Precision medicine in 2020: what barriers remain for drug developers? – pharmaceutical-technology.com

High costs, data management issues and recruitment problems are some of the main challenges for personalised medicine in clinical development. Credit: US Air Force/Kemberly Groue Genome sequencing costs are falling fast, opening the playing field for developing highly personalised drug candidates. Personalised medicine in the 21st century offers the promise of therapies customised based on the study of what truly makes us unique: our DNA.

The importance of the individual has been widely established in medicine since time immemorial. The well-worn adage that physicians should treat the patient, not the disease has been around since the 19th century, and the awareness of that message is far older than that. Even Hippocrates, the father of Western medicine who treated patients in the fifth century BC, stressed the importance of treating each patient as an individual.

For the sweet [medicines] do not benefit everyone, nor do the astringent ones, nor are all patients able to drink the same things, Hippocrates wrote.

Hippocrates might have tailored his rudimentary treatments based on the patients age, physique and other easily observable factors, but personalised medicine in the 21st century offers the promise of therapies customised based on the study of what truly makes us unique: our DNA.

Advancements in genomics, proteomics, data analysis and other fields both medical and technical are gradually facilitating the development of laser-focused drugs, as well as the ability to predict peoples personal risk factors for particular diseases and how individual responses to various treatments might differ.

After years of anticipation, there is now evidence that governments around the world have clocked the importance of personalised medicine and are driving efforts to the build the genetic data sets and biobanks that are required to push the science forward. Former US President Barack Obama launched the Precision Medicine Initiative to great fanfare in 2015; the scheme has since evolved into the All of Us research programme, which aims to gather health data from more than a million US volunteer-citizens to unlock new insights.

In the UK, the 100,000 Genomes Project reached its goal of sequencing 100,000 whole genomes from 85,000 NHS patients with cancer or rare diseases. Genomics England has noted that so far, analysis of this data has revealed actionable findings in around one in four rare disease patients, while about 50% of cancer cases suggest the potential for a therapy or clinical trial.

You can match a blood transfusion to a blood type that was an important discovery, said Obama at the launch of the Precision Medicines Initiative, summarising the broad appeal of personalised therapies and diagnostics. What if matching a cancer cure to our genetic code was just as easy, just as standard? What if figuring out the right dose of medicine was as simple as taking our temperature?

The stage might be set for personalised healthcare to dramatically transform public health, but few in the medical field would deny that the world is hardly ready yet. Transitioning from the traditional one-cure-fits-all treatment model to new processes that leverage patients genetics, lifestyles and environmental risk factors is an immense task that presents challenges in both the laboratory and the clinic.

Oncology is, by a landslide, the field that has been most impacted by developments in precision medicine; around 90% of the top-marketed precision treatments approved in 2018 were cancer therapies, while other therapeutic areas have lagged far behind. The majority of approved precision medicines in oncology achieve something of a halfway house between the old way and the new they fall short of being tailored to a specific individual, but they allow for more detailed stratification of patients by the oncogenic mutations of their tumours, which may be driving cancer cell survival and growth.

Common examples of these mutations are HER-2 in certain breast and stomach cancers, BRAF in melanoma and EGFR in lung cancer. High expression of these proteins at cancer sites can be targeted by precision treatments, such as Roches monoclonal antibody Herceptin (trastuzumab) for HER-2, Genentechs BRAF inhibitor Zelboraf (vemurafenib), and Roches EGFR inhibitor Tagrisso (osimertinib). Regulators such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are also increasingly approving tumour-agnostic treatments the first and most famous of which is Mercks immunotherapy Keytruda (pembrolizumab) which target specific biomarkers regardless of tumour location.

But despite the availability of a growing menu of personalised cancer treatments, actually matching patients up to the right therapy can be difficult. According to a survey of US acute care organisations conducted by Definitive Healthcare and published in December 2019, just over 20% had established precision medicine programmes. Investment in genomic testing is vital to quickly get patients on the best treatment course, but financial and operational barriers remain.

The foremost among these is the cost associated with genomic sequencing and the use of companion diagnostic devices, cited by 28% of Definitive Healthcares respondents as the biggest challenge for already-established precision medicine schemes. Lack of expertise is another obstacle, as many physicians may struggle to accurately interpret test results without specialist assistance another major cost driver for clinics and hospital departments trying to build pathology teams that are up-to-date with the newest tests. A 2018 survey of 160 oncologists by Cardinal Health found that 60% of physicians who dont use genomic tests avoid them because of the difficulty of interpreting the data.

In clinical research and development, too, there are growing pains associated with moving the pharmaceutical pipeline towards drugs targeting smaller patient sub-groups. Again, cost is a central issue companion diagnostics dont come cheap, finding and validating biomarkers to guide targeted therapies is a lengthy task, and analysing vast amounts of data often requires new teams with specialised knowledge.

The expense of incorporating a host of new processes into innovative trial designs not to mention the cost of manufacturing cell and gene therapies obviously has an impact on the list price of personalised drugs that win approval. This is most clearly seen in the eye-watering prices of some of the worlds first truly individualised cancer treatments, chimeric antigen receptor T-cell (CAR-T) therapies.

Treatments such as Novartiss Kymriah and Gileads Yescarta remove T-cells from the patients blood, modify them to target tumour cell antigens and then infuse them back into the blood stream. These therapies have achieved impressive results in rare and advanced cancers, but cost upwards of $400,000 per patient, limiting their reimbursement options among both private and public payers. Promising advances in CAR-T manufacturing and potential off-the-shelf T-cell production could help bring these costs down in the years to come, but for now the problem remains.

As for the broader clinical trial eco-system, these studies have been historically set up to assess a drug candidates safety and efficacy in an increasingly large segment of the patient population, building evidence towards the regulatory approval process. Bringing a personalised medicine through the clinical development process is a new paradigm in a number of ways; as well as the aforementioned cost drivers, there can be an extra enrolment burden to identify and recruit patients this is already a common cause of trial failure, but its all the more difficult when youre looking to access a small patient sub-group with the appropriate biological profile.

The difficulty of providing sufficient evidence of safety and efficacy can also present issues where current regulations struggle to accommodate new innovations in personalised medicine. Smaller trial designs present statistical problems in terms of understanding a drugs definitive risk-benefit profile, and while some personalised applications can be discovered as part of larger trials that fail to meet their endpoints outside of a select patient group with particular biomarkers, many current regulations dont accept post hoc analysis and would require an entirely new trial.

Personalised medicine developers desire better guidance on how best to design a successful clinical trial for a personalised therapy, because absent guidance, they risk presenting suboptimal evidence regarding stratification options, reads a 2017 study on personalised medicine barriers, published in the Journal of Law and Biosciences. Designing clinical trials for differently responding subgroups (for example, biomarker-positive and biomarker-negative groups) requires additional time and resources. Companies are reluctant to make this investment without a commensurate increase in the certainty of regulatory approval.

The increasing use of surrogate endpoints, conditional approvals and real-world data is helping to address these issues, but theyre not yet an ideal solution. Conditional approvals rely on very careful post-marketing observation and analysis, while the value of surrogate endpoints has been questioned, adding to the tension between accelerating approvals and ensuring patient safety.

The ultimate benefits of creating more personalised treatments are clear, and their advantages for human health could, in the long-term, be matched by their economic returns. After all, quickly treating patients with the right therapy for them or, even better, using knowledge of a patients genetic risk profile to prevent illness in the first place would be a huge financial gain for overburdened health systems.

Todays costs are gradually falling, as NIH data on DNA sequencing costs demonstrate. But there is still a long way to go before we can wave goodbye to the blanket drug development that has dominated modern pharma for decades, even in the advanced field of oncology, let alone other therapeutic areas. Only a sustained and holistic push from regulators, drug developers, clinicians, governments and others will be enough to bring us over the line.

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Precision medicine in 2020: what barriers remain for drug developers? - pharmaceutical-technology.com

Hemophilia Treatment Market outlook with industry review and forecasts – Technology Magazine

Hemophilia Treatment Market will exceed USD 14.8 billion by 2025; as per a new research report.

Increasing burden of bleeding disorders including hemophilia will stimulate global hemophilia treatment market growth in upcoming years. According to the World Federation of Hemophilia, the global prevalence of hemophilia was depicted to be around 184,723 in the year 2016. Currently, the number of individuals in the U.S. suffering from hemophilia is predicted to be about 20,000. Individuals with hemophilia A is anticipated to be around 149,764 and individuals with hemophilia B are around 29,712. According to the Center for Disease Control and Prevention, average age for diagnosis of hemophilia is 36 months for mild hemophilia, 8 months for moderate hemophilia and 1 month for severe hemophilia. Thus, rise in prevalence of hemophilia will boost the hemophilia treatment business growth in the forthcoming period.

Rise in initiatives and educational programs by the government will further boost the hemophilia treatment market. Organizations such as World Federation of Hemophilia (WFH) along with government of several countries carry out diagnosis program. One such program is Cornerstone initiative by WFH for underprivileged people. Furthermore, increase in R&D activities along with novel drug launches will upsurge the growth of hemophilia treatment market; for instance, gene therapy is under clinical investigation.

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Additionally, increasing adoption of prophylaxis treatment by individuals suffering from hemophilia is contributing to the growth of the market. However, high-costs associated with hemophilia drugs will limit the growth of hemophilia treatment market. Furthermore, lack of awareness and adequate treatment in developing and under-developed countries will have negative impact on the market growth.

Hemophilia A segment will hold a notable revenue size of USD 6,743.7 million in the year 2018. The prevalence rate is high in developing and underdeveloped economies. According to the National Hemophilia Foundation, hemophilia A is four times more common than hemophilia B. Thus, increase in number of individuals suffering from hemophilia A along with rise in awareness regarding treatment of hemophilia will positively impact the growth of segment.

Recombinant coagulation factor concentrates market is estimated to dominate the market in the coming years and grow at 5% over the estimation period. Additionally, recombinant coagulation factor concentrates have a low risk of transmitting an infectious disease and are used to prevent viral infection. Also, recombinant coagulation factor concentrates increases the safety of replacement therapy, increasing its adoption rate leading to increased business growth over the forecast period.

Prophylaxis treatment market segment will hold a significant revenue size of USD 7,219.9 million in 2018 and is estimated to hold majority market share over the forthcoming years owing to rise in adoption of prophylaxis treatment. Patients with severe hemophilia are recommended prophylactic treatment to avoid bleeding episodes. Furthermore, some recent studies have proven prophylactic treatment to be quite effective with incidence of joint pain getting reduced by prophylactic treatment. Thus, better efficacy and merits over other treatment will increase its adoption rate, thereby leading to upsurge in prophylaxis segment revenue.

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Factor replacement therapy market will grow at a significant pace owing to its preference over other therapies. Recombinant factor replacement therapy is the first line treatment for patients with moderate to severe hemophilia. Increasing R&D investments and new drug launch in replacement therapy is estimated to drive the growth of segment. Furthermore, increasing awareness amongst the patients regarding factor replacement therapy will boost the market growth.

Coagulation factors market held a significant market share and was valued at USD 9,948.3 million in the year 2018. Coagulation factors are used in replacement therapy. The rise in prevalence of moderate to severe hemophilia will spur the demand of replacement therapy, thereby boosting the demand for coagulation factors.

Injectables route of administration is projected to witness growth at 5% over the estimation period owing to majority of medications that are dispensed in injectable form. Also, bioavailability of intravenously administered drug is 100%, thus, intravenous route is preferable for quick action. Nasal spray segment is anticipated to foresee momentous growth in the coming years due to various advantages offered by nasal route over injectables such as ease of administration.

Hemophilia treatment in adult will dominate the patient segment and was valued at USD 6,778.2 million in 2018, due to rise in prevalence of hemophilia in adults. According to one recent article, around 65% of the individuals suffering from hemophilia are adults. Also, the chances of developing moderate to severe hemophilia are high in adults. Thus, growing age results in high occurrence of hemophilia, thereby impacting the growth of segment.

Hemophilia treatment centers (HTCs) is expected to grow at 4.6% in the coming years owing to increase preference of these centers over hospitals and clinics. Hemophilia treatment centers are primarily aimed to provide comprehensive hemophilia treatment to the underprivileged patients. Additionally, HTCs focus on organizing initiatives for spreading awareness regarding hemophilia in developing and under-developed countries. Also, special care provided by HTC's will increase its preference, thereby fueling its growth.

U.S. hemophilia treatment market was valued at USD 3,539.3 million in the year 2018. According to the National Hemophilia Foundation, the number of individuals suffering with hemophilia are estimated to be around 20,000. Furthermore, favorable reimbursement policies provided by hemophilia treatment centers and insurance in the country will raise the adoption rates of hemophilia treatment.

India hemophilia treatment market is expected to grow at 9.2% in the coming years owing to rise in awareness regarding hemophilia treatment in developing economies. Furthermore, rise in government initiatives, increase in healthcare expenditure and educational programs conducted by government will further increase the market growth.

Some of the major players involved in the global hemophilia treatment market are Sanofi SA, Swedeish Orphan Biovitrum AB, Biotest AG., Genentech, among others. Companies are implementing several business strategies such as strategic collaborations, mergers and acquisition to increase their product portfolio and market footprint. For instance, in June 2018, Biotest opened two plasma collection centers in Czech Republic to complement its plasma collection capacity.

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Hemophilia Treatment Market outlook with industry review and forecasts - Technology Magazine

Precision Cancer Therapies Market Size, Status and Forecast 2025 Trends, Size, Drivers, Strategies, Products and – News Times

Precision Cancer Therapies Market research now available at Brand Essence Research encompasses an exhaustive Study of this business space with regards to pivotal industry drivers, market share analysis, and the latest trends characterizing the Precision Cancer Therapies industry landscape. This report also covers details of market size, growth spectrum, and the competitive scenario of Precision Cancer Therapies market in the forecast timeline.

The Precision Cancer Therapies Market Report provides key strategies followed by leading Precision Cancer Therapies industry manufactures and Sections of Market like- product specifications, volume, production value, Feasibility Analysis, Classification based on types and end user application areas with geographic growth and upcoming advancement. The Precision Cancer Therapies market report provides comprehensive outline of Invention, Industry Requirement, technology and production analysis considering major factors such as Revenue, investments and business growth.

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The well-established players in the market are:

Abbott Laboratories, Bayer HealthCare, GlaxoSmithKline, OncoGenex Pharmaceuticals, Hospira, Boehringer Ingelheim, AstraZeneca, Aveo Pharmaceuticals

This report for Precision Cancer Therapies Market discovers diverse topics such as regional market scope, product market various applications, market size according to specific product, sales and revenue by region, manufacturing cost analysis, Industrial Chain, Market Effect Factors Analysis, market size forecast, and more.

Drivers & Hindrances of the Precision Cancer Therapies market: How does the report explicate on the same

The report unveils the driving parameters affecting the commercialization chart of this industry.

The Precision Cancer Therapies market research report further illustrates the various challenges that this market is prone to as well as its impact on the market trends.

An important aspect that the report sets focus on is the market concentration ratio for the predicted timeframe.

Important Points Mentioned In the Precision Cancer Therapies Market

Manufacturing Analysis:The report comprises descriptive information after analyzing multiple segments of Precision Cancer Therapies market, which include product type and applications, among others. Precision Cancer Therapies market report includes a separate chapter emphasizing thorough analysis of the manufacturing process authenticated via primary information gathered from key officials of reputed industries and several industry analysts.

Sales and Revenue Estimation:By implementing several top-down and bottom-up approaches on the historical sales & revenue data and the current market status, the researchers have forecasted the market growth and size in key regions. Moreover, the report includes a comprehensive study on classified and prominent types as well as end-use industry. The report even provides significant information related to regulatory policies and macroeconomic factors that determine Precision Cancer Therapies industry evolution and predictive analysis.

Demand & Supply Assessment:Precision Cancer Therapies report also offers important information on product & service distribution, manufacturing, Consumption, and Export & Import (EXIM) ** if applicable.

Competitiveness:Precision Cancer Therapies report provides key information based on the product portfolio, company profile, product & service cost, potential, and sales & revenue generated by the global and regional leading companies.

Market segment by Type, the product can be split into

Hormone Therapy, Immunotherapies, Targeted Therapy, Monoclonal Antibody Therapy, Gene Therapy

Market segment by Application, split into

Hospitals, Diagnostic Centers, Oncology Clinics, Research Institutes

Market segment by Regions/Countries, this report covers

North America (United States, Canada and Mexico)

Europe (Germany, UK, France, Italy, Russia and Turkey etc.)

Asia-Pacific (China, Japan, Korea, India, Australia, Indonesia, Thailand, Philippines, Malaysia and Vietnam)

South America (Brazil etc.)

Middle East and Africa (Egypt and GCC Countries)

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Key highlights and essential features of the report:

1) Which major players are presently listed in the report?Here are the companies that are presently listed in the report: Abbott Laboratories, Bayer HealthCare, GlaxoSmithKline, OncoGenex Pharmaceuticals, Hospira, Boehringer Ingelheim, AstraZeneca, Aveo Pharmaceuticals

**List of the firms stated above might differ in the final report dependent on a merger, name change, and other factors.

2) Can you list or add new firms as per our requirement?Yes,we can list or add new firm as per the requirement by client in the report. The final confirmation regarding the same must be provided by the research team subject to difficulty of survey.

**Availability of data will be confirmed after research in case of a privately held firm. Maximum 3 companies can be included at no additional charge.

3) Which all regional categorization are covered? Is it possible to add any specific country?Presently, our research report offers special focus and attention on the following areas:Europe, United States, Japan, China, India, Southeast Asia, and Central & South America

**Maximum one country of specific interest can be added at no extra charge. Charges will be applied for the addition of extra countries or regions.

4) Can the addition of extra Market breakdown or segmentation is possible?Yes, the addition of extra Market breakdown or segmentation is possibly dependent on the difficulty of survey and availability of data. On the other hand, detailed sharing of the requirements with our research team is a must before providing final confirmation to the client.

More Details on this Report: https://brandessenceresearch.biz/Medical-Devices-and-Consumables/Global-Precision-Cancer-Therapies-Industry-Market-Research-2019/Summary

There are 15 Chapters to display the Global Precision Cancer Therapies market

Chapter 1, Definition, Specifications and Classification of Precision Cancer Therapies , Applications of Precision Cancer Therapies , Market Segment by Regions;

Chapter 2, Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;

Chapter 3, Technical Data and Manufacturing Plants Analysis of Precision Cancer Therapies , Capacity and Commercial Production Date, Manufacturing Plants Distribution, R&D Status and Technology Source, Raw Materials Sources Analysis;

Chapter 4, Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);

Chapter 5 and 6, Regional Market Analysis that includes United States, China, Europe, Japan, Korea & Taiwan, Precision Cancer Therapies Segment Market Analysis (by Type);

Chapter 7 and 8, The Precision Cancer Therapies Segment Market Analysis (by Application) Major Manufacturers Analysis of Precision Cancer Therapies ;

Chapter 9, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;

Chapter 10, The Consumers Analysis of Global Precision Cancer Therapies ;

Chapter 11, Precision Cancer Therapies Research Findings and Conclusion, Appendix, methodology and data source;

Chapter 12, 13 and 14, Precision Cancer Therapies sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

https://www.marketwatch.com/press-release/europe-busbar-market-2019-industry-challenges-key-vendors-drivers-trends-and-forecast-to-2025-2020-03-03

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Precision Cancer Therapies Market Size, Status and Forecast 2025 Trends, Size, Drivers, Strategies, Products and - News Times