Special Access Scheme Research Articles

Managing medicine shortages.

Medicine shortages are increasingly common and disruptive to the optimal delivery of health care. They are caused by a variety of factors, including manufacturing and supply-chain issues, regulatory and trade issues, and fluctuations in demand. Prescribers and pharmacists in Australia can manage a shortage by switching to another brand, strength or dosage form of the same medicine, switching to a different registered medicine, or accessing an unregistered medicine that has been made available via section 19A of the Therapeutic Goods Act 1989 or through the Special Access Scheme. There are a range of resources and tools that can assist clinicians with identifying and managing medicine shortages in Australia. Shortages are managed most effectively when prescribers, pharmacists and nurses work together, in collaboration with patients, to develop, implement and monitor strategies to manage the shortage.

A provisional evaluation of Australia's medical cannabis program

In 2016, the Australian Government legislated to allow cannabis to be prescribed to patients as an unapproved medicine under the special access provisions of the Therapeutic Goods Act. This paper compares the Australian regulatory approach with other national approaches, outlines the main provisions of the Special Access Scheme for medical cannabis, describes how the program has evolved since 2017, includes an analysis of adverse events reported to the Therapeutic Goods Administration, and discusses the barriers that remain for patients who wish to access medical cannabis. It assesses how well the Australian program has addressed the challenges of providing patients with easier access to medical cannabis while ensuring that high-quality products are used safely and effectively under medical guidance.

Medicinal cannabis for psychiatry-related conditions: an overview of current Australian prescribing.

Objective: Evidence is accumulating that components of the Cannabis sativa plant may have therapeutic potential in treating psychiatric disorders. Medicinal cannabis (MC) products are legally available for prescription in Australia, primarily through the Therapeutic Goods Administration (TGA) Special Access Scheme B (SAS-B). Here we investigated recent prescribing practices for psychiatric indications under SAS-B by Australian doctors. Methods: The dataset, obtained from the TGA, included information on MC applications made by doctors through the SAS-B process between 1st November 2016 and 30th September 2022 inclusive. Details included the primary conditions treated, patient demographics, prescriber location, product type (e.g., oil, flower or capsule) and the general cannabinoid content of products. The conditions treated were categorized according to the Diagnostic and Statistical Manual of Mental Disorders, 5th edition, text revision (DSM-5-TR). Trends in prescribing for conditions over time were analyzed via polynomial regression, and relationships between categorical variables determined via correspondence analyses. Results: Approximately 300,000 SAS-B approvals to prescribe MC had been issued in the time period under investigation. This included approvals for 38 different DSM-5-TR defined psychiatric conditions (33.9% of total approvals). The majority of approvals were for anxiety disorders (66.7% of psychiatry-related prescribing), sleep-wake disorders (18.2%), trauma- and stressor-related disorders (5.8%), and neurodevelopmental disorders (4.4%). Oil products were most prescribed (53.0%), followed by flower (31.2%) and other inhaled products (12.4%). CBD-dominant products comprised around 20% of total prescribing and were particularly prevalent in the treatment of autism spectrum disorder. The largest proportion of approvals was for patients aged 25-39years (46.2% of approvals). Recent dramatic increases in prescribing for attention deficit hyperactivity disorder were identified. Conclusion: A significant proportion of MC prescribing in Australia is for psychiatry-related indications. This prescribing often appears somewhat "experimental", given it involves conditions (e.g., ADHD, depression) for which definitive clinical evidence of MC efficacy is lacking. The high prevalence of THC-containing products being prescribed is of possible concern given the psychiatric problems associated with this drug. Evidence-based clinical guidance around the use of MC products in psychiatry is lacking and would clearly be of benefit to prescribers.

Standardised treatment and monitoring protocol to assess safety and tolerability of bacteriophage therapy for adult and paediatric patients (STAMP study): protocol for an open-label, single-arm trial

IntroductionThere has been renewed interest in the therapeutic use of bacteriophages (phages); however, standardised therapeutic protocols are lacking, and there is a paucity of rigorous clinical trial data assessing efficacy.Methods...

The Rise and Rise of Medicinal Cannabis, What Now? Medicinal Cannabis Prescribing in Australia 2017-2022.

Medicinal cannabis was legalised in Australia in November 2016. By August 2022, there were 5284 specialist physician and general practitioner (GP) prescribers who submitted Special Access Scheme (SAS) applications to the Therapeutic Goods Administration (TGA) for the provision of medicinal cannabis prescriptions their patients. In this article we examine the impact of the delivery of publicly available clinical guidance documents, provision of education to prescribers, establishment of the TGA online portal, and launching of cannabis clinics on the number of applications approved by the TGA over time. We considered these findings in the context of the need to align the interventions facilitating the prescribing of medicinal cannabis with the establishment of processes to enable the systematic monitoring of patient outcomes. The cumulative number of medicinal cannabis Special Access Scheme-B (SAS-B) prescription approvals from January 2017 to June 2022 was 258,926. SAS-B approvals increased at an average rate of 208.55% p < 0.000, (95% CI 187.25–229.85) per month. Conclusion: There has been a rapid growth in prescribing since the legalisation of medicinal cannabis in Australia and this expansion has not been accompanied by parallel processes for the monitoring of medicinal cannabis. The capture of more highly granulated data, as found in the electronic medical record (EMR), patient smartphone applications, and social media provide an opportunity to monitor medicinal cannabis effectiveness and safety across multiple prescribing indications.

Medicinal Cannabis Prescribing in Australia: An Analysis of Trends Over the First Five Years.

A regulatory framework allowing legal access to medicinal cannabis (MC) products has operated in Australia since November 2016. MC prescribing by healthcare practitioners (HCPs) is primarily conducted through the Special Access Scheme - Category B (SAS-B) pathway, through which prescribers apply to the Therapeutic Goods Administration (TGA–the federal regulator) for approval to prescribe a category of product to an individual patient suffering from a specific indication. The dataset collected by the TGA provides a unique opportunity to examine MC prescribing trends over time in the Australian population. Here we analysed this TGA SAS-B dataset since inception with respect to age, gender, product type (e.g., oil, flower, etc.), CBD content, indication treated, and prescriber location. Results are presented descriptively as well as being analysed using non-linear regression models. Relationship between variables were explored via correspondence analyses. Indications were classified with reference to the International Statistical Classification of Diseases and Related Health Problems (10th Revision). As of 31 August 2021, a total of 159,665 SAS-B approvals had been issued for MC products, 82.4% of were since January 2020. Leading indications for approvals were for pain, anxiety, and sleep disorders. Oil products were the most popular product type, while CBD-dominant products (≥98% CBD) accounted for 25.1% of total approvals. Approvals for flower products increased markedly during 2020–2021, as did approvals involving younger age groups (18–31 years old), male patients, and non-CBD dominant products. A disproportionate number of SAS-B MC applications (around 50%) came from HCPs in the state of Queensland. Associations between patient gender and age and/or indication with product type were found. For example, approvals for oil products were commonly associated with approvals for pain. While, overall prescribing increased dramatically over the last 2 years of analysis, stabilization of approval numbers is evident for some indications, such as pain. Current prescribing practices do not always reflect provided TGA guidance documents for MC prescribing. While acknowledging some limitations around the SAS-B dataset, it provides a unique and valuable resource with which to better understand current prescribing practices and utilisation of MC products within Australia.

Cannabis and its therapeutic value in the ageing population: Attitudes of health-care providers.

To explore the attitudes of health-care providers and the perceived barriers and facilitators regarding the use of cannabis-containing medicines (CCMs) in older patients. Semi-structured interviews with eleven health-care providers in rural New South Wales (NSW). A thematic analysis was performed. Themes and recommendations include attitudes towards CCMs for therapeutic purposes, their perceived uses, side effects, barriers to use and prescription (stigma, regulation, access and driving), difficulties encountered with the Special Access Scheme (SAS) and the need for ongoing research and education. An ecological framework demonstrated the complex interactions between patients and health-care providers and their social, community and political environment. Providers' attitudes towards CCM use in older patients are generally positive; however, there is a need for further high-quality evidence on efficacy and safety, alongside improved CCM education and training, to reduce barriers to their implementation. Barriers to prescription and access continue to evolve on personal, organisational and systemic levels.

Medicinal psychedelics for mental health and addiction: Advancing research of an emerging paradigm.

The medical use of psychedelic substances (e.g. psilocybin, ayahuasca, lysergic acid diethylamide and 3,4-methylenedioxymethamphetamine) is attracting renewed interest, driven by a pressing need for research and development of novel therapies for psychiatric disorders, as well as promising results of contemporary studies. In this Viewpoint, we reflect upon the 'Clinical Memorandum on Psychedelics' recently released by the Royal Australian and New Zealand College of Psychiatrists and note subsequent developments including the application for down-scheduling of psilocybin and 3,4-methylenedioxymethamphetamine presently being considered by the Therapeutic Goods Administration and approvals for access via the Special Access Scheme. We suggest that this field is worthy of rigorous research to assess potential benefits, address safety parameters and clarify therapeutic mechanisms. To this end, we outline recent research findings, provide an overview of current knowledge relating to mechanisms of action and discuss salient aspects of the psychedelic-assisted psychotherapy treatment model. The sum of this research points towards medicinal psychedelics as a potential new class of psychiatric treatments when used within a medically supervised framework with integrated psychotherapeutic support. However, before widespread translation into clinical use can occur, appropriately designed and sufficiently powered trials are required to detect both potential positive and negative outcomes. Unique safety and regulatory challenges also need to be addressed. As for any new medical therapy, psychedelic research needs to be conducted in a rigorous manner, through the dispassionate lens of scientific enquiry. Carte blanche availability to practitioners, without specific protocols and appropriate training, would be potentially harmful to individuals and detrimental to the field.

Treatment of in-transit melanoma metastases using intralesional PV-10

Melanoma in-transit metastases (ITMs) can sometimes be difficult to manage by surgical excision due to their number, size or location. Treatment by intralesional injection of PV-10, a 10% solution of rose bengal, has been reported to be a simple, safe and effective alternative, but more outcome data are required to confirm its value in the management of ITMs. Two hundred and twenty-six melanoma ITMs in 48 patients were treated with intralesional PV-10 supplied under a special-access scheme. By 8 weeks a complete response in all injected ITMs was achieved in 22 patients (46%) and a partial response in 19 patients (40%). Of 19 patients who had uninjected metastases, 3 (16%) had a response in these. The most common adverse event was transient localised pain in injected tumours. New ITMs developed in 25 patients within 8 weeks, and later in another 8 patients. Repeat injection cycles were given to 21 patients: 13 of these received repeat injection into partially responding or nonresponding tumours, 5 had new ITMs, as well as partially-responding lesions injected, and 3 received injection into new ITMs only. Twenty-two patients received subsequent systemic therapy. At 1 year 37 of the 48 patients were alive, 28 with melanoma, and at 2 years 27 were alive, and 19 with melanoma. Injection of PV-10 was simple and safe and resulted in tumour involution in most patients and sometimes in noninjected tumours. However, many patients developed new lesions; these were treated by further PV-10 injections or with alternative therapies.

Estimating the utilisation of unregistered antimicrobials in Australia.

To identify and estimate the usage of unregistered antimicrobial drugs in Australian clinical practice. A descriptive pharmaco-epidemiological study, utilising three data sources: analysis of Special Access Scheme (SAS) applications for unregistered antimicrobials included in clinical guidelines over a five year period, analysis of antimicrobials dispensed from South Australian public hospital pharmacy departments over a two year period and analysis of National Antimicrobial Utilisation Surveillance Program (NAUSP) data for reported inpatient usage of unregistered antimicrobials in Australian hospitals over the last 5 years. 59 unregistered antimicrobials were identified using the mixed methods. 18,362 Special Access Scheme applications were submitted between May 2012 and April 2017 to access the 20 unregistered antimicrobials identified in the Therapeutic Guidelines® (eTG complete); 51.4% were determined by the prescriber to be for life-threatening indications. Annual applications more than doubled over the five years. 34 unregistered antimicrobials were dispensed from South Australian public hospitals between July 2015 and June 2017. On average, 1.1% of total antimicrobial usage (Defined Daily Doses) per month was accessed via the SAS, of which 87.7% were for outpatients or discharged patients. 34 unregistered antimicrobials for systemic use identified in the NAUSP database were used in Australian hospitals between 2013 and 2018. The use of unregistered antimicrobials in Australian clinical practice is not uncommon. With increasing antimicrobial resistance, there will be a continued reliance on older less-used antimicrobial agents and an increasing need for novel drugs, therefore regulatory pathways need to facilitate security of supply and assurance of medicine quality and safety.

PDCT-07. EARLY RESULTS OF THE EMULATE THERAPEUTICS HÆLO™ SYSTEM IN PEDIATRIC BRAIN TUMORS

Abstract BACKGROUND The EMulate Therapeutics Hælo system is an investigational non-sterile, non-invasive, non-thermal, non-ionizing, portable, home-use medical device that uses a specific, localized ultra-low radio frequency energy (ulRFE®) cognate for the treatment of pediatric brain tumors. METHODS Sixteen patients with brain tumors consisting of diffuse midline glioma/diffuse intrinsic pontine glioma (DMG/DIPG, n=14), recurrent medulloblastoma (n=1), or anaplastic astrocytoma (n=1) – were treated with the Hælo under FDA’s single-patient compassionate use pathway, as protocol deviations in a glioblastoma trial, or under TGA’s Special Access Scheme. Baseline information and on-treatment safety and exposure data were collected. RESULTS Patients ranged in age from 4 to 28 years (median = 8 years) and were diagnosed 91 – 1399 days (median = 397 days) prior to treatment with the Hælo system. Patients were treated for 2 – 52 weeks (median = 15 weeks), with 4 patients still alive (all with a diagnosis of DMG/DIPG), and 3 still on treatment (ranging 18 – 52 weeks). Two out of the 16 patients reported mild-moderate adverse events - one patient reported nausea, fatigue, and excessive sleepiness, and one patient reported vomiting. No device-related serious adverse events were reported. Other adverse events reported were generally associated with progressive disease. Unsolicited, anecdotal reports from some parents/caregivers noted improvements in mobility, speech, and visual acuity while on treatment. CONCLUSIONS The Hælo system appears to be safe and feasible for the treatment of pediatric brain tumors. Given that therapy is delivered non-invasively and no device-related serious adverse events were reported, further prospective study of the investigational device is warranted.

Clinical indications treated with unregistered antimicrobials: regulatory challenges of antimicrobial resistance and access to effective treatment for patients

Objective Increasing antimicrobial resistance and a concurrent paucity of new antimicrobials marketed increases the risk that patients will develop infections resistant to currently available drugs. This study aimed to determine the range of clinical indications for which unregistered antimicrobials are prescribed at two tertiary hospitals in South Australia to identify any trends over a 2-year period. The effects of recent regulatory changes to the Special Access Scheme (SAS) were assessed. Methods Data were extracted from application forms submitted to the Therapeutic Goods Administration to access unregistered antimicrobials via the SAS pathway at two Australian tertiary hospitals for the period July 2015-June 2017. Average weighted antimicrobial prices were retrieved from the hospital iPharmacy (DXC Technology, Macquarie Park, NSW, Australia) dispensing system. To estimate the effect of a new access pathway (Category C), the SAS classification for each application was retrospectively assessed over time with each regulatory change. Results Between July 2015 and June 2017, 477 SAS applications for 29 different antimicrobials were submitted for 353 patients at the two hospitals. The most common indications were tuberculosis (43.6%) and refractory Helicobacter pylori (10%). Regulatory changes reduced the proportion of applications requiring preapproval for access. Conclusions Although the introduction of a new pathway has decreased the administrative burden when accessing unregistered antimicrobials, this study highlights the range of clinical conditions for which there are no registered drugs available in Australia. What is known about the topic? With increasing antimicrobial resistance and a paucity of novel antimicrobials entering the market, access to older, previously less-used antimicrobials is increasingly important in clinical practice. Accessing unregistered antimicrobials is common practice in Australian hospitals, but the range of clinical indications for which they are used is unclear. What does this paper add? Increasing antimicrobial resistance and a concurrent paucity of new antimicrobials being marketed globally is increasing the risk that patients may develop infections that cannot be treated with registered products. This study describes the range of clinical conditions for which registered antimicrobials are not available or appropriate, illustrating the challenges associated with sustainable access to effective treatments. What are the implications for practitioners? Access to effective antimicrobials in a timely manner is essential for optimal patient outcomes. Reliance on unregistered products is associated with increased risks regarding timely access to safe, quality-assured, effective medicines.

New detection of flinders island spotted fever (due to Rickettsia honei infection) in new south wales: A case of fulminant septic shock and multi-organ failure

We report a case of a new location and severe infection for Rickettsia honei. A 76-year-old man, five days after returning from a camping trip to Grafton and Coffs Harbour, presented to the emergency department with two-day history of widespread rash, fever, rigors, confusion and decreasing mobility with rapid deterioration. He eventually progressed into multi-organ failure requiring dialysis, vasopressor support and intubation. On day 2, rickettsial infection was suspected. Therefore, a blood ethylene diamine triacetic acid (EDTA) tube for rickettsial polymerase chain reaction (PCR) and serology (microimmunofluorescence assay) were sent to the Australian Rickettsial Reference Laboratory. He was started on oral doxycycline 100 mg BD via nasogastric tube but did not improve. On day 4, his treatment was escalated to intravenous azithromycin 500 mg daily and intravenous doxycycline 100 mg BD (Special Access Scheme Category A) given non-improvement and doubtful absorption of oral doxycycline considering high vasopressor support. He rapidly improved 48 hours later with reducing organ support, extubated and subsequently discharged from ICU on Day 8. Subsequently, his serology and PCR suggested Spotted Fever Group Rickettsia. Further amplification of the citrate synthase and outer membrane protein A genes confirmed Rickettsia honei infection, which had not been detected in New South Wales previously.

Outcomes of cinacalcet withdrawal in Australian dialysis patients.

Secondary hyperparathyroidism (SHPT) in chronic kidney disease is associated with cardiovascular and bone pathology. Measures to achieve parathyroid hormone (PTH) target values and control biochemical abnormalities associated with SHPT require complex therapies, and severe SHPT often requires parathyroidectomy or the calcimimetic cinacalcet. In Australia, cinacalcet was publicly funded for dialysis patients from 2009 to 2015 when funding was withdrawn following publication of the EVOLVE study, which resulted in most patients on cinacalcet ceasing therapy. We examined the clinical and biochemical outcomes associated with this change at Australian renal centres. To assess changes to biochemical and clinical outcomes in dialysis patients following cessation of cinacalcet. We conducted a retrospective study of dialysis patients who ceased cinacalcet after August 2015 in 11 Australian units. Clinical outcomes and changes in biochemical parameters were assessed over a 24- and 12-month period, respectively, from cessation of cinacalcet. A total of 228 patients was included (17.7% of all dialysis patients from the units). Patients were aged 63 ± 15 years with 182 patients on haemodialysis and 46 on peritoneal dialysis. Over 24 months following cessation of cinacalcet, we observed 26 parathyroidectomies, 3 episodes of calciphylaxis, 8 fractures and 50 deaths. Eight patients recommenced cinacalcet, meeting criteria under a special access scheme. Biochemical changes from baseline to 12 months after cessation included increased levels of serum PTH from 54 (interquartile range 27-90) pmol/L to 85 (interquartile range 41-139) pmol/L (P < 0.0001), serum calcium from 2.3 ± 0.2 mmol/L to 2.5 ± 0.1 mmol/L (P < 0.0001) and alkaline phosphatase from 123 (92-176) IU/L to 143 (102-197) IU/L (P < 0.0001). Significant increases in serum PTH, calcium and alkaline phosphatase occurred over a 12-month period following withdrawal of cinacalcet. Longer-term follow up will determine if these biochemical and therapeutic changes are associated with altered rates of parathyroidectomies and cardiovascular mortality and morbidity.

1642. Safety and Efficacy of Bacteriophage Therapy: Analysis of Clinical Case Series Data

BackgroundBacteriophage therapy (BT) is a re-emerging strategy to treat antibiotic-resistant infections. Here, we describe our initial experience with intravenous (IV) and inhaled BT to treat life-threatening Staphylococcus aureus and Pseudomonas aeruginosa infections not responding to antibiotic therapy. Emergency Investigational New Drug application approvals (United States) or Special Access Scheme Category A notifications (Australia) and informed consent from the patients were obtained.MethodsPatients were treated with AB-SA01 (3-phage product targeting S. aureus) and AB-PA01 (4-phage product targeting P. aeruginosa) produced in a Good Manufacturing Practice-certified facility. Pre- and posttreatment bacterial isolates were tested for phage susceptibility during BT. In all cases, concomitant antibiotics were continued. Safety was assessed clinically and using laboratory parameters with up to 90 days of follow-up. Samples to assess bacterial loads, bacteriophage kinetics in blood, and immune responses to phage were collected.ResultsAs of April 2018, 8 patients were treated with BT; 5 with AB-SA01 (bacteremia, n = 4; endocarditis, n = 1) and 3 with AB-PA01 (lung infection, n = 3). Median duration of BT was 14 days and treated patients received over 90 IV doses of AB-SA01 (3 × 109 PFU/dose) and over 490 IV and nebulized doses of AB-PA01 (4 × 109 PFU/dose). BT was well tolerated, with no treatment-related adverse events. Clinical treatment success was documented in 75% of patients. Isolates collected during therapy showed ongoing susceptibility to the BT products with changes in sensitivity to the individual phage components observed in some cases. Bacteriophage kinetics revealed bloodstream clearance within a few hours after IV infusion, with an inferred initial bacteria:bacteriophage ratio of ~200 for the bacteremia patients.ConclusionBT was well tolerated as an adjunct to antibiotics, with several examples presented of microbiological eradication and improvement of objective clinical criteria. BT appears to be a safe adjunct to antibiotic therapy in life-threatening S. aureus and P. aeruginosa infections and is a promising candidate for controlled clinical trials.Disclosures S. Morales, AmpliPhi Biosciences: Employee, Salary. S. Lehman, AmpliPhi Biosciences: Employee, Salary. S. Branston, AmpliPhi Biosciences: Employee, Salary. A. Petrovic Fabijan, AmpliPhi Biosciences: Collaborator, Research support. C. L. Langlais Furr, AmpliPhi Biosciences: Employee, Salary. F. Rosas, AmpliPhi Biosciences: Employee, Salary. I. Bilinsky, AmpliPhi Biosciences: Employee and Shareholder, Salary. P. Grint, AmpliPhi Biosciences: Board Member, Employee and Shareholder, Salary. J. Iredell, AmpliPhi Biosciences: Collaborator, Research support.

Vape Club: Exploring Non-Profit Regulatory Models for the Supply of Vaporised Nicotine Products.

Vaporised nicotine products (VNPs) that are not approved as therapeutic goods are banned in some countries, including Australia, Singapore, and Thailand. We reviewed two non-profit regulatory options, private clubs and the Australian Therapeutic Goods Administration Special Access Scheme (SAS) that have been applied to other controlled substances (such as cannabis) as a potential model for regulating VNPs as an alternative to prohibition. The legal status of private cannabis clubs varies between the United States, Canada, Belgium, Spain, and Uruguay. Legal frameworks exist for cannabis clubs in some countries, but most operate in a legal grey area. Kava social clubs existed in the Northern Territory, Australia, until the federal government banned importation of kava. Access to medical cannabis in Australia is allowed as an unapproved therapeutic good via the SAS. In Australia, the SAS Category C appears to be the most feasible option to widen access to VNPs, but it may have limited acceptability to vapers and smokers. The private club model would require new legislation but could be potentially more acceptable if clubs were permitted to operate outside a medical framework. Consumer and regulator support for these models is currently unknown. Without similar restrictions applied to smoked tobacco products, these models may have only a limited impact on smoking prevalence. Further research could explore whether these models could be options for regulating smoked tobacco products.

Access to unregistered drugs in Australia.

Drugs can usually only be prescribed for patients if they have been approved by the Therapeutic Goods Administration for inclusion in the Australian Register of Therapeutic Goods. Unregistered drugs can be obtained through the Special Access Scheme, the Authorised Prescriber Scheme or by personal importation. Almost any drug can be accessed through these schemes, if it is considered clinically justified. The use of unregistered drugs should be considered experimental. Written informed consent from the patient is therefore required and any adverse events need to be reported to the Therapeutic Goods Administration.

Access to anticancer drugs: many evidence‐based treatments are off‐label and unfunded by the Pharmaceutical Benefits Scheme

The off-label use of a drug refers to a use outside the terms of its approval by the Therapeutic Goods Administration (TGA). It is also possible to prescribe unlicensed drugs under the TGA's special access scheme. A high rate of off-label prescribing has previously been reported in cancer. Our study aimed to document the disparity between evidence-based clinical guidelines for anticancer therapy, product approval and funding status of these agents within an academic tertiary/quaternary cancer centre. All chemotherapy protocols approved for use in our specialist oncology centre were assessed to determine if the drugs were off-label or unlicensed for that indication based on review of their current product information. The Pharmaceutical Benefits Scheme (PBS) funding status for each protocol was subsequently assessed. A total of 448 protocols containing 82 different drugs across 15 tumour groups was identified. Overall, 189 (42.2%) of protocols were off-label, and three (0.7%) were unlicensed. This resulted in all 192 protocols being unfunded by the PBS. Of the 189 off-label protocols, 132 (69.9%) were based on established evidence-based treatment guidelines, and a further 39 (20.6%) was based on phase II or III clinical trial data. Over 90% of off-label protocols are supported by established treatment guidelines or published peer-reviewed research even though the medications are not approved for that particular use by the TGA. However, these off-label protocols are unfunded by the PBS; this results in a marked inequality of access to appropriate medications for cancer patients across Australia.

SS01-04 - Specific experience from Australia

The disabling nature of depression, the frequency of treatment resistance, the threat of persistence or frequent recurrence and the commonality of discontinuation early and later in treatment due to intolerable side-effects provide constant challenges for those specialists prescribing existing antidepressant therapies. New treatment options are urgently required. The new antidepressant agomelatine, a MT 1 , MT 2 receptor agonist and a 5-HT 2C antagonist with chronobiotic effects, targets the circadian system which is involved in mood disorders. Agomelatine was made available to Australian psychiatrists for compassionate use through a Special Access Scheme (SAS) prior to its registration in 2010. The SAS was restricted to patients with chronic or recurrent Major Depressive Disorder and those who had no possible therapeutic alternative. Psychiatrists were required to apply for approval to both the Australian regulatory authority and to Servier Laboratories, which was granted on a case-by-case basis. Requests for entry into the SAS were received from 84 psychiatrists for over 370 patients. The average age of all patients was 46 years, the majority of patients were female, all were treatment resistant and presented with either moderate (53%) or severe (41%) MDD as assessed by their treating psychiatrist. This presentation will focus on the difficulties associated with managing patients with treatment resistant depression. Agomelatine has been used in these patients and discussion will centre on the initial analysis of its clinical benefit to these patients as assessed by their treating psychiatrists. We will provide an insight into the potential evidence for the value of agomelatine in this population.

Dose-modified lenalidomide induces sustained hematological response in patients with intermediate to high risk myelodysplasia

No Abstract Available