Compassionate access to orphan drugs in the USA

The “lottery” process for rare disease patients

NORD, the National Organization for Rare Disorders in the USA, has many programs of advocacy, education, and research. It also offers medication assistance programs for uninsured patients or for people who are privately insured but cannot afford expensive orphan drugs, depending on what health insurance they have. The programs also target recipients of Social Security Disability Insurance, who are too disabled to work, and parents of sick children, who have had to stop working in order to care for their child. 'The programs started in 1998 when the manufacturer of an expensive drug asked NORD to ensure that uninsured and low-to-middle income patients would not be denied their medication if they could not afford it,' says Maria Harding, NORD Vice President for Patient Services. 'The manufacturer thought it would be better if NORD, an unbiased independent charity, made the decisions as to who would get the free drugs.' And that's how the programs started. In April 2006, NORD is managing 26 programs for a variety of drugs. These programs include medication assistance (for uninsured patients), medicare and insurance top up (when health insurance is not enough to cover the cost of the drugs), emergency programs (for drugs in short supply), and expanded access programs (for compassionate use).

Compassionate use can be granted for experimental drugs when patients do not participate in a clinical trial and when the pharmaceutical company is willing to provide the drug. The expanded access programs for compassionate use administered by NORD work differently from the European programs.

 The process is the following:

• The doctor refers the patient in need of a drug to a Contract Research Organisation (CRO), which is an entity that collects scientific data for protocols.
• The CRO collects demands from doctors and sends them to NORD.
• NORD checks that each demand is complete (e.g. it must include a patient consent form; the patients must comply with other rules of the protocol; and they must qualify under strict medical criteria).
• NORD then enters the demands in a database and performs a “computerized random selection” to select the patients that will benefit from the program.
• NORD notifies non-selected and selected patients, as well as their doctors and the pharmaceutical company.

These programs are sometimes referred to as lotteries, because the selection process is totally random. 'The problem is that there aren't enough drugs for everybody,' says Maria Harding. 'This process is therefore considered a fair process.' The frequency and number of selections are determined by the pharmaceutical company. The percentage of patients selected varies according to the amount of the drug given by the company and the number of demands. It often reaches 90%, but in some cases, for example when the drug is new and not yet available, it can be as low as 20%. 'Patients whose names are not selected continue to be eligible for subsequent selections,' says Maria Harding, 'so that in many cases, everyone ends up accessing the drug. Each selection can be replicated for FDA¹ inspection; this is important in terms of credibility and reliability.'

'The expanded access programs (EAPs) have faced challenges. First, there is always a limited drug supply. And then, the randomized access has not always been well accepted in the community. There have also been some issues with the size of a program once it is started.' NORD is putting forward a set of questions regarding the EAPs to the community, rare disease organisations and pharmaceutical companies. Some of these questions include: Should there be a threshold of demand before an EAP is created? Can guidelines be developed to avoid reinventing the wheel with each new EAP? In any case, the controversy is ongoing, and the word “lottery” will keep ringing in many ears.

For more information:
NORD's Medication Assistance Programs

¹US Food and Drug Administration

This article was previously published in the June 2006 issue of our newsletter.

Author: Jerome Parisse-Brassens
Photos : doctor © 2004 Mayo Foundation for Medical Education and Research ; pills © 2004 budgetstockphoto.com
Graphics: NORD logo © NORD - National Organization for Rare Disorders