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At the start of 2025, NICE updated its real-world evidence (RWE) framework on guidance for qualitative studies, showing NICE’s increasing openness to qualitative evidence submissions, particularly in rare conditions where RWE can be scarce. A new appendix dedicated to planning, conducting and reporting qualitative research to support NICE decision-making was introduced.

Clinical endpoints are critical in evaluating the effectiveness of new treatments. Traditionally, endpoints in trials have focused on objective, clinical measures of disease. However, these endpoints do not always capture patient experiences, treatment burden, or quality of life factors that may influence treatment adherence and overall outcomes.

Engaging patients and caregivers in Health Economics and Outcomes Research (HEOR) is crucial for developing healthcare solutions that truly address patient needs.

Metabolic dysfunction-associated steatotic liver disease (MASLD), formerly known as non-alcoholic fatty liver disease (NAFLD), is a growing global concern.

Throughout 2024, Prime HCD has continued to publish research addressing challenges faced globally, across diverse groups, and explored the potential impacts of transformative treatments.

Huntington disease (HD) is a rare, inherited neurodegenerative disorder that progressively affects motor skills, cognition, and behaviour. Currently, there is no cure for HD. Existing treatments can alleviate movement disorders and help manage psychiatric symptoms. However, these treatments do not halt disease progression.

Over the past 30 years, antiretroviral therapy (ART) has transformed HIV into a manageable condition, drastically reducing mortality where access is available. Yet, the disease remains a significant challenge, particularly in low- and middle-income countries. Excitingly, curative therapies (CTx) might be within reach in the next 10-20 years. But addressing challenges related to pricing and financing will be essential for ensuring global access. To address these, we need to understand the economic value of CTx and develop a decision-analytic model to estimate long-term costs, health outcomes, and a value-based price for each country.

SLE can be relentless. An inflammatory autoimmune disease that affects multiple systems within the body. It challenges patients across ethnicities, leaving them grappling with symptoms that disrupt their lives. While SLE predominantly affects black women, it also impacts Asian and Caucasian women. Unfortunately, there’s no magic cure. Instead, treatment focuses on symptom management. But what about the quality of life (QoL) of these patients? How does SLE affect their daily experiences, work, and overall well-being?

Gene therapy (GTx) offers a revolutionary treatment option for patients with haemophilia. It’s a one-time treatment that could potentially provide continuous protection for years. This significant treatment decision warrants a thorough understanding and careful consideration of all relevant information.

Global Prevalence: Sickle Cell Disease (SCD) is a widespread genetic blood order, affecting millions worldwide. The majority of cases are found in sub-Saharan Africa and Southeast Asia.