Applying Quality by Design to the Rare Disease Population

When applied to clinical development, Quality by Design is an approach that focuses effort on prospectively identifying and managing risk to improve clinical trial quality and outcomes.

The application of Quality by Design principles is particularly important in rare diseases, where the limited, geographically-dispersed, and often vulnerable patient pool leaves little margin for error. By building quality into the design of a clinical trial program from day one, sponsors of rare disease studies can enhance clinical trial execution while improving efficiency and meeting their regulatory and ethical obligations.

Quality by Design (QbD) is a comprehensive, systematic approach to biopharmaceutical development and manufacturing that employs statistical, analytical, and risk management methodologies to ensure the quality of medicines. While QbD principles have been used successfully in the biopharmaceutical manufacturing industry for decades, sponsors have had difficulty translating this approach to research and development (R&D) and clinical trial programs.

QbD is an approach that focuses on:

  • Examining the objectives of a trial,
  • Defining critical factors to achieving those objectives, and
  • Creating a plan to prevent risks to those factors from negatively impacting outcomes.

In essence, developing a detailed understanding of the data and processes that form the underpinnings of a successful trial is a prerequisite to identifying and managing risks and improving the quality and outcomes for clinical trials.

The application of QbD-based risk management principles is relevant to virtually any clinical trial, in any therapeutic area. However, there are special considerations that must be taken into account when applying QbD concepts for drugs and biological products intended to treat the rare disease population. For example, it is important to consider that in the rare disease model – also known as the Orphan Drug Act in the U.S. and “Orphan Designation” in the EU – there may be limited opportunity to study the disease and replicate clinical trial results due to the inherently small eligible patient population.

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