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Pharmaceutical Medicine - Health Technology Assessment

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MAESTrO: About MAESTrO. About MAESTrO MAESTrO is an online market access data resource and consultancy based in Sydney, Australia.


Building on Michael’s 20 years’ experience in health technology assessment, we constantly invest in expert research, analysis, customer support and design to create a comprehensive database of global market access submissions and outcomes. Thomas Mulvaney Development Thomas Crowley Development manager Mark Reuten User Experience Our people MAESTrO’s growing team reflects the growing demand for our unique services. Emily Tjeuw Database Manager Rob Gower Marketing manager About Michael Michael gained his B.Sc (Hons) in Biochemistry & Pathology from the University of Melbourne and a B.

Since 2011, Michael has been an independent consultant on biopharmaceutical reimbursement and pricing matters. Michael has a strong record of speaking and publishing on medical and health economy topics, including: Wonder M, 2014. Wonder M, Milne R, 2014. Wonder M, Backhouse M, Hornby E., 2013. Wonder MJ, 2013. Health Economic Evaluation Alongside Stepped Wedge Trials: A Methodological Systematic Review. MSAC - 1634 - Comprehensive genomic profiling of non-small cell lung cancer tumour tissue specimens using next generation sequencing assays. Application Detail.

MSAC - 1634 - Comprehensive genomic profiling of non-small cell lung cancer tumour tissue specimens using next generation sequencing assays.

Paying For Value From Costly Medical Technologies: A Framework For Applying Value-Based Payment Reforms. Budget 2020-21: Improving Access to Medicines – Unique Identification framework for PBS medicines. Principles for deliberative processes in health technology assessment. UK testing Netflix-style payments to boost antibiotics development. Dive Brief: Health authorities in the United Kingdom are testing out a new payment model they hope will better incentivize drug companies to develop new antibiotics.The subscription-style model would offer participating drugmakers an upfront payment in exchange for access to their antibiotics. This differs from the current system, which bases payments on the volume of antibiotics sold to the U.K.'s National Health Services.NHS said the subscription model makes it "more attractive" for industry to invest in antibiotics at a time when antimicrobial resistance, or AMR, poses significant health and economic risks.

A World Health Organization report released in April found drug-resistant diseases result in upwards of 700,000 deaths each year, and concluded AMR may cause economic damage similar to the 2008-2009 global financial crisis. Dive Insight: Officials are slowly warming to new drug purchasing systems like the Netflix-style subscription model. To that point, Merck & Co. NICE and PBAC – TACS Healthcare. The current review of NICE is a useful foil upon which to consider the announced refresh of the Australian National Medicines Policy.

NICE and PBAC – TACS Healthcare

Both are being driven by concern that access processes are not keeping pace with biomedical innovation. The table below provides a side by side comparison of the two appraisal systems and HTA reimbursement environments. Of interest is the relatively recent increase in NHS responsibility and focus on overall budget impact of reimbursing a new technology.

The need to effectively manage uncertainty whilst still making decisions is a key challenge of providing timely access, especially as patient populations are becoming smaller and more targeted. References and Notes: Bmj.l7058. MSAC - 1587 - YESCARTA™ (axicabtagene ciloleucel [KTE-C19]) for the treatment of refractory or relapsed CD19-positive lymphoma. Announcement MSAC stakeholder meeting for CAR-T cell therapy for relapsed or refractory Diffuse Large B Cell Lymphoma, Primary Mediastinal B-cell Lymphoma and Follicular Lymphoma The Medical Services Advisory Committee (MSAC) will hold a stakeholder meeting on 12 November 2019 on CAR-T cell therapy for relapsed or refractory Diffuse Large B Cell Lymphoma (DLBCL), Primary Mediastinal B-cell Lymphoma (PMBCL), and Follicular Lymphoma (FL).

MSAC - 1587 - YESCARTA™ (axicabtagene ciloleucel [KTE-C19]) for the treatment of refractory or relapsed CD19-positive lymphoma

The purpose of the meeting is to allow MSAC to seek advice on patient eligibility for treatment and other aspects of patient management, in light of the recent MSAC consideration of Kymriah® (tisagenlecleucel, sponsored by Novartis Australia) and the future MSAC consideration of Yescarta® (axicabtagene ciloleucel, sponsored by Gilead Sciences). MSAC has invited treating clinicians, consumers, and representatives of Novartis and Gilead and the Commonwealth and State/Territory health departments to attend the meeting. Potential Solutions To Current Pricing Models For Cell And Gene Therapies. By Erin Harris, Editor-In-Chief, Cell & Gene Follow Me On Twitter @ErinHarris_1 Curative cell & gene therapies are changing modern medicine as we know it, but are their high price tags fair to the patient?

Potential Solutions To Current Pricing Models For Cell And Gene Therapies

Move over, religion and politics. Drug prices are one of the new polarizing topics on the minds of anyone paying attention. Headlines about high-priced cell and gene therapies specifically can cause sticker shock for the patient; exhibit A — Novartis’ Zolgensma, the spinal muscular atrophy (SMA) gene therapy treatment priced at $2.125 million is the world’s most expensive drug to date. But cell and gene therapies are their own animal; the differences between them and traditional biologics are too numerous to count. The State of Current Pricing Models. Alternative pricing models a fantasy for gene therapies? This feature is part of a series focused on gene therapy.

Alternative pricing models a fantasy for gene therapies?

To view other posts in the series, check out the spotlight page. Once upon a time, gene therapy was thought to be the miracle of science that was going to change everything. After safety issues and patient deaths, that didn’t work out quite like the industry had hoped. Yet, 20 years later, the next generation of gene therapies is coming of age.

These drugs so far have proven to be safer and more effective, but are raising new issues around cost. Medicines Status Website. Role of Health Technology Assessment in Drug Policies: Korea. Earlier access to cheaper medicines for 1.6 million Australians. Date published: 1 January 2020 Media type: Media release The Morrison Government has delivered on its election commitment, significantly lowering the threshold for the Pharmaceutical Benefits Scheme (PBS) Safety Net from 1 January 2020.

Earlier access to cheaper medicines for 1.6 million Australians

This means from today, more than 1.6 million concesional and general patients will get earlier access each year to free or significanrly cheaper PBS medicines. The PBS Safety Net eases the financial burden on Australians who spend significant amounts on PBS medicines, such as people with chronic conditions. Once people reach the safety net threshold, they can apply to get free or discounted PBS medications for the remainder of the calendar year.

The safety net thresholds have been lowered from: $390.00 to $316.80 for concessional patients.$1550.70 to $1486.80 for general patients. The changes mean concessional patients will need to get 12 fewer scripts filled in a year in order to reach the PBS Safety Net, and two fewer scripts for general patients. Vitrakvi deemed ‘not cost-effective’ by NICE. Bayer’s Vitrakvi (larotrectinib) has been rejected by The National Institute for Health and Care Excellence (NICE), as at its current price, it doesn’t have the “potential to be cost-effective.”

Vitrakvi deemed ‘not cost-effective’ by NICE

The guidance service published the decision in regards to the drug being used in advanced neurotrophic tyrosine receptor kinase (NTRK) fusion-positive solid tumours in adults and children who have no satisfactory treatment options. The organisation, however, encourages further data collection on similar “game changing” histology independent cancer drugs, with Meindert Boysen, director of the centre for health technology evaluation saying that the meds are “an exciting new development in cancer care.” Another histology independent treatment, Roche’s entrectinib, which NICE is also appraising could become the first histology independent treatment to be available to patients, provided it receives its marketing authorisation. Are Payers Ready, Willing, and Able to Provide Access to New Durable Gene Therapies?

Introduction After several decades of development, the US Food and Drug Administration (FDA) approved innovative new gene therapies in 2017.

Are Payers Ready, Willing, and Able to Provide Access to New Durable Gene Therapies?

Two chimeric antigen receptor (CAR) T-cell therapies, tisagenlecleucel (Kymirah/Novartis) and axicabtagene ciloleucel (Yescarta/Kite Pharma), with listing prices of $475 000 and $373 000, respectively,1 provide new hope to patients with certain types of leukemia and lymphoma. In late December 2017, voretigene neparvovec-rzyl (Luxturna/Spark Therapeutics) became the first pharmacologic gene therapy approved to treat an inherited retinal disease.

Its list price is $425 000 per eye or $850 000 per patient, as the majority of patients will need treatment for both eyes.2. The High Cost of New Cancer Therapies—A Challenge of Inequality for All Countries. When people or governments cannot afford effective and potentially life-saving or life-prolonging treatments, we usually think of this situation affecting low-resource countries in the Third World.

The High Cost of New Cancer Therapies—A Challenge of Inequality for All Countries

When antiretroviral HIV medications increased life expectancy almost to normal throughout much of the world, hundreds of thousands died in African countries, in part due to the high cost of medications. Ford et al1 in 2011 stated, “Early concerns about the cost and complexity of treatment were overcome thanks to the efforts of a global coalition of health providers, activists, academics, and people living with HIV/AIDS, who argued that every effort must be made to ensure access to essential care when millions of lives depended on it.” Avoiding the Tragedy of the Commons in Health Care. A Beginner's Guide to Understanding Curative Therapies. Headlines from late-breaking sessions at scientific conferences herald exciting advances in medicine.

Yet, what is often whispered between physicians, and more obvious to market access specialists and health technology assessors, are the relatively small health gains associated with most new medical treatments. This is not a fault of innovation—advances in nutrition, sanitation, and improved socioeconomic conditions have already created a high bar for life expectancy in developed countries.1 Similarly, we have already benefitted from advances in medicine introduced decades ago—vaccines and antibiotics—curing and eradicating prematurely fatal conditions that have long plagued humanity.

The past 50 years also has marked significant declines in cardiovascular mortality and improvements in cancer outcomes. Not surprisingly then, scientific discovery has shifted its attention toward the outliers—rare occurrences of disease that lead to significant early loss of life or prolonged suffering. New Approaches to Value Assessment: Towards More Informed Pricing in Healthcare—An Introduction. NCCN clinical practice guidelines in oncology (NCCN Guidelines) with NCCN Evidence Blocks™.

National Comprehensive Cancer Network L.E. Schnipper, H.E. Davidson, D.S. Wollins, et al.Updating the American Society of Clinical Oncology value framework: revisions and reflections in response to comments received. Defining and Managing High-Priced Cures: Healthcare Payers’ Opinions. Introduction Recent regulatory approvals of several highly effective, potentially curative, but high-cost treatments have brought these therapies to the forefront of health policy discussions. Some treatments, such as the new generation of direct-acting antiviral therapy for hepatitis C, are major advances in both efficacy and tolerability from previously available treatments for a large population.

The list prices for the marketed curative treatments are often high as evidenced by the suggested $475 000 list price for the CAR T-cell therapy, tisagenlecleucel for acute lymphoblastic leukemia,10 and $850 000 for the gene therapy voretigene nepavarac for inherited retinal disease.11, 12 When combined with highly prevalent conditions or expanding indications, these high prices can have substantial effects on overall health spending. Results Interviewee Description. Addressing the Value of Gene Therapy and Enhancing Patient Access to Transformative Treatments.

Gene therapy is a radical shift in our approach to disease treatment. By modifying the expression of a patient’s genes or repairing abnormal genes, gene therapy often addresses the root cause of diseases. Even though several new gene and cell therapies have received U.S. Food and Drug Administration (FDA) approvals over the past 20 years, the field has recently experienced a turning point.

Three gene therapies were approved for human medical use in the United States in 2017,1, 2, 3 including the first in the country for an inherited condition. Concise Review: The High Cost of High Tech Medicine: Planning Ahead for Market Access - Driscoll - 2017 - STEM CELLS Translational Medicine - Wiley Online Library. Policy Strategies for the “New Normal” in Healthcare to Ensure Access to High-Quality Cancer Care in: Journal of the National Comprehensive Cancer Network Volume 17 Issue 2 (2019)

Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs—a Systematic Literature Review. Next Steps in Methodology Development? Introduction. Propensity Score Methods in Health Technology Assessment: Principles, Extended Applications, and Recent Advances. A review of rare disease policies and orphan drug reimbursement systems in 12 Eurasian countries. Marcin Czech1, 2, Aleksandra Baran-Kooiker3*, Kagan Atikeler4, 5,

Critical Review of European Health-Economic Guidelines for the Health Technology Assessment of Medical Devices. Introduction. Commercial Framework v1. Cancer medicine funding: Parallel assessment. From 1 January 2020, suppliers can apply to PHARMAC for funding of cancer medicines at the same time as they’re assessed by Medsafe. We predict that parallel assessment will reduce the time it takes for cancer medicines to go from submission to Medsafe to being ranked by PHARMAC by 12 to 15 months. ZonMw – The Netherlands Organisation for Health Research and Development. The Hague, The Netherlands "An active involvement of health care professionals, patients, researchers and healthcare decision-makers is essential in HTA research to identify priorities, perform high quality HTA and facilitate implementation". – Mr. Pharmaceutical Benefit–Risk Communication Tools: A Review of the Literature.

Obscure Model Puts a Price on Good Health—and Drives Down Drug Costs. ICER Identifies Costliest US Drug-Price Hikes That Are Not Supported by New Clinical Evidence - ICER. — In 2017 and 2018, out of nine identified drugs that had substantial price increases on top of already high current spending, seven drugs had no new important evidence to support their price increases. The net price increases on these seven drugs alone cost American insurers and patients an additional $5.1 billion over two years — BOSTON, October 8, 2019 – The Institute for Clinical and Economic Review (ICER) today published its first annual report on Unsupported Price Increases (UPI) of prescription drugs in the United States. Humira, Rituxan top list of U.S. drugs with biggest price increases: report. Italy announces a new reimbursement model for the first CAR-T-cell therapy. National report - May 2019. Context matters global. Australian Government Department of Health. The Value of Vaccines: Ensuring Australia keeps pace with community values and international practice.

Value-Based Pricing for Emerging Gene Therapies: The Economic Case for a Higher Cost-Effectiveness Threshold: Journal of Managed Care & Specialty Pharmacy: Vol 25, No 7. Pricing and Paying for Cures: Early Experience with HTA of Gene Therapy in the USA. In a new OHE Seminar Briefing, Dr Steven Pearson, President of the Institute for Clinical and Economic Review (ICER), discusses early experiences of using HTA to assess Gene Therapy, setting out four challenges. First, uncertainty about clinical effectiveness makes it difficult for ICER to suggest an appropriate price. The Limitations of QALY: A Literature Review. Health Technology Assessment as Part of a Broader Process for Priority Setting and Resource Allocation. Keeping medicines affordable.

Broadening the Evidence report 2019. - Good Research Practices for Measuring Drug Costs in Cost Effectiveness Analyses: Issues and Recommendations. List all terms. Australian Health Care. Gsk viiv the pbs in australia feb 2018. UHC2030 - Accelerating progress towards Universal Health Coverage. Mirror, Mirror 2017: International Comparison. Health Technology Assessment in Japan: A Pharmaceutical Industry Perspective - Kaori Kido, Naoki Matsumaru, Katsura Tsukamoto, 2018. Health Technology Assessment and Appraisal of Therapies for Rare Diseases.

Health technology assessment in Saudi Arabia: Expert Review of Pharmacoeconomics & Outcomes Research: Vol 18, No 4. Health Technology Assessment and Its Use in Drug Policies in China. - PubMed - NCBI. Ensuring access to medicines: How to redesign pricing, reimbursement and procurement? - NCBI Bookshelf. Prescription drug coverage in Canada: a review of the economic, policy and political considerations for universal pharmacare. Database Access - UNSW Library. Database Access - UNSW Library. Factors Influencing Delays in Patient Access to New Medicines in Canada: A Retrospective Study of Reimbursement Processes in Public Drug Plans. HTA - Health Technology Assessment, Consumers and Communities. HiT pharmaceutical regulation 15 European countries. PBS Process Improvements. Voisin Consulting Life Sciences. PBS Process Improvements. Public Consultation – Draft Procedure Guidance and Forms for Listing Medicines on the PBS.

Excessive Pricing in Generic Drug Markets. A Look Inside Trump's 4-Point Plan For Curbing Drug Prices. The Trump Administration's Latest Drug Pricing Initiatives. China updates key drug list in boost for Big Pharma. Public Consultation – Draft Cost Recovery Implementation Statement (CRIS) for Listing Medicines on the PBS and Designated Vaccines on the NIP. Rogs 2019 parte chapter10. ATOM Report 2018 FINAL. MA Compare final. Glossary. Addressing the challenge of high-priced prescription drugs in the era of precision medicine: A systematic review of drug life cycles, therapeutic drug markets and regulatory frameworks.

Bcp. High cost of new drugs. Directory of HTA Organizations Worldwide. Publications. Hans Rosling's 200 Countries, 200 Years, 4 Minutes - The Joy of Stats - BBC Four. Opioid Epidemic: U.S. Life Expectancy Drops As Crisis Leaves Mark. Life expectancy rises 'grinding to halt' Guidelines for the Economic Evaluation of Health Technologies: Canada. How is the price of medicine worked out in Australia? Database Access - UNSW Library. Database Access - UNSW Library. Database Access - UNSW Library. Untitled. The value of self medication summary of existing evidence. Deloitte au economics new wave immunotherapy cancer medicines 121017. Real-World Evidence. Postgraduate study - Taught degree programmes A‑Z - Health Technology Assessment.

Trial Detail - UK Clinical Trial Gateway. PROSPERO. Methods for the development of NICE public health guidance (third edition) Securing good health for the whole population: Final report - February 2004 : Department of Health - Publications.