Getting It Right from the Start: Applying QbD to Rare Disease Studies

Quality and effective risk management are vital to every clinical trial, and their importance is greater still when working in rare indications. Your patient population is small, vulnerable, and geographically dispersed, so there’s limited data and little opportunity to replicate clinical trial results.

It’s as if the phrase “get it right the first time” had been coined just for you.

We understand these challenges, having conducted 100 rare disease studies in the past five years, and we’ve mastered the use of quality by design to maximize their effectiveness. We’re sharing this experience in our white paper,  Applying Quality by Design to the Rare Disease Population: Special Considerations.

In it, we describe a statistical and analytical approach to trial development that builds in quality from the start. It’s a roadmap for addressing the unique needs of rare diseases and incorporating QbD principles into your next study.

A detailed understanding of the data and processes that underpin a successful trial is foundational to identifying and managing risks and improving outcomes. Thus, QbD focuses on examining study objectives, defining critical factors in meeting those objectives, and creating a plan to mitigate risk.

Compounding the challenge
Rare diseases are usually combinations of multiple indications of varying progression, severity, and manifestation. This heterogeneity makes them even more difficult to diagnose, and consequently, patients often have life-threatening afflictions when they enter a trial. That makes it difficult to develop an outcome assessment model that accurately measures the study’s effectiveness.

QbD outlines the requirements for a well-designed and -executed trial. They include:

  • Data and literature reviews to support orphan designation.
  • Detail about the drug’s novelty and previous experience with the compound.
  • Definition of the afflicted population.
  • Outcome measures that are specific and sensitive to changes in disease manifestation.
  • Development of new or optimized biomarkers to support proof of concept, guide dose selection, identify safety concerns, and provide evidence of efficacy.

What might go wrong?
The QbD model is a good tool for ensuring that rare disease trials incorporate quality from day one. Carefully followed, these principles will guard against potential threats, both known and unknown. They also can demonstrate to regulators that you incorporated robust controls and early risk detection and prevention measures.

We describe the systematic use of information to identify hazards using historical data, theoretical analysis, informed opinions, and the concerns of stakeholders. It’s all aimed at addressing the question, “What might go wrong” — and then uncovering the possible consequences.

You’ll walk through every stage in the risk management continuum: identifying, analyzing, evaluating, and managing potential pitfalls through a wide lens that involves physicians, statisticians, investigators, patients, and patient advocacy groups throughout the project life cycle, from study design to close-out.

You’ll review so much more in the full white paper. We all know that in rare disease research, every data point matters, so paying close attention to preventing errors can dramatically improve the likelihood of a successful outcome. Get the full story by downloading the white paper now.

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