Industry
Life Sciences
Patient-centered Care Models Taking Center Stage in Life Sciences
Highlights
- Clinical research increasingly focuses more on real-world evidence, with patient-centricity at the core.
- The potential of personalized medicine—a combination of diagnostics and therapies—is becoming evident in enabling healthcare systems to shift toward a more comprehensive prototype of care, delivering better patient outcomes.
- Industry regulations will be imperative in realizing this potential. There is a need for more adaptable regulatory pathways to promote personalized treatments, and regulatory affairs will play a pivotal role in their design.
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The medical model for personalized care
The personalized care approach demands a deep understanding of the patient’s needs, medicinal preferences, and behavioral care.
While life sciences and healthcare industries focus on patient-centricity, a medical model of personalized medicine has emerged. This model uses a person’s phenotypic and genotypic characteristics such as molecular profiling, medical imaging, and lifestyle data to customize the right therapeutic strategy for the person at the right time.
This medical model promises personalized care but has challenges in meeting regulators’ expectations; there is a clear need for more adaptable regulatory pathways to promote personalized treatments. We believe the regulatory affairs (RA) teams of pharma majors can play a pivotal role in influencing the regulatory landscape design that aids faster approvals for this line of treatment.
A promise for life
Personalized medicine helps determine a person’s predisposition to a disease and facilitates timely and targeted prevention and/or treatment.
Personalized medicine offers the promise of good health, of longevity. Aside from improving the overall health of a patient, it helps make medical decisions and improves treatment accuracy. Based on learnings gathered from an individual’s genotype, phenotype, lifestyle, and environment, personalized medicine ensures access to tailored and focused care.
Personalized medicine provides rational pharmacotherapy, which means prescribing the right drug to the right patient in the right dose at the right time, thereby lowering the overall burden of medical expenses. It helps patients receive optimal treatment, lowering the risk of induced damage. Given that personalized medicine capitalizes on the developments in genetics, molecular biology, and biochemistry, its scope and sphere of influence are increasing by the day.
The future of personalized medicine
While opportunities galore, there are challenges that will need to be tackled in order to reap the benefits of personalized medicine.
Some of the most evident challenges are:
- Lack of individual health information: Currently, the market and healthcare system do not offer personalized medicine as they do not adhere to the individualized healthcare approach. In addition, responsible, safe, and optimal use of individual health information required for personalized medicine has yet to become routine in clinical settings.
- Finding the right candidates: Personalized vaccines, for example, require biomarker-negative and biomarker-positive subjects and a waiver to negative biomarkers can happen if the sponsor provides well-analyzed data and justifies excluding participants with a negative biomarker.
- Unclear regulatory pathways for in vitro diagnostic products (IVD) or companion diagnostics (CDx): The well-entrenched regulatory path applies to plentiful drugs on the market. However, the regulatory path for IVD or CDx is often unclear to many test developers and pharmaceutical sponsors. Moreover, the current IVD tests focus on immunohistochemistry (IHC) or next-generation sequencing (NGS), the regulatory path of which is complex for the NGS test. Overall, the IVD regulatory pathways are not as established as the conventional diagnostics, such as blood glucose.
- Different approval times for therapeutic products and CDx: Proper planning for therapeutic products and companion diagnostics (CDx) applications to respective authorities to expect same-time approval may avoid market delays of the personalized medicine. However, this might not be within the applicant’s control. For example, in the US FDA, the pre-market review and approval of the therapeutic drug is by Center for Drug Evaluation and Research (CDER) or Center for Biologics Evaluation and Research (CBER), and the companion diagnostic device is by Center for Devices and Radiological Health (CDRH).
The need for a more defined approach
Pharma companies rely on their regulatory affairs team for more defined regulatory approaches for patients to benefit from personalized treatments.
The expected inputs from regulatory affairs are:
- Conduct discussions with business units highlighting the patient and regulatory requirements and mapping them to the scientific and technological capability of the product manufacturing company. This will facilitate an advanced regulatory approach for personalized medicine’s quick access to the market.
- Standard clinical trial designs evaluate the efficacy of medicines in a large subject size of the generalizable patient population. In meeting the goals of personalized medicine, it would be practical to design a regulatory approach that includes smaller populations of patients with customized therapies. Doing so can overcome the problem of selecting the wrong participants in the correct trial designs.
- Recognizing that many personalized medicines require regulatory intelligence of Health Authority requirements and guidelines (for example for FDA approval - CDER, CDRH, and CBER) and knowledge of drugs and companion diagnostics, pharma companies can develop an internal, cross-knowledge working team to develop personalized medicine approvals. The team can address complex issues associated with the regulation of drug or device combinations. The regulatory affairs team develops this capability with the help of empirical data, to understand patient-defined insights and outcomes not only after approval but during development as well as submission (for regulatory review).
- Provide input for developing firm guidelines and policies with a clear path using empirical data. These guidelines and policies need to be agreed by all major regulatory agencies of the world to follow harmonized guidelines and policies.
- Strategic planning to get approvals of both drug and device at the same time for personalized medicine—which is a combination product—so that it can be launched in the market on time.
Personalized medicine can truly transform how serious diseases are diagnosed and treated. The input from the regulatory affairs teams can help develop a well-defined and established regulatory framework with clear pathways, which can speed up registration management with the health authorities and improve patients’ access to personalized medicine.
It is pertinent to note that while regulatory affairs teams help organize the regulations side of things, pharma companies must work toward adopting newest technologies to streamline operations leading to regulatory compliance and improved outcomes. Advancements in artificial intelligence, big data, blockchain, and data analytics are revolutionizing patient risk predictions, and a well-defined and established regulatory framework offers greater potential for innovations meeting tomorrow’s needs.
