A proposed population‑based justice model for ethical gene transfer expanded access policies

Investigational clinical programs using somatic gene‑altering therapies to treat rare and ultra‑rare diseases with unmet need can carry greater risk than other modalities due to emerging technologies with limited safety data and the irreversible nature of the treatments that may rule out future approved gene treatments. The window of opportunity for patients affected by these diseases is narrow and is affected by variables like availability, manufacturing and delivery constraints, and limited research programs. Individuals with life threatening genetic conditions may seek to gain access to unapproved experimental products and processes outside of a clinical trial. In isolation, arguments for expanded access from autonomy offer insufficient grounds to introduce additional risk to a promising clinical development pathway that could result in treatment ultimately being closed to others. The Belmont principle of justice can be expanded beyond equitable enrollment and representation to also incorporate distribution and access at the population level to alleviate suffering, ameliorate adverse associated outcomes, and to prevent early mortality associated with rare genetic conditions. The broadest interpretation of equality of opportunity under distributive justice requires that ethically sound manufacturer policies governing expanded access should increase the range of opportunities for genetically disadvantaged present and future populations to receive a treatment they otherwise would not.

Autonomy alone cannot justify expanded access to investigational gene therapies. Distributive justice at the population level is the appropriate ethical standard.

What you will learn

01

Why autonomy is insufficient justification for expanded access

02

How individual access can harm entire rare disease populations

03

How distributive justice can guide ethical manufacturer policies

The ethical framework

1

Belmont principles & gaps

2

Risk to patients & programs

3

Autonomy vs distributive justice

4

Population-based policy

Key findings

Gene therapy's irreversibility means expanded access can permanently close future treatment options for the patient and others

For ultra-rare diseases, a handful of expanded access cases can threaten trial integrity and program viability for the entire eligible population

Policies must be transparent, population-centred, and independently reviewed — not shaped by public pressure

Key interests

Gene Therapy

Expanded Access

Rare Diseases

Bioethics

CGT Policy

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