In This Week’s Issue:
1. PIPC Chairman's Corner: Accelerating Cures: A Patient-Centered Approach to Innovation, click here to view the blog post.
2. PIPC Chairman Coelho Comments on Evidence-Based Medicine and Patient-Centeredness, click here to view the full response.
3. Inside Health Policy: Rare Disease Group Releases First Patient-Initiated Drug Development Guide, click here to view the article (subscription only).
4. PCORI Blog: What’s Happening in Merit Review, click here to view the blog post.
5. PCORI Blog: National Cancer Institute Designates PCORI As An Approved Funding Organization, click here to view the blog post.
6. The Pink Sheet: ASCO Method For Valuing Cancer Drugs To Reflect Biopharma, Patient Input, click here to view the article (subscription required).
7. KevinMD.com: Don't Mistake Patient Satisfaction for Patient-Centeredness, click here to view the blog post.
8. Inside Health Policy: Comparative Effectiveness Research Group Takes On Hep C Rx Sovaldi, click here to view the article (subscription required).
1. PIPC Chairman's Corner: Accelerating Cures: A Patient-Centered Approach to Innovation, click here to view the blog post.
2. PIPC Chairman Coelho Comments on Evidence-Based Medicine and Patient-Centeredness, click here to view the full response.
3. Inside Health Policy: Rare Disease Group Releases First Patient-Initiated Drug Development Guide, click here to view the article (subscription only).
4. PCORI Blog: What’s Happening in Merit Review, click here to view the blog post.
5. PCORI Blog: National Cancer Institute Designates PCORI As An Approved Funding Organization, click here to view the blog post.
6. The Pink Sheet: ASCO Method For Valuing Cancer Drugs To Reflect Biopharma, Patient Input, click here to view the article (subscription required).
7. KevinMD.com: Don't Mistake Patient Satisfaction for Patient-Centeredness, click here to view the blog post.
8. Inside Health Policy: Comparative Effectiveness Research Group Takes On Hep C Rx Sovaldi, click here to view the article (subscription required).
1. PIPC Chairman's Corner: Accelerating Cures: A Patient-Centered Approach to Innovation
Last week, PIPC Chairman Tony Coelho discussed PIPC’s comments to the House Energy and Commerce Committee regarding their 21st Century Cures Initiative. “We believe that to effectively support innovation, the Committee must consider both regulatory reforms to accelerate innovation and focus on supporting a delivery system that can accommodate and provide patient access to these advances. Innovation and access are equally important to patients. Therefore, PIPC made several explicit recommendations to the committee… Meeting the principles of patient-centeredness will require a new way of approaching health care policy. A patient-centered health system will value patient engagement, and will applaud patients that are empowered and active in their own health.” Click here to view the blog post.
2. PIPC Chairman Coelho Comments on Evidence-Based Medicine and Patient-Centeredness
In a recent BMJ article on evidence-based medicine, the authors warn that an over-dependence on clinical evidence could inappropriately drive clinical decisions according to a rule rather than encouraging providers to respond to the needs of the patient. “Inflexible rules and technology driven prompts may produce care that is management driven rather than patient centered,” the authors comment. “Evidence based guidelines often map poorly to complex multimorbidity.” Responding to the article, PIPC Chairman Tony Coelho agrees that evidence-based medicine should inform individual patients and their providers about the range of treatment options, while ensuring that care is patient- and not management-driven.
Chairman Coelho comments, "We agree that, particularly as health care continues to move to models in which providers are at risk for hitting financial targets and relies on evidence-based protocols and pathways to meet those targets, keeping EBM centered on patient needs and values is more challenging than ever. In this environment, the patient voice is going to be more important than ever. We recognize and seek to champion the importance of the patient voice, not only in research but in clinical practice. Evidence-based medicine should inform individual patients and their providers about the range of treatment options and their impact on people with similar characteristics and preferences. Access to those treatment options that are best suited for the individual patient must be built into any payment or delivery system that calls itself patient-centered. More data is one part of the solution to achieve patient-centeredness. Engaging and empowering patients in the process is a core component to ensure that a) data is capturing patient needs, outcomes and preferences b) that research findings are being implemented through shared decision-making tools that are similarly patient-centered, and c) care is patient-driven and not ‘management driven.’” Click here to view the full response.
3. Inside Health Policy: Rare Disease Group Releases First Patient-Initiated Drug Development Guide
According to an article published last week in Inside Health Policy, “Parent Project Muscular Dystrophy submitted to FDA Wednesday (June 25) the first patient-developed guidance outlining what the agency and sponsors can do to accelerate the development of new treatments for Duchenne muscular dystrophy patients, a move viewed by the group as a model for others seeking to accelerate development of orphan drugs. The group and FDA have been working together to develop a risk-benefit framework for the condition and FDA is expected to issue a separate guidance responding to the group's recommendations next year… PPMD President Pat Furlong called the guidance a major milestone that incorporates the patient voice in ‘well-documented and quantifiable ways.’ … The PPMD guidance recommends patients, caregivers and others complete surveys on meaningful benefit-risk tolerance and acceptable trade-offs to inform FDA's risk-benefit analysis… PPMD further suggests sponsors validate existing patient-reported outcome measures or develop new measures.” Click here to view the article (subscription only).
4. PCORI Blog: What’s Happening in Merit Review
Last week, Tsahai Tafari and Kirstin Margosian discussed PCORI’s grant review process on The PCORI Blog. “For each funding cycle, reviewers do a preliminary assessment of the applications, then we bring all the reviewers together for in-person discussion. The patient, other stakeholder, and scientific reviewers meet in a plenary session before breaking into the panels that evaluate and rank proposals received in response to different funding announcements. We began our Winter 2014 session with a new activity—a moment of recognition—to highlight the importance of patient-centeredness, patient engagement, and the inclusion of patients and other stakeholders in our review process. Two of our veteran reviewers—a patient and the daughter of a patient—each spoke for a few minutes describing their experiences with health care and telling the packed ballroom why they’re participating in PCORI.” Click hereto view the blog post.
5. PCORI Blog: National Cancer Institute Designates PCORI As An Approved Funding Organization
Writing for The PCORI Blog, Tsahai Tafari, Victoria Szydlowski, and Lori Frank, announced that “the National Cancer Institute (NCI) has officially designated PCORI as an approved peer review and funding organization. In its evaluation, NCI assessed our merit review process, including conflict of interest protections, to ensure that it conforms to National Institutes of Health standards. Our designation as a NCI-approved funding organization enables award recipients to include PCORI research awards as part of their research-base calculations to earn or maintain Cancer Center designations, access shared Cancer Center Support Grant resources, and have opportunities to obtain additional federal funding for cancer research. To apply to become an NCI-designated Cancer Center, an institution must have at least $10 million in annual direct costs of approved peer-reviewed, cancer-related funding. There are currently 68 NCI-designated Cancer Centers across the United States.” Click here to view the blog post.
6. The Pink Sheet: ASCO Method For Valuing Cancer Drugs To Reflect Biopharma, Patient Input
Cathy Kelly of The Pink Sheet reported last week about a new plan from leading oncologists’ organization to evaluate the treatment value of cancer drugs. “The American Society for Clinical Oncology plans to release a proposed methodology for valuing cancer drugs later in 2014 to gather comments from stakeholders, including the biopharmaceutical industry and patient groups, that will help inform a final version.[...][ASCO Value in Cancer Care Task Force Chairman Lowell Schnipper] explained the methodology is intended to help determine a value ‘score’ for cancer drugs, which could help physicians and patients become better aware of how the cost of a treatment relates to its benefits. The methodology will take into account clinical benefit, toxicity, quality-of-life measures and cost.” Click here to view the article (subscription required).
7. KevinMD.com: Don't Mistake Patient Satisfaction for Patient-Centeredness
Last week, physician Stanley Frencher commented in the blog KevinMD.com that perhaps it is more important to improve a patient’s health than to give them a Magic Kingdom-like experience. “Now, is there a need for customer service to be a part of good, high quality patient care? Absolutely. Are the goals of patient-centeredness important? Yes. But our preeminent job as clinicians and as a health care system is to make people well, not just happy. A recent study found that, in fact, patient satisfaction scores are not linked to higher quality care. Moreover, this analysis showed that while higher patient satisfaction led to less ED use, it also led to higher costs, more inpatient admissions, and higher rates of mortality.” Click here to view the blog post.
8. Comparative Effectiveness Research Group Takes On Hep C Rx Sovaldi
In an article published last week in Inside Health Policy, reporter John Wilkerson highlights how the development of a cure for Hepatitis C has pivoted to discussions that could limit access to the drug. “Some believe Sovaldi is a prime candidate for comparative effectiveness research, but the drug industry typically opposes comparing the value of drugs and was instrumental in preventing government-funded research from considering the cost of treatments… Gilead is selling far more of Sovaldi than anticipated in part because it is estimated to cure Hepatitis C in more than 90 percent of people who are infected… Most of the attention to Sovaldi has been on the cost, and less attention has been on the amount and quality of clinical and economic evidence available on the drug… Obamacare set up the Patient Centered Outcomes Research Institute to do comparative effectiveness research, but PCORI is prohibited from considering cost.” Click here to view the article (subscription required).
Last week, PIPC Chairman Tony Coelho discussed PIPC’s comments to the House Energy and Commerce Committee regarding their 21st Century Cures Initiative. “We believe that to effectively support innovation, the Committee must consider both regulatory reforms to accelerate innovation and focus on supporting a delivery system that can accommodate and provide patient access to these advances. Innovation and access are equally important to patients. Therefore, PIPC made several explicit recommendations to the committee… Meeting the principles of patient-centeredness will require a new way of approaching health care policy. A patient-centered health system will value patient engagement, and will applaud patients that are empowered and active in their own health.” Click here to view the blog post.
2. PIPC Chairman Coelho Comments on Evidence-Based Medicine and Patient-Centeredness
In a recent BMJ article on evidence-based medicine, the authors warn that an over-dependence on clinical evidence could inappropriately drive clinical decisions according to a rule rather than encouraging providers to respond to the needs of the patient. “Inflexible rules and technology driven prompts may produce care that is management driven rather than patient centered,” the authors comment. “Evidence based guidelines often map poorly to complex multimorbidity.” Responding to the article, PIPC Chairman Tony Coelho agrees that evidence-based medicine should inform individual patients and their providers about the range of treatment options, while ensuring that care is patient- and not management-driven.
Chairman Coelho comments, "We agree that, particularly as health care continues to move to models in which providers are at risk for hitting financial targets and relies on evidence-based protocols and pathways to meet those targets, keeping EBM centered on patient needs and values is more challenging than ever. In this environment, the patient voice is going to be more important than ever. We recognize and seek to champion the importance of the patient voice, not only in research but in clinical practice. Evidence-based medicine should inform individual patients and their providers about the range of treatment options and their impact on people with similar characteristics and preferences. Access to those treatment options that are best suited for the individual patient must be built into any payment or delivery system that calls itself patient-centered. More data is one part of the solution to achieve patient-centeredness. Engaging and empowering patients in the process is a core component to ensure that a) data is capturing patient needs, outcomes and preferences b) that research findings are being implemented through shared decision-making tools that are similarly patient-centered, and c) care is patient-driven and not ‘management driven.’” Click here to view the full response.
3. Inside Health Policy: Rare Disease Group Releases First Patient-Initiated Drug Development Guide
According to an article published last week in Inside Health Policy, “Parent Project Muscular Dystrophy submitted to FDA Wednesday (June 25) the first patient-developed guidance outlining what the agency and sponsors can do to accelerate the development of new treatments for Duchenne muscular dystrophy patients, a move viewed by the group as a model for others seeking to accelerate development of orphan drugs. The group and FDA have been working together to develop a risk-benefit framework for the condition and FDA is expected to issue a separate guidance responding to the group's recommendations next year… PPMD President Pat Furlong called the guidance a major milestone that incorporates the patient voice in ‘well-documented and quantifiable ways.’ … The PPMD guidance recommends patients, caregivers and others complete surveys on meaningful benefit-risk tolerance and acceptable trade-offs to inform FDA's risk-benefit analysis… PPMD further suggests sponsors validate existing patient-reported outcome measures or develop new measures.” Click here to view the article (subscription only).
4. PCORI Blog: What’s Happening in Merit Review
Last week, Tsahai Tafari and Kirstin Margosian discussed PCORI’s grant review process on The PCORI Blog. “For each funding cycle, reviewers do a preliminary assessment of the applications, then we bring all the reviewers together for in-person discussion. The patient, other stakeholder, and scientific reviewers meet in a plenary session before breaking into the panels that evaluate and rank proposals received in response to different funding announcements. We began our Winter 2014 session with a new activity—a moment of recognition—to highlight the importance of patient-centeredness, patient engagement, and the inclusion of patients and other stakeholders in our review process. Two of our veteran reviewers—a patient and the daughter of a patient—each spoke for a few minutes describing their experiences with health care and telling the packed ballroom why they’re participating in PCORI.” Click hereto view the blog post.
5. PCORI Blog: National Cancer Institute Designates PCORI As An Approved Funding Organization
Writing for The PCORI Blog, Tsahai Tafari, Victoria Szydlowski, and Lori Frank, announced that “the National Cancer Institute (NCI) has officially designated PCORI as an approved peer review and funding organization. In its evaluation, NCI assessed our merit review process, including conflict of interest protections, to ensure that it conforms to National Institutes of Health standards. Our designation as a NCI-approved funding organization enables award recipients to include PCORI research awards as part of their research-base calculations to earn or maintain Cancer Center designations, access shared Cancer Center Support Grant resources, and have opportunities to obtain additional federal funding for cancer research. To apply to become an NCI-designated Cancer Center, an institution must have at least $10 million in annual direct costs of approved peer-reviewed, cancer-related funding. There are currently 68 NCI-designated Cancer Centers across the United States.” Click here to view the blog post.
6. The Pink Sheet: ASCO Method For Valuing Cancer Drugs To Reflect Biopharma, Patient Input
Cathy Kelly of The Pink Sheet reported last week about a new plan from leading oncologists’ organization to evaluate the treatment value of cancer drugs. “The American Society for Clinical Oncology plans to release a proposed methodology for valuing cancer drugs later in 2014 to gather comments from stakeholders, including the biopharmaceutical industry and patient groups, that will help inform a final version.[...][ASCO Value in Cancer Care Task Force Chairman Lowell Schnipper] explained the methodology is intended to help determine a value ‘score’ for cancer drugs, which could help physicians and patients become better aware of how the cost of a treatment relates to its benefits. The methodology will take into account clinical benefit, toxicity, quality-of-life measures and cost.” Click here to view the article (subscription required).
7. KevinMD.com: Don't Mistake Patient Satisfaction for Patient-Centeredness
Last week, physician Stanley Frencher commented in the blog KevinMD.com that perhaps it is more important to improve a patient’s health than to give them a Magic Kingdom-like experience. “Now, is there a need for customer service to be a part of good, high quality patient care? Absolutely. Are the goals of patient-centeredness important? Yes. But our preeminent job as clinicians and as a health care system is to make people well, not just happy. A recent study found that, in fact, patient satisfaction scores are not linked to higher quality care. Moreover, this analysis showed that while higher patient satisfaction led to less ED use, it also led to higher costs, more inpatient admissions, and higher rates of mortality.” Click here to view the blog post.
8. Comparative Effectiveness Research Group Takes On Hep C Rx Sovaldi
In an article published last week in Inside Health Policy, reporter John Wilkerson highlights how the development of a cure for Hepatitis C has pivoted to discussions that could limit access to the drug. “Some believe Sovaldi is a prime candidate for comparative effectiveness research, but the drug industry typically opposes comparing the value of drugs and was instrumental in preventing government-funded research from considering the cost of treatments… Gilead is selling far more of Sovaldi than anticipated in part because it is estimated to cure Hepatitis C in more than 90 percent of people who are infected… Most of the attention to Sovaldi has been on the cost, and less attention has been on the amount and quality of clinical and economic evidence available on the drug… Obamacare set up the Patient Centered Outcomes Research Institute to do comparative effectiveness research, but PCORI is prohibited from considering cost.” Click here to view the article (subscription required).
