McKinsey May 13, 2021
Björn Albrecht, Emily Capra, Meredith Reichert, and Pablo Salazar

The small number of people with rare diseases raises challenges for the development and launch of all therapies for such conditions, but rare cancers face extra barriers to widespread treatment.

Greater understanding of disease biology and revolutionary diagnostic and therapeutic technologies have unlocked increasingly effective treatments for people with cancer. Diseases for which the only recourse was palliative care now have treatment-response rates of more than 80 percent.1 Notwithstanding those encouraging improvements, there are a number of rare cancers, many of them genetically defined, for which there are still no effective treatments. For example, KRAS, an oncogene commonly mutated in non-small-cell lung cancer (NSCLC) and other diseases that affect thousands of people, has historically been considered an “undruggable” target.2 However,...

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