Precision medicine is a novel and targeted approach for disease treatment and prevention taking into account individual differences in a patient’s genes, environment, and lifestyle either at the clinical level or within drug discovery and development. It is pivotal in alleviating unnecessary suffering related to medical care, due to unintended side effects which can result from the current one-size-fits-all approach. It is moving towards patient engagement and disease management thereby promoting early diagnosis and reducing the overall cost of treatment. Thus precision medicine can rightly be considered an outcome-based approach.
Precision medicine is commonly used in cancer treatment. It is also used to detect and treat human immunodeficiency virus (HIV) and cystic fibrosis. Examples of precision medicine also include using targeted therapies to treat specific types of cancer cells, such as hormone receptor-positive/human epidermal growth factor receptor 2 negative (HR+/HER2-) breast cancer cells or using tumor marker testing to help diagnose other cancer types. For example, Novartis’ drug, Piqray targets the effect of PIK3CA mutations (most commonly mutated gene in HR+/HER2- breast cancer) and may help overcome endocrine resistance in HR+ advanced breast cancer.
Although precision medicine’s adoption can be found in several areas of diseases, its role in day-to-day healthcare remains relatively limited.
Barriers in the way of precision medicine
- Cost is a crucial issue. The cost of incorporating a host of new processes into innovative trial designs, not to mention the cost of manufacturing gene and cell therapies, evidently has an impact on the list price of personalized drugs that win approval. For example, treatments such as Gilead’s Yescarta and Novartis’s Kymriah remove T-cells from a patient’s blood, modify them to target tumor cell antigens, and then infuse them back into the bloodstream. These therapies have attained remarkable results in rare and advanced cancers, but a cost of $400,000 per patient, limits their reimbursement options among both private and public payers
- Precision medicine depends on companion diagnostics which often require testing on biomarkers and marker-negative patients, resulting in a need for larger patient pools. In general validating biomarkers to guide targeted therapies is a prolonged task
- Analyzing huge data sets often requires new teams with specialized skills in data science and bioinformatics
How can hospitals prepare for adoption of precision medicine?
- Improve data recording capabilities: For precision medicine purposes added patient information is needed, like a person’s diet, family health history, lifestyle, allergies, race, and maybe even records collected from wearable technologies. These data can be rendered useless unless the hospital electronic health records (EHRs) can support robust and coded data recording, storage, and distribution
- Standardize patient data: Most EHR systems support the Common Clinical Data Set (CCDS), a list of patient reporting standards required for getting a certification on a health technology. It is high time to essentially start using these standards routinely as they are to become industry-wide and will be a basic foundation of precision medicine data
- Integrate data access and sharing via application programming interfaces (APIs): Hospitals can deploy Fast Healthcare Interoperability Resource (FHIR) services which use RESTful APIs to different parties for the healthcare data exchange. Hospitals can set up FHIR services in the cloud using ready-made solutions or build it on top of their EHRs
- Providing genetics training to clinicians: Hospitals can start by raising awareness through the introduction of genetics into primary care
Follow through/setting standards
Novartis has been one of the torch bearers in precision medicine. As part of its commitment to ensuring eligible patients have access to Kymriah:
- The company has collaborated with the Centers for Medicare and Medicaid Services (CMS) on the creation of an appropriate value-based pricing approach
- To ensure all hospitals and their associated clinics are aware of how to manage the risks of cytokine release syndrome (CRS) and neurological toxicities, Novartis has made Kymriah available through a Risk Evaluation and Mitigation Strategy (REMS) program. The REMS program serves to inform and educate healthcare professionals about the risks that may be associated with Kymriah treatment.
- To support safe patient access, Novartis has established a network of certified treatment centers throughout the US which are fully trained on the use of Kymriah and associated appropriate patient care.
Advances in the area of precision medicine are happening faster than the ability of hospitals and health systems to keep pace. Therefore, drug companies should come up with various patient programs, value-based pricing initiatives, and resources to support safe and timely access for patients and address the full range of their needs.
#precisionmedicine #valuebasedpricing
Sources:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6909265/
https://www.fda.gov/drugs/precision-dosing-defining-need-and-approaches-deliver-individualized-drug-dosing-real-world-setting
https://www.researchgate.net/publication/338079839_Precision_dosing_to_avoid_adverse_drug_reactions
https://www.biospace.com/article/precision-medicine/