Patient Reported Outcomes

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Patient Reported Outcomes

Anantha Naik Nagappa and Uday Venkat Mateti

Manipal College of Pharmaceutical Sciences, Manipal University. Manipal 576104

What are Patient Reported Outcomes (PROs)?

In a clinical scenario the voice of the patients usually unheard and prominent clinical outcomes are dominating. The models of financing and submissive attitude of patients are one of the causes for poor demand for PRO. The PROs are collective views and opinions expressed by patients regarding the outcomes of treatments itself. Here the patients were served with validated questionnaires and the data is subjected to statistical methods to minimize the bias and get an opinion what is there in the patients mind.

How to measure PROs?

The PROs is measured using validated instruments which are broadly classified as generic and disease specific. The most popular way is to administer both type of instruments and evaluate to understand the opinion of the patients. The questionnaires for capturing the data on a particular aspect for example the severity of pain is asked to rate on a numerical scale. The idea here is to convert the qualitative pain to a measurable numerical scale.

What are the instruments required to measure PROs?

Due to interventional therapeutic patients are likely to have outcomes in variety of domains like physical functional, social, role physical, emotional, etc. The generic questionnaire is likely to cover in general above domains, whereas disease specific has instruments focused on brining out special domains of the disease for example in CKD the KDQOL focuses of quality of life among dialysis patients. The examples of generic and disease specific instruments were summarized in the table 1.

Table 1: Examples of Generic and Disease Specific Instruments

Generic instruments

Short Form (SF-36) health survey

Quality of Well-Being (QWB) Scale

Short Form (SF-12) health survey

Health Utilities Index (HUI)

World Health Organization quality of life Instruments (WHOQOL-100 and WHOQOL-Bref)

Euro-QoL Instrument (EQ-5D)

Disease specific instruments

The Mac-New

Kidney Disease Quality of Life Short Form (KDQOL)

HIV/AIDS-Targeted Quality of Life Instrument

Diabetes Quality of Life measure (DQOL)

Asthma Quality of Life Questionnaire (AQLQ)

European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C 30

The most commonly used PRO questionnaires assess one of the following constructs:

v Symptoms (impairments) and other aspects of well-being

v Functioning (disability)

v Health status

v General health perceptions

v Quality of life (QoL)

v Health related quality of life (HRQoL)

v Reports and Ratings of health care.

How do we evaluate and administer questionnaires?

The validated questionnaires available with various agencies can be purchased and utilized to perform PROs. The questionnaires administration involves the ethics committee approval of the protocol of the study. The protocol should contain the informed consent form which should be taken before recruiting the patients in the study. The questionnaire should be preferably in mothers tongue. So that patient can understand the questionnaire. The questionnaire translated should be subjected to forward and backward translation to ensure the errors in translation. The validity of questionnaires can be established be test and re-test. The reliability of questionnaires is ascertained by Cronbach's alpha test and Internal Consistency Reliability.

What is the necessity for performing PROs?

The outcomes of PROs are essential prima facie to establish the value of treatment perceived by the patients. In all circumstances directly or indirectly the sponsorship of treatment is by the patients. As the patients may differ in their views and may choose a particular treatment if choices are offered to them. There is a necessity of PROs results as this can be a major issue in policy framing and budget allocation for healthcare delivery.

How does the PROs play role in pharmaceutical marketing?

The pharmaceutical marketing being competitive looks for additional benefits as evidences over competing drugs. The clinical outcomes are the major predictors for decision making by care provider. The economic and PROs are important for policy decisions which a regulator or patients can influence the pharmaceutical marketing decisions along with clinical outcomes.

How to harmonize the PROs results to an international data base?

The barriers in utilizing the data across the globe arise due to geopolitical, socioeconomic and cultural divergence. So there is a need to identify these differences and formulate a scale of equity which can be extended to the global scenario. For example the pharmacoeconomic evaluations of USA cannot be extended to India. However the WHO has attempted to harmonize the QALY and other data to international scenario.

What is the regulatory status of PROs submissions?

The PROs data along with pharmacoeconomic data are becoming important due to the utility and primary evidence it makes available for rational decision making. Currently the PROs and pharmacoeconomic data are desirable in case they are derived during clinical studies and submitted along with clinical safety and efficacy data. The Pharmacovigilance data submission has become already mandatory in most of the countries as regulatory approval.

What are the utilities of PROs in patient centric health care?

The PROs are generated by patients in themselves who express their experiences of treatment. The PROs are likely to indicate the patient preferences, discomforts and their opinion regarding appropriateness of treatment. Based on this the treatment and policy of treatment can be suitably updated so that the patients are happy with the treatments.

Who should lead PRO projects?

The PRO project leader should be a person with clinical background along with management and statistical expert. The PROs projects are social experiments in which the complexities of healthcare settings make it extremely difficult to collect data. It is multidisciplinary by nature as clinical, cost and as well humanistic outcomes data's need to be collected and subjected to stastical analysis. There is a need to develop a robust model of analysis which can support analysis. For example Markov modeling and decision tree model.

What is the difference between HRQoL and QoL studies?

The Health related quality of Life is a subset of Quality of life. The quality of life includes apart from physical health, the Psychological, Social, Economic and Spiritual domains. For example HRQoL measures the quality of life pertaining to health, where as Quality of life may include other domains mentioned above.

What are the major organizations involved in promoting PROs?

The major organizations involved in promoting PROs were International Society of Pharmacoeconomics and Outcomes Research (ISPOR), International Society of Quality of Life Research (ISOQoL), Research and Development (RAND), European Quality of Life (Euro-QoL) Foundation and International Society of Pharmacoepidemiology (ISPE), and International Health Economics Association (iHEA).

References

1. U.S Department of Health and Human Services Food and Drug Administration Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. U.S. FDA, Clinical/Medical 2009. Available form: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Last cited on [20 July 2014].

2. Deshpande PR, Rajan S, Sudeepthi B L, Abdul Nazir C P. Patient-reported outcomes: A new era in clinical research. Perspect Clin Res 2011;2:137-44.

3. Chin R, Lee BY. Economics and patient reported outcomes, Principles and practice of clinical trial medicine, London, Amsterdam, Burlington, San Diego: Elsevier Inc;2008 p.145-66.

4. Development and Testing of PRO Measures. Available from: http://www.isoqol.org/about-isoqol/what-is-health-related-quality-of-life-research. Last cited on [15 July 2014].

5. Quality of life and health related quality of life - is there a difference?. Available from : http://blogs.bmj.com/ebn/2014/01/27/quality-of-life-and-health-related-quality-of-life-is-there-a-difference/. Last cited on [25 July 2014].

About the Author

Anantha Naik Nagappa's picture

I am professor, intrested in developing the community pharmacy services in India, We have an association called ASSOCIATION OF COMMUNITY PHARMACISITS OF iNDIA. CHECK AT www.acpi.in for further detials

Comments

Albert Ana-Maria's picture

What are the minor organization involved in promoting PROs? Do you believe that patients have only to gain from them?

Anantha Naik Nagappa's picture

The Major organizations emphasizing on PROs are EORTC, RAND, and WHO. The latest trend is the regulatory authority is also taking keen interest in PRO and pharmacoeoconomiv data. One should not be surprised if the regulators start demanding mandatory submission of PRO data along with clinical trial data as prerequisite for licensing and approval

Prof Anantha Naik Nagappa

My Page : http://www.pharmainfo.net/ananthanaik

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