The much-hyped phrase of the global pharma majors – ‘meeting unmet needs of patients’, is very often used to create an aura around newer patented drugs of all kinds, from original to banal, including evergreen varieties such as:
| Evergreen Drug/Brand | Medical Condition | Original Drug/Brand |
| Levocetirizine (Vozet) | Allergies | Cetirizine (Zyrtec) |
| Escitalopram (Lexapro) | Depression | Citalopram (Celexa) |
| Esomeprazole (Nexium) | Acid reflux | Omeprazole (Prilosec) |
| Desloratadine (Clarinex) | Allergies | Loratadine (Claritan) |
| Pregabalin (Lyrica) | Seizures | Gabapentin (Neurotonin) |
I do not have any terrible issue with this usage, as many stakeholders, including various governments, have already started differentiating between the ‘Chalk’ and the ‘Cheese’ kinds of patented products and contemplating future course of action, accordingly. The recent development in South Africa is one such example.
That said, there is now a greater need to ponder over the much bigger picture in the same context and direction, which would improve predictability of treatment outcomes by manifold. Simultaneously, such R&D initiatives would help reducing the overall cost, especially for dreaded diseases like cancer, mainly through highly targeted drugs and consequently avoiding the risk and associated wastage, as often happens with the prevailing ‘trial and error’ therapy approach, thereby benefitting the patients immensely. This is mainly because no drug is 100 percent effective with inconsequential side-effects for all patients of any disease type.
Genetics and Genomics Science made it possible:
With already acquired knowledge in genetics, genomics and genome sequencing capability, it is now possible to precisely predict a person’s susceptibility to various disease types and proactively working out measures to help either avoiding ailments, such as, non-infectious life threatening and chronic diseases altogether, if not, making their treatment more predictable and less expensive, as stated above.
If organized efforts are made to extend the application and benefits of this science to a larger section of population, those R&D initiatives can really be construed, unquestionably, as ‘meeting unmet needs of the patients’, just as ‘first in kind’ category of innovative drugs are recognized by the scientific community and the civil society as a whole.
A treatment revolution in the offing:
Expectations are rapidly building up that evolving genetics and genomics science based technological know-how would ultimately revolutionize the practice of medicine ushering-in the pathway of personalized medicine for a large number of patients.
Definition:
A report from the Tufts Center for the Study of Drug Development defines personalized medicine as “Tailoring of medical treatment and delivery of health care to individual characteristics of each patient, including their genetic, molecular, imaging and other personal determinants. Using this approach has the potential to speed accurate diagnosis, decrease side effects, and increase the likelihood that a medicine will work for an individual patient.”
The aim:
The aim of personalized medicine is, therefore, to make a perfect fit between the drug and the patient. It is worth noting that genotyping is currently not a part of clinically accepted routine. However, it is expected to acquire this status in the western world, shortly.
To give a very quick example, genetic differences within individuals determine how their bodies react to drugs such as Warfarin – a blood thinner taken to prevent clotting. It is of utmost importance to get the dosing right, as more of the drug will cause bleeding and less of it will not have any therapeutic effect.
In the field of cancer, genetic tests are now being done by some oncologists to determine which patients will be benefited most; say with Herceptin, in the treatment of breast cancer.
Thus, with personalized medicine the health of a patient will be managed based on personal characteristics of the individual, including height, weight, diet, age, sex etc. instead of defined “standards of care”, based on averaging response across a patient group. Pharmacogenomics tests like, sequencing of human genome will determine a patient’s likely response to drugs.
Disease prevention:
In addition, such medicines would help identifying individuals prone to serious ailments such as, metabolic, cardiac, endocrine, auto-immune, psychosomatic, including cancer of various types; enabling physicians to take appropriate preventive measures much before disease manifestations and in that process would help containing the overall treatment cost.
Cost of genome sequencing:
Sir John Bell, Professor of Medicine at Oxford University, reportedly said in early December 2012 that personalized medicine for all could soon be a clear possibility, as everybody will be able to have their entire DNA make-up mapped for as little as £100 (Rs.10, 000 approx.).
This estimate seems to be realistic, as the price of genome sequencing has fallen by 100,000-fold in 10 years. This cost is expected to further decline, as genome of any person essentially remains unchanged over time. Thus, this information might become a part of an individual’s medical record allowing the doctors to use it as necessary.
Summary of key advantages:
To summarize, the expected benefits from personalized medicine, besides very early diagnosis as stated above, are the following:
1. More Accurate Dosing: Instead of dose being decided based on age and body weight of the patients, the physicians may decide and adjust the dose of the medicines based on the genetic profiling of the patients.
2. More Targeted Drugs: It will be possible for the pharmaceutical companies to develop and market drugs for patients with specific genetic profiles. In that process, a drug needs to be tested only on those who are likely to derive benefits from it. This in turn will be able to effectively tailor clinical trials, expediting the process of market launch of these drugs.
3. Improved Healthcare: personalized medicine would enable the physicians to prescribe ‘the right dose of the right medicine the first time for everyone’ without any trial or error approach, resulting in much better overall healthcare.
Current use:
Though these are still the early days, initial usage of personalized medicine is now being reported in many areas, such as:
Genetic analysis of patients dealing with blood clots: Since 2007, the U.S. Food and Drug Administration has been recommending genotyping for all patients being assessed for therapy involving Warfarin.
Colorectal cancer: For colon cancer patients, the biomarker that predicts how a tumor will respond to certain drugs is a protein encoded by the KRAS gene, which can now be determined through a simple test.
Breast cancer: Women with breast tumors can now be effectively screened to determine which receptors their tumor cells contain.
In addition, this approach would also help clinicians to determine which particular therapy is most likely to succeed on which patient.
Present outlook:
A September 2013 article published in Forbes Magazine titled, “Personalized Medicine May Be Good For Patients But Bad For Drug Companies’ Bottom Line” says, although personalized medicine offers tremendous potential for patients, because of the dual burdens of expensive clinical trials and diminished revenue potential, the concept may become unsustainable in the long term, the attitude of regulators will be critical to drug companies’ willingness to embrace personalized medicine, and to its wider application.
In my view, for greater interest of patients to ‘meet their unmet needs’ global pharma, majors, academics, respective governments and the drug regulators should find a way out in this new direction, sooner.
Indian initiatives:
Some companies, both well known and lesser known, are making collaborative progress, keeping low profile, in the genome sequencing area in India, which will ultimately make expensive treatments, such as cancer, more predictable and simultaneously affordable to many.
The concerns:
While the progress in the field of personalized medicine is quite heartening, some experts have reportedly been sounding a note of caution. They strongly feel that DNA code sequencing brings to light a “very real privacy concerns” of individuals.
The key argument being, if genome sequencing is extended to entire population, individuals and their relatives could then be identified and tracked by matching their DNA with the genome stored in the respective health records. This move, as contemplated by the opponents, could “wipe out privacy” with a significant impact on the society.
A paper published in ‘Scientific American’ dated January 2014, titled “What Fetal Genome Screening Could Mean for Babies and Parents” deliberated that today doctors are closer than ever before to routinely glimpsing the full genetic blueprints of a fetus just months after sperm meets egg. That genomic reconstruction would reveal future disease risk and genetic traits even as early as the first trimester of pregnancy – raising another ethical issue that could hugely impact parents’ decision threshold for deciding to terminate a pregnancy or influencing how they rear their child.
Thus, all these ethical and social issues in the development and usage of personalized medicine must be appropriately addressed under a well deliberated ethical, social, legal and regulatory framework of each country.
Conclusion:
Though in Europe and to some extent in the United States, treatments based on personalized medicine have already been initiated, we are still in a nascent stage for this novel concept to get translated into reality for the benefit of a much wider population across the world.
Lot of grounds may still need to be covered, especially in the realm of medical research and also to work out the regulatory pathways for personalized medicine in healthcare by the pioneers of this great concept and more importantly by effectively addressing the ethical concerns raised on this subject.
If collaborative initiatives are taken jointly by academia, R&D based global pharma majors and medical diagnostic players towards this new direction with a clearer focus and supported by the law makers, a huge unmet needs of patients will truly be met, giving yet again a fresh impetus to the much hyped phrase “Meeting Unmet Needs of Patients”, though in a refreshingly new direction.
By: Tapan J. Ray
Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.