Economics Of Personalized Medicine

These days, you will read various articles going round and round about how personalized medicine could possibly prove to be a boon for medical sciences in terms of diagnosis and treatment. While on the other hand you will find many expressing their disappointment about how it hasn’t lived up to its promise pop up.

But a more interesting question is why exactly is personalized medicine such a big challenge?

The reasons might seem obvious — genes are really very complex and it’s hard to get enough people to study any particular target. A disease in which personalized therapy has been touted, probably results from a combination of many genetic mutations and it very difficult to deduce which one to blame.

Once we get to know the mechanics of personalized medicine, various questions tend to arise in our minds, like – will it work for us? If yes, then how? But the most important of them all is whether it really is worth our time and money?

First, let us throw some light on some of the many reasons why personalized medicine seems so tricky;

  • It is very hard to know which mutations are actually causing the disease and which are passive abnormalities that just happen to be present with no connection to the disease at all. For example, most patients with advanced cancer have p53 mutations. Right now, there is no drug that targets p53, and there’s no saying that taking aim at p53 will be useful.
  • Responses to the targeted therapy are often short-lived and it’s not easy to move these into meaningful extensions for healthy survival.
  • When you target one abnormality or pathway, cells might develop another means of growth, a process also known as treatment resistance.
  • Gene sequencing costs about $1,000 per patient, but handling and storage could increase the cost up to $10,000.

 

Now having stated the above issues, one cannot simply ignore the need to find better ways for patients to take care of themselves and better ways for physicians to help them do this. Today, most of the doctors continue to practice traditional trial-and-error medicine. In contrast, personalized medicine uses much more refined diagnostic testing to identify the exact cause of disease. Then, to select the best treatment and determine the right dosage, doctors who use the personalized medicine approach take into account the patient’s unique physiology, if applicable, of the tumor, virus, or bacteria; and the patient’s ability to metabolize particular drugs.

What really is hindering the transition from trial-and-error medicine to personalized are the following issues-

  • The pharmaceutical industries follow their blockbuster model, which focuses on developing and marketing drugs for as broad a patient population as possible and it discourages the development of therapies that aim at smaller subpopulations and the diagnostic tests that can identify them.
  • A regulatory environment that causes too many resources to be dedicated to phase-three clinical trials and too few to monitoring and assessment after the U.S. Food and Drug Administration has approved a drug.
  • The dysfunctional payment system, which pays physicians for completing procedures and prescribing drugs rather than for early diagnosis and prevention.
  • Physician behavior that is deeply rooted in trial-and-error medicine.

 

We need to consider and implement some straight forward and some complex solutions to overcome the aforementioned barriers, in a prudent manner.

Transforming pharmaceutical giants:

Big pharmaceutical companies can take three steps to speed the introduction of personalized medicine,

  • Abandon the blockbuster business model,
  • Forge alliances with diagnostic companies, and
  • Step up efforts to communicate the safety and efficacy advantages of targeted therapies.

 In long terms, the targeted drug business model would increase sales and profits for several reasons:

  • Once a highly effective therapy for a disease is available, more of the affected patients visit their physicians, who will then be aware of and willing to provide the treatment.
  • If a pharmaceutical company can demonstrate that its drug lowers the overall cost of treating a subpopulation with a disease, private and government insurers will be willing to pay for the diagnostic test and to pay a higher price for the drug treatment.
  • Since clinical trials now consume more than half the money spent on drug development, focusing clinical trials on targeted subpopulations would decrease their size, duration, and cost.

Given the trends, large pharmaceutical companies have little choice but to change. Those that stick with the blockbuster model face a frustrating future of declining sales and profits.

Role of FDA:

  • The FDA should motivate pharmaceutical companies to develop diagnostics and targeted drugs together.
  • The agency needs to implement practical regulations that continue to encourage industry innovation but maintain high standards of quality. 

Paying for benefit:

The cost of diagnostic tests might be high initially, but that pales in comparison with the potential benefits that tag along with personalized medical care.

When you have an expensive drug, rather than giving it to everybody, the act of individualizing that therapy will actually reduce the overall cost. If we increase the cost but have better outcomes, people are more likely to accept the change.

Changing physicians’ habits:

To get physicians accustomed to personalized medicine, medical schools must focus on genomics, diagnostic testing, and targeted therapies. This change alone will play a critical role in moving personalized medicine into mainstream practice.

The slow progress of personalized medicine in the past years could be discouraging, but it’s not very surprising given how complex of the health care system actually is. Given the higher stakes involved in personalized medicine—people’s lives and the viability of health care systems—it would be unreasonable to expect the widespread adoption of personalized medicine to happen swiftly.

References-

http://theincidentaleconomist.com/wordpress/personalized-medicine-will-it-bend-the-cost-curve-down-or-up/

http://www.researchandmarkets.com/reports/39077/personalized_medicine_scientific_and_commercial

http://www.wired.com/2013/02/personalized-medicine/

http://www.forbes.com/forbes/welcome/

The Many Reasons Why Personalized Medicine Is So Tricky

http://www.cnbc.com/2015/12/04/personalized-medicine-better-results-but-at-what-cost.html

https://hbr.org/2007/10/realizing-the-promise-of-personalized-medicine

Understanding Personalized Medicine: Drug Development and Usage

Every day, millions of people are taking medications that will not help them. The drugs currently being prescribed are optimally beneficial to as few as 4% of the population consuming them. The realization that physicians need to take individual variability into account is driving huge interest in ‘precision’ medicine or Personalized medicine. In this time of technological advancements and unprecedented scientific breakthroughs, personalized health care has the ability to look at a patient on an individual basis so as to detect the onset of disease at its earliest stages, and at the same time increase the efficiency of the health care system by improving quality, accessibility, and affordability.

Every person has a unique genome and personalized medicine relies on technologies that confirm a patient’s biology at the molecular level with DNARNA, or protein, which ultimately leads to the diagnosis of the disease. Having an individual’s genomic information can be significantly useful for developing drugs. These days, it’s common for physicians to use a trial and error strategy until they find the treatment that is most effective for their patient, whereas with personalized medicine, we can:

  • Specifically, formulate a treatment for an individual and have insight into how their body will respond to the drug.
  • Use detailed information of the person  genotype to decide the treatment prescriptions, which will be more cost-effective and accurate.
  • Shift the emphasis in medicine from reaction to prevention.
  • Direct the selection of optimal therapy and reduce trial-and-error prescribing.
  • Help avoid adverse drug reactions.
  • Improve the quality of life.
  • Reveal additional or alternative uses for medicines and drug candidates.
  • The decrease in the overall cost of health care due to small and fast trials.

Studies that focus on a single person are known as N-of-1 trials, where enough genomic data of an individual is collected. This data provides the blueprint for the production of various proteins in the body that may have an important role in drug development for one of the several reasons, including the following:

  • The protein plays a role in breaking down the drug.
  • It helps with the absorption or transportation of the drug.
  • The protein that is the actual target of the drug.
  • It has some role in a series of molecular events triggered by the drug.

 

When researchers compare the genomes of individuals taking the same drug, they may discover that a set of people who share a certain genetic variation also share a common treatment response, such as:

  • A greater risk of side effects
  • Severe side effects at relatively low doses
  • The need for a higher dose to achieve a therapeutic effect
  • No benefit from the treatment
  • A greater or more likely benefit from the treatment
  • The optimal duration of treatment

In N-of-1 trials, the appropriate crossover designs, in which different interventions are administered to the same person alternately (possibly with ‘wash-out’ periods in between to allow the drugs’ effects to wear off) would also enable experimenters to compare the effect of different drugs in the same person.

Well-designed N-of-1 trials could be useful in the early stages of clinical drug development and for studies investigating the safety and appropriate dosages of drugs. Currently, phase I and II clinical trials usually involve giving different amounts of an FDA-approved drug to a small group of healthy volunteers.

FDA’s (U.S. Food and Drug Administration) role is to ensure the accuracy of genetic tests, many of which are acquired from the next generation sequencing (NGS), that poses novel regulatory issues for FDA. Recognizing these challenges, FDA is working out an optimum regulatory platform, by issuing discussion papers and holding workshops that will encourage innovation while ensuring accuracy. In addition, FDA has created precisionFDA, a community research and development portal that allows testing, piloting, and validating existing and new bioinformatics approaches to NGS process.

There are still various barriers to bringing N-of-1 trials mainstream, such as:

  • Regulatory agencies, researchers, and physicians are wary of moving away from classical clinical trials.
  • Pharmaceutical companies tend to focus on drugs that are likely to be used by thousands or millions of people.
  • Tailoring treatments to patients is costly as there is a lot of work to be done on biomarkers, monitoring devices, study designs and data analysis methods.

The fact remains still that these well-designed trials could save the millions of dollars that are spent on inappropriate interventions, the management and treatment of persistent or recurring diseases, and on conventional phase III trials. And the best part is that the researchers, as well as doctors, are interested in exposing people’s unique characteristics at the molecular level to deduce better alternatives to the already existing treatment procedures.  Also, cheap and efficient devices that collect health data are becoming available along with the increasing support of the governments and life-sciences funding bodies worldwide. All we require is a team effort by innovators, entrepreneurs, regulators, payers, and policymakers to overcome the barriers and move personalized medicine forward.

References-

http://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/pmc_the_case_for_personalized_medicine.pdf

 

https://en.wikipedia.org/wiki/Personalized_medicine

 

http://www.fda.gov/ScienceResearch/SpecialTopics/PrecisionMedicine/default.htm

 

http://www.mayoclinic.org/healthy-lifestyle/consumer-health/in-depth/personalized-medicine/art-20044300

 

http://www.nature.com/news/personalized-medicine-time-for-one-person-trials-1.17411

 

Personalized vs. Mainstream Medicine.

Have you ever noticed how different people react differently to any drug?

How a person A may be fit as a fiddle upon popping a single pill but person B goes through the whole bottle and still be the same? What about person C, who might just be allergic and for whom the pill would do more bad than good? On average, any given prescription drug on the market nowadays only works for half of those who take it.

The underlying concept behind this different responsiveness to drugs is a combination of each person’s genetic make-up and the influence of environmental factors.

Conventional healthcare is based on the evidence-based practice of diagnosing and treating diseases. The drugs and treatments thus devised are tested on broad populations and prescribed using statistical averages. While these may work perfectly on some, others aren’t that lucky and for some, they may even prove fatal. In fact, there have been more than a million deaths due to ‘adverse drug reactions’ in the last 10 years!

Advancements in technology and drug discovery have led to the inception of a previously-deemed-science-fiction concept- Customized Health Care using personalized medicine. Yes, you read that right, customized treatment plans designed for every individual. Though still far from being commonly practiced, the roots of this new practice have begun to take hold.

Personalized medicine uses predictive tools to evaluate health risks and to design personalized health plans to help patients mitigate risks, prevent disease and to treat it with precision when it occurs. Techniques such as genome sequencing can reveal mutations in DNA that influence diseases ranging from cystic fibrosis to cancer.  This emerging science has the potential to truly customize healthcare to the patient, enabling providers to match drugs to patients based on their genetic profiles, to identify which health conditions an individual is susceptible to, and to determine how a given patient will respond to a particular therapy.

Although there have been quite a few encouraging signs of change, not many health systems apart from a few pioneers have yet embraced this practice. This concept seems like a boon for all those who are included in the minority percentage of people dealing with adverse effects of traditional medicine.

To see how it compares to our trusty old mainstream medicine, let’s take a look at the pros and cons of each:

Mainstream Medicine

Pros-

  •  Pharmaceutical Medicines go through strict trials to become licensed. It takes years of research and bundles of paperwork AND laboratory testing sessions to finally launch a new drug, did you know?
  • The physicians prescribing these medicines are highly skilled, trained professionals. Might as well trust that they know what they’re doing!
  • Both the Medical Profession and the Pharmaceutical Industry have very strict guidelines to abide by.  Any doctor not adhering to these guidelines is struck off and unable to practice again.  Pharmaceutical Companies are subject to massive fines if found to be in breach.
  • Doctors have access to highly accurate diagnostic equipment nowadays and they are trained to recognize and diagnose disease. Medicine is not a stagnant science, you know! We’ve come a long way from the days of Hippocrates.

Cons-

  • One of the most obvious problems- side effects. Some patients get stuck in a cycle of taking more Drugs to deal with the unwanted effects of the drugs they were originally prescribed.
  • Mainstream medicine focuses on dealing with the symptoms of the disease. Rather than curing the problem, most of the time it just suppresses it. This can lead to a lifetime need for drug therapy.
  • You ever notice how Doctors earn well? Yes, medical treatment is expensive, and without access to health insurance, can be out of the reach of many.  Even in countries where there is free access to health care, certain drugs or treatments may not be available, due to the local health care services being unable to pay for them.
  • Being different poses a problem. Dosing and regimen are standardized, which is ok for the majority of patients, but not for those who fall outside the norm. The treatment doesn’t work as well for them or they then have tolerability problems.

Personalized Medicine

Pros-

  • It can be used to predict a person’s risk for a particular disease, based on the analysis of their genome. The physician can thus initiate preventative treatment before the disease even presents itself in the patient. For example, if it is found that a DNA mutation increases a person’s risk of developing Type 2 Diabetes, the person can begin lifestyle changes that will lessen their chances of developing the disease later in life.
  • The detailed account of genetic ‘intel’ from the individual will help prevent adverse reactions or unfortunate events, allow for appropriate dosages, and create maximum efficacy with drug prescriptions.
  • Having information about an individual’s genetic makeup can be a major asset in deciding if a patient can be chosen for inclusion or exclusion in the final stages of a clinical trial. Such selective testing will prevent any adverse outcomes in patients. Not only will this allow for smaller and faster trials, it will lead to lower costs- a win-win situation!

Cons-

  • The validity of genomic tests, given the complexity of gene expression, would be surrounded by uncertainty, no matter how advanced the technology used, which is a major concern. No test is 100% accurate!
  • There is no guarantee against possible mishandling of private genomic information by providers and discrimination based on genomic information (by, for example, insurance companies, private companies, and the healthcare system).
  • Genetic tests can only provide limited information about a condition; they cannot determine if or when a person will show symptoms of a disease, how severe the symptoms will be, or whether the disorder will progress over time.

Both mainstream and personalized medicines have their own positive and negative aspects. Instead of being narrow-minded and choosing one over the other, it would be wiser to explore all the options available to you, the more choices you have the better, right? Since none is without its own flaws, it rests with the patient to decide which course they want to give a try. And why choose one? Just take what each offers and try to integrate them together! It’s not much of a risk, it would definitely be worth it…. but do consult with your physician first, don’t just take our word for it!

References-

http://alternativemedicineanswers.com/articles/Oct_Dec_08/WesternVsAlternativeMedicine.html

The Pros and Cons of Modern Medicine

 

http://www.pwc.com/us/en/view/issue-13/what-it-means-for-patient-centered-healthcare-and-how-to-address-its-current-challenges.html

https://en.wikipedia.org/wiki/Personalized_medicine

 

https://www.jax.org/genetics-and-healthcare/personalized-medicine/benefits-of-personalized-medicine

 

http://medical-dictionary.thefreedictionary.com/mainstream+medicine

http://www.acpm.org/?GeneticTestgClinRef#Limitations

https://www.personalizedmedicinebulletin.com/2013/10/07/clinical-adoption-of-personalized-medicine/

Personalized Medicine In India

Personalized medicine in India is a young but rapidly advancing field of healthcare that is informed by each person’s unique clinical, genetic, genomic, and environmental information. It is about making the treatment as individualized as the disease. It involves identifying genetic, genomic, and clinical information that allows accurate predictions to be made about a person’s susceptibility of developing the disease, the course of the disease, and its response to treatment.

Specific advantages that personalized medicine may offer patients and clinicians include:

 Ability to make more informed medical decisions

 Higher probability of desired outcomes thanks to better-targeted therapies

 Reduced probability of negative side effects

 Focus on prevention and prediction of disease rather than reaction to it

 Earlier disease intervention than has been possible in the past

 Reduced healthcare costs

Advances in Genomics Technology Have Set the Stage for a Revolution in Personal Genomics that will ultimately work towards preventive healthcare, disease burden reduction and Personalized Medicine in India. Positive hqdefaultBioscience has partnered with Medanta – The Medicity to launch India’s first personal genomics clinic, which offers state-of-the-art facilities and services in preventive healthcare and personalized medicine within the country. This partnership seeks to offer comprehensive personal genomics services to the customers along with advice from leading experts associated with the Medanta – The Medicity under one roof.

Advances in genomics now enable scientists to examine the DNA of each-and-every individual and then predict if the person would be susceptible to a particular disease or whether a particular medicine is suitable for that person in case he falls sick. This is where pharmacogenomics comes into play. Companies have started investing in pharmacogenomics as it offers several advantages such as the elimination of the unpredictable nature of drug development, bring new products to the market and the company could also benefit up to $200-to-$500 million for each drug.

main-thumb-t-265506-200-aduztyxvjxylkhxrkxzmaztfmjqdxhyxA start-up company called Xcode Lifesciences has come up with the InDNA technology to provide solutions to lifestyle-related diseases such as coronary, diabetes and obesity. A person has to order the test online after which a saliva kit will be shipped to the customer. This saliva kit is completely non-invasive and safe. DNA extracted from the saliva will then be used to determine the allelic information of the individual using high-throughput genotyping techniques.

sgdsdgssfgsdfgdNutraGene launched the country’s first commercial genetic test for type 2 diabetes. NutraGene’s Type 2 Diabetes Genetic Scan is a DNA testing service that screens DNA variations that have been widely replicated as risk factors for type 2 diabetes. It is based on a buccal (cheek) swab sample and the methodology of targeted mutation screening (genotyping).

AvesthagenAvesthagen is another major player focusing on pharmacogenomics field. One of the major projects of the company is the AVESTAGENOME Project. It is a system biology-based study of the Parsi population to determine the genetic basis of longevity and age-related disorders. This study aims to develop a model for pharmacogenomics-based therapies, development of biomarkers for predictive diagnostics and drug discovery and to enable the archiving of the genome of the community. Avesthagen started this Rs125 crore project in 2007.

Despite the optimism expressed regarding the impact that this field might have on the health of people, many barriers need to be crossed. Researchers, diagnostic firms and regulatory authorities need to establish methodologies by which to judge their effectiveness. Application of genomic and personalized medicine in India needs a change in the regulatory system.