Rare diseases collectively affect an estimated 300 – 400 million people worldwide, despite each individual condition impacting only a small patient population. While scientific advances in biologics, gene therapies and targeted therapeutics have transformed the treatment landscape for some rare conditions, many diseases still lack effective treatment options. Drug–device combination products (DDCs) are emerging as an important strategy to bridge this gap by improving drug delivery, enhancing adherence and supporting patient self-administration in increasingly complex therapeutic regimens.