An edited transcript of the presentation from Professor Julia Downing, CEO of the International Children’s Palliative Care Network, at the launch of the rapid communication for the ‘WHO guideline on balanced national policies on controlled medicines to ensure medical access and safety’ at the World Health Assembly on 23rd May 2025. The document can be accessed on the WHO website. The full document will be launched later in the year.
It was both an honour and a responsibility to be co-chairing the development of these guidelines along with my colleague, Dr Afarin Rahimi. We would like to thank everybody involved in the development of these guidelines: all those in the Guidelines Development Group (GDG), the external and internal reviewers, the team at the World Health Organization, those doing the systematic review, along with everyone who made it possible to launch these guidelines at the World Health Assembly. The guidelines were launched at a side event that was supported by the International Association of Hospice and Palliative Care, the Worldwide Hospice and Palliative Care Alliance, the International Children’s Palliative Care Network, the Union for International Cancer Control, the International Association for the Study of Pain, the Global Commission on Drug Policy, along with the WHO member states of Belgium and Switzerland.
Challenges in Access to Controlled Medicines
Sadly, we all know of places where people who need access to controlled medicines do not have them, whether the medicines are for pain, palliative care, mental health disorders, anesthetics, epilepsy – whatever the reason.
In terms of pain and palliative care, in 2021 over 80% of the world’s morphine was distributed in high-income countries, and yet, when we looked at the need, it was greatest in low- and middle-income countries (LMICs), where there is the lowest access to the world’s morphine. In terms of mental health and neurological disorders, 75% of those with epilepsy in LMICs could not access the medicines they needed. In terms of substance use disorder, medicines such as methadone and buprenorphine are not registered in over 50% of countries. Getting access to controlled medicines is a challenge in emergency and humanitarian settings, as well as for vulnerable populations such as children.
We hope that these guidelines will help reduce these inequities and enable medicines to get to the people who need them, in the place that they need them, wherever they live around the world.
Previous WHO Guidelines and New Developments
In 2019 two previous WHO guidance documents relating to pain medicines were withdrawn following concerns about perceived conflicts of interest and that the advice did not reflect current evidence. One was replaced with a new guideline on the management of chronic pain in children and this new guideline relating to ensuring balance in national policies on controlled substances will replace the other withdrawn document.
Prior to the development of these guidelines, there was a public consultation about what was needed, and the scope of the guidelines was expanded to all controlled medicines in all clinical settings, not limited to opioids for the management of pain.
In developing the guidelines and in terms of the scientific rigor, we followed the WHO gold standard methodology for the development of recommendations and good practice statements – the GRADE system – underpinned by systematic reviews. In order to manage conflicts of interest, there was an open call for people to be involved and following an open call, members of the GDG was selected according to their technical/clinical expertise, geographic (including representation from LMICs) and gender representation, with any conflicts of interest being addressed before selection. The draft guidelines were reviewed by an external advisory group. The new guidelines were developed to align with the WHO roadmap for access to medicines, vaccines and other health products.
Development Process and Key Questions
It was a long process and a lot of hard work, and of course, we began the work just as COVID hit. There were four key phases in the development of the guidelines: a) guideline planning, b) the systematic review, c) guideline formulation, and d) the finalisation, review and approval process.
We began by thinking about two key questions that the guidelines were going to address:
- What national policies and implementation strategies are effective in improving appropriate access to controlled medicines?
- What national policies and implementation strategies are effective to ensure safe use of controlled medicines (including limiting non-medical use)?
We then thought about the populations that would be included and it was agreed that it should include all patient groups affected by conditions in which the use of controlled medicines is deemed to be medically appropriate according to evidence based guidelines. Thus, it included a wide range of conditions including: pain, anesthesia, palliative care, the management of mental health disorders, the management of substance use disorders, the management of neurological disorders including epilepsy, humanitarian or health emergencies, along with other clinical research settings. In addition, it was felt that we needed to cover the whole life course, i.e. neonates, children, adolescents, young people, adults, the elderly. We therefore had a large scope with a wide range of conditions and across all ages.
Purpose and Aim of the Guidelines
The purpose of these guidelines is to support WHO Member States and partners in developing and implementing national policies on controlled medicines—ensuring accessibility, availability, and affordability for medical and scientific use, while minimising the risk of non-medical use. They aim to focus on policies for conditions where controlled medicines are medically appropriate, based on evidence-based clinical guidelines. These are policy rather than clinical guidelines, aiming to ensure availability rather than instruct on how to prescribe.
The recommendations cover all types of controlled medicines with authorised medical or scientific purposes, including opioids, benzodiazepines, barbiturates, dissociative anaesthetics, cannabinoids, hallucinogens, and amphetamine-type stimulants. Throughout, the document stresses that medication use must be based on clinical need—is it the right medication for the individual’s type of epilepsy or pain? Has a good assessment been carried out to confirm these medicines suit that particular need?
Guiding Principles
The guidelines are underpinned by some key guiding principles which the GDG feel are key to underpinning the guideline and apply to all national policies pertaining to controlled medicines.
- All people have the right to enjoyment of the highest attainable standard of health, a state of complete physical, mental and social well-being and not merely the absence of disease or infirmity.
- Controlled medicines are crucial for managing many health conditions and treating illness. Access to essential controlled medicines is a component of the rights to both health and life.
- National policies pertaining to controlled medicines should be balanced to enable safe and appropriate use, ensuring access for medical and scientific need (that is uninterrupted, sustainable and continuous) while avoiding harmful consequences for individuals and societies.
- All national policies should be tailored to the needs and requirements of the social context and resources of the population, while recognizing individuals’ right to the highest attainable standard of health.
- Member States and health-care providers should ensure that patients, their families and their caregivers know their rights to self-determination, non-discrimination, accessible and appropriate health services and confidentiality.
- Governments should invite patients, advocacy groups, health professionals, academia, professional societies, civil society and other affected groups to participate in formulation of health policy.
Two core principles guided the development of these guidelines: ensuring access and avoiding harm. The full Recommendations and Good Practice Statements in the rapid communication.
Summary of the Recommendations and Best Practice Statements
We identified seven different domains to be covered:
- Development of a national policy on controlled medicines,
- Pricing and financing of controlled medicines and related healthcare services,
- Medicines selection,
- Procurement and supply chain management,
- Medicines, regulation and control,
- Prescribing, dispensing and administration, and
- Education, knowledge and attitudes.
These domains were then divided into two groups. For domains 1-3 a narrative review was undertaken and we decided to use the existing up to date recommendations and good practice statements from the WHO. For the remaining domains (4-7) a systematic review was undertaken, and new recommendations and good practice statements were developed based around the evidence.
The first area addressed through new recommendations and good practice statements was Domain 4: Procurement and Supply Chain Management. The GDG agreed that quantification of controlled medicines should be based on need, using the best available data. Regular updates to estimates are essential, and any changes in need must be communicated promptly. Countries must have a responsive system for reporting, ordering, and estimating controlled medicines to ensure consistent access. There should be a sufficient, safe, high-quality, and cost-effective supply of controlled medicines, available at sustainable prices. Governments should use simple, appropriate technologies and tools to manage supply chains, ensuring full geographical coverage, minimising waste and shortages, and addressing inequities in access. Adequate infrastructure, technology, funding, and workforce capacity are essential. Governments should also weigh the health, financial, and social benefits—as well as potential risks—of local production of quality-assured controlled medicines.
Next Domain 5 focused on Medicine Regulation and Control. Controlled medicines must be available in acceptable, affordable, and accessible formulations for those with clinical need. Packaging should prevent accidental use by children or vulnerable adults without creating barriers to legitimate access. The GDG recommended a review of laws and regulations that may hinder access, with revisions where necessary to ensure patients with clinical need are not denied treatment. Healthcare professionals must be authorised to possess and transport controlled medicines—for example, when visiting patients at home—without risk of legal repercussions. Similarly, patients using these medicines legitimately should have legal protection. Drug scheduling should be based on robust scientific evidence and include input from health authorities, professional associations, patients, families, and other stakeholders. Scheduling must aim to optimise health outcomes without restricting access. On import and export, governments should streamline regulatory procedures, adopt electronic authorisations, and ensure that controlled medicines can be rapidly supplied in humanitarian crises, especially for emergency response organisations.
Domain 6 focuses on Prescribing, Dispensing, and Administration. Clinical guidelines for controlled medicines should be grounded in the best available evidence. A key recommendation is that opioid agonist treatment (OAT) must be available for people with opioid dependence—including those in the community, prisons, and other closed settings. In areas with high non-medical use, diversion control policies must not restrict legitimate access. Regulations should support safe, effective, equitable, and convenient access to controlled medicines, while maintaining proportionate safeguards. Health professionals should be able to prescribe, dispense, and administer these medicines without unnecessary barriers, within their scope of practice. Governments should implement robust national systems—ideally electronic where feasible and affordable—to monitor controlled medicines without exposing patients, caregivers, or professionals to unwarranted scrutiny. There should also be active safety monitoring. This domain also addresses pharmaceutical industry relations. Governments should ban misleading or unethical marketing of controlled medicines and enforce transparent, conflict of interest policies in training, guideline development, and product promotion. These safeguards should also apply to legislators, regulators, and officials involved in policymaking.
The final domain – Domain 7 – is Education, Knowledge and Attitudes. The first recommendation calls for comprehensive training for healthcare professionals on both adequate access to and the safe use of controlled medicines, aligned with clinical guidelines. This training should be included in core curricula and continuing professional education. In parallel, there must be delivery of balanced, accurate information about controlled medicines to patients, families, caregivers, and the public. Public messaging is often skewed toward either access or safety, but it’s vital that communication reflects both priorities equally.
Challenges and Research Gaps
It was a challenge developing these guidelines as in many areas of policy there is no published evidence or the evidence that does exist is focused on high income countries. In the full document to be released later in the year we have identified several reasons that this research gap might exist:
- It is difficult to design studies to test national policies, especially when these policies are shaped by international law
- There are challenges to assessing and quantifying an unmet clinical need for those conditions needing controlled medicines
- Non-medical use is often illegal, so it is hard to undertake research within that setting;
- It is challenging to have research which looks at both access and safety in parallel, for example research that looked at ‘access’ did not look at the safety-related outcomes if access was increased and likewise, research that looked at increasing safety measures did not look at the implications of this on access-related outcomes.
The majority of the studies had been undertaken in high income settings and studies in LMICs were underrepresented. Throughout the document there are also implementation considerations with regards to how each country can implement the recommendations and good practice statements. Cost-related issues are also discussed along with how we ensure access and safety for all, in particular within humanitarian crisis settings and for vulnerable populations.
A key consideration throughout the development of the guidelines was balancing the need for access and safe use as access to medicines is so key to the attainment of universal health coverage, which is central to the health-related sustainable development goal.
Conclusion and Call to Action
There needs to be action around the world if we are going to increase access to controlled medicines for those in clinical need, whilst maintaining safety. It’s important that these guidelines do not just sit on the shelf and gather dust. They need to be used. We need to be aware of the implementation information. We need to think about the research gaps. We need to think about the evidence in all that we do. At the launch of the guidelines, Dr Deusdedit Mubangizi, from the WHO Department of Health Policies and Standards, said:
“Together, we must all work to ensure the effective implementation of the recommendations in these guidelines so that safe, affordable medicines are available to all”.
We need to make sure that people around the world, wherever they live, whatever their condition, whether it’s in a low-income country or a high-income country, if they need controlled medicines for clinical need they should be able to access them. We hope that these guidelines will be useful, that they will be used, and that they will help us in ensuring access and maintaining safety.
Thank you.
Prof Julia Downing
Co-Chair Guidelines Development Group
Professor Downing’s note:
We would like to thank everybody involved in their development: all those in the Guidelines Development Group, the external and internal reviewers, the team at the World Health Organization, those doing the systematic review, along with everyone who made it possible to launch these guidelines at the World Health Assembly.
Please note that any views, commentary or opinions expressed about the guideline are not WHO’s official position but my viewpoint as an independent expert who served as the chair of the GDG that advised WHO on the recommendations.