Browsing by Author "Mamdani, Muhammad"

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    Estimating the clinical cost of drug development for orphan versus non-orphan drugs
    (2019-01-10) Jayasundara, Kavisha; Hollis, Aidan; Krahn, Murray; Mamdani, Muhammad; Hoch, Jeffrey S; Grootendorst, Paul
    Abstract Background High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool. However, estimates of the cost of orphan drug development are sparse. Methods Using publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs. Results We found that the out-of-pocket clinical costs per approved orphan drug to be $166 million and $291 million (2013 USD) per non-orphan drug. The capitalized clinical costs per approved orphan drug and non-orphan drug were estimated to be $291 million and $412 million respectively. When focusing on new molecular entities only, we found that the capitalized clinical cost per approved orphan drug was half that of a non-orphan drug. Conclusions More discussion is needed to better align on which cost components should be included in research and development costs for pharmaceuticals.
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    Intrapleural Dornase and Tissue Plasminogen Activator in pediatric empyema (DTPA): a study protocol for a randomized controlled trial
    (2017-06-24) Livingston, Michael H; Mahant, Sanjay; Ratjen, Felix; Connolly, Bairbre L; Thorpe, Kevin; Mamdani, Muhammad; Maclusky, Ian; Laberge, Sophie; Giglia, Lucy; Walton, J. M; Yang, Connie L; Roberts, Ashley; Shawyer, Anna C; Brindle, Mary; Parsons, Simon J; Stoian, Cristina A; Cohen, Eyal
    Abstract Background A randomized controlled trial of adults with empyema recently demonstrated decreased length of stay in hospital in patients treated with intrapleurally administered dornase alfa and fibrinolytics compared to fibrinolytics alone. Whether this treatment strategy is safe and effective in children remains unknown. Methods/design This study protocol is for a superiority, placebo-controlled, parallel-design, multicenter randomized controlled trial. The participants are previously well children admitted to a children’s hospital with a diagnosis of empyema requiring chest tube insertion and fibrinolytics administered intrapleurally. Children will be randomized after the treating physician has decided that pleural drainage is required but prior to chest tube insertion. After chest tube insertion, participants in the treatment group will receive intrapleurally administered tissue plasminogen activator (tPA) 4 mg followed by dornase alfa 5 mg. Participants in the placebo group will receive tPA 4 mg followed by normal saline. Study treatments will be administered once daily for 3 days. All participants, parents or caregivers, clinicians, and research personnel will remain blinded. The primary outcome is length of stay from chest tube insertion to discharge from hospital. Secondary outcomes include time to meeting discharge criteria, chest tube duration, fever duration, need for additional procedures, adverse events, hospital readmission, cost of hospitalization, and mortality. Discussion This multicenter randomized controlled trial will assess the safety, effectiveness, and cost-effectiveness of combined treatment with dornase alfa and fibrinolytics compared to fibrinolytics alone for the treatment of empyema in children. Trial registration ClinicalTrials.gov: NCT01717742 . Registered on 8 October 2012.