In the global scheme of medicines provision, low- and middle-income countries (LMICs) face difficulties in ensuring medicine access. Aside from the improvements still needed to healthcare infrastructure, financing, and service delivery, these countries also face issues in prioritizing their health services and ensuring the sustainability of their benefits packages. These issues are complex, but even more so when considering inclusion of off-label medicines and the benefits, risks, and political considerations they need to consider.
So, what are off-label medicines?
Off-label use of medications involves “prescribing currently available and marketed medications but for an indication (e.g., a disease or a symptom) that has not been approved by a country’s drug administration”. The term can also apply to the use of a marketed medication in a patient population (e.g., pediatric), dosage, or dosage form that does not have a country formal approval.” It can be common practice in countries, especially amongst patient populations that are less likely to be included in clinical trials, such as pregnant women, children, extremely elderly and mental health patients. Medicines may also be registered for a certain indication in some countries but not in others, which begs the question on their registration and availability in countries that may have more stringent regulations on off-label medicines. They can be controversial because they have not undergone a rigorous assessment process to ensure safety and clinical benefit. Nevertheless, obtaining approval for a new medical indication can be resource and time consuming and industry players are not willing to invest, especially when medicines are not patented.
Using off-label medicines for benefits packages is an unexplored area for healthcare priority setting, though it can have a significant positive or negative effect to health outcomes and efficiency of healthcare systems. Countries may have regulations to manage off-label medicines use; however, the full understanding of its current situation is not often evaluated. To address this, two academic teams in Indonesia and Thailand explored the laws, regulations, and use of off-label medicines in Australia, Indonesia, Singapore, Thailand, and the United Kingdom as well as interviewed stakeholders and conducted focus group discussions (in Indonesia) to understand the political economy of off-label prescription and identify policy solutions for Indonesia and other countries that may have legal constraints regarding the use of off-label medicines. They found that these countries, except Indonesia, legally allow for the use of off-label medicines in their public health insurance schemes given their potential benefits. To mitigate the risk, Australia and the UK developed a clear process and rigorous mechanism for priority-setting of off-label medicines, while Singapore and Thailand apply general approaches of health technology assessments (HTA) to consider inclusion of off-label medicines in their medicines formulary.
These results were presented to Indonesian policymakers and stakeholders on March 17, 2017, in Jakarta, Indonesia. Several policy recommendations for relevant actors were put forward based off the results of the studies, such as recommending healthcare authorities and payers to: fund the use of off-label medicines with strong scientific evidence for the benefits package; control the marketing and use of off-label medicines with no evidence of clinical benefit and safety, or, if there is clear evidence of harm; provide incentives (e.g. limit the cost and effort required) for industry to register medicines for off-label medicine indications; and, implement national guidelines on the use of off-label medicines at the policy level and for individual physicians. While this issue is still emerging in Indonesia and other countries, this policy forum shows that the movement is starting.
 Ten Common Questions (and Their Answers) About Off-label Drug Use. Christopher M. Wittich, MD, PharmD; Christopher M. Burkle, MD, JD;and William L. Lanier, MD
 Health technology assessment (HTA) refers to the systematic evaluation of properties, effects, and/or impacts of health technology. It is a multidisciplinary process to evaluate the social, economic, organizational and ethical issues of a health intervention or health technology. The main purpose of conducting an assessment is to inform a policy decision making.http://www.who.int/medical_devices/assessment/en/