Is Canada Moving towards a More Agile Regulatory Approval and Reimbursement Process with a Shifting Role for Real-World Evidence (RWE) for Oncology Drugs?

Canada is known to have a complex pathway for new drug approval and reimbursement, resulting in delayed access for patients with serious and life-threatening diseases, such as cancer. Several recent publications from key stakeholders, including patients, physicians and policymakers, highlight patient helplessness, physician frustrations and policymakers entangled in a massive network of bureaucracy unable to make headway. Several quantitative and qualitative assessments using time from regulatory approvals to successful reimbursements confirm long review times and high rejection rates for oncology drugs, especially those receiving conditional approvals.

A pilot study of the role of the BDNF Val66Met polymorphism in response to exercise-augmented exposure therapy for posttraumatic stress disorder.

Brain-Derived Neurotrophic Factor (BDNF) is implicated in extinction learning, which is a primary mechanism of exposure therapy for posttraumatic stress disorder (PTSD). Brief aerobic exercise has been shown to promote BDNF release and augment extinction learning. On the premise that the Val allele of the BDNF Val66Met polymorphism facilitates greater release of BDNF, this study examined the extent to which the Val allele of the BDNF polymorphism predicted treatment response in PTSD patients who underwent exposure therapy combined with aerobic exercise or passive stretching.

Timeliness of Health Technology Assessments and Price Negotiations for Oncology Drugs in Canada.

Purpose: To evaluate whether time targets for Canadian Agency for Drugs and Technologies in Health (CADTH) reimbursement reviews and pan-Canadian Pharmaceutical Alliance (pCPA) price negotiations are being achieved for oncology drugs.

Materials And Methods: Recommendations, dates of submission and publication, and indications for oncology medicines issued between January 2014 and December 2023 were recorded from CADTH's reimbursement reports webpage. The date any negotiation began and the date it was completed (successfully or not), or when a decision was made not to pursue negotiation was extracted from the pCPA's webpage.

New Anticancer Drugs: Reliably Assessing "Value" While Addressing High Prices.

Countries face challenges in paying for new drugs. High prices are driven in part by exploding drug development costs, which, in turn, are driven by essential but excessive regulation. Burdensome regulation also delays drug development, and this can translate into thousands of life-years lost.

Integrating the patient's voice into the research agenda for treatment of chemosensory disorders.

World-wide some 658 million people were infected with coronavirus disease 2019 (COVID-19) and millions suffer from chemosensory impairment associated with long COVID. Current treatments for taste and smell disorders are limited. Involving patients has the potential to catalyze the dynamic exchange and development of new ideas and approaches to facilitate biomedical research and therapeutics.

Topical platelet-rich plasma as a possible treatment for olfactory dysfunction-A randomized controlled trial.

Background: Olfactory dysfunction (OD) affects many survivors of COVID-19. Prior studies have investigated the use of platelet-rich plasma (PRP) injections for OD. We describe the first randomized controlled trial investigating topical PRP for OD treatment and contribute to existing literature illustrating PRP as an emerging therapeutic.

Worldwide study of the taste of bitter medicines and their modifiers.

The bitter taste of medicines hinders patient compliance, but not everyone experiences these difficulties because people worldwide differ in their bitterness perception. To better understand how people from diverse ancestries perceive medicines and taste modifiers, 338 adults, European and recent US and Canada immigrants from Asia, South Asia, and Africa, rated the bitterness intensity of taste solutions on a 100-point generalized visual analog scale and provided a saliva sample for genotyping. The taste solutions were five medicines, tenofovir alafenamide (TAF), moxifloxacin, praziquantel, amodiaquine, and propylthiouracil (PROP), and four other solutions, TAF mixed with sucralose (sweet, reduces bitterness) or 6-methylflavone (tasteless, reduces bitterness), sucralose alone, and sodium chloride alone.

Access to Oncology Medicines in Canada: Consensus Forum for Recommendations for Improvement.

Patient access to new oncology drugs in Canada is only possible after navigating multiple sequential systemic checkpoints for national regulatory approval, health technology assessment (HTA) and collective government price negotiation. These steps delay access and prevent health care providers from being able to prescribe optimal therapy. Eighteen Canadian oncology clinicians from the medicine, nursing and pharmacy professions met to develop consensus recommendations for defining reasonable government performance standards around process and timeliness to improve Canadian cancer patients' access to best care.

Cognitive Behavior Therapy vs Mindfulness in Treatment of Prolonged Grief Disorder: A Randomized Clinical Trial.

Importance: Although grief-focused cognitive behavior therapies are the most empirically supported treatment for prolonged grief disorder, many people find this treatment difficult. A viable alternative for treatment is mindfulness-based cognitive therapy.

Objective: To examine the relative efficacies of grief-focused cognitive behavior therapy and mindfulness-based cognitive therapy to reduce prolonged grief disorder severity.

Human papillomavirus associated oropharyngeal cancer now the most common mucosal head and neck cancer in Queensland.

Introduction: The profile and outcomes of head and neck cancer throughout Australia has changed over the past decade. The aim of this study was to perform a population-based analysis of incidence, demographics, stage, treatments and outcomes of patients diagnosed with oropharyngeal squamous cell carcinoma (OPSCC), with a particular focus on HPV-associated disease.

Methods: This was a retrospective analysis of prospectively collected data within the Queensland Oncology Repository (QOR) and analysed by the Queensland Cancer Control Analysis Team.

The impact of willingness-to-pay threshold on price reduction recommendations for oncology drugs: a review of assessments conducted by the Canadian Agency for Drugs and Technologies in Health.

Since late 2020, the Canadian Agency of Drugs and Technologies in Health (CADTH) has been using a threshold of $50,000 (CAD) per quality-adjusted life-year (QALY) for both oncology and non-oncology drugs. When used for oncology products, this threshold is hypothesized to have a higher impact on the time to access these drugs in Canada. We studied the impact of price reductions on time to engagement and negotiation with the pan-Canadian Pharmaceutical Alliance for oncology drugs reviewed by CADTH between January 2020 and December 2022.

Thiazolidinediones are Partially Effective Bitter Blockers.

Purpose: The bad bitter taste of some medicines is a barrier to overcoming noncompliance with medication use, especially life-saving drugs given to children and the elderly. Here, we evaluated a new class of bitter blockers (thiazolidinediones, TZDs).

Methods: In this study, 2 TZDs were tested, rosiglitazone (ROSI) and a simpler form of TZD, using a high-potency sweetener as a positive control (neohesperidin dihydrochalcone, NHDC).

Chronic social defeat stress broadly inhibits gene expression in the peripheral taste system and alters taste responses in mice.

Human studies have linked stress exposure to unhealthy eating behavior. However, the mechanisms that drive stress-associated changes in eating behavior remain incompletely understood. The sense of taste plays important roles in food preference and intake.

Patient Insights into the Diagnosis of Smell and Taste Disorders in the United States.

Objectives: Diagnosis of smell/taste dysfunction is necessary for appropriate medical care. This study examines factors affecting testing and diagnosis of smell/taste disorders .

Methods: The online was made available to US patients with smell/taste disorders between April 6-20, 2022.

Primary Culture of the Human Olfactory Neuroepithelium and Utilization for Henipavirus Infection In Vitro.

The olfactory receptor neurons (ORNs) are a unique cell type involved in the initial perception of odors. These specialized epithelial cells are located in the neuroepithelium of the nasal cavities and directly connect the nasal cavity with the central nervous system (CNS) via axons, which traverse the cribriform plate to synapse within the olfactory bulb. ORNs are derived from precursor cells that lie adjacent to the basal lamina of the olfactory epithelium.

Thiazolidinediones are partially effective bitter blockers.

Purpose: The bad bitter taste of some medicines is a barrier to overcoming non-compliance with medication use, especially life-saving drugs given to children and the elderly. Here we evaluated a new class of bitter blockers (thiazolidinediones; TZDs).

Methods: In this study, two TZDs were tested, rosiglitazone (ROSI) and a simpler form of TZD, using a high-potency sweetener as a positive control (neohesperidin dihydrochalcone, NHDC).

Canadians Need Improved Access to Drugs for Rare Diseases, Not More Denial.

Sirrs et al. (2023a) discuss what they consider "explosive growth" (p. 11) in the research and development (R&D) and commercialization of expensive drugs for rare diseases (DRDs).

Habituation of distress during exposure and its relationship to treatment outcome in post-traumatic stress disorder and prolonged grief disorder.

Reliving distressing memories is a core component of treatments for post-traumatic stress disorder (PTSD) and prolonged grief disorder (PGD). There is little understanding of how reliving these memories functions in the treatment of these disorders. This study investigated whether reliving functions comparably in the treatment of PTSD and PGD, and whether it is comparably related to treatment outcome.

Proof-of-concept: SCENTinel 1.1 rapidly discriminates COVID-19-related olfactory disorders.

It is estimated that 20%-67% of those with COVID-19 develop olfactory disorders, depending on the SARS-CoV-2 variant. However, there is an absence of quick, population-wide olfactory tests to screen for olfactory disorders. The purpose of this study was to provide a proof-of-concept that SCENTinel 1.

Augmenting trauma-focused psychotherapy for post-traumatic stress disorder with brief aerobic exercise in Australia: a randomised clinical trial.

Background: Although exposure therapy is central in most front-line psychotherapies of post-traumatic stress disorder (PTSD), many patients do not respond to this treatment. We aimed to investigate the effects of brief aerobic exercise on the efficacy of exposure therapy in reducing the severity of PTSD.

Methods: We did a single-blind, parallel, randomised controlled trial in Sydney, NSW, Australia.

Health technology assessment and price negotiation alignment for rare disorder drugs in Canada: Who benefits?

Background: Since 2014, the Canadian Agency for Drugs and Technologies in Health (CADTH), which performs health technology assessments for all federal, provincial and territorial government drug programs (except Quebec's) and the pan-Canadian Pharmaceutical Alliance (pCPA), which conducts price negotiations with manufacturers for all government drug programs, have been aligning their processes.

Objective: To examine trends in CADTH recommendations for non-oncology drugs for rare disorders (DRDs) released between 2014 and 2021, results of pCPA negotiations for the same drugs, and listings in government drug plans to assess who benefits from the alignment.

Results: Recommendations were positive in 87% of the reviews, although all included clinical criteria for use and/or economic conditions.

Proof-of-concept: SCENTinel 1.1 rapidly discriminates COVID-19 related olfactory disorders.

It is estimated that 20-67% of those with COVID-19 develop olfactory disorders, depending on the SARS-CoV-2 variant. However, there is an absence of quick, population-wide olfactory tests to screen for olfactory disorders. The purpose of this study was to provide a proof-of-concept that SCENTinel 1.

Effectiveness of a Videoconferencing-Delivered Psychological Intervention for Mental Health Problems during COVID-19: A Proof-of-Concept Randomized Clinical Trial.

Introduction: Anxiety and depression have increased markedly during the COVID-19 pandemic. There is a lack of evidence-based strategies to address these mental health needs during the pandemic.

Objective: We aim to conduct a proof-of-concept trial of the efficacy of a brief group-based psychological intervention delivered via videoconferencing for adults in Australia distressed by the pandemic.

Evidence that human oral glucose detection involves a sweet taste pathway and a glucose transporter pathway.

The taste stimulus glucose comprises approximately half of the commercial sugar sweeteners used today, whether in the form of the di-saccharide sucrose (glucose-fructose) or half of high-fructose corn syrup (HFCS). Therefore, oral glucose has been presumed to contribute to the sweet taste of foods when combined with fructose. In light of recent rodent data on the role of oral metabolic glucose signaling, we examined psychopharmacologically whether oral glucose detection may also involve an additional pathway in humans to the traditional sweet taste transduction via the class 1 taste receptors T1R2/T1R3.