Definition and Introduction:
Precision Medicine is a new approach to illness treatment and prevention that combines molecular biology tools with system biology. It is opposed to the broad development of a medicinal treatment in which disease treatment and prevention methods are designed for the typical person with less consideration for individual characteristics. Diagnostic testing is frequently used in precision medicine to determine appropriate and optimal therapeutics based on a patient's genetic content or other molecular or cellular studies. Precision medicine techniques include molecular diagnostics, imaging, and analytics. It is a strategy to prevent and diagnose diseases based on an individual's genetic, environmental, and behavioural variability. It helps forecast illness vulnerability, improve disease detection and progression, personalise methods, and prescribe effective medications. It also supports in the making of related healthcare decisions, enhancing treatment quality, and decreasing inefficiencies caused by trial and error. Precision medicine is also widely used in detecting chromosomal abnormalities in the foetus and cancer, guiding therapy for chronic diseases, and predicting the risk of transferring genetic problems to offspring, as it minimises the time, cost, and failure rate of pharmaceutical clinical trials. The words "precision medicine" and "personalised medicine" have a lot of overlap. According to National Research Council, "Personalized medicine" is an older phrase with a meaning similar to "precision medicine”. However, there was concern that the term "personalised" could be misinterpreted to mean that treatments and preventions are being developed individually for each person; Precision medicine aims to figure out which treatments will work best for which patients based on genetic, environmental, and lifestyle factors. As a result, the Council prefers the phrase "precision medicine" rather than "personalised medicine." Some people, however, continue to use the terms interchangeably.
Advantage and Application
Benefits associated with precision medicine is:
- It is a new technique to safeguarding study participants' privacy and data confidentiality, particularly for patients.
- New tool for constructing, evaluating, and exchanging enormous volumes of medical data are being developed.
- Improved FDA oversight of tests, medications, and other technology to encourage innovation while also ensuring that these goods are safe and effective.
- Lead to new collaborations between scientists from various specialities, as well as representatives from patient advocacy groups, universities, pharmaceutical corporations, and others and A million people will have the opportunity to contribute to the advancement of scientific research as well.
- Doctors' capacity to use patients' genetic and other molecular information as part of ordinary medical care is expanded.
- Improved capacity to forecast which medicines will work best for certain patients, as well as a better understanding of the diseases' underlying causes.
- Improved methods for avoiding, diagnosing, and treating a variety of illnesses as well as better integration of electronic health records (EHRs) in patient care, making medical data more accessible to doctors and researchers.
Precision medicine application encompass Oncology, CNS, Immunology, Respiratory and others. Respiratory disorders are diseases that damage the organs and tissues of the lungs, making gas exchange problematic in humans. Precision medicine aids in the development of unique therapeutic limitations for the treatment of respiratory disorders. Precision medicine applications are mostly aimed at improving oncological disease therapy. Oncology is a medical speciality concerned with the prevention, diagnosis, and treatment of cancer. Precision medicine has proven to be quite effective in the treatment of cancer. As a result it see continuous funding and grants from public/private intuitions for instance, the National Institutes of Health awarded Washington University School of Medicine a USD 3.7 million grant in 2019 to develop an open-source database with the goal of boosting precision medicine and cancer genomic research. According to American Cancer Society, Precision medicine is being used to determine which tests and treatments are best for certain cancers. Doctors may be able to assist them by using precision medicine, correctly detect a specific form of cancer and choose the best treatment choices also determine who is at a high risk for cancer and take steps to prevent certain types of cancer.
According to a report from the Personalized Medicine Coalition (PMC), the FDA approved or cleared 12 personalised medicines and seven diagnostics in 2019, supporting physicians in addressing disease's underlying causes and incorporating precision medicine into clinical care. Since 2014, personalised medications have accounted for more than one out of every four drugs authorised by the FDA. This represents a huge increase from 2005, when tailored medicines made up only 5% of new therapies licenced each year. In 2019, the FDA authorised new customised medicine indications for previously approved pharmaceuticals, in addition to the newly approved personalised medicines. The approvals change who the drugs are for and give patients more individualised treatment options, according to PMC. This shift in healthcare from one-size-fits-all, trial-and-error medicine to a targeted approach based on each patient's molecular data is accelerating, as the US Food and Drug Administration approves diagnostic tools and treatments that push the boundaries of personalised medicine more frequently and quickly.
Important Drivers of Precision Medicine:
1. The Medical Emphasis Is Shifted from Reaction To Prevention
Precision Medicine introduces the potential to detect disease risk or presence via molecular markers before clinical signs and symptoms develop. As a result, it allows to focus on illness prevention and early intervention rather than reaction at later stages. Early clinical interventions can save lives in a variety of situations. Women who carry particular BRCA1 or BRCA2 gene variants, for example, have an 85 percent lifetime risk of breast cancer and a 60 percent lifetime risk of ovarian cancer. Preventive interventions such as increased mammography frequency, prophylactic surgery, and chemoprevention can all be guided by the BRCA1 and BRCA2 genetic tests.
2. Reduces Trial and Error Prescribing
Precision medicine reduces the trial and error prescribing. Up to 50% of patients do not respond well to the initial medicine prescribed for them. Different genes that code for drug-metabolizing enzymes, drug transporters, or pharmacological targets have been associated to variances in response in studies. Genetic and other forms of molecular screening allow doctors to choose the best therapy the first time, avoiding the time-consuming and costly practise of trial-and-error prescribing. Women with breast cancer have been one of the most common beneficiaries of this treatment. Overexpression of a cell surface protein termed human epidermal growth factor receptor 2 is found in about 30% of breast cancer cases (HER2). When administered in conjunction with chemotherapy, an antibody medication called Herceptin® (trastuzumab) can cut the recurrence of a tumour in half in women with this type of cancer. Patients who will benefit from Herceptin® and other HER2-targeting medications like Tykerb® are identified using HER2 molecular diagnostic testing (lapatinib). Oncotype DX® and MammaPrint®, for example, are two complicated diagnostic procedures that use genetic information to assist clinicians in determining the best course of treatment for breast cancer patients.
Similarly, Many people with SMA, Duchenne muscular dystrophy, acute hepatic porphyria, cystic fibrosis, and sickle cell disease now have therapies that target the underlying molecular pathways of diseases that previously had no therapy choices.
3. Helps Avoid Adverse Drug Reactions
Variations in genes that code for drug-metabolizing enzymes, such as cytochrome P2C9, cause many ADRs (CYp2C9). Genetic differences in this drug-metabolizing enzyme and an enzyme that activates vitamin K complicate the administration of warfarin, which is used to prevent blood clots (VKORC1). According to many studies, ADRs account for roughly 5.3 percent of all hospital admissions.
4. Increases Patient Adherence To Treatment
Noncompliance with therapy by patients has negative health consequences and raises overall health-care expenses. Patients, on the other hand, may be more willing to stick to their treatments if individualised medicines are more successful and have fewer adverse effects.
5. Reveals Additional Or Alternative Uses For Drug Candidates
When a medicine's use is restricted to genetically defined patient populations, it may demonstrate better benefits than when it is used in a broader generalised patient population. For example, after failing to show a survival advantage in a general population of patients in clinical trials, the lung cancer medicine Iressa® (gefitinib) was pulled from the market after receiving fast approval. However, the sponsoring company has employed pharmacogenetics to demonstrate benefit in around 10% of patients who test positive for epidermal growth factor mutations, and it has been approved as a first-line treatment for that patient population in the United Kingdom.
Challenges and Constraints:
There are multiple constraints including, economical, regulatory, social, and technical issues that need novel solutions. Some of these are listed and explained below.
1. Economic Feasibility:
The cost of adding a variety of new techniques into innovative trial designs, as well as the cost of producing cell and gene treatments, has an evident impact on the list price of personalised pharmaceuticals that gain clearance. This is particularly evident in the exorbitant costs of chimeric antigen receptor T-cell (CAR-T) therapies, the world's first totally tailored cancer treatments. Additionally, identifying and confirming biomarkers to guide targeted therapy is time-consuming, and analysing large amounts of data frequently necessitates the formation of new teams with specialised knowledge. Also, there are growing problems in clinical research and development can be a major constraint.
2. Regulatory Uncertainty:
Current regulations may struggle to accommodate future advances in personalised medicine since it is difficult to provide sufficient evidence of safety and efficacy. While some 'personalised' applications can be discovered as part of bigger trials that fail to reach their endpoints outside of a select patient population with specific biomarkers, many existing regulations do not accept post-hoc analysis and would necessitate an altogether new trial.
3. Other Challenges and constraints:
- Evidence-based support for precision medicine. More research is currently needed to support the adoption of precision medicine, which would result in dramatically improved outcomes.
- To increase data collection, storage, exchange, and integration with EHR, significant resources are required.
- Integrating genetic data into clinical care and research is still a work in progress, but it will be critical in many health-care systems.
- Because many physicians lack confidence in their capacity to make therapeutic choices when genetic or genomic information is involved, education is a major concern. There is a need for educational activities throughout the various sub-disciplines of medicine.
Global trends
Globally precision medicine is gaining a lot of importance, according to the Digital Health Coalition report around 89% of physicians agree science will produce more personalized medications over the next 5 years and 63% of physicians agree pursuing precision medicine for their patients is a top priority. The physicians employ precision medicine to investigate each patient's gene, cell, and biochemical makeup in order to gain a more comprehensive knowledge of their illness. This information assists in making the accurate diagnosis the first time, treating and preventing diseases more efficiently, and assisting patients from all over the world. As a result, there are multiple initiatives taken in different region for the development of Precision medicine.
Initiatives in U.S.
According to the data published by American Medical Association (AMA) there are over 30 million consumer that have taken Direct-to-consumer (DTC) genetic test with over 42thousand test per day many with actionable gene and/or FDA clearance. AMA also estimated that one in ten American have taken the test in 2020 and that 10 test are introduced every day while the total genetic test offered currently is predicted to be over 75000.
The United States' precision medicine initiatives include ongoing efforts by the Department of Veterans Affairs (VA), which enlisted over 450,000 veterans in the Million Veteran Program (MVP), a participant-driven research cohort to construct one of the world's largest precision medicine databases. Blood samples from 1 million veterans throughout the country are being sought by the VA. Moreover, the country formed an interagency working group in March 2015 with the purpose of developing the following Privacy and Trust Principles as part of its commitment to guarantee that privacy is integrated into the core of the Precision Medicine Initiative, such initiatives are predicted to boost the country market.
Furthermore, there is advancement in the research and development wherein multiple nation and private institutes are encouraged to perform study of precision medicine in U.S. for instance the Precision Medicine Initiative which is a long-term research project combining the National Institutes of Health (NIH) and a number of other research institutions. Its purpose is to figure out how a person's genetics, environment, and lifestyle affect the best ways to prevent and treat disease. As a result it has both short- and long-term objectives as a part of the Precision Medicine Initiative. The short-term objectives include advancing precision medicine in cancer research. Researchers at the National Cancer Institute (NCI) want to employ a better understanding of cancer's genetics and biology to develop new, more effective treatments for the disease's numerous forms. The Precision Medicine Initiative's long-term goals include applying precision medicine to all aspects of health and healthcare on a wide scale. To that purpose, the National Institutes of Health (NIH) has started the All of Us Research Program, which includes a group (cohort) of at least 1 million volunteers from around the country. Moreover, according to the US National Clinical Trial Registry (NCT), there were roughly 191 registered clinical studies linked to precision medicines as of June 1, 2020, which is projected to promote industry growth in the near future. Similarly, the US Food and Drug Administration (FDA), the Office of the National Coordinator for Health IT, and the Office for Civil Rights announced that they would build research and data capacities, technical tools, and policies to accelerate precision medicine, thereby boosting the country's overall market growth.
Initiatives in Europe
European countries hope to combine research and health policy to accelerate the adoption of personalised medicine with the formation of the International Consortium for Personalized Medicine, which brought together health research funders and policymaking groups.
Reflections on customised medicine began at the EU level in 2010 with a series of workshops on various research fields that could contribute to this new form of practising medicine. Several projects are presently underway in EU Member States to develop the framework for personalised medicine. In the United Kingdom, for example, the Academy of medical science held a series of workshops and conferences and issued publications on stratified medicine. A study on customised medicine was published by the German Academy of Sciences Leopoldina, and an Action Plan for Individualized Medicine was released by the German Ministry of Education and Research. AVIESAN, the French National Alliance for Life Sciences and Health, has released its Genomic Medicine 2025 strategy, which was commissioned by French Prime Minister Manuel Valls. Similarly Personalized medicine is being implemented in healthcare in a number of European countries, including Estonia, Scotland, and others.
Additionally, an initiative "Personalized Medicine 2020 and Beyond - Preparing Europe to Lead the Way" was launched by the European Commission. This programme was conceived with the goals of increasing stakeholder knowledge and empowerment, integrating information and ICT solutions, encouraging clinical research, and shaping healthcare around precision medicine in Europe. Because of its application in the treatment and diagnosis of a number of diseases in this region, including cancer, diabetes, and rare metabolic diseases, Europe has been in the forefront of encouraging investment, innovation, and research in this new field. As a result, the market for precision medicine in Europe is expected to grow exponentially in the coming years.
Initiatives in APAC
The West has paved the path for precision medicine over the last two decades. As a result, the existing databases are largely made up of data from Caucasians. APAC will need to acquire enough data on APAC genotypes before it can fully benefit from precision medicine. APAC will need to acquire enough data on APAC genotypes before it can fully benefit from precision medicine. Only then can diseases that disproportionately afflict this region be diagnosed and treated appropriately. The quality of the data acquired will be just as crucial; there must be enough variation to reliably represent the genetic features of certain subsets of the APAC population.
Precision medicine is predicted to experience a huge increase in APAC-led innovation. China has already made great progress in genomics research and technology, announcing its precision medicine project in 2016 and pledging a US$9 billion investment by 2030. Other countries in the region are also attempting to break new ground in this area. Singapore recently launched its national precision medicine plan, and it already has a number of organisations and programmes in place to promote precision medicine innovation. For example, the SingHealth Duke-NUS Institute of PRecIsion Medicine (PRISM) is creating a genome/phenome database for Asian patients, and the National University Cancer Institute Singapore's (NCIS) Integrated Molecular Analysis of Cancer Programme is matching cancer patients' genetic profiles to early-phase clinical trials of new drugs.
The bustle of industry activity currently taking place in APAC is reassuring. Precision medicine offers a way to give cost-effective care and lessen the significant healthcare burden the region is prepared to face as the population ages and chronic diseases grow more widespread. Precision medicine might have a huge economic impact compared to the current one-size-fits-all strategy, and APAC is leading the charge to make it a reality.
Which region will lead the advancement of precision medicine?
From how information is controlled (for example, GDPR) to how medical services are paid for to how novel cures are evaluated and authorised, macro issues will invariably define the trajectory of precision healthcare innovation. These elements will have an impact at least on a regional level, but more often on a national or international one. China is spending extensively and has a considerably less stringent regulatory environment than the United States, which has traditionally seen the most money, creative technology, and therapy development. Many European countries, interestingly, have healthcare models that are most aligned with the value proposition of precision medicine, and have been aggressive in building programmes around individual patient data collecting, particularly for huge, de-identified patient data sets (for example, UK Biobank). However, data privacy concerns and regulations may stifle this growth. Given the diversity of possible approaches, North America precision Medicine Market is analysed to be the largest while APAC region is predicted to accelerate quickest in the future.
Opportunity:
1. Assimilation and analytics of the data from the precision medicine use
The companies should be able to store and interpret large, divergent data sets accurately and efficiently since they operate with such large, disparate data sets. Genomic or proteomic data (large-scale studies of organic proteins), scholarly literature, clinical studies, and data from universities and laboratories, all in various formats and from numerous sources, should be assimilated and analysed.
Digital healthcare data and analytics capabilities are spreading as a result of recent technology advancements. These include (but are not limited to) individual-level data, as well as capabilities that link genetic data with other health indicators, such as medical records and even nonmedical data. Furthermore, the general computing trend towards artificial intelligence could potentially increase the value of these larger yet linked data sets.
2. Upgradation in the technology
As precision medicine requires heavy number crunching technology upgradation such as In-memory computing, that has a lot of promise for precision medicine organisations who are dealing with a lot of data will be very resourcefully. These platforms that analyses data stored in main memory rather than having to retrieve it from a standard database, will significantly boost the speed of analysis. Furthermore, in-memory technology allows for speedier data deletion. It enables researchers to fine-tune and change their algorithms in real time, improving data quality and speeding up the ability to calculate study outcomes.
3. Use of precision medicine in obesity treatment
While precision medications that are customised to patients' genetic profiles have revolutionised the treatment of many diseases, this technique has yet to be applied to illnesses such as obesity in clinical practise. Targeting the glucagon-like peptide 1 receptor (GLP1R), which regulates appetite, is currently the most used therapeutic method in obesity pharmacological management.
This mechanism is used by Novo Nordisk's Saxenda (liraglutide), which has been a top global obesity medication since 2014, and its replacement, Wegovy (semaglutide), which was introduced in the United States in June 2021. Wegovy has now been approved in the United Kingdom and Canada, and the European Medicines Agency's Committee for Medicinal Products for Human Use has recommended it for marketing authorization (CHMP). The medicine, which boasts a convenient once-weekly dosing regimen and produced unprecedented efficacy outcomes in Phase III trials, has sparked a lot of interest in the obesity area. Despite some early manufacturing issues that will result in a shortage of the drug in the first half of this year in the United States, Wegovy is expected to become a blockbuster.
Despite the fact that Novo Nordisk presently dominates the obesity business, a slew of other companies are vying for a piece of the pie. According to recent study, there are 363 investigational candidates being developed by 233 businesses in the research and development (R&D) sector. The obesity pipeline is not only large, but also diversified, with 150 different molecular targets discovered. Such efforts towards developing obesity treatment using precision medicine is predicted to create more opportunity for the market.
4. Emergence of Artificial Intelligence in precision medicine
Advances in genetic disease and precision oncology have boosted demand for predictive assays that allow patients to be selected and stratified for therapy throughout the last decade. The discovery of functionally useful biomarkers based on a single gene or protein is complicated by the huge variety of signalling and transcriptional networks governing cross talk between healthy, sick, stromal, and immunological cells.
Machine learning (ML), as well as network and systems biology, have made significant contributions to biomedical discovery and are now being seamlessly integrated into the process. Translation of patient data to therapies is a major goal of medical Artificial intelligence (AI). It is feasible to accelerate the drug development pipeline and pick structures that can be generated on automated systems and made available for biological testing by tightly combining database knowledge, AI, and lab automation, allowing for quick hypothesis testing and confirmation. Drug perturbation experiments can be used to predict the actions of drugs on seemingly unrelated biological systems using computational analysis.
ML can help with drug mechanism, establishing biomarkers, repurposing existing medications, optimising drug candidates, designing clinical trials, and even recruiting for clinical trials. Even when a chemical library was repurposed in conjunction with high-content picture screening, image-based drug fingerprints were shown to provide biological activity prediction for drug discovery. The potential uses of predictions offered by installed computer models extended far beyond the original compound screen's intended purpose.
Developments:
- FDA approves Novartis Scemblix® (asciminib), with novel mechanism of action for the treatment of chronic myeloid leukemia. Scemblix is a precision medicine used to treat adults with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitor (TKI) medicines.
- August 2021: Precision for Medicine, Clinical research organization partnered with Trialbee, a patient matching and enrollment platform to accelerate the future of precision medicine. This partnership will expand Precision for Medicine's ability to match patients who could benefit from life-saving therapies, particularly those who could be a match for trials in oncology, rare and orphan diseases, including cell and gene therapy trials, will be enhanced as a result of the partnership. Trialbee allows for accurate and quick enrollment, enhancing the patient experience and assisting in meeting patient enrolment deadlines.
- June 2021: Wegov, the first and only once-weekly GLP-1 therapy for weight management, approved in the US. Wegovy™ is precision medicine and is the first and only once-weekly glucagon-like peptide-1 (GLP-1) receptor agonist therapy approved for weight management for people living with obesity.
- Illumina, Inc. inked a national-level pilot programme agreement with the Belgian Society of Medical Oncology (BSMO) in February 2021 to evaluate Comprehensive Genomic Profiling (CGP) in advanced metastatic cancer patients.
- Diaceutics PLC established DXRX – The Diagnostic Network in October 2020, with the goal of lowering the time to peak biomarker test acceptance for cancer testing from years to months, hence accelerating the end-to-end research and commercialization of precision medicine diagnostics.
- Infosys released a brand-new Personalized Medicine solution for the pharmaceutical sector in June 2020. To derive intelligent insights, the solution uses SAP S/4HANA, SAP C/4HANA, SAP Analytics Cloud, and the SCI platform. This allows pharmaceutical businesses to satisfy important business and regulatory obligations while providing individualised experiences to patients.
- FDA approved a digital medical device development tool for customised medicine tests in 2019. The OsiriX CDE Software Module is the first biomarker test for brain damage, and it could allow inventors more quickly enrol patients in clinical trials of therapeutic medical devices intended to treat mild traumatic brain injury based on their unique characteristics.
- In 2017, 2bPrecise partnered with Mayo Clinic to include new clinical protocols using Mayo Clinic's electronic phenotyping algorithms. 2bPrecise was able to use these new methods in studies to measure outcomes in patients with hereditary cardiovascular disease because to this collaboration.
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