Conditional approvals for autologous stem cell-based interventions: Conflicting norms and institutional legitimacy

Tsung Ling Lee, Tamra Lysaght

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

Demands from patients, health-care professionals, and industry to streamline the market approval process for promising new therapies has prompted the introduction of programs that can provide more rapid access to stem cell-based products before evidence of safety and efficacy has been demonstrated in clinical trials. These products may be approved for marketing under “conditional authorizations,” while uncertainty around safety and efficacy is reduced through the collection of clinical data in observational trials or registries. The rationale for conditional approval programs assumes that patients with unmet medical needs will benefit with rapid access to novel stem cell therapies. It also assumes that data gathered in actual clinical contexts is inherently better at reducing uncertainty than conventional clinical trial methods of demonstrating safety and efficacy. These assumptions may be overly optimistic and do not account for the broader societal burdens of prematurely releasing high-cost therapies with uncertain safety risks and benefits on to health-care markets. This essay focuses on the introduction of conditional approval programs for autologous somatic stem cell therapies and argues that these programs may conflict with, and potentially undermine, the normative commitments of regulatory agencies charged with promoting population health and protecting vulnerable groups from harm and exploitation. It concludes with suggestions of how programs designed to accelerate access to potentially helpful but experimental interventions could be reconfigured to be more equitable.

Original languageEnglish
Pages (from-to)59-75
Number of pages17
JournalPerspectives in Biology and Medicine
Volume61
Issue number1
Publication statusPublished - Dec 1 2018
Externally publishedYes

Fingerprint

Illegitimacy
Stem Cells
Safety
Health Care Sector
Cell- and Tissue-Based Therapy
Uncertainty
Clinical Trials
Adult Stem Cells
Marketing
Registries
Patient Care
Costs and Cost Analysis
Legitimacy
Health
Therapeutics
Therapy
Population
Efficacy

ASJC Scopus subject areas

  • Issues, ethics and legal aspects
  • Health Policy
  • History and Philosophy of Science

Cite this

Conditional approvals for autologous stem cell-based interventions : Conflicting norms and institutional legitimacy. / Lee, Tsung Ling; Lysaght, Tamra.

In: Perspectives in Biology and Medicine, Vol. 61, No. 1, 01.12.2018, p. 59-75.

Research output: Contribution to journalArticle

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