Medicine development for rare diseases, including inborn errors of metabolism (IEMs) is challenging. Many academic innovations fail to reach the patient, either by stranding in the translational... Show moreMedicine development for rare diseases, including inborn errors of metabolism (IEMs) is challenging. Many academic innovations fail to reach the patient, either by stranding in the translational stage or due to suboptimal patient access related to pricing or uncertain effectiveness. Expanding and solidifying the role of the academic in public-private partnerships (PPPs) may present an innovative solution to help overcome these complexities. This narrative review explores the literature on traditional and novel collaborative approaches to medicine development for rare diseases and analyzes examples of PPPs, with a specific focus on IEMs. Several academic institutions have introduced guidelines for socially responsible licensing of innovations for private development. The PPP model offers a more integrative approach toward academic involvement of medicine development. By sharing risks and rewards, failures in the translational stage can be mutually absorbed. If socially responsible terms are not included, however, high pricing can impede patient access. Therefore, we propose a framework for socially responsible PPPs aimed at medicine development for metabolic disorders. This socially responsible PPP framework could stimulate successful and accessible medicine development for IEMs as well as other rare diseases if the establishment of such collaborations includes terms securing joint data ownership and evidence generation, fast access, and socially responsible pricing. Show less
Al-Mansoori, R.; Arjomand, B.; Larik, J.; Petcu, C.; Ponzio, R.; Siddique, M.; Torrez, P. 2020
The Doha Forum Report 2020 examines the leadership deficit in multilateral cooperation toward both the coronavirus pandemic and other existing and emerging global challenges, including runaway... Show moreThe Doha Forum Report 2020 examines the leadership deficit in multilateral cooperation toward both the coronavirus pandemic and other existing and emerging global challenges, including runaway climate change, rising political violence, menacing cyber-attacks, and growing inequality within and between countries. It finds that present international efforts are too often fragmented, delayed, ad hoc, and under-resourced, while pointing to promising new public-private partnerships to spur innovation through new technologies and to promote decent job and wealth creation opportunities. The report also recommends a roadmap for a durable, green, and broad-based global recovery and institutional revitalization, culminating in a 2023 World Summit on Inclusive Global Governance. With the recent news on the expected arrival of one or more effective vaccines, the road to recovery must chart a bold new course and avoid a return to the “old normal” of unsustainable practices. Show less
Hmlinen, I.; Trnwall, O.; Simell, B.; Zatloukal, K.; Perola, M.; Ommen, G.J.B. van 2018
