National medicines policies – a review of the evolution and development processes

OBJECTIVES: Continuous provision of appropriate medicines of assured quality, in adequate quantities, and at reasonable prices is a concern for all national governments. A national medicines policy (NMP) developed in a collaborative fashion identifies strategies needed to meet these objectives and provides a comprehensive framework to develop all components of a national pharmaceutical sector. To meet the health needs of the population, there is a general need for medicine policies based on universal principles, but nevertheless adapted to the national situation. This review aims to provide a quantitative and qualitative (describing the historical development) study of the development process and evolution of NMPs. METHODS: The number of NMPs and their current status has been obtained from the results of the assessment of WHO Level I indicators. The policy formulation process is examined in more detail with case studies from four countries: Sri Lanka, Australia, former Yugoslav Republic of Macedonia and South Africa. RESULTS: The number of NMPs worldwide has increased in the last 25 years with the highest proportional increase in the last 5–10 years in high-income countries. Higher income countries seem to have more NMP implementation plans available and have updated their NMP more recently. The four case studies show that the development of a NMP is a complex process that is country specific. In addition, it demonstrates that an appropriate political window is needed for the policy to be passed (for South Africa and the FYR Macedonia, a major political event acted as a trigger for initiating the policy development). Policy-making does not stop with the official adoption of a policy but should create mechanisms for implementation and monitoring. The NMPs of the FYR Macedonia and Australia provide indicators for monitoring. CONCLUSIONS: To date, not all countries have a NMP since political pressure by national experts or non-governmental organizations is generally needed to establish a NMP. Case studies in four countries showed that the policy process is just as important as the policy document since the process must create a mechanism by which all stakeholders are brought together and a sense of collective ownership of the final policy may be achieved

Chronicity of sleep problems in children with chronic illness: a longitudinal population-based study

<p>Abstract</p> <p>Background</p> <p>The aim of this study was to examine the chronicity of sleep problems in children with chronic illness, and potential predictors of sleep problems.</p> <p>Methods</p> <p>Using data from a longitudinal total population study in Norway, The Bergen Child Study, data on sleep problems, chronic illness and potential confounders were assessed at ages 79 and 1113.</p> <p>Results</p> <p>295 of 4025 (7.3%) children had a chronic illness, and the prevalence of chronic sleep problems was significantly higher in this group compared to children without chronic illness (6.8% versus 3.6%). Sleep problems at the first wave increased the risk of sleep problems at the second wave, also when adjusting for potential confounders (odds-ratio = 5.41). Hyperactivity and emotional problems were also independent risk factors for later sleep problems.</p> <p>Conclusion</p> <p>These findings call for increased awareness and development of treatment strategies of sleep problems in children with chronic illness.</p

Evaluation of sleep, puberty and mental health in children with long-term melatonin treatment for chronic idiopathic childhood sleep onset insomnia

OBJECTIVES: To establish whether long-term use of melatonin influences pubertal development, sleep quality and mental health development in children as compared with the normal Dutch population of the same age. METHODS: This follow-up research study was conducted in children included in a previous melatonin dose-finding trial. Outcomes were measured using questionnaires (Strength and Difficulties Questionnaire (SDQ), Children's Sleep Habits Questionnaire (CSHQ) and Tanner Stages) adopted for Dutch children. Mean duration of therapy, persistence of effect, adverse events and (other) reasons leading to cessation of therapy were additional objectives of this study. RESULTS: Mean years of usage (n = 51) was 3.1 years (min 1.0 year, max 4.6 years), mean dose 2.69 mg (min 0.3 mg, max 10 mg). Mean SDQ score, mean CSHQ score and Tanner Stages standard deviation scores did not differ in a statistically significant way from published scores of the general Dutch population of the same age and sex. CONCLUSIONS: This follow-up study demonstrates that melatonin treatment in children can be sustained over a long period of time without substantial deviation of the development of children with respect to sleep quality, puberty development and mental health scores, as compared with the general Dutch population

Dose finding of melatonin for chronic idiopathic childhood sleep onset insomnia: an RCT

Contains fulltext : 86695.pdf (publisher's version ) (Open Access)Rationale Pharmacokinetics of melatonin in children might differ from that in adults. Objectives This study aims to establish a dose–response relationship for melatonin in advancing dim light melatonin onset (DLMO), sleep onset (SO), and reducing sleep onset latency (SOL) in children between 6 and 12 years with chronic sleep onset insomnia (CSOI). Methods The method used for this study is the randomized, placebo-controlled double-blind trial. Children with CSOI (n=72) received either melatonin 0.05, 0.1, and 0.15 mg/kg or placebo during 1 week. Sleep was assessed with log and actigraphy during this week and the week before. Outcomes were the shifts in DLMO, SO, and SOL. Results Treatment with melatonin significantly advanced SO and DLMO by approximately 1 h and decreased SOL by 35 min. Within the three melatonin groups, effect size was not different, but the circadian time of administration (TOA) correlated significantly with treatment effect on DLMO (rs=-0.33, p=0.022) and SO (rs=-0.38, p=0.004), whereas clock TOA was correlated with SO shift (r=-0.35, p=0.006) and not with DLMO shift. Conclusions No dose–response relationship of melatonin with SO, SOL, and DLMO is found within a dosage range of 0.05–0.15 mg/kg. The effect of exogenous melatonin on SO, SOL, and DLMO increases with an earlier circadian TOA. The soporific effects of melatonin enhance the SO shift. This study demonstrates that melatonin for treatment of CSOI in children is effective in a dosage of 0.05 mg/kg given at least 1 to 2 h before DLMO and before desired bedtime.13 p

Competitive Tendering In The Netherlands: Central Planning Or Functional Specifications?

Institute of Transport and Logistics Studies. Faculty of Economics and Business. The University of Sydne

Improved clinical investigation and evaluation of high-risk medical devices: the rationale and objectives of CORE-MD (Coordinating Research and Evidence for Medical Devices)

: In the European Union (EU) the delivery of health services is a national responsibility but there are concerted actions between member states to protect public health. Approval of pharmaceutical products is the responsibility of the European Medicines Agency, whereas authorizing the placing on the market of medical devices is decentralized to independent 'conformity assessment' organizations called notified bodies. The first legal basis for an EU system of evaluating medical devices and approving their market access was the medical device directives, from the 1990s. Uncertainties about clinical evidence requirements, among other reasons, led to the EU Medical Device Regulation (2017/745) that has applied since May 2021. It provides general principles for clinical investigations but few methodological details-which challenges responsible authorities to set appropriate balances between regulation and innovation, pre- and post-market studies, and clinical trials and real-world evidence. Scientific experts should advise on methods and standards for assessing and approving new high-risk devices, and safety, efficacy, and transparency of evidence should be paramount. The European Commission recently awarded a Horizon 2020 grant to a consortium led by the European Society of Cardiology and the European Federation of National Associations of Orthopaedics and Traumatology, that will review methodologies of clinical investigations, advise on study designs, and develop recommendations for aggregating clinical data from registries and other real-world sources. The CORE-MD project (Coordinating Research and Evidence for Medical Devices) will run until March 2024; here we describe how it may contribute to the development of regulatory science in Europe

Off-label use of medicinal products

Medicijnen kunnen worden voorgeschreven voor andere ziekten of groepen patiënten dan waar ze voor zijn goedgekeurd. Dat noemen we 'off-labelgebruik' en is onder voorwaarden wettelijk toegestaan. Dit wordt meestal gedaan wanneer er geen andere geschikte behandelmogelijkheden zijn. Zo komt off-labelgebruik bijvoorbeeld vaak voor bij kinderen of bij ernstige ziekten waarvoor nog geen goedgekeurde therapie bestaat. De mate waarin dit voorkomt verschilt per aandoening maar kan omvangrijk zijn. Off-labelgebruik van medicijnen voorziet dus in een medische behoefte, maar kan nog op meerdere punten worden verbeterd. Dit blijkt uit onderzoek van het RIVM. In de praktijk blijkt bijvoorbeeld dat artsen hun patiënten niet altijd informeren en om toestemming vragen wanneer zij geneesmiddelen off-label voorschrijven, ook al zijn ze daar wettelijk toe verplicht. Verder is het belangrijk de kennis over off-labeltoepassingen toegankelijker te maken voor artsen en apothekers; deze kennis is nu versnipperd en niet altijd volledig beschikbaar. Om kennis te vergroten is het ook van belang beter bij te houden wat de werking en eventuele bijwerkingen van off-labelgebruik zijn. Het heeft uiteraard de voorkeur om off-labelgebruik van een medicijn on-label te maken. Hiervoor moet de geneesmiddelfabrikant het initiatief nemen om het bewijs voor de effectiviteit en veiligheid van het gebruik bij de overheid aan te leveren. Als een fabrikant dit initiatief niet neemt, wordt het off-label-gebruik niet genoemd in de bijsluiter. Fabrikanten zouden meer kunnen worden aangespoord om het bewijs te leveren. Ook zou onderzocht kunnen worden of dit initiatief door anderen dan de fabrikant genomen kan worden.Medicinal products may be prescribed for other diseases, or groups of patients, than the ones that they are approved for. This is what we call 'off-label use', a practice which is legally permitted under certain conditions and usually takes place when there are no other suitable treatment options available. Off-label use, for example, frequently occurs in the treatment of children or patients who have serious illnesses for which there is no approved therapy. The extent to which this course of action is taken differs per disease area but can be substantial. The off-label use of medicinal products thus fulfils a medical need, but can still be improved on in several ways. This has been illustrated in research conducted by RIVM. For example, in practice, doctors do not always inform their patients or ask for permission when prescribing medicines off-label, even though they are required to do so by law. Furthermore, it is important to make information about off-label applications more accessible to doctors and pharmacists; currently this knowledge is fragmented and availability is patchy. It is also important to increase levels of awareness about the efficacy of these medicines by keeping track of their performance and recording any possible side effects of off-label use. Of course, it is preferable to make off-label use of a medicinal product 'on label'. To do this, the drug manufacturer must take the initiative to provide proof of the product's efficacy and safety of use to the government. If a manufacturer does not take this initiative, the off-label use is not mentioned in the patient information leaflet. Manufacturers could be encouraged to provide proof. Whether this initiative could be taken by anyone other than the manufacturer might also be investigated.Onderzoeksprogramma Geneesmiddelenkete

Kosteneffectiviteit van implantaten wordt zwaar overschat: Meer onderzoek nodig naar maatschappelijke kosten en bijwerkingen

Nieuwe technologie brengt in de regel hogere kosten met zich mee. In kosteneffectiviteitsstudies worden daarom de kosten en de verwachte opbrengsten in de vorm van gezondheidswinst of kwaliteit van leven tegen elkaar afgezet. Maar welke studies bestaan er voor implantaten en hun (langetermijn)bijwerkingen? En welke rol speelt kosteneffectiviteit bij de afweging om een implantaat wel of niet toe te passen? Het RIVM besloot een verkenning te starten