Introduction

Key challenges

From initial discovery to regulatory approval, life sciences companies face formidable obstacles, including high costs, lengthy timelines, and a high rate of failure. Clinical research is typically costly for sponsors and burdensome for providers and patients. However, new methods and approaches, such as integrated evidence generation using real-world data and new technologies, such as study automation platforms, are accelerating the path from start-up to readout and reducing burden.

Key challenges with traditional research approaches

1. High costs and lengthy timelines

2. Patient retention, recruitment, and population diversity

3. Evidence gaps

Clinical Research: Key Challenges

1. High costs and lengthy timelines

The cost and time required to develop new therapies are staggering, with estimates exceeding billions of dollars and more than a decade from inception to approval. A recent report estimated that R&D costs will increase by 3% each year, reaching over $203 billion by 2024.

High failure rates during clinical trials contribute significantly to these costs. Low participation rates increase study time and cost, with 90% of trials doubling their initial timelines in order to meet enrollment goals.

12 years

Estimated time frame for a therapeutic agent to go through clinical testing to FDA approval

$1-1.8 billion

Estimated cost to develop a new therapy

$600k–$8 million

Estimated cost to sponsors for each day that a trial delays a product’s development and launch²

^2.www.ncbi.nlm.nih.gov

Clinical Research: Key Challenges

2. Patient retention, recruitment, and population diversity

Patients enrolled in clinical trials often represent a narrow subset of the real-world population, limiting the generalizability of trial results. Additionally, underpowered clinical trials with small sample sizes can fail to demonstrate the efficacy needed for regulatory approval. At the same time, registries have become too 'trialized' and can introduce bias and underrepresentation for some patient populations.

Clinical Research: Key Challenges

3. Evidence Gaps

Various stakeholders, including clinicians, payers, and patients, rely on clinical evidence to support different aspects of treatment decision-making, patient access, and informed choices:

Clinicians depend on evidence to make optimal treatment decisions that align with the unique needs and circumstances of their patients.

Payers require evidence to justify patient access to specific therapies, ensuring that healthcare resources are allocated efficiently and effectively.

Patients seek evidence to comprehend how a therapy aligns with their needs, enabling them to make informed decisions about their own care.

While traditional clinical research studies, such as randomized controlled trials (RCTs), play a critical role in establishing efficacy, their limitations become apparent when attempting to translate their findings into real-world clinical practice. RCTs often evaluate therapies in carefully selected populations under ideal conditions, which may not fully represent the diverse patient population and real-world treatment complexities. As a result, an evidence gap emerges, and clinical trial results alone may not provide sufficient evidence to effectively inform clinical decisions. Traditionally, registries are used to fill the real-world evidence gap, but they have become too trialized and burdensome in many cases for both providers and patients.