Personalized Medicine (also referred as Precision Medicine) is a tailored approach of deciding and predicting treatment practices based on the molecular analysis of patient’s proteins, metabolites and genetic makeup. This specific treatment approach can be considered as an extension of traditional approaches to understanding and treating disease.
In 2015, Personalized Approach of treatment came in to limelight when Former United States President Mr. Obama announced the Precision Medicine Initiative during his State of the Union address. The Former US president has also depicted the prodigious benefits of this approach in enabling the physicians to better able to tailor medical treatments to individual patient requirement. The approach focuses on the unique attributes of each patient – his environment, lifestyle, and, most notably, his genetic characteristics and thereby allowing targeted treatment therapies to be deployed which are more likely to be efficacious, less likely to lead to elicit adverse side effects, and, in many circumstances, can be more cost-effective for both the patient and society at large than current approaches to many different illnesses.
In 2017, US FDA has grabbed the attention of scientific and investor’s community, by granting approval to more than 17 precision medicine therapies. Here are some prominent highlights of US FDA approvals based on personalized medicine last year –
- Merck’s Keytruda has set a glaring example by receiving an expanded approval for all solid tumor types in advanced cancers with microsatellite instability-high or mismatch repair deficiency. Also, this incident has marked as the first time in oncology, when a drug approval was based on a biomarker, regardless of where the tumor is being located in the body.
- Keytruda approval based on personalized approach has paved the way of further FDA approval of other noteworthy targeted drugs in Oncology such as Zejula, Nerlynx, Idhifa, Verzenio, Bavencio, Kisqali, Imfinzi, Rydapt and Alunbrig.
- Ingrezza, Austedo, Brineura, Vosevi, Mepsevii and Hemlibra were the other striking approvals in the specialty therapeutics area.
- Trastuzumab-dkst became the first personalized biosimilar approved for the treatment of HER2-positive breast cancer.
Howbeit, these granted approvals were not only demarcated to targeted and specialty therapies as, there were 3 notable gene therapies which managed to win US FDA approval last year-
- Kymriah became the first and foremost approved personalized CAR-T gene therapy. However, this gene therapy grabbed many eyeballs due to its “half million” dollar price tag, and imposed as a paradox of cost efficient treatments, despite of being the first approved gene therapy in US.
- Other remarkable noteworthy personalized gene therapies which has received FDA approval last year, include Kite Therapeutics Yeskarta and Luxturna by Spark Therapeutics.
At present, several leading Pharmaceuticals giants have pulled up their socks and started working on personalized medicine. A few of them has put on an large amount of investment, such as-
- Novartis Molecular Diagnostics, a unit of Novartis Pharma is working to develop innovative companion diagnostics and other tests to inform physician decision-making regarding patient care and treatment.
- Merck is working on the companion diagnostics and The Precision Medicine Initiative. The company has also developed a companion diagnostics guide that is applied across the company to its all drug discovery projects. As stated by Merck, it is committed to advance the personalized medicine through the continual development of targeted therapies, innovative research and through ensuring the availability and accessibility of its therapies to patients who need them
- AstraZeneca is also working on the personalized medicine approach and aims to develop innovative, targeted medicines for every patient who needs them
- Roche is idiosyncratically positioned to make personalized medicine a reality.
The potential competitors list goes on with a focus to achieve a maximum market share in respective therapy areas with a forefront in Oncology, ease to gain therapy approval, to better design the clinical trials and to achieve maximum effectiveness of an intervention during the trial.
Although, few personalized-medicine tests have achieved high levels of clinical adoption in the regular traditional treatment practice approaches but, as like the drawbacks of everything, personalized medicine has its shortfalls too. These shortfalls ranges from scientific challenges regarding the lack of good understanding of molecular mechanisms or a lack of molecular markers associated with some diseases to poorly lined up incentives and operational and financial issues. However, these Operational and financial issues can often be largely redressed within a particular stakeholder group and financial grants by respective health authorities.