Access to unlicensed medicines, who should pay when they are not provided for free?

The rising cost of clinical development, license submissions, commercial product launches, and affiliate management in all countries around the world, coupled with the ethical obligation to ensure that eligible patients have access to new treatments, has led some pharmaceutical and biopharmaceutical companies to review their approach to access to medicine. The traditional US first launch, followed by European Union approval and then a strategic launch process, can eventually ensure access in the key markets with developed healthcare systems. For many other countries, providing access via the current legislation available for unlicensed medicine supply can provide a solution for increasing access. This option can be considered for broadening access to a greater number of eligible patients in more countries where unlicensed supply may be the only option, for example, if no clinical trials or commercial product supplies are available. This article looks specifically at the key financial and reimbursement considerations for unlicensed medicines and how some companies are adopting a “charged for” early access model that can be sustainable and affordable from their perspective. It is also important to consider how sustainable a charged program would be for the patient and the relevant payer, as they may expect an unlicensed treatment is provided free of charge. However, if the sponsor or manufacturer simply cannot afford to run a free supply program, the patient is faced with a more serious problem, that of no access at all, either charged or free. The objective of this article is to raise awareness amongst interested stakeholders from different perspectives, including the patients. Unlicensed medicines are usually only prescribed when there is a serious or life-threatening unmet need, and the implications for the company, physician, patient, and payer should be clear if access to treatment depends on the ability to pay.

Unlicensed medicines

Richard Griffith, Senior Lecturer in Health Law at Swansea University, discusses the law relating to the supply and administration of unlicensed medicines to meet the special need of a patient.