Managing Drugs for Rare Genetic Diseases: Trends and Insights

This article evaluates recent trends and challenges in health system management of exceedingly rare genetic diseases, from the perspective of the manufacturer, managed care organization, physician, and actuary.

Mark Zitter, MBA
Chief Executive Officer, The Zitter Group, Millburn, N.J.

ABSTRACT: Managed care organizations generally pay for expensive drugs that treat rare genetic diseases because few patients have these conditions, the conditions are often life threatening, and the benefit design mandates coverage. In most cases, the cost-control measures and management restrictions applied to many other specialty pharmaceuticals do not make sense for the orphan drugs used to treat extremely rare genetic conditions because treatment alternatives usually are lacking for rare conditions (e.g., Gaucher's disease) and because the per member per month costs for truly rare conditions are low. The increasing number of biologic and injectable therapies for more common conditions, however, is prompting managed care decision makers to manage these new therapies more actively. There is a potential that treatments for rare disorders will be swept up in this broad-based response. This article provides definitions, background, and stakeholder perspectives on this topic and describes recent trends and challenges in health system management of exceedingly rare genetic diseases. The author suggests that current protocols are appropriate for managing rare and ultra-rare diseases, and that applying more active management practices to less rare diseases is neither efficient nor productive; in fact, it may be counterproductive.

Author correspondence:
Mark Zitter, CEO
The Zitter Group
75 Main St., Suite 303
Millburn, NJ 07041

Disclosure: The author has a consulting relationship with Genzyme Corp.
This monograph was supported by an educational grant from Genzyme Corp.

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HAP, a subsidiary of Henry Ford Health System, is a nonprofit health plan providing coverage to individuals, companies and organizations. This executive develops strategies to meet membership and revenue targets through products, pricing, market segmentation and advertising.  Aligns business among Business Development, Commercial Sales, Medicare and Public Sector Programs and Product Development. Seeks to enhance and be responsible for business development and expansion through the development of an effective product portfolio, strong interpersonal relationships and service excellence.

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