Excellence in Rare Diseases or Orphan Drugs

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This category relates to work in support of any drug for a rare disease or disorder or classified orphan drug at any phase of the product life cycle.

Entrants will need to demonstrate a thorough understanding of the specific healthcare environment; show how they tailored their approach to the specific challenge of rare diseases/orphan drugs and show an appropriate approach to deliver value to stakeholders and so drive the business forward.

Judges are looking for evidence of true collaboration with patient groups and healthcare professionals/advocates.

Work may be local, regional, national, multi-national or global countries or UK are eligible.

The entry should relate to activities carried out between July 2023 and June 2025.

Below is where the judges will be focusing their scores. Write your entries accordingly.

Judging criteria

Executive Summary – 200 words (not scored)

If you are nominated for an award, PMGroup may publish extracts from this summary, so ensure that it contains no confidential or sensitive information. No other part of your entry will be reproduced and the main content of your entry will always remain confidential.

Context – 400 words (20 points)

  • Demonstrate how you gained deep and meaningful insights to prepare
  • Demonstrate how you set about to address the strategy and planning
  • Outline specific needs and aims

Objectives – 400 words (20 points)

  • Demonstrate how your objectives related to all stakeholders and reinforced each other
  • The rigour and ambition of your objective setting/shared objectives
  • Were your objectives SMART (specific, measurable, achievable, realistic and timely)

Approach – 400 words (20 points)

  • How the organisation’s approach to strategy development has changed to suit the work
  • Evidence of how you created or developed a step-wise approach to your project

Measures of Success – 400 words (20 points)

  • Demonstrate how metrics have been applied to measure outcomes, take further action and enable learning
  • Demonstrate how data captured is being leveraged and used to adapt strategy and tactics on an ongoing basis
  • Evidence of engagement and positive support statements
  • Evidence of changed perceptions and behaviours that will deliver improved health outcomes