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PATIENT ADVOCACY AND CHANGING PARADIGM IN DRUG ACCESS
A294
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 1 - A 3 1 8
The fourth and final level captures complete virtual healthcare systems, which incorporate both virtual add-ons to an existing healthcare system and comprehensively virtual healthcare systems. We aim to shed light on the virtual healthcare by conceptually categorizing the innovative and diverse ways these virtual initiatives are currently being leveraged in order to more effectively generate cost-effectiveness studies in the future. Virtual healthcare aims to increase patient access and convenience, especially in rural areas with minimal specialized care, while assisting in prevention and continuity of care in disease management. PHP210 PHRMACEUTICAL DEVELOPMENT: AFRICA AN EMERGENT MARKET Mahi L , Amoura L Axelys Sante DZ, Alger, Algeria .
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With population of more than 1 Billion, Africa is the world’s second largest continent. The transition that African countries are undergoing is a real challenge for healthcare authorities. 24% of the global disease burden is accounted in Africa. The diseases that prevailed in the 60s and 70s have not completely disappeared, tuberculosis is still endemic as well as transmissible diseases like HIV/AIDS and Hepatitis still remained as a major problem while some others, such as cancers, cardiovascular and metabolic disorders are dramatically increasing, requiring the implementation of effective healthcare programs and leading to an increasing demand for drugs treating. In 2012, African Pharmaceutical market revenue was USD 18 Billion and expected to reach USD 30 Billion by 2016. Established countries in both Sub-Saharan and North African countries (South Africa, Nigeria, Cote d’Ivoire, Senegal, Egypt, Morocco, Algeria and Tunisia) are contributing # 80% of the pharma market in Africa. Despite maintaining regional offices within Africa, many major Pharma and device manufacturers frequently overlook the continent when sponsoring clinical studies. Cultural barriers, political upheaval and uneven infrastructure are certainly causes for the lack of interest. But Africa offers tremendous expertise and opportunity for drug and device companies looking for cost-effective study sites and appropriate patient drug market populations. Currently more than 45% of the whole continent’s clinical trials are being conducted in South Africa and hence the need for the next generation clinical trial destination for a drug and device manufacturers. These companies can also consider technology transfer by partnering with local drug manufacturers and research centers to diversify their business portfolio. PHP211 ESTIMATION OF HEALTH BENEFITS FROM SUCCESSFUL PREVENTION OF AN ILLNESS: METHODS AND EXAMPLES Wang J 1, Hung M 2, Hwang J 3 Cheng Kung University College of Medicine, Tainan, Taiwan, 2Division of Cancer Prevention and Control, US Centers for Disease Control and Prevention, Chamblee, GA, USA, 3Acadmia Sinica, Taipei, Taiwan .
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1National
In classical epidemiology, people usually quantify and compared mortality rates of different diseases for priority decision of prevention and control. After the second half of 20th century, most illnesses have become chronic and one must also consider quality of life (QOL) for patient-centered decision. We propose to quantify potential health benefits of successful prevention of an illness by estimating the loss of quality-adjusted life expectancy (loss-of-QALE) and the expected years of life lost (EYLL) plus expected years of living with disability (EYLD) that could be saved. Lung cancer and stroke were used as empirical examples. We derive theoretically that the loss-of-QALE would be close to EYLL plus EYLD, if disability is the major determinant for QOL and the QOL utility for general population is close to one throughout all ages. The discrepancies of these two estimates were larger for patients with stroke than those with lung cancer, because the former’s life expectancy and co-morbidities would be higher than the latter. While loss-of-QALE can be directly compared with benefits of clinical services, EYLL plus EYLD that could be saved sheds light on the associated productivity losses and the financial demands of long-term care. Both are useful in health policy decision for prevention but in different contexts.
Objectives: To identify evidence-based practices related to health and clinical psychology for being included in clinical practice guidelines. Methods: A systematic review of literature including review papers in 7 databases, published between 2010 and 2015, incorporating recommendations for evidence-based psychological intervention as part of the comprehensive health care. Results: 147 scientific articles were found, 16 of which met the inclusion criteria. Psychological interventions most commonly recommended are related to treatment compliance or adherence, and coping to any health condition and control of symptoms of anxiety or depression associated with diseases. All recommendations had moderate or weak level of evidence. Conclusions: The psychological constructs are often difficult to measure and intervene, especially when associated with chronic or infectious disease. Evidence-based recommendations limited to address three main topics: anxiety/ depression, adherence, acceptance and coping. Studies of efficacy or effectiveness of psychological interventions are not frequently found. Other topics of great importance as quality of life, social support, disease-related emotional impact or health education are less common or are not considered as sources of psychological evaluation and intervention. This indicates that it is necessary to deepen studies provide strong evidence for effectiveness of health psychology interventions as part of comprehensive care models and clinical practice guidelines. PHP214 OUTCOMES-BASED ARRANGEMENTS IN VALUE-BASED CARE: AN ENGAGEMENT FRAMEWORK Nazareth T 1, Yu T 1, Brunken C 1, Arcona S 1, Sasane R 1, Petrilla A 2, Velez E 2, Hughes K E 2 1Novartis Pharmaceuticals, East Hanover, NJ, USA, 2Avalere Health LLC, Washington, DC, USA .
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Background: Ensuring access to quality treatment options which meet the diverse needs of patients is paramount. As such, interest in agreements linking outcomes with payment is growing across stakeholder groups. Currently, available literature focuses primarily on the outlook, trends and challenges; here, key considerations for engagement are shared. Objective: Outcomes-based agreements may be implemented between diverse healthcare stakeholders, e.g. health plan and manufacturer, in order to increase alignment on value, accountability, and risk mitigation regarding real-world effectiveness of a treatment. Thoughtful agreements, executed well, may enhance mutual stakeholder benefit for partners, providers and patients. Description: Performance is measured in a defined cohort over a finite period of time using a key outcome; this outcome, indicating effectiveness, may be observed via reductions in clinical events or healthcare resource utilization or may be inferred by use of an established proxy (e.g. therapeutic compliance). The key outcome should resonate with both partners (e.g. tie to quality / performance measures). Value-based payments are dependent on measured performance vs. contractual metrics. Recommendations: Shared objectives, clear incentives, availability and quality of required data, and committed resources are important factors in securing interest and appropriate partners. The following elements are also critical to address: outcomes-based benefit design (standalone / supplemental to formulary access), applicable patient segment, analytic resources and support, timing, format and utilization of results, associated communications, and anticipated touchpoints. A comprehensive and pragmatic analytic roadmap guiding the outcome evaluation should be in place, detailing data elements and metrics involved in reporting. Ongoing dialogue, collaboration and transparency to ensure mutual understanding, efficiencies, and successful progression are integral. Conclusions: These elements are essential to increase success. The relationship that is often built simultaneously provides an opportunity to strengthen foundational understanding and ties between diverse stakeholders in the direction of common healthcare goals and additional innovative solutions. PHP215 A PROPOSED RUBRIC FOR PATIENT ENGAGEMENT IN DRUG DEVELOPMENT: WHAT CONSTITUTES “SOUND” ENGAGEMENT? Perfetto E M 1, Oehrlein E M 2, M-CERSI PFDD Planning Committee U M 2 of Maryland, School of Pharmacy, Baltimore, MD, USA, 2University of Maryland School of Pharmacy, Baltimore, MD, USA .
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PHP212 PATIENT ADVOCACY AND CHANGING PARADIGM IN DRUG ACCESS
1University
Dang A , Likhar N , Vsn M , Mothe R K , Sawant A , Esam H MarksMan Healthcare Solutions LLP, Health Economics and Outcomes Research (HEOR) and RWE (Real World Evidence), Navi Mumbai, India
Objectives: A movement to include the patient voice in health care research and decision-making is underway. All stakeholders agree that patient engagement activities should be meaningful and not token actions, however no official guidance has been issued on what constitutes meaningful engagement in drug development. The objective of this project was to develop a rubric for assessing the quality of patient engagement in drug development to help assess when meaningful engagement takes place. Methods: Key opinion leaders representing patient advocacy organizations, the biopharmaceutical industry, regulatory bodies, payers, and academia discussed characteristics of meaningful engagement via regularly scheduled teleconferences over a 6-month period in 2015. The committee crafted the rubric through an iterative process during teleconferences and as a product of one-day conference on patient engagement. Results: The rubric characterizes the patient role, continuity, depth and temporality, and the diversity of populations engaged. “Patients as Partners” explores how patients, caregivers, and other relevant people (e.g., persons at risk for the disease) are recognized as partners; “Continuous Patient Engagement” examines patient engagement throughout the product lifecycle continuum; “Meaningful Patient Engagement,” emphasizes thoughtful dialogue and evidence their input is used; “The Right Patients are Engaged,” includes assessment of whether the population is generalizable to the population of interest; and “The Right Time to Engage” examines whether engagement is appropriate given the natural history of disease. Conclusion: The experiences of patients can be heterogeneous and an individual patient’s perspective may differ from that of other patients, and may change with time as personal circumstances and his or her state of disease or condition changes. A rubric for assessing the soundness of patient engagement has been developed. The rubric may be implemented in various settings by patients and those who wish to engage patients to ensure that meaningful interactions are taking place.
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.
.
.
.
In the process of drug development and drug approval, the pharmaceutical industry is challenged with increasing cost constraints, a growing need for real-world evidence and heavier regulatory scrutiny. Patient advocacy groups can be a valuable partner to the pharmaceutical industry in speeding up drug access. Patient advocates possess practical rather than purely scientific knowledge that is valuable for both drug development and drug approval. It was recognized quite early that the goals of patient advocacy groups are aligned with those of the pharmaceutical industries. This understanding resulted in supporting patient involvement across the entire drug development continuum, from discovery and U.S. FDA approval to market by legal and regulatory requirements. Support received by the patient advocacy groups include fundraising for R&D; organizing patients to expedite clinical trials; lobbying government and payer organizations to create public awareness and building support for research funding, patient advocacy group also back research for “orphan” diseases where there is a desperate need for new therapies. The recent success of Vertex and Sprout Pharmaceutical highlights the power of the patient voice in healthcare decision-making especially for orphan diseases. Working with patient groups can thus lead to a win-win situation for both the FDA and pharmaceutical industry PHP213 IMPROVING THE CLINICAL PRACTICE GUIDELINES: WHY INCLUDE PSYCHOLOGICAL EVALUATION AND INTERVENTION? Díaz-Sotelo O D Universidad El Bosque, Bogotá D.C., Colombia .
.
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 1 - A 3 1 8
The fourth and final level captures complete virtual healthcare systems, which incorporate both virtual add-ons to an existing healthcare system and comprehensively virtual healthcare systems. We aim to shed light on the virtual healthcare by conceptually categorizing the innovative and diverse ways these virtual initiatives are currently being leveraged in order to more effectively generate cost-effectiveness studies in the future. Virtual healthcare aims to increase patient access and convenience, especially in rural areas with minimal specialized care, while assisting in prevention and continuity of care in disease management. PHP210 PHRMACEUTICAL DEVELOPMENT: AFRICA AN EMERGENT MARKET Mahi L , Amoura L Axelys Sante DZ, Alger, Algeria .
.
With population of more than 1 Billion, Africa is the world’s second largest continent. The transition that African countries are undergoing is a real challenge for healthcare authorities. 24% of the global disease burden is accounted in Africa. The diseases that prevailed in the 60s and 70s have not completely disappeared, tuberculosis is still endemic as well as transmissible diseases like HIV/AIDS and Hepatitis still remained as a major problem while some others, such as cancers, cardiovascular and metabolic disorders are dramatically increasing, requiring the implementation of effective healthcare programs and leading to an increasing demand for drugs treating. In 2012, African Pharmaceutical market revenue was USD 18 Billion and expected to reach USD 30 Billion by 2016. Established countries in both Sub-Saharan and North African countries (South Africa, Nigeria, Cote d’Ivoire, Senegal, Egypt, Morocco, Algeria and Tunisia) are contributing # 80% of the pharma market in Africa. Despite maintaining regional offices within Africa, many major Pharma and device manufacturers frequently overlook the continent when sponsoring clinical studies. Cultural barriers, political upheaval and uneven infrastructure are certainly causes for the lack of interest. But Africa offers tremendous expertise and opportunity for drug and device companies looking for cost-effective study sites and appropriate patient drug market populations. Currently more than 45% of the whole continent’s clinical trials are being conducted in South Africa and hence the need for the next generation clinical trial destination for a drug and device manufacturers. These companies can also consider technology transfer by partnering with local drug manufacturers and research centers to diversify their business portfolio. PHP211 ESTIMATION OF HEALTH BENEFITS FROM SUCCESSFUL PREVENTION OF AN ILLNESS: METHODS AND EXAMPLES Wang J 1, Hung M 2, Hwang J 3 Cheng Kung University College of Medicine, Tainan, Taiwan, 2Division of Cancer Prevention and Control, US Centers for Disease Control and Prevention, Chamblee, GA, USA, 3Acadmia Sinica, Taipei, Taiwan .
.
.
1National
In classical epidemiology, people usually quantify and compared mortality rates of different diseases for priority decision of prevention and control. After the second half of 20th century, most illnesses have become chronic and one must also consider quality of life (QOL) for patient-centered decision. We propose to quantify potential health benefits of successful prevention of an illness by estimating the loss of quality-adjusted life expectancy (loss-of-QALE) and the expected years of life lost (EYLL) plus expected years of living with disability (EYLD) that could be saved. Lung cancer and stroke were used as empirical examples. We derive theoretically that the loss-of-QALE would be close to EYLL plus EYLD, if disability is the major determinant for QOL and the QOL utility for general population is close to one throughout all ages. The discrepancies of these two estimates were larger for patients with stroke than those with lung cancer, because the former’s life expectancy and co-morbidities would be higher than the latter. While loss-of-QALE can be directly compared with benefits of clinical services, EYLL plus EYLD that could be saved sheds light on the associated productivity losses and the financial demands of long-term care. Both are useful in health policy decision for prevention but in different contexts.
Objectives: To identify evidence-based practices related to health and clinical psychology for being included in clinical practice guidelines. Methods: A systematic review of literature including review papers in 7 databases, published between 2010 and 2015, incorporating recommendations for evidence-based psychological intervention as part of the comprehensive health care. Results: 147 scientific articles were found, 16 of which met the inclusion criteria. Psychological interventions most commonly recommended are related to treatment compliance or adherence, and coping to any health condition and control of symptoms of anxiety or depression associated with diseases. All recommendations had moderate or weak level of evidence. Conclusions: The psychological constructs are often difficult to measure and intervene, especially when associated with chronic or infectious disease. Evidence-based recommendations limited to address three main topics: anxiety/ depression, adherence, acceptance and coping. Studies of efficacy or effectiveness of psychological interventions are not frequently found. Other topics of great importance as quality of life, social support, disease-related emotional impact or health education are less common or are not considered as sources of psychological evaluation and intervention. This indicates that it is necessary to deepen studies provide strong evidence for effectiveness of health psychology interventions as part of comprehensive care models and clinical practice guidelines. PHP214 OUTCOMES-BASED ARRANGEMENTS IN VALUE-BASED CARE: AN ENGAGEMENT FRAMEWORK Nazareth T 1, Yu T 1, Brunken C 1, Arcona S 1, Sasane R 1, Petrilla A 2, Velez E 2, Hughes K E 2 1Novartis Pharmaceuticals, East Hanover, NJ, USA, 2Avalere Health LLC, Washington, DC, USA .
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.
.
.
.
.
.
.
Background: Ensuring access to quality treatment options which meet the diverse needs of patients is paramount. As such, interest in agreements linking outcomes with payment is growing across stakeholder groups. Currently, available literature focuses primarily on the outlook, trends and challenges; here, key considerations for engagement are shared. Objective: Outcomes-based agreements may be implemented between diverse healthcare stakeholders, e.g. health plan and manufacturer, in order to increase alignment on value, accountability, and risk mitigation regarding real-world effectiveness of a treatment. Thoughtful agreements, executed well, may enhance mutual stakeholder benefit for partners, providers and patients. Description: Performance is measured in a defined cohort over a finite period of time using a key outcome; this outcome, indicating effectiveness, may be observed via reductions in clinical events or healthcare resource utilization or may be inferred by use of an established proxy (e.g. therapeutic compliance). The key outcome should resonate with both partners (e.g. tie to quality / performance measures). Value-based payments are dependent on measured performance vs. contractual metrics. Recommendations: Shared objectives, clear incentives, availability and quality of required data, and committed resources are important factors in securing interest and appropriate partners. The following elements are also critical to address: outcomes-based benefit design (standalone / supplemental to formulary access), applicable patient segment, analytic resources and support, timing, format and utilization of results, associated communications, and anticipated touchpoints. A comprehensive and pragmatic analytic roadmap guiding the outcome evaluation should be in place, detailing data elements and metrics involved in reporting. Ongoing dialogue, collaboration and transparency to ensure mutual understanding, efficiencies, and successful progression are integral. Conclusions: These elements are essential to increase success. The relationship that is often built simultaneously provides an opportunity to strengthen foundational understanding and ties between diverse stakeholders in the direction of common healthcare goals and additional innovative solutions. PHP215 A PROPOSED RUBRIC FOR PATIENT ENGAGEMENT IN DRUG DEVELOPMENT: WHAT CONSTITUTES “SOUND” ENGAGEMENT? Perfetto E M 1, Oehrlein E M 2, M-CERSI PFDD Planning Committee U M 2 of Maryland, School of Pharmacy, Baltimore, MD, USA, 2University of Maryland School of Pharmacy, Baltimore, MD, USA .
.
.
.
.
.
PHP212 PATIENT ADVOCACY AND CHANGING PARADIGM IN DRUG ACCESS
1University
Dang A , Likhar N , Vsn M , Mothe R K , Sawant A , Esam H MarksMan Healthcare Solutions LLP, Health Economics and Outcomes Research (HEOR) and RWE (Real World Evidence), Navi Mumbai, India
Objectives: A movement to include the patient voice in health care research and decision-making is underway. All stakeholders agree that patient engagement activities should be meaningful and not token actions, however no official guidance has been issued on what constitutes meaningful engagement in drug development. The objective of this project was to develop a rubric for assessing the quality of patient engagement in drug development to help assess when meaningful engagement takes place. Methods: Key opinion leaders representing patient advocacy organizations, the biopharmaceutical industry, regulatory bodies, payers, and academia discussed characteristics of meaningful engagement via regularly scheduled teleconferences over a 6-month period in 2015. The committee crafted the rubric through an iterative process during teleconferences and as a product of one-day conference on patient engagement. Results: The rubric characterizes the patient role, continuity, depth and temporality, and the diversity of populations engaged. “Patients as Partners” explores how patients, caregivers, and other relevant people (e.g., persons at risk for the disease) are recognized as partners; “Continuous Patient Engagement” examines patient engagement throughout the product lifecycle continuum; “Meaningful Patient Engagement,” emphasizes thoughtful dialogue and evidence their input is used; “The Right Patients are Engaged,” includes assessment of whether the population is generalizable to the population of interest; and “The Right Time to Engage” examines whether engagement is appropriate given the natural history of disease. Conclusion: The experiences of patients can be heterogeneous and an individual patient’s perspective may differ from that of other patients, and may change with time as personal circumstances and his or her state of disease or condition changes. A rubric for assessing the soundness of patient engagement has been developed. The rubric may be implemented in various settings by patients and those who wish to engage patients to ensure that meaningful interactions are taking place.
.
.
.
.
.
.
.
In the process of drug development and drug approval, the pharmaceutical industry is challenged with increasing cost constraints, a growing need for real-world evidence and heavier regulatory scrutiny. Patient advocacy groups can be a valuable partner to the pharmaceutical industry in speeding up drug access. Patient advocates possess practical rather than purely scientific knowledge that is valuable for both drug development and drug approval. It was recognized quite early that the goals of patient advocacy groups are aligned with those of the pharmaceutical industries. This understanding resulted in supporting patient involvement across the entire drug development continuum, from discovery and U.S. FDA approval to market by legal and regulatory requirements. Support received by the patient advocacy groups include fundraising for R&D; organizing patients to expedite clinical trials; lobbying government and payer organizations to create public awareness and building support for research funding, patient advocacy group also back research for “orphan” diseases where there is a desperate need for new therapies. The recent success of Vertex and Sprout Pharmaceutical highlights the power of the patient voice in healthcare decision-making especially for orphan diseases. Working with patient groups can thus lead to a win-win situation for both the FDA and pharmaceutical industry PHP213 IMPROVING THE CLINICAL PRACTICE GUIDELINES: WHY INCLUDE PSYCHOLOGICAL EVALUATION AND INTERVENTION? Díaz-Sotelo O D Universidad El Bosque, Bogotá D.C., Colombia .
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