Expanded Access – Frequently Asked Questions
What good does an expanded access program do?
A well-designed expanded access program offers a treatment option to patients who frequently have no other options. In addition, it can provide information – safety, quality of life – about the drug in a ‘real world' setting, and increase patient/provider knowledge and ‘buzz' about the drug before it hits the market. Thus, both patients and companies can benefit from a properly designed and executed expanded accessprogram.
When should expanded access be considered? What about drugs that are being tested in several forms of cancer?
Expanded access should be considered when a population of patients who might benefit from the treatment can be defined – for example, after Phase 2 trial results are known, or once interim Phase 3 trial results show a benefit.
A drug that shows benefit in one form of cancer may not produce a similar benefit in another cancer, which is why some level of efficacy data is important. As targeted treatments become more common, the argument could be made that any type of tumor with the appropriate molecular markers might respond to treatment with a drug. This is a good argument that should be tested in clinical trials.
Some argue that compassionate use gets in the way of clinical trials.
Exclusion criteria for expanded access can state that if a patient is eligible for the trial, he is ineligible for expanded access.
Some argue that compassionate use is a bad use of resources. In other words, they feel that in cases where developmental drugs are in short supply, surplus drug should be used for additional research. In addition, the programs are expensive especially if additional data (safety, quality of life) is gathered.
There are times, especially with large molecule drugs, where the resource supply issue is valid. Manufacturing biologics as opposed to drugs can be complex and expensive. Both time and money are required to qualify a new manufacturing plant under FDA guidelines. There are verifiable supply constraints that may greatly limit the amount of drug available to both clinical trials and expanded access programs.
At the same time, many expanded access programs when a manufacturer begins to ramp up supply of a drug prior to expected approval. In this situation, expanded access is possible.
The expense of the programs is a very real issue. On the other hand, EAPs offer an opportunity to learn tremendous amounts about the drug prior to large-scale marketing. A clear understanding of safety, side effects and quality of life issues benefits patients tremendously.
Some argue that compassionate use is actually cruel, because in most cases it's not clear that the drug will actually benefit the patient. In addition, if the drug is in short supply and a lottery is used to allocate the drug, the stress involved is very difficult for patients to handle.
Cancer is cruel. No cancer drugs are effective for all patients. Searching for treatment options is stressful. Patients have the right to make fully informed decisions about their treatment options, including the use of developmental drugs when appropriate.
Why expanded access instead of single patient compassionate use?
Individual compassionate use can be much more difficult for a company to deal with than a defined program with well-described qualification procedures. Expanded access makes an experimental drug available to people within a defined population. When supplies are limited, an expanded access program can operate via a completely objective allocation system, like a lottery. A single patient access approach is, by nature, not objective. At the same time, there are times, especially for patients with very rare cancers, when single patient compassionate use may be appropriate.
Some have suggested that drug manufacturers should be allowed to profit from selling experimental drugs prior to completing required safety and efficacy testing. Wouldn't this be a better approach than EAPs?
Unfortunately, history is full of examples where people with life-threatening illnesses are exploited for short-term profits. In fact, there have been very few dramatic advances in the treatment of cancer that have not required long patient testing, optimization, and careful assessment of safety risks.
We believe that profits are appropriate only after a rigorous assessment of both safety and efficacy has demonstrated that a new drug has value in treating the target disease and works for a reasonable proportion of the target population. In addition, experience gained with the “accelerated approval” mechanism has demonstrated that early profits don't necessarily correlate with speedier development of effective new treatments for cancer or other life-threatening diseases.
Properly designed EAPs offer a good opportunity to make promising experimental drugs available to patients who have exhausted other options without interfering with the clinical research process. In fact, EAPs have the potential to enhance the clinical research process because of the additional information they provide about use of the treatment in a real-world population.