Development of an outcomes-focused data platform to empower policy makers and clinicians to optimize care for patients with hematologic malignancies

Topic description

Specific Challenge:

Hematologic malignancies (HMs) account for about one-third of cancer cases in children and about one-third of cancer deaths. Due to the rarity of the conditions and the diverse healthcare practice across EU, current healthcare systems are challenged with

• lack of definition and alignment on outcomes
• policy makers having limited benchmark data to evaluate the risk/benefit ratio and value,
• clinicians having to make treatment choices based on short-term, surrogate and often not comparable data
• payers having the need to make reimbursement decisions on life prolonging options with limited data and finite budgets.

This can lead to patients not having access to the right treatment at the right time.

Key objectives include:

• define standard sets of outcomes for HMs that are relevant and meaningful to patients and clinicians
• communicate and support the use of the defined standard outcomes data sets on selected HMs
• develop a data sharing platform that empowers clinicians and policy stakeholders to improve decision making
• propel key medical and policy initiatives through fact based decisions based on large, diverse harmonised data sets.

Improved clinical development based on clear definition of endpoints, outcome measures leading to consistent assessments across EU;

• better understanding of the natural history of the diseases;
• ability to prove the value, safety and effectiveness of therapies, therefore faster patient access to these therapies.