Value of Cancer Medicines: Benefiting Society
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View the full infographic: Value of Cancer Medicines. (Source: PHRMA)
Source: PHRMA - November 18, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
John F P Bridges, Ph.D. on PDUFA
For many, terms like patient-centered outcomes research and patient-focused drug development are new. For others, they are simply buzzwords that are aimed at putting a new spin on existing practices. As a researcher who has dedicated the majority of my career to the scientific study of the patients’ point of view, I rejoice in renewed interest in the patients’ perspective.
The formal study of patient preferences emerged in the 1990s as a more scientific alternative to qualitative approaches to patient input. Using methods like conjoint analysis and discrete-choice experiments, this early literature challenged tradition...
Source: PHRMA - November 13, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
John F P Bridges, Ph.D.
Associate ProfessorJohn F P Bridges, PhD is an international leader in the application of stated-preference methods. He is the founding editor of The Patient – Patient Centered Outcomes Research and has worked with numerous patient groups, health technology assessment agencies, regulators and international aid agencies to advance and apply these methods to document the preferences of patients and other stakeholders. Within the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) he founded the Conjoint Analysis Working Group (2006-2011), the Conjoint Analysis Task Force (2008-2010) and was the first a...
Source: PHRMA - November 13, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Kim McCleary on PDUFA
A paradigm shift is occurring. Patients were once viewed as passive, deferential recipients of medical products and services developed for them. Today, there is growing recognition that patients are a vital force for transformative change to address serious unmet medical needs and improve public health.
To deliver on the promise of a patient-focused biomedical system, stakeholders across the research and care enterprise are working – mostly independently – to define and scale effective patient engagement, develop instruments to measure patient-reported outcomes, quantify preferences, and incorporate patient perspective...
Source: PHRMA - November 13, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Kim McCleary
Director of Strategic InitiativesKim McCleary is FasterCures’ Director of Strategic Initiatives. She leads a new FasterCures program to expand patient engagement in FDA’s assessment of benefits and risks for medical products. Kim also works closely with FasterCures’ network of patient-focused venture philanthropy organizations, The Research Acceleration and Innovation Network (TRAIN). Prior to joining FasterCures’ staff, Kim was President & CEO of the CFIDS Association of America from 1991 until 2013. She has participated in every opportunity organized by the FDA to shape its Patient-Focused Drug Development In...
Source: PHRMA - November 13, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Ryan Fischer on PDUFA
Parent Project Muscular Dystrophy has long held the belief that new approaches are required to expedite the development of treatments for rare diseases like Duchenne. Patient advocates have worked tirelessly with Congress, over time through legislation, to increase the engagement of the patient voice within the regulatory review process for therapeutics. Passed in 2012, The Food and Drug Administration Safety and Innovation Act (FDASIA) was a culmination of those advocacy efforts by patients. The legislation included a number of provisions under the patient-focused drug development and patient preference headings that ...
Source: PHRMA - November 13, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Sharon Terry on PDUFA
This is not rocket science! Running a molecule through all of the traps is really hard. Understanding what people feel and think and experience with regard to the drugs they take and those they need is simple. In most other areas of our lives, various industries know quite well what features we want on our smartphones, that we suddenly all want Greek yogurt, and that we don’t want to pay to use the bathroom on a plane. In medicine, we have not yet realized the same dynamic and robust methods, tools and studies for understanding the needs and values of the end user. The needs, wants, values and goals of the end ...
Source: PHRMA - November 13, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Sharon Terry
President and CEOSharon F. Terry is President and CEO of Genetic Alliance, a network of more than 10,000 organizations, of which 1,200 are disease advocacy organizations. Genetic Alliance engages individuals, families and communities to transform health.
She is the founding CEO of PXE International, a research advocacy organization for the genetic condition pseudoxanthoma elasticum (PXE). PXE affects Terry’s two adult children. As co-discoverer of the gene associated with PXE, she holds the patent for ABCC6 to act as its steward and has assigned her rights to the foundation. She developed a diagnostic test and cond...
Source: PHRMA - November 13, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Ryan Fischer
Vice President of Advocacy and Community OutreachRyan Fischer serves as the Vice President of Advocacy and Community Outreach for Parent Project Muscular Dystrophy and has been with the organization for 10 years. Parent Project Muscular Dystrophy is the leading organization in the fight to end Duchenne.
Ryan oversees strategic community engagement and advocacy, educating the patient community on the best ways to interact with members of Congress and federal agencies. He acts as an interpreter for Duchenne patients and families, explaining complex issues in public policy and about federal legislation with the goal of making...
Source: PHRMA - November 12, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Sascha on PDUFA
As important stakeholders in the drug development and approval process, patients provide a unique and valuable perspective when considering the benefits and risks of potential new and innovative medicines. Patients can identify areas of unmet medical need, provide critical perspective on the impact of a disease and its manifestations, and uniquely inform the development of outcome measures that are meaningful to patients. A scientific approach to gathering patient input is necessary to realize the shared goal of a patient-centered approach to drug development and regulatory review. PhRMA believes that advancing the s...
Source: PHRMA - November 12, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Sascha Haverfield
VP, Scientific and Regulatory AffairsSascha Haverfield is the Vice President for Scientific and Regulatory Affairs at PhRMA. He leads PhRMA's activities on global regulatory policy issues and is the architect of our engagement on the implementation of the Prescription Drug User Fee Act (PDUFA). He previously worked in the biopharmaceutical industry on drug discovery, translational medicine and regulatory affairs with a focus on global drug development issues.
Sascha completed his undergraduate studies in biological sciences at the University of Marburg, Germany and received a doctorate in genetics and cell biology ...
Source: PHRMA - November 12, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
How can we best advance the science of patient input to improve the efficiency of the drug development and regulatory review process?
Marc Boutin on PDUFA
Submitted by Emily on Tue, 11/11/2014 - 09:30
An astute marketer will tell you that success of any product is dependent on the wants and needs of the end user. In health care, we need a similar mindset. By collaborating with patients early in drug development, there is an opportunity to improve the success rates in the preclinical stage of the development, which could ultimately accelerate the delivery of new treatments to patients and enhance patient adherence to their treatment plans. The end result will be a win for all stakeholders.
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Source: PHRMA - November 11, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Robert Metcalf on PFDD
The most recent reauthorization of the Prescription Drug User Fee Act (PDUFA) included new efforts by the Food and Drug Administration (FDA) to enable patients and patient groups to become actively involved in FDA evaluations of new medicines. Two programs in particular--the Patient-Focused Drug Development (PFDD) Initiative and Benefit/Risk Assessment--work in tandem to inform the FDA's decisions on whether or not to approve a medicine for patient use.
There’s no doubt that the feedback gained through the PFDD Initiative gives the FDA deeper insight into patient preferences, especially regarding the benefits and risks o...
Source: PHRMA - November 11, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Peter L. Saltonstall on PDUFA
We at NORD believe that patients need to play a central and much more active role in the drug development process, from start to approval. Until very recently, the process has largely ignored the patient as an active participant.
Drug developers, whether researchers, industry or government, would benefit immensely from understanding better the needs of patients -- what symptoms trouble them most, what is their tolerance for risk, how do their diseases progress and how do patient needs change as a disease progresses. The issue is especially important for patients with rare diseases, since there often is a lack of informa...
Source: PHRMA - November 11, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
Peter L. Saltonstall
President and CEOPeter L. Saltonstall is the President and CEO of the National Organization for Rare Disorders (NORD). He joined NORD in 2008 after having served for more than 30 years as a senior official in both for-profit and not-for-profit healthcare environments.
Under his leadership, NORD has forged new relationships between the patient community and the Congress, FDA, NIH and Social Security Administration, as well as with drug/device companies and the medical/academic and investment communities. His efforts to build collaborations stems from his view that advances for the rare disease patient can be achieved be...
Source: PHRMA - November 11, 2014 Category: Pharmaceuticals Authors: Emily Source Type: news
