Em Oehrlein

Patient-Focused Drug Development: A New Direction for Collaboration.

Context:Patient-Focused Drug Development (PFDD) is a new initiative from the Food and Drug Administration (FDA) intended to bring patient perspectives into an earlier stage of product development. The goal is that patients will be able to provide context for benefit-risk assessments and input to review divisions, and also aid in the development of new assessment tools, study endpoints, and risk communications. This paper provides a summary on what is known to date about FDA’s PFDD initiative and describes implications for patients, researchers, payers, and the biopharmaceutical industry. It also provides a roadmap for stakeholders to consider in defining their role in and in shaping PFDD’s direction, and for expanding PFDD principles to conditions beyond the current 20 under FDA consideration. Methods:A search was conducted of the peer-reviewed and gray literature using PubMed and Google. This included laws, FDA guidance documents, the peer-reviewed literature, and FDA presentations for content relevant to the search term “patient-focused drug development.” Findings:Currently, FDA activities within PFDD are limited to gaining patient insights through 20 disease-specific meetings. However, many stakeholders see the initiative much more generally as representing a broad shift toward patient centeredness in biopharmaceutical product development. Conclusions:Depending upon the trajectory taken and whether or not all PFDD aims are eventually addressed, the initiative has the potential to change product development in fundamental ways. Further research should explore how patient input on disease manifestation and treatment options is best ascertained from patients and documented before initiating and during drug development.

Value to Whom? The Patient Voice in the Value Discussion

BACKGROUND Professional societies and other organizations have recently taken a visible role trying to define treatment value via value frameworks and assessments, providing payer or provider recommendations, and potentially impacting patient access. Patient perspectives routinely differ from those of other stakeholders. Yet, it is not always apparent that patients were engaged in value framework development or assessment. OBJECTIVES To describe the development and content of the National Health Councils (NHCs) Rubric, a tool that includes criteria for evaluation of value frameworks specifically with regard to patient-centeredness and meaningful patient engagement. METHODS The NHC held a multistakeholder, invitational roundtable in Washington, DC, in 2016. Participants reviewed existing patient-engagement rubrics, discussed experiences with value frameworks, debated and thematically grouped hallmark patient-centeredness characteristics, and developed illustrative examples of the characteristics. These materials were organized into the rubric, and subsequently vetted via multistakeholder peer review. RESULTS The resulting rubric describes six domains of patient-centered value frameworks: partnership, transparency, inclusiveness, diversity, outcomes, and data sources. Each domain includes specific examples illustrating how patient engagement and patient-centeredness can be operationalized in value framework processes. CONCLUSIONS The NHC multistakeholder roundtables recommendations are captured in the NHCs Rubric to assess value framework and model patient-centeredness and patient engagement. The Rubric is a tool that will be refined over time on the basis of feedback from patient, patient group, framework developer, and other stakeholder-use experiences.

Increasing uptake of comparative effectiveness and patient-centered outcomes research among stakeholders: Insights from conference discussion

The goal of comparative effectiveness research (CER) and patient-centered outcomes research (PCOR) is to improve health outcomes by providing stakeholders with evidence directly relevant to decision making. In January 2017, the Pharmaceutical Research and Manufacturers Association Foundation, alongside the Academy for Managed Care Pharmacy, organized a conference aimed at engaging experts and opinion leaders representing clinicians, patients and payers to identify and discuss barriers and strategies to enhancing uptake and use of CER/PCOR. This report summarizes the conference discussion in the following sections: preconference survey; summary of barriers and strategies to the uptake of CER/PCOR identified by conference attendees; and future perspectives on the field.

National Trends in ADHD Diagnosis and Treatment: Comparison of Youth and Adult Office-Based Visits

OBJECTIVES The study objective was to assess national trends in the diagnosis of attention-deficit hyperactivity disorder (ADHD) in outpatient visits by comparing adults and youths. Also examined were recent stimulant prescribing patterns for ADHD visits by youths and adults. METHODS Databases from the 1999-2010 National Ambulatory Medical Care Survey and National Hospital Ambulatory Medical Care Survey were used in this cross-sectional study to analyze outpatient visit data of youths (ages two to 17 years; unweighted N=112,404) and adults (ages 18-64; unweighted N=426,209). The 12-year trends in ADHD visits were assessed as a proportion of youth and adult visits. The interaction of time period and age group was added to multivariable and weighted logistic regression models to assess whether trends in ADHD diagnosis differed by age group. RESULTS As a percentage of total visits, those involving an ADHD diagnosis were more common among youths than adults. However, from 1999 through 2010, the percentage of total visits involving a diagnosis of ADHD increased proportionally more among adult visits (from .3%, unweighted N=363 of 132,065, to .7%, unweighted N=1,015 of 154,764; adjusted odds ratio [AOR]=2.7, 95% confidence interval [CI]=2.1-3.7) than among youth visits (from 3.9%, unweighted N=2,033 of 36,263, to 5.2%, unweighted N=2,609 of 37,906; AOR=1.3, CI=1.1-1.6; p<.001). ADHD visits by adults compared with those by youths represented significantly greater proportions of females, Caucasians, patients with private insurance, and visits with a psychiatrist. Stimulant prescribing was common in ADHD visits regardless of age group (>70%). CONCLUSIONS As a percentage of total office-based visits, those at which ADHD was diagnosed increased more among adults than among youths from 1999 to 2010. Further research is warranted on the appropriateness, benefit-risk, and policy implications of stimulant use among adults with ADHD.

Patient-Reported Outcome Measures in the Food and Drug Administration Pilot Compendium: Meeting Today’s Standards for Patient Engagement in Development?

BACKGROUND In 2016, the Food and Drug Administration (FDA) released a Pilot Clinical Outcome Assessment Compendium (COA Compendium) intended to foster patient-focused drug development (PFDD). However, it is unclear whether patient perspectives were solicited during development or validation of the included patient-reported outcome (PRO) measures. OBJECTIVE To examine the pedigree of a sample of measures included in the COA Compendium. METHODS PROs included in chapters 1 or 2 of the COA Compendium were extracted and three reviewers independently searched PubMed and Google to identify information on measure pedigree. Data on method and stage of measure development where patient engagement took place were documented. RESULTS Among the 26 evaluated PRO measures, we were unable to identify information on development or validation on nearly half the sample (n = 12). Among the remaining 14 measures, 5 did not include any evidence of patient engagement; 2 engaged patients during concept elicitation only; 1 engaged patients during psychometric validation only; and 6 engaged patients during both concept elicitation and cognitive interviewing. Measures either previously qualified or submitted for qualification were more likely to include patient engagement. CONCLUSIONS For the FDA Pilot COA Compendium to fulfill its purpose of fostering PFDD, it needs fine-tuning to reflect todays standards, improving transparency and facilitating clear identification of included measures so that the level of patient engagement, among other factors, can be properly assessed. Suggested improvements include identifying clinical trials that correspond to the COA Compendiums use in drug development; more clearly identifying which measure is referred to; and including only those measures that already qualified or undergoing qualification.