Offering Compassionate Care While Alleviating Ethical Concerns: How Some Pharmaceutical Companies Are Meeting Both Demands

In recent years, families and friends of terminally ill patients have launched highly visible social media campaigns to secure access to potentially life-saving medicine, before those experimental drugs are approved. Pharmaceutical companies that are developing these investigational medicines often face difficult ethical and business relations dilemmas: there are limited exceptions for non-approved drug dissemination and the costs and consequences attendant on the exceptions can make either choice unpalatable. Companies and caregivers alike have struggled with how to fairly provide access to experimental drugs without negatively impacting long term drug development or approval.

Pharmaceutical companies’ development of new drugs is highly regulated, as is approval of drugs for marketing by the Food and Drug Administration (“FDA”). Moreover, investigational medicines are frequently in limited supply, and are only provided to tightly controlled clinical trials after satisfying limited safety and efficacy minimums. In restricted circumstances, the FDA permits distribution of as-of-yet unapproved drugs for compassionate use, which is defined as “a pathway for patients to gain access to investigational drugs, biologics, and medical devices for serious diseases or conditions. Such investigational drugs have not yet been approved by the FDA and they have not been proven to be safe and effective.” The exceptions include expanded access for individual patients, including for emergency use; expanded access for intermediate-size patient populations; and expanded access for widespread treatment use. A treating physician must submit an individual new drug request, which the FDA then reviews and considers if the patient or patients to be treated have a serious or immediately life-threatening disease or condition before approving or denying the request.

Accordingly, the compassionate use exception is limited, and not without risks. The inclusion of a broad class of people desperate for potentially life-saving medicine could alter the outcomes of the clinical trials, thus impacting potential approval by the FDA due to the need to report adverse events. Alternatively, distribution based on social media campaigns may result in unfair access to the investigational medicine by those who publicize their needs in a more sophisticated manner, as opposed to distribution based on medical necessity or likely efficacy. Thus, pharmaceutical companies, while sympathetic to the fears and needs of people who may not meet the entry criteria into a clinical trial, find difficulty in determining who should receive access, whether by lottery or on a case-by-case basis. Concerns about fairness as well as access to drugs create issues with either an individualized or randomized selection process.

Pharmaceutical companies, where permitted by local laws, have tried to employ programs that have permitted expanded access to investigational medicines for patients with serious illnesses upon the drug achieving certain milestones in development. As a new potential solution, one major pharmaceutical company has announced that it has begun a pilot program that will utilize a third party to review requests for investigational medicines, beginning with one particular drug, while holding out the opportunity for the model to expand across other treatment areas.

This pharmaceutical company, partnering with a respected local medical school, indicates that it will defer to the advice of a newly established advisory committee on compassionate use of experimental drugs. The committee will consist of internationally recognized medical experts, bioethicists, and patient representatives, to help prioritize access to a limited supply of investigational medicines based on the goals of equality, need, and efficacy. By transferring the decision of who should receive access outside of the company controlling the product, pharmaceutical companies may have an ethical way to provide compassionate care without also carrying the burden of allocation.

George M. Gould is Counsel in the Gibbons Intellectual Property Department.
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