James Chambers has put collectively a sequence of papers well being features (and value effectiveness) utilizing the Tufts Price Effectiveness Evaluation Registry. In these research , he finds that remedies for uncommon illnesses, specialty medicine, and people who obtain expedited evaluation are extra seemingly to supply the biggest well being profit (≥0.9 QALYs gained versus comparator).
Chambers et al. (2020) exhibits that is true for remedies for uncommon illness:
Chambers et al. (2017) exhibits that is true for remedies receiving expedited evaluation:
and Chambers et al. (2014) exhibits that that is true for specialty medicine.
Observe that the bigger well being features are sometimes correlated with increased drug costs. Within the case of uncommon illness, these extremely useful remedies are much less prone to be price efficient, resulting from excessive therapy price. This shouldn’t be shocking, nevertheless, as increased costs are wanted to fund R&D efforts since–by definition–market sizes are smaller for uncommon illnesses. Then again the Chambers et al. (2014) research exhibits that whereas specialty medicine price extra, price effectiveness is similar to conventional medicine as a result of well being features are increased on common.