Orphan Drugs

DEFINITION OF ORPHAN DRUGS

Orphan drugs are those that are designed to diagnose, prevent or treat rare diseases.
In the EU further defined as life-threatening or chronically debilitating conditions.
The definition also encompasses therapies that are not likely to be developed under usual market conditions, where the firm can demonstrate that a return on research investment will not be feasible.
To be eligible for orphan drug status, there must not be a satisfactory treatment or, as an alternative, the proposed new treatment is to be presumed to be of major benefit.
To be eligible as an orphan indication, the incidence of the disease must be less than 5 in 10 000 people in the EU, less than 50 000 affected in Japan, or less than 200 000 affected in the USA.

SIGNIFICANCE OF ORPHAN DRUGS

1. Delivering Life-Saving Treatments

Numerous rare diseases are progressive, severe, and life-threatening (e.g., Duchenne Muscular Dystrophy, ALS).
Orphan drugs ensure prolonged survival and enhance the quality of life.

2. Filling Unmet Medical Needs

Rare diseases do not have available treatment until an orphan drug is developed.
The drugs bring hope to families and patients with otherwise incurable illnesses.

3. Facilitating Research and Development

It is costly and has poor commercial return to develop drugs for rare diseases.
The government offers incentives (tax credits, grants, market exclusivity) to prompt drug firms to invest in the research of orphan drugs.

4. Increasing Medical Knowledge

Scientific discoveries due to research on orphan drugs may help other diseases.
Knowledge about genetic and metabolic disorders can be useful in personalized medicine.

5. Global Health Impact

Even though rare individually, rare diseases impact more than 300 million people globally.
Orphan drugs make sure that no patient is left behind, irrespective of the prevalence of the disease.

6. Economic and Social Benefits

Investment in orphan drugs saves long-term healthcare costs as it prevents complications of the disease.
It enhances patient productivity and alleviates the burden on caregivers and families.

HISTORY OF ORPHAN DRUGS

The evolution of orphan drug development has progressively shifted in time due to the growing cognizance of rare diseases and the pressing need for more specialized treatment approaches. Here is a brief history of the subject:

1. Early Peculiarities (Until 1980s)

Before the '80s, pharmaceutical companies largely ignored rare diseases on account of little commercial attraction.
Patients suffering from rare diseases mostly had little or no treatment options and thus serious health consequences and early mortality.
Certain drugs were used "off-label" (drugs that were approved for other conditions used for the treatment of rare diseases).

2. Orphan Drug Act (1983, USA)

A significant turning point in orphan drug development was the passing of the Orphan Drug Act in 1983 in the U.S.
This act was pushed by some patient advocacy groups, with the main force being amplified by the National Organization for Rare Disorders.
Important incentives introduced were:

Market exclusivity for 7 years for approved orphan drugs.

Tax credits for R&D expenses incurred during clinical investigations.

Waivers of FDA fees and grants for research.

The introduction of the ODA greatly increased development for orphan drugs, thus promising therapies for previously neglected rare diseases.

3. Global Adoption of Orphan Drug Policies

Inspired by the success of the U.S. Orphan Drug Act, other countries put or began to advocate similar policies.
European Union (2000): provided 10 years of market exclusivity and other research incentives.
Japan (1993): formed the Orphan Drug Designation System.
Australia (1998): introduced the orphan drug regulation under TGA.
China (2019): enacted rare disease policies to advance orphan drug development.

4. Forthcoming Developments, New Discoveries (2000-Now)

Advances in biotechnology and genetics enable the development of targeted orphan drugs.
The number of FDA-approved orphan drugs changed dramatically (more than 600 since 1983).
Gene therapy and precision medicine have led to breakthrough treatments for such diseases as Spinal Muscular Atrophy and Duchenne Muscular Dystrophy.
Pharmaceutical companies are investing more in orphan drugs due to greater incentives and new technologies.

5. Future of Orphan Drug Development

Attention is being drawn to gene therapy, stem cell treatments and personalized medicine.
Assessment policies will be refined for better global access and affordability of orphan drugs.
Ongoing advocacy will be essential to guarantee that no rare disease is left without treatment.

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