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Oncology and Haematology

Targeted small molecules, biologics and cell or gene therapies are revolutionising the treatment of solid and haematologic tumours. This revolution in cancer treatment is turning once imminently life-threatening advanced cancers into increasingly chronic conditions—bringing new hope to patients.

What is the evidence for this?

Of the 41 positive opinions for new active substances granted by the Committee for Medicinal Products for Human Use in 2022, 12 (~30%) of those were for new cancer medicines.

However, regulatory challenges appear in bringing new cancer medicines to patients, as the treatment landscape segments and new medicines target progressively less common subsets of patients. We are experts in navigating the strategic regulatory challenges which need to be addressed in the modern era of cancer drug development.

We support a complete spectrum of regulatory activities for new cancer medicines that target rare, biomarker-driven subsets, and utilise Phase 1/2 or Phase 2 studies that become the pivotal efficacy and safety registrational clinical trials.

Case Study

Tyrosine kinase inhibitor – solid tumour oncology


A pharmaceutical company planned to submit a marketing authorisation application in Europe for a tyrosine kinase inhibitor.


We wrote a detailed regulatory strategy to gain approval using Phase 2 data. We supported the planning and messaging within the common technical document as part of the global filing team. We also wrote the initial submission of the paediatric investigation plan and successfully applied for an orphan designation.


Planning for the orphan strategy in the context of a non-orphan and orphan indication was pivotal to the regulatory strategy and marketing authorization was granted.

Questions you may have if you are developing an oncology/haematology medicine

  • What is the success rate of oncology clinical trials and oncology drug development?
  • What are the specific regulatory pathways and requirements for drug/biologic approval in the oncology/haematology target market?
  • What are the specific regulatory guidelines relevant to developing drugs/biologics in oncology/haematology?
  • What are the precedent targeted therapies in our related oncological disease?
  • Are there any regulatory authority schemes or accelerated pathways?
  • What is the most appropriate clinical trial design given the nature of oncological and haematological diseases?
  • How do regulatory agencies support the development of drugs for oncology and haematology?

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