Australian perspective

One of the aims of the Australian government is to have equal access to medications for all patients. However, in the 1990's, the Australian Drug Evaluation Committee found that there was a lack of access to medications for children so in 1995 the Working Party on the Registration of Drugs for Use in Children was established. Although several recommendations were made, the lack of incentives did not encourage their uptake.³ In 1997, the Orphan Drug Program was established. The TGA defines an "orphan drug"

as a medicine, vaccine or in vivo diagnostic agent that is intended to treat, prevent or diagnose a rare disease that is not commercially viable to supply to treat, prevent or diagnose another disease or condition.⁹³ Although the Orphan Drug Program was not specific to children, it aimed to encourage drug companies to provide essential products for rare conditions to Australia, while ensuring the same level of safety, efficacy and quality as other products and involved evaluation fees being waivered for drugs with small patient populations (< 2000). As the concerns around paediatric medicines continued, the Australian Health Minister's Advisory Council (AHMAC) set up a working party to consider issues relating to registration of paediatric

pharmaceuticals and appropriate access.³ This led to the establishment of the Paediatric Medicines Advisory Group (PMAG) by the Department of Health and Ageing.⁹⁴ The PMAG has identified a priority list of medicines requiring access for paediatric use in Australia, which is reviewed and updated at each meeting.³

A list of priority drugs and their clinical need for consideration by the PMAG was prepared in one of the initial meetings in October 2007 (Table 1.1).⁹⁵ As a result of the work by the PMAG, by 2008, clarithromycin powder and levetiracetam were listed on the Pharmaceutical Benefits Scheme (PBS).⁹⁶ In an outcome statement in June 2012, the PMAG reported that as a result of its work, a number of new medicines had been listed on the PBS including arthemether with lumefantrine dispersible tablet, clarithromycin powder for oral liquid, cefuroxime oral suspension, fluconazole powder for oral suspension, lansoprazole tablet (orally disintegrating), levetiracetam oral solution, ondansetron syrup, tocilizumab concentrate for injection and voriconazole powder for oral suspension. Access to diazoxide oral suspension via the Special Access Scheme had improved as a result of the PMAG's work and there had also been several amended listings on the PBS, including risperidone oral solution, methylphenidate hydrochloride, ciprofloxacin ear drops, dornase alfa solution for inhalation, albendazole chewable tablets, nevirapine oral suspension, terbinafine, deferasirox dispersible tablet, ribavirin with pegylated interferon and atenolol 50mg/10mL oral liquid.⁹⁷ The PMAG continues to liaise with expert paediatric groups and pharmaceutical companies to discuss medications on the PMAG's priority list. The most recent list, as at October 2012, is shown in Table 1.2.⁹⁸

Table 1.1 - Medicines under PMAG consideration as at October 2007.⁹⁵

Medicine Clinical Need as agreed by the PMAG aciclovir suspension to treat herpes simplex infections/ prevent recurrent

attacks of herpes simples infections

atenolol solution beta-blocker used to treat hypertension and angina calciferol/

cholecalciferol

vitamin D deficiency

calcitriol drops renal bone disease ciprofloxacin

suspension

cystic fibrosis, atypical mycobacterial infections and urinary tract infections

clarithromycin powder pertussis and atypical mycobacterial infections, particularly immunosuppressed patients

clindamycin suspension

Community acquired MRSA and osteomyelitis

diclofenac dispersible tablets

NSAID used to treat pain and inflammation

flecainide solution used to treat cardiac arrhythmias fusidic acid

suspension

anti-infective used to treat MRSA

gabapentin suspension

used for neuropathic pain and as anticonvulsant

melatonin tablets sleep disorders midazolam oral

suspension

sedative

nitrofurantoin suspension

antibiotic for prophylaxis and treatment of complicated urinary tract infections

spironolactone suspension

congenital heart disease in neonates and chronic lung disease

trimethoprim suspension

antibiotic for prophylaxis and treatment of complicated urinary tract infections

Another priority of the PMAG was the development of a national paediatric prescribing manual. This was achieved with support from the AHMAC, as part of the Paediatric Pharmaceuticals Prescribing Resource Project and led to the publication of the Australian Medicines Handbook (AMH) Children's Dosing Companion in 2013.⁹⁹ The resource provides detailed dosing information for around 230 drugs and will be updated with more drugs every six months. Dosages are provided by indications and/or age groupings from toddlers to teens. Other specific information relating to each drug's paediatric use is included, as well as off-label use and all content is evidence-based and peer reviewed. Further, the Australian Medicines Handbook Children's Dosing Companion⁹⁹ makes reference to the proposed framework published in the Australian Medical Journal in 2006 as a guide for clinicians and others for the off-label use of medicines.⁷⁷ According to the article, off-label prescribing may be considered appropriate if there is high-quality evidence supporting its use, within formal research or in exceptional use in an individual patient (e.g. if there is a serious underlying disease or condition).⁷⁷

The recent initiatives in Australia with the availability of an evidence-based and peer reviewed paediatric prescribing information resource, the guidelines published in the Medical Journal of Australia and the PMAG national decision-making framework have led to some improvements in the use and access to children's medicines.⁷⁷ However, despite strong professional advocacy on many fronts, there is still a lack of any legislative and regulatory reforms addressing paediatric medicines.^{78, 85} Unlike the United States and Europe, there is currently no specific government commitment to give high priority to paediatric medicines issues.⁸⁵

Table 1.2 - Medicines under PMAG consideration as at October 2012.⁹⁸

Medicine Clinical Need as agreed by the PMAG abatacept polyarticular juvenile idiopathic arthritis

adalimumab uveitis related to juvenile idiopathic arthritis, enthesitis and Crohn disease

anakinra systemic juvenile idiopathic arthritis

bosentan pulmonary hypertension

calciferol/

cholecalciferol

vitamin D deficiency

clindamycin suspension

Community acquired MRSA and osteomyelitis

clobazam tablets resistant epilepsy diazepam mixture chronic spasticity glycopyrrolate oral

solution

drooling

infliximab ankylosing spondylitis

Kindergen® medicinal food for older children leflunomide juvenile idiopathic arthritis

leuprorelin precocious puberty

melatonin sleep disorders particularly those with neurological impairment/ cortical blindness

mycophenolate sodium

nephrotic syndrome

natalizumab refractory multiple sclerosis 6-mercaptopurine/

thioguanine suspension

acute leukaemia

tacrolimus suspension organ transplant triamcinolone

hexacetonide

steroid joint injections for juvenile idiopathic arthritis