scholarly journals Why licensing authorities need to consider the net value of new drugs in assigning review priorities: Addressing the tension between licensing and reimbursement

2008 ◽  
Vol 24 (02) ◽  
pp. 140-145 ◽  
Author(s):  
Christopher McCabe ◽  
Karl Claxton ◽  
Anthony O'Hagan

Pharmaceutical regulators and healthcare reimbursement authorities operate in different intellectual paradigms and adopt very different decision rules. As a result, drugs that have been licensed are often not available to all patients who could benefit because reimbursement authorities judge that the cost of therapies is greater than the health produced. This finding creates uncertainty for pharmaceutical companies planning their research and development investment, as licensing is no longer a guarantee of market access. In this study, we propose that it would be consistent with the objectives of pharmaceutical regulators to use the Net Benefit Framework of reimbursement authorities to identify those therapies that should be subject to priority review, that it is feasible to do so and that this would have several positive effects for patients, industry, and healthcare systems.

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5823-5823
Author(s):  
Daniel Chakos ◽  
Azam Farooqui ◽  
Kimberly McCormick ◽  
Abhishek R Chilkulwar

Introduction Per the National Cancer Institute epidemiological data, the incidence of multiple myeloma has not changed significantly over the past decade. Mortality from myeloma has not changed significantly between 2007-2016 with an estimated 5-year survival of 55.2 % between 2009-2015. The treatment of multiple myeloma has improved significantly over the past decade with the approval of several new drugs. In the recent IFM trial, the 4-year overall survival in a select group of myeloma patients was 82% when treated with bortezomib, lenalidomide and dexamethasone followed by autologous stem cell transplant and maintenance therapy. These numbers may further improve with approval of monoclonal antibodies and several new drugs in clinical trials. The cost of healthcare has been growing consistently for the past several years. Per CMS data, healthcare spending grew 3.7% in 2017 reaching $3.5 trillion, or $10,739 per person. National health spending is projected to grow at a rate of 5.5% per year from 2018-2027 reaching nearly $6.0 trillion. Health share of the GDP is expected to rise from 17.9% to 19.4%. Pharmaceutical costs have been rising consistently over the past several years with cancer therapies being some of the costliest. Most new cancer therapy costs more than $100,000 per year with the triplet combinations for myeloma costing upwards of $200,000 per year. Methods Our goal was to evaluate the utilization of the newer drugs approved for myeloma by the Medicare population and assess the economic burden of growing drug prices on the health care system. We queried the national CMS database from 2013-2017 to obtain information on Medicare payments made, number of providers making claims and consulting fees for bortezomib, carfilzomib, daratumumab, elotuzumab, ixazomib, lenalidomide, panobinostat and pomalidomide. Results Between 2013-2017 there has been a significant increase in the annual Medicare payments made for myeloma drugs. The amount of payments has tripled between 2013-2017. The number of claims made and providers making those claims have increased. There has also been an exponential increase in consulting fees paid by various pharmaceutical companies to promote prescription of the aforementioned drugs. The amount of money paid in consultation has nearly quadrupled since 2013. Results are consolidated in Table 1 and Figure 1. Discussion This past decade has been an exciting period in cancer research and drug approvals, with myeloma being one of the many malignancies benefitting from this progress. This was made possible by the collaboration between pharmaceutical companies and academic institutions. With the need for continuous combination therapy in multiple myeloma along with increasing prices of existing drugs and high upfront market prices for newly approved drugs, there is significant financial burden placed on the healthcare system. Based on the published literature substitution of carfilzomib for bortezomib or addition of monoclonal antibodies in induction would double cost. Some of these drug approvals are based solely on surrogate endpoints of overall response rates and progression free survival benefit of few months. This is without proven overall survival data while harboring significant toxicity. Conclusion: With pharmaceutical companies spending millions of dollars for drug development there has been a trend towards higher drug pricing. These companies also pay large amounts of consulting fees to physicians dedicated to aggressively market these newly approved drugs which are priced higher than existing therapy. Some of the current cancer guidelines include authors who have financial conflicts of interest and receive consulting fees from pharmaceutical companies. It would be interesting to investigate the correlation between the consulting fees and its effect on prescription patterns. Even the cancer care guidelines do not consider the cost effectiveness of equally effective regimens. There has been a push from various pioneers in the field to incorporate value-based recommends into their guidelines. Given the trajectory of rising costs within our healthcare system, it is imperative for all physicians to be mindful of value-based care while seeking therapy options that provide true benefit to their vulnerable patient populations. Disclosures No relevant conflicts of interest to declare.


2017 ◽  
Vol 13 (02) ◽  
pp. 114 ◽  
Author(s):  
Nils Wilking ◽  
Gilberto Lopes ◽  
Klaus Meier ◽  
Steven Simoens ◽  
Wim van Harten ◽  
...  

Oncology is facing a crisis of affordability that is not sustainable. The economic burden of cancer is growing, as a result of the rising cancer incidence and increased survival, alongside growing investment in disease prevention, diagnosis and treatment. The prices of new cancer drugs continue to increase, placing growing pressure on many healthcare systems. The aim of this article is to explore the reasons why the cost of cancer care is increasing, and why this increase may become unsustainable unless changes are made. There are only limited options for future health spending. Finding ways to improve the allocation of existing resources to achieve the best outcomes for patients will be key to achieving sustainability, whilst safeguarding the continued development of new, effective cancer treatments. Currently, too many cancer drugs are approved without robust evidence of value, and spending more on treatments does not necessarily translate to improvements in health. For all new drugs, in addition to efficacy and safety, there should be a focus on value, with measured outcomes and pricing that ensures these drugs are affordable. Fundamental changes to healthcare systems and industry are needed to sustain cancer care and allow continued access to effective and safe treatments for all patients.


2019 ◽  
Vol 2019 (3) ◽  
pp. 47-53
Author(s):  
Галина Глембоцкая ◽  
Galina Glembockaya ◽  
Станислав Еремин ◽  
Stanislav Eremin

In order to identify promising strategic development possibilities for the pharmaceutical industry in the Russian Federation, a pilot study was conducted, which has analyzed the main trends in the development of innovative medicines. As a result of the content analysis of available sources of scientific literature, the characteristics of options used in the world practice for increasing the innovative activity of individual subjects and the pharmaceutical market as a whole are presented. Possible reserves for the further development of the innovative component of the pharmaceutical market within the framework of the concept of personalized medicine according to the P4 principle (predictive - personalized - preventive - participatory) are identified and structured. The results of use by individual pharmaceutical companies of scientifically and practically justified approaches to optimizing the costs of development and promoting drugs are presented. The advantages and real prospects of a generally accepted method to reduce the cost of development by «expanding the pharmacological effect» (label expansion) of already existing drugs with a known safety profile in the world practice are shown. A scientific generalization and structuring of the goals and results of the post-registration phase of clinical trials to expand the pharmacological action of a number of drugs already existed at the market have been carried out.


2021 ◽  
Vol 22 (9) ◽  
pp. 4688
Author(s):  
Mootaz M. Salman ◽  
Zaid Al-Obaidi ◽  
Philip Kitchen ◽  
Andrea Loreto ◽  
Roslyn M. Bill ◽  
...  

Neurodegenerative diseases (NDs) including Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, and Huntington’s disease are incurable and affect millions of people worldwide. The development of treatments for this unmet clinical need is a major global research challenge. Computer-aided drug design (CADD) methods minimize the huge number of ligands that could be screened in biological assays, reducing the cost, time, and effort required to develop new drugs. In this review, we provide an introduction to CADD and examine the progress in applying CADD and other molecular docking studies to NDs. We provide an updated overview of potential therapeutic targets for various NDs and discuss some of the advantages and disadvantages of these tools.


2003 ◽  
Vol 29 (4) ◽  
pp. 525-542
Author(s):  
Merri C. Moken

The use of pharmaceutical products in the United States has increased more than the use of any other health resource from 1960 to 1990. In excess of 9,600 drugs were on the market in 1984, and the Food and Drug Administration (“FDA”) approves approximately 30 new drugs and countless new applications for alterations of already existing drugs each year. In 2001, the $300 billion pharmaceutical industry sold $154 billion worth of prescription drugs in the United States alone, nearly doubling its $78.9 billion in sales in 1997. With such a rapid increase in market domination and expenditures, the U.S. government and many hospitals have focused their attention on the sales and pricing practices of pharmaceutical companies, as well as other potential factors contributing to these escalating prices. One such cause of the steadily increasing prices of brand name pharmaceuticals is the sale of fake or counterfeit pharmaceuticals (also called “look-alike” drugs).


Biomolecules ◽  
2021 ◽  
Vol 11 (1) ◽  
pp. 74
Author(s):  
Lúcio Ricardo Leite Diniz ◽  
Yunierkis Perez-Castillo ◽  
Hatem A. Elshabrawy ◽  
Carlos da Silva Maia Bezerra Filho ◽  
Damião Pergentino de Sousa

The coronavirus disease 2019 (COVID-19) pandemic is caused by a novel coronavirus; the Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2). Millions of cases and deaths to date have resulted in a global challenge for healthcare systems. COVID-19 has a high mortality rate, especially in elderly individuals with pre-existing chronic comorbidities. There are currently no effective therapeutic approaches for the prevention and treatment of COVID-19. Therefore, the identification of effective therapeutics is a necessity. Terpenes are the largest class of natural products that could serve as a source of new drugs or as prototypes for the development of effective pharmacotherapeutic agents. In the present study, we discuss the antiviral activity of these natural products and we perform simulations against the Mpro and PLpro enzymes of SARS-CoV-2. Our results strongly suggest the potential of these compounds against human coronaviruses, including SARS-CoV-2.


2019 ◽  
Vol 143 (1) ◽  
pp. 73-77
Author(s):  
Anat Gafter-Gvili ◽  
Ariadna Tibau ◽  
Pia Raanani ◽  
Daniel Shepshelovich

The prevalence of safety-related postmarketing label modifications of medications for hematological malignancies is unknown. We identified 35 new drugs indicated for hematological malignancies approved by the US Food and Drug Administration between January 1999 and December 2014. Characteristics of supporting trials and safety-related label modifications from approval to December 2017 were collected from drug labels. Regulatory review and approval pathways were also collected. New drug approvals were supported by trials with a median of 167 patients (interquartile range 115–316). All drugs were approved based on surrogate endpoints. Twenty-seven drug approvals (77%) were not supported by randomized controlled trials. All drugs received orphan drug designation, and most were granted fast track designation, priority review, and accelerated approval (83, 74, and 60%, respectively). A total of 28 drugs (80%) had postmarketing safety-related label modifications. Additions to black box warnings, contraindications, warnings and precautions, and common adverse reactions were identified in 31, 11, 77, and 46% of drugs, respectively. Five drugs (14%) were permanently or temporarily withdrawn from the US market. Drugs for hematological malignancies are often approved based on limited evidence through expedited regulatory pathways with incomplete safety profiles. Hematologists should be vigilant for unrecognized side effects when prescribing newly approved drugs.


2002 ◽  
Vol 74 (1) ◽  
pp. 145-150 ◽  
Author(s):  
PETER R. SEIDL

The use of products extracted from plants for medicinal purposes can be traced to the beginnings of civilization and up until the end of the nineteenth century natural products were the principal source of medicines. Since then their relative importance has oscillated according to the strategies of large pharmaceutical companies. Now that these strategies are changing, there are new opportunities for countries like Brazil, in which a large proportion of the world's biodiversity is located. There are, however, new circumstances that must be taken into consideration: material must be collected by groups which are formally authorized to do so and under the conditions of the Convention of Biological Diversity, the discovery process is being successively outsourced to smaller specialized firms and there is a growing integration with producers of cosmetics and phytomedicines.


2021 ◽  
Vol 13 (7) ◽  
pp. 3599
Author(s):  
Yoshimi Harada ◽  
Huayi Wang ◽  
Kota Kodama ◽  
Shintaro Sengoku

Biotech startup firms developing pharmaceutical seeds from scientific and technological innovation are burdened by significant Research & Development (R&D) expenses, long-term R&D operations, and low probability of R&D success. To address these challenges while sustainably creating innovations and new drugs, business alliances with existing pharmaceutical companies are one of the most important issues on the management agenda. The present study explores the necessity and significance of business alliances with pharmaceutical companies for the development of drug-discovery by Japanese biotech startup firms under high uncertainty. This study investigates the types of alliances to understand the origins of sustainability of these creative activities. First, we investigate and analyze the details of the partnership and its impact on the products under development based on the publicly available information of 16 drug discovery biotech startup firms in Japan that had become public since 2010. As a result, all firms continued their operations with the funds obtained from the business alliances with pharmaceutical firms at the time of their initial public offering (IPO). In addition, 56% of these firms’ alliance projects (n = 73) were seeded-out, and 32% seeded-in, indicating that they had adopted flexible alliance strategies not limited to seed-out ones. For sustainable going concern of the biotech startup business, it is valuable to consider multiple strategic options: “in-licensing and value up”, “best-in-class”, “platform leadership” and “first-in-class” depending on the characteristics of seeds and environmental restrictions.


PEDIATRICS ◽  
1992 ◽  
Vol 90 (2) ◽  
pp. 281-282
Author(s):  
LEWIS H. MARGOLIS

In Reply.— In my essay1 I suggested that the acceptance of gifts from pharmaceutical companies violates a duty of justice because gifts represent resources inappropriately taken from patients through the added costs of the drugs that they buy. Dr Procopio raises the larger question of how other types of promotional activities increase the cost of drugs in particular and medical care in general. His letter makes clear, however, that the contribution of an activity such as the distribution of samples to the cost of care is difficult to determine because several different purposes are served.


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