All medicinal products introduced on the market are the result of lengthy and costly R&D conducted by pharmaceutical and biotech
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
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'
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.
Authorities have set standards concerning the minimal data that is necessary to corroborate a marketing authorization application.
At the same time, they have introduced exceptions to allow patients in need to benefit from a promising experimental treatment
prior to completion of full testing or prior to its launch onto the market.
Resources for Monitoring Compassionate Use Information
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