After going from doctor to doctor, I tried to think of how the doctors must have felt…. This is
what I think:“This woman is presenting this odd disease [lymphangioleiomyomatosis] that no …

Patient access to new cancer drugs in the EU involves centralised licensing decisions by
regulators as well as reimbursement recommendations in the context of national healthcare …

Importance Ensuring patients have access to safe and efficacious medicines in a timely
manner is an essential goal for regulatory agencies, one which has particular importance in …

Background: The tyrosine kinase inhibitor ponatinib is the only treatment option for chronic
myelogenous leukemia patients with T315I (gatekeeper) mutation. Pharmacovigilance …

Purpose: Anticancer drug development is inefficient, but genetically engineered murine
models (GEMM) and orthotopic, syngeneic transplants (OST) of cancer may offer …

To date, five cancer treatment modalities have been defined. The three traditional modalities
of cancer treatment are surgery, radiotherapy, and conventional chemotherapy, and the two …

Highlights•New approvals for cancer have increased dramatically since
1990.•Biotechnology takes the dominant role in early-stage development, whereas …

Background To assess uncertainty in regulatory decision-making for orphan medicinal
products (OMP), a summary of the current basis for approval is required; a systematic …

Conditional marketing authorization (CMA) in the European Union (EU) is an early access
pathway for medicines that show promising therapeutic effects, but for which comprehensive …

Several large pharmaceutical companies have selectively downsized their neuroscience
research divisions, reflecting a growing view that develo** drugs to treat brain diseases is …