid coverage across states: The coverage of gene and cell therapies is inconsistent across states. In a study of over 12 states, it was discovered that coverage for Zolgensma was more restrictive compared with the FDA label, which may ultimately create delays and denials in patient access to Zolgensma.