Patient Reported Outcomes (PROs)

Patient reported outcome (PRO) measures are important secondary end-points in many therapeutic areas and PRO data can offer competitive advantage through labeling and promotional claims. Our PRO studies have enabled our clients and stakeholders to understand critical dimensions related to a patients’ health, functional status (physical function, mental function, social function etc), symptom burden, treatment preferences, treatment satisfaction and quality of life.

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Observational Burden of Illness Studies

When our clients’ research questions and information needs extended beyond the scope of data available in retrospective databases, we provided solutions through the design and conduct of large scale observational studies in US and Ex-US countries.

Study objectives have ranged from direct and indirect costs including profiling costs associated with over-the-counter medications, alternative treatments, therapeutic procedures, surgeries, and impaired work productivity; functional and quality of life assessments for patients with specific medical conditions or taking specific treatments; long-term effectiveness and safety of bio-pharmaceutical products and medical devices; and patient preference, compliance, persistence and satisfaction with treatments.

Our observational studies have comprised longitudinal and cross-sectional patient surveys, physician and caregiver surveys, medical chart data abstraction, and patient, physician, and caregiver focus groups. Patients in our studies were recruited through their physicians, health plans, membership of advocacy groups, or from the general population using television or print media.

We undertake all aspects of these studies including:

  • Protocol development
  • Development of data collection instruments including case report forms for completion by patients, caregivers, physicians, nurses or other healthcare professionals
  • Study site identification
  • Physician/investigator screening and recruitment
  • Managing all aspects of Institutional Review Board (IRB) submissions
  • Investigator training and study monitoring
  • Data collection and management
  • Data analysis and study report development
  • Publication and dissemination of study findings

Below are examples of some questions related to health outcomes associated with medical conditions and healthcare interventions our observational studies have been instrumental in answering for our clients:

  • What are the unmet clinical needs in a particular disease population?
  • What are the comorbidity profiles of patients with specific medical conditions?
  • What are the rates of patient satisfaction with current treatments?
  • What factors determine patient satisfaction with a particular treatment?
  • How important are “ease of use” attributes in patient and physician adoption of new treatments or technologies?
  • What is the patient preference for specific product attributes?
  • What are the reasons for patient compliance or persistence on certain therapies?
  • What is the quality of life impact of specific medical conditions or treatments?
  • How do specific medical conditions or treatments affect patient functional status?
  • What is the effect of certain diseases on patients’ ability to work? and do some treatments affect work productivity differently than others?
  • What are the commonly cited reasons for discontinuation or switching off different therapies?
  • Do some side-effects of therapies bother patients more than others?
  • How do product attributes like size and shape of an oral tablet, or sensory attributes of a nasal spray affect patient adoption of these treatments?
  • To what extent do patients use alternative treatment strategies?
Stand-alone PRO Studies

When PROs for a new treatment or a medical device were of particular significance, we have supported our clients with designing and conducting stand-alone PRO studies. Our PRO study designs were either randomized controlled trials where the primary endpoint was a PRO measure, or naturalistic studies where PROs for various treatments were evaluated under actual or ordinary conditions of use.

We undertake all aspects of these studies including:

  • Protocol development
  • Development of case report forms
  • Study site identification
  • Physician/investigator screening and recruitment
  • Managing all aspects of Institutional Review Board (IRB) submissions
  • Investigator training and study monitoring
  • Data collection and management
  • Data analysis and study report development
  • Publication and dissemination of study findings

Examples of questions our PRO studies answered include:

  • What is the effect of comparator treatments on health-related quality of life (HRQOL)?
  • Are improvements in patient functional status comparable across therapies?
  • Is treatment A better than treatment B in improving specific dimensions of HRQOL (e.g., physical well-being, emotional well-being, etc)?
  • What is the effect of comparator treatments on patient functional status?
  • Does treatment with a specific medication lead to a greater improvement in overall patient functioning compared to other treatments?
  • Is a particular treatment significantly better than competing treatments in improving certain critical dimensions of patient functioning (e.g., physical function, mental function, etc)?
  • Does treatment A lead to better symptom improvement than treatment B?
  • Are patients taking treatment A more satisfied with therapy than those taking treatment B?
  • Do patients taking a specific treatment have better outcomes in terms of work productivity and presenteeism compared with those on comparator treatments?
  • What are the rates of patient satisfaction on comparator treatments?
  • Does patient preference for specific product attributes (e.g., sensory attributes of a nasal spray, or aftertaste of an oral solution) influence treatment compliance?
Development of New PRO Measures

When appropriate PRO measures were not available, our clients have relied on our outcomes researchers to develop new PRO measures. Our methodology for development of new PRO measures is outlined below.

  • Development of draft measures based on literature, and primary research with patients, physicians, and caregivers through focus groups or study panels
  • Administration of the draft measures in a pilot study
  • Refining and revising draft measures based on statistical analyses and input from study participants
  • Re-administration of the revised measures in a second pilot study (measures might be incorporated in Phase II studies as a part of the second pilot study)
  • Finalizing the measures based on statistical analyses

We perform all of the following aspects of a PRO measure development study:

  • Screening and recruiting appropriate participants for the focus groups or panels
  • Developing screening questionnaires for recruitment
  • Moderating the focus groups or study panels
  • Summarizing and transcribing the focus groups/panel discussions
  • Development of the pilot study protocols and statistical analysis plan
  • All aspects of conducting the pilot studies including patient recruitment, administration (including instruction for study questionnaire completion) and monitoring
  • Data management and statistical analyses
Validation/Translation of Existing PRO Measures

In addition to developing new PRO measures, our outcomes researchers have also assisted our clients with various aspects of validating and/or translating existing PRO measures. The types of projects we have undertaken include:

  • Translation of existing measures into one or more languages
  • Validation of translated measures
  • Modification/adaption and/or validation of existing measures in previously untested sub-groups or languages
  • Identification of clinically meaningful cut-points for existing measures

Data collection for validation studies entailed mail or telephone surveys, in-person completion of study questionnaires in physician offices, or web-based questionnaire completion.

PRO Data Collection alongside RCTs

Our clients have often requested our services for management and analysis of PRO data collected as a part of their pivotal studies. The scope of our support has ranged from all or some aspects of the following:

  • Recommending the appropriate PRO measures
  • Determining the PRO data collection schedule
  • Development of the statistical analysis plan
  • Data analysis including instrument scoring
  • Development of study reports
  • Publication and dissemination of study findings
Strategic & Implementation Plans

Our outcomes researchers have developed many strategic and implementation plans that underscore the PROs essential for effective commercialization and adoption of bio-pharmaceutical products by patients and physicians. Our plans have also proposed studies essential to obtaining the necessary PRO data prior to product launch.

Environmental Analyses

We have undertaken numerous environmental analyses for our clients to answer a number of fundamental questions key to understanding the drug development and commercialization landscape. These analyses were conducted for a specific product or for products within and across therapeutic specialties:

  • For any particular therapeutic area, which PROs are the most valued by payers, patients, and decision makers?
  • If competitors had PRO claims, to what extent were they used in promotional materials?
  • How does PRO data for a specific product stack up against it competitors?
  • What are the strengths and weakness of competitor PRO data?
  • What are the potential opportunities and challenges for new products when it comes to PROs?
  • What label claims do competitor products have and how important are these for product approval? and subsequently market access and penetration?
  • What are the PRO value messages for competitor treatments? and how effective have these messages been with different stakeholders?
Literature Reviews, Backgrounders, Meta-analyses
  • Over the years, we have assisted our clients with numerous critical appraisals and reviews of pertinent health outcomes literature. Our reviews have been disease area-specific or product-specific and have informed the development of internal documents, background sections of study protocols and product dossiers, and supported the design of PRO studies and development of case report forms and other data collection instruments.
  • We have conducted several meta-analyses with the aim of understanding and quantifying PRO data on competitor products.
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Therapeutic Areas

  • Anti-infectives
  • CNS
  • CVS
  • Dermatology
  • Endocrine and Metabolic Disorders
  • GI Disorders
  • Oncology
  • Ophthalmology
  • Organ Transplant
  • Pain
  • Respiratory