Developing drug-device combination products for rare diseases presents unique challenges and opportunities for biopharmaceutical companies. These advanced drug delivery products which integrate a drug and a device into a single treatment, can be particularly beneficial for patients with rare diseases, offering novel solutions that may improve treatment efficacy, patient adherence, and quality of life. However, the development process for these products is complex, requiring careful consideration of regulatory requirements, technical feasibility, clinical efficacy, and patient-centred design.
Understanding the Unique Needs of Rare Disease Populations
Rare diseases, by definition, affect fewer than 200,000 people in the U.S, or less than one in 2,000 in Europe but globally, they impact some 300 million people.1 It is estimated there are more than 7,000 rare diseases. Yet, with a mere five to seven percent of these conditions having an FDA-approved drug, they remain largely untreated.
People with rare diseases face numerous challenges, including the pursuit of an accurate diagnosis, accessing suitable and sufficient medical care, and working toward better social inclusion and independence. Each condition has its own unique characteristics, progression, and symptoms, which often require tailored treatments. These variations necessitate a personalised approach in designing drug-device combination products.
Key considerations:
• Conduct thorough research on the rare disease to understand its pathology, symptoms, and treatment challenges.
• Engage with patient advocacy groups to gain insights into patient needs and preferences.
• Design drug-device combination products with flexibility to accommodate variations in disease presentation and severity.
Regulatory Pathways and Requirements
Navigating the regulatory landscape is arguably the most critical aspect of developing and commercialising drug-device combination products. Regulatory bodies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have established specific guidelines to ensure the safety and efficacy of these complex products.
First, companies must determine the regulatory pathway applicable to their product. Understanding the regulatory classification of a drug-device combination product is essential and depends on its principal mode of action. This will dictate whether the product will be regulated as a medical device, a drug, or a combination product. This determination influences the type of regulatory submission required and the specific requirements for demonstrating safety and efficacy.
