Showing Value in the Design of Clinical Trials

As RCTs undergo rethinking by pharma, the industry must better understand the impetus for change. In this report, you will discover:

  • The importance of delivering value propositions that meet health outcomes, cost-effectiveness and patient outcomes
  • How multi-faceted clinical development programmes can incorporate new parameters in support of traditional RCTs
  • New trends in trial design, including adaptive licensing, real-world data-gathering and composite clinical trials

The pharmaceutical industry’s view of RCTs is changing. In Designing Clinical Trials to Show Value, you will:

  • Understand how new concepts of value created by cost-conscious healthcare stakeholders are transforming clinical development programmes
  • Gain insight into predictions of emerging trends in big data, patient involvement in trial design and the relationship between drug regulators and health technology assessment bodies
  • Be able to plan for multi-faceted clinical development programmes that include new value parameters
  • Understand new trends in clinical trials that promise to deliver product value sought by regulators, health systems and patients
  • Designing Clinical Trials to Show Value answers key questions:
  • What does ‘value’ mean in the current, cost-consciousregulatory and healthcare environment?
  • How is ‘value’ understood by stakeholders who increasingly hold sway over market access and product uptake?
  • How can the industry adapt traditional RCT design to deliver more sophisticated value across a range of levels?
  • What can be gleaned about product development programmes from composite trials such as the Salford Lung Study?
  • How can pharma manage its various functions to deliver comprehensive value though clinical development programmes?


1. Executive summary
2.Introducing new conceptions of value
2.1. The RCT standard
2.2. Disruptive change
3.Defining value
3.1. A multi-faceted understanding of value
3. 1.1. A shift towards the supply of outcomes
3.2. Payer priorities
3. 2.1. Economic modelling
4.Creating genuine value for stakeholders
4.1. Understanding variations in value assessment
4. 1.1. Other elements in the value equation
4. 1.2. Quality of life as a value component
4.2. The shifting balance of power among stakeholders
4.3. Patients and payers
4. 3.1. The rise of the payer
4.3. 1.1. Value for payers
4.3. 1.2. Prevailing market conditions
4.4. Value convergence between stakeholders
4.5. Value from RCTs
4. 5.1. Making RCTs more efficient
4.5. 1.1. Increased complexity in CTs
4. 5.2. The benefits of data-mining
4. 5.3. Building value into RCTs
4. 5.4. Patient-reported outcomes
4.5. 4.1. New technologies
4.5. 4.2. The patient experience
4.5. 4.3. Promoting adherence
5.Social media
5.1. PatientsLikeMe
6.Patient involvement in trial design
6.1. Patient influence
6.2. Huge appetite for patient involvement
6.3. Patient advocates
7.Big data and composite trials
7.1. Levels of application for big data
7.2. Leveraging CPRD
7.3. The Salford Lung Study
8.Pragmatic trials
9.Adaptive licensing
9.1. Binary system
9.2Adaptive licensing in practice
10.Combining RCTs with real-world, pragmatic studies and adaptive licensing needs early discussions
1 0.1. Challenges and advantages of an early value strategy
10.2.alking to regulators and payers
1 0.3. Payer direction on value
1 0.4. Regulator-HTA convergence
1 0.5. Balancing global programmes with local demands

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