Patient focused drug development

Patient-focused drug development is a pragmatic shift in the drug development process incorporating patients’ participation right from the first stage of drug development to the end. Patient-centered drug development now has become the talking point in the clinical research industry. It helps to provide meaningful information from participants to researchers in order to perform research more effectively and efficiently.

The FDA states that patient-focused medication development aims to be a thorough strategy of gathering patient opinions on the severity of an illness or an unmet medical need in a therapeutic area to aid the drug review process.

In other words, the analysis of the severity of the condition, therapy, and treatment status for a particular disease will be based on the patient’s perspective and provide context for regulatory decision-making. 

The main objectives of patient-focused drug development

  • Promote the use of approaches to obtain substantial and valuable patient and caregiver input to more consistently inform regulatory and drug development decision-making.
  • Ease patient recruitment and lessen the pressure on patients.
  • Become more knowledgeable about the proper use of techniques for collecting information on patient preferences.
  • To best support their decision-making, patients must determine what information about the benefits, risks, and burdens of their chosen course of treatment is necessary and how to best communicate it.

Characteristics of patient-focused or patient-centered drug development

  • Patients are regarded as co-researchers when making judgments about unmet needs, trial outcomes, and device design. trial design and execution, and the translation and distribution of information.
  • In all stages of development, developers carefully look for, comprehend, and include patients’ perceptions of value. benefit, and risk.
  • The study process is more transparent, and patients are more aware of it.
  • Patients find it more convenient and see it as a treatment choice to participate in clinical research.
  • The viewpoints of the carers are part of the development.

Role of FDA in patient input in drug development

  • The FDA is working to include patient feedback in the regulatory review process following the 2012 renewal of the Prescription Drug User Fee Act (PDUFA).
  • In recent years, the FDA has made progress toward its goal of gathering patient opinions on particular disease states and incorporating patient input into its pharmaceutical assessment process.
  • The FDA has agreed to hold 24 PFDD sessions with participating patients and patient groups.
  • FDA incorporates patients’ viewpoints on their diseases into its decision-making. 
  • In the FDA’s 2015 approval of new melanoma therapy, patient feedback on the aesthetic and psychological benefits of seeing melanoma lesions decreased and played a crucial role in the advisory committee’s choice of medication with an overall favorable benefit-risk profile for patients.

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The FDA’s framework for patient-centered drug development

FDA provides several guidance documents for patient-focused drug development and briefly let’s look at the FDA’s framework for drug development.

Preparation phase


  • A thorough understanding of the patient’s path, preferences for outcomes, and the sickness or condition from their point of view
  • Databases of patients
  • Determine an unmet need
  • Education on productive partnerships for patients, the community, or researchers
  • Evaluate the success of the current treatment or specific demographics.

Research Questions

  • Create a research question based on the interests of the patients.
  • The subject orders the research questions.
  • The patient offers input on possible indications

Pre-clinical development

  • Compile or create study materials (PROs, ClinROs, PerfOs, and ObsROs); 2. Patients indicate any potential recruiting or participation barriers.
  • Determine who, when, and how the patient will be involved.
  • Patient comments on the study’s endpoints.

Execution Phase

Clinical development

  • Kind assistance with recruitment and comments on participant experiences
  • Patients serve on the board that oversees data security.

FDA approval process.

  • Patients serve as patient advocates, participate in benefit or risk debates, and serve in advisory groups.

Communication phase

Post approval surveillance

  • Patient input on risk assessment and mitigation techniques (REMS)
  • The patient offers input on the phase four studies
  • The patient is aware of how to report unfavorable incidents.

Evidence communication

  • Under FDAMA 114, the patient must offer context for information.
  • The patient offers input on the patient counseling materials, medication guidelines, package inserts, and usage directions.

FDA Patient-Focused Drug Development Guidance

A change for a more systematic PCCD

First, we require a cultural transformation that appreciates and consistently considers patient preferences, beliefs, needs, and experiences throughout all stages of medication development.

In all stages of development, including planning, carrying out, and disseminating the evidence, patients must be seen and acknowledged as co-researchers. Trust in the research community, research process, and pharmaceutical industry will increase if the patient is at the center of all we do.

Second, we require transparent governance and regulations that support collaboration between patients and researchers throughout the development lifecycle. FDA has committed to convening more patient-focused medication development meetings with individuals and groups of patients.

  • Use of COAs in the manufacture of drugs, including PROs on labels.
  • Application of modern technology in researchers.
  • Efficient utilization of evidence from the real world.
  • Innovative, adaptable, and safe privacy and trust in

Third, for data, knowledge, and proof to flow, we need the technological infrastructure – the connectivity and the standards. We cannot transition to systematic PCDD without a linked system because our current fragmented health ecosystem makes it impossible for us to use health data, information, and evidence reliably and efficiently.

Finally, effective patient inclusion in all facets of medication development requires clear procedures and best practices. We must figure out how to take into account the various patient viewpoints.

What is the role of LHS in helping PCCD?

LHS is known as a learning health system (LHS). To improve patient care, LHS facilitates the evidence-collection and knowledge-generation process for disease‐based research and for the diagnosis, classification, or treatment of each disease subtype.

  • To improve the flow of data, information, and evidence, the LHS will be a global network of networks that connects the fragmented health ecosystem, including healthcare delivery, health research, and individual experiences in the real world.
  • Since the healthcare delivery system and the field of health research will be more interconnected, the connected LHS will enable the system to learn from each individual’s experiences and hasten learning across the board. 

Governing and policy

  • The LHC policy and governance Initiative is developing plans for running the LHS in a way that facilitates its operations, fosters and upholds stakeholder trust, and encourages continual innovation.
  • This concept will benefit all stakeholders in understanding how to access and share data, information, and evidence while ethically participating in the LHS.
  • As a result, research will participate in a more systematic PCDD since there will be trust, shared objectives, and communication guidelines.


All stages of drug development, including hypothesis creation, study planning and execution, medication and device design, and the translation and distribution of evidence, are actively and consistently included in patient-centered drug development. 

The FDA has prioritized including patient input in medication development. The FDA is aware that there are many more illness categories than we can cover with constrained meeting space and staff.

To improve the benefits of the FDA’s PFDD initiative, the FDA added a new “externally-led” meeting option. The FDA welcomes patient organizations to locate and organize patient-focused collaborations to collect public input on other disease areas using the procedure established through the FDA’s PFDD initiative as a mode of operation.

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