Orphan Drugs and Humanitarian Use Technology

Orphan Drugs

The Orphan Drug Act of 1983 (ODA) defines an “orphan drug” as a product that treats a rare disease affecting fewer than 200,000 Americans. Since the Orphan Drug Act passed, over 100 orphan drugs and biological products have been brought to market.

The ODA is intended to stimulate the research, development, and approval of products that treat rare diseases. This mission is accomplished through a combination of direct funding, tax incentives, and protected markets for drugs developed under the provisions of the act such as:

The “Sponsor” (the drug maker) is granted 7 years of marketing exclusivity after approval of its orphan drug product.

Sponsors also are granted tax incentives for clinical research they have already undertaken.

FDA’s Office of Orphan Products Development coordinates research study design assistance for sponsors of drugs for rare diseases and also encourages sponsors to conduct open protocols, allowing patients to be added to ongoing studies.

Grant funding is available to defray costs of qualified clinical testing expenses incurred in connection with the development of orphan products.

Despite its noble intentions, the Orphan Drug Act has been criticized since it was first introduced into Congress. These criticisms generally relate to the market protection and tax incentives awarded to developers. See Urch Publishing’s Orphan Drugs to 2008 for the European Union perspective on orphan drugs and markets.

As of July 31, 2006 the FDA listed 1,617 drugs / biological compounds on its “active list” as being designated or approved as an orphan drug with some 250-odd products being marketed in the . The most recent listings can be found at the FDA’s List of Orphan Designations and Approvals.

Drugs Developed under the Orphan Drug Act

Some drugs developed under the provisions of the ODA have been recertified for use in the treatment of conditions other than their original targets. Each drug in the following list was once an orphan but now has worldwide sales of at least $1 billion/year.

Avonex(TM) (multiple sclerosis)

Epogen(TM) (originally for anemia associated HIV infection, now for other anemias)

Intron A(TM) (originally for the treatment of Kaposi’s sarcoma, now other cancers and viral diseases)

Neupogen(TM) (originally for the treatment of neutropenia [low white blood cell levels] associated with bone marrow transplant, now for neutropenia of other origins)

Remicade(TM) (originally for the treatment of Crohn’s disease, now for specific types of arthritis and colitis)

Humanitarian Use Devices and Exemptions

The United States Food and Drug Administration has defined a Humanitarian Use Device (HUD) as a device that is intended to treat or diagnose a disease (or a condition) that affects fewer than 4,000 individuals per year in the United States. FDA regulations (21 CFR 814.124) make a provision for the submission of a Humanitarian Device Exemption (HDE) in which the manufacturer is not required to provide the results of scientifically valid clinical investigations demonstrating that the device is effective for its intended purpose prior to marketing.

Take note of the phrase scientifically valid clinical investigations in the previous sentence. This means that the device manufacturer and the patient’s physician are still responsible for monitoring the device’s performance and possible complications that may arise from its use but the manufacturer does not have to submit an extensive evaluation as would be required for unrestricted use.

Companies with Products Meeting the FDA Humanitarian Use Device and Humanitarian Device Exemption Regulations

Abiomed (implantable artificial heart and heart assistance): Despite advances in both the medical and surgical treatment of heart disease, the dream of an implantable artificial heart has proven elusive. Most research in this area has been directed to “heart rest and recovery” devices, meaning artificial heart technology that takes over part of the heart’s workload until the patient is clinically stable or as a “bridge to transplant.” Abiomed recently received a FDA Humanitarian Device Exemption for its AbioCor(TM) II implantable artificial heart.

Cyberkinetics Neurotechnology Systems (devices that improve quality of life in brain/spinal cord injury patients): Cyberkinetics’ Andara(TM) Oscillating Field Stimulator (Andara(TM) OFS Device) is currently in limited clinical trials as an investigational device. Cyberkinetics is actively working toward combining its implantable devices with newer neurophilic (promoting nerve cell growth) technologies with the goal being the first effective spinal cord injury treatment system.

Medtronic(Enterra(TM) Implantable Gastric Stimulation Device): Gastroparesis is a condition in which the stomach fails to properly empty following meals. It is found primarily in diabetes (particularly Type I or insulin-dependent diabetes) although it has been reported in Parkinson’s disease and following injury or inflammation to the Vagus nerve. The primary symptoms of Gastroparesis are persistent nausea and vomiting, abdominal pain, heartburn, and erratic blood glucose levels.

Although there have been reports documenting successful treatment of gastroparesis with medication and dietary changes, the use of a gastric stimulation device (a stomach “pacemaker”) may result in a dramatic improvement in the patient’s symptoms. The patient’s gastroenterologist will be happy to discuss the latest published studies regarding gastric stimulation as well as potential benefits that can be reasonably expected if this device is used.


The information presented in this article and its included links is of an informational nature only and is not intended as a recommendation of any changes in the reader’s health care program. Before making any changes in diet, medications, or other treatments the reader is strongly advised to consult with their health care provider.

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