Compassionate use programs facilitate patient access to promising medicinal products at an early stage in the drug development process, when the information already gathered for the drug benefit/risk profile suggests that patients will not be exposed to safety hazards.
This article reviews the relevant legislative framework in Canada, the EU, and United States, and highlights the applicable regulatory agencies' efforts to provide more efficient programs by revising their current regulation on compassionate use.A long and pricey haul
Drugs under investigation must undergo highly regulated nonclinical and clinical research to prove their quality, efficacy, and safety in the target population before they can be made commercially available. Additionally, pricing, reimbursement, and distribution discussions might delay the availability of the product in some countries even after a medicinal product has been successfully reviewed and approved by the local regulatory agencies.
Thus, the major challenge for industry is to market their product with as short a delay as possible in order to recuperate the huge financial sums invested during the R&D phase. In comparison, the hopes and expectations of the patient community are even greater, as they could possibly have at their disposal a new therapeutic arm to fight against fatal illness or seriously debilitating conditions.
All in all, the overall process for getting a drug on the market can take up to 10 years. This delay is far too long, especially for patients suffering from a seriously debilitating disease/condition and who lack efficacious treatment. Even when suitable treatment exists, the timely intervention of new and more satisfactory treatment could markedly improve the quality of patients' lives.
Exception to the rules
Although opportunities exist for patients to benefit from experimental or nonmarketed drugs—for example, by entering clinical trials or by importing the drug for personal use—they account for only a small number of cases. The actual need for access to such treatment is considerable and has brought about effective and transparent initiatives on the part of health authorities and regulatory agencies.
This exception, often referred to as compassionate use of medicinal products, is in force in many countries and has been warmly welcomed by patients, patient organizations, and activists but has unexpectedly raised many issues for pharmaceutical companies unable to deal with and satisfy the increasing demands and who face more and more difficulties in recruiting patients for clinical trials. In an effort to meet all parties' expectations and adapt to growing needs, compassionate use and related legislation/guidelines have or are being revamped in the major regions (i.e., the EU, United States, and Canada).
Compassionate use in the EU
The origins of compassionate use in the EU can be traced back to 1989, when the possibility for Member States to allow the supply of unauthorized medicinal products "in response to a bona fide unsolicited order, formulated in accordance with the specifications of an authorized health care professional and for use by his individual patients on his direct personal responsibility" was introduced in Directive 89/341/EEC.1