Well being positive factors from uncommon illness, specialty and expedited assessment medication – Healthcare Economist

James Chambers has put collectively a sequence of papers well being positive factors (and price effectiveness) utilizing the Tufts Value Effectiveness Evaluation Registry. In these research , he finds that therapies for uncommon ailments, specialty medication, and people who obtain expedited assessment are extra probably to supply the biggest well being profit (≥0.9 QALYs gained versus comparator).

Chambers et al. (2020) reveals that is true for therapies for uncommon illness:

Chambers et al. (2017) reveals that is true for therapies receiving expedited assessment:

and Chambers et al. (2014) reveals that that is true for specialty medication.

Observe that the bigger well being positive factors are sometimes correlated with larger drug costs. Within the case of uncommon illness, these extremely useful therapies are much less prone to be price efficient, as a consequence of excessive remedy price. This shouldn’t be stunning, nevertheless, as larger costs are wanted to fund R&D efforts since–by definition–market sizes are smaller for uncommon ailments. Alternatively the Chambers et al. (2014) research reveals that whereas specialty medication price extra, price effectiveness is corresponding to conventional medication as a result of well being positive factors are larger on common.