Building A Stronger Foundation For A New Century Of Treatments And Cures

Sebelius, Kathleen

doi:10.1377/hlthaff.2010.0995

Cited by 4 publications

(4 citation statements)

References 1 publication

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Public-private partnerships (PPPs) are an alternative potentially important resource to address the antibiotic market failure. US HHS Secretary Kathleen Sebelius has written that PPPs are considered critical to overcome the "chokepoints in our medical pipelines" [39]. PPPs may consist of nonprofit corporations funded by both public and private revenues, such as the Global Alliance for TB Drug Development.…”

Section: Methods To Promote Antibiotic Developmentmentioning

confidence: 99%

Seven Ways to Preserve the Miracle of Antibiotics

Bartlett

Gilbert

Spellberg³

2013

Clinical Infectious Diseases

392

266

View full text Add to dashboard Cite

Antibiotic resistance is a well-acknowledged crisis with no clearly defined comprehensive, national corrective plan. We propose a number of interventions that, collectively, could make a large difference. These include collection of data to inform decisions, efforts to reduce antibiotic abuse in people and animals, great emphasis on antibiotic stewardship, performance incentives, optimal use of newer diagnostics, better support for clinical and basic resistance-related research, and novel methods to foster new antibiotic development.

show abstract

Section: Methods To Promote Antibiotic Developmentmentioning

confidence: 99%

Seven Ways to Preserve the Miracle of Antibiotics

Bartlett

Gilbert

Spellberg³

2013

Clinical Infectious Diseases

392

266

View full text Add to dashboard Cite

show abstract

“…The December 2011 NDSI-IEM workshop participants pointed to a need for improved coordination among the federal agencies, a sentiment also articulated by Health and Human Services Secretary, Kathleen Sebelius in Health Affairs [57]. The participants highlighted two primary areas for coordination: within the agencies’ solicitation processes and during the process of product development.…”

Section: Need For Novel Approaches For Iem Product Developmentmentioning

confidence: 99%

Expanding research to provide an evidence base for nutritional interventions for the management of inborn errors of metabolism

Camp

Lloyd-Puryear

Yao

et al. 2013

Molecular Genetics and Metabolism

View full text Add to dashboard Cite

A trans-National Institutes of Health initiative, Nutrition and Dietary Supplement Interventions for Inborn Errors of Metabolism (NDSI-IEM), was launched in 2010 to identify gaps in knowledge regarding the safety and utility of nutritional interventions for the management of inborn errors of metabolism (IEM) that need to be filled with evidence-based research. IEM include inherited biochemical disorders in which specific enzyme defects interfere with the normal metabolism of exogenous (dietary) or endogenous protein, carbohydrate, or fat. For some of these IEM, effective management depends primarily on nutritional interventions. Further research is needed to demonstrate the impact of nutritional interventions on individual health outcomes and on the psychosocial issues identified by patients and their families. A series of meetings and discussions were convened to explore the current United States’ funding and regulatory infrastructure and the challenges to the conduct of research for nutritional interventions for the management of IEM. Although the research and regulatory infrastructure are well-established, a collaborative pathway that includes the professional and advocacy rare disease community and federal regulatory and research agencies will be needed to overcome current barriers.

show abstract

“…Developing drugs for rare disease can be challenging due to specific rare disease characteristics such as small heterogeneous patient populations, long time-frames for disease progression, a poor understanding of disease natural history, and a lack of prior clinical studies. Recent advances in medical science have enhanced the understanding of these disorders at the biochemical and pathophysiologic levels and created more opportunities to address unmet needs by developing specific therapeutic options for rare disease patients [ 3 ]. Improving and adapting the development process for these rare diseases is now an important part of assuring that many of the rarest and most difficult-to-treat rare diseases have specific drugs developed.…”

Section: Introductionmentioning

confidence: 99%

Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints

Kakkis¹,

O’Donovan

Cox

et al. 2015

Orphanet J Rare Dis

View full text Add to dashboard Cite

For rare serious and life-threatening disorders, there is a tremendous challenge of transforming scientific discoveries into new drug treatments. This challenge has been recognized by all stakeholders who endorse the need for flexibility in the regulatory review process for novel therapeutics to treat rare diseases. In the United States, the best expression of this flexibility was the creation of the Accelerated Approval (AA) pathway. The AA pathway is critically important for the development of treatments for diseases with high unmet medical need and has been used extensively for drugs used to treat cancer and infectious diseases like HIV.In 2012, the AA provisions were amended to enhance the application of the AA pathway to expedite the development of drugs for rare disorders under the Food and Drug Administration Safety and Innovation Act (FDASIA). FDASIA, among many provisions, requires the development of a more relevant FDA guidance on the types of evidence that may be acceptable in support of using a novel surrogate endpoint. The application of AA to rare diseases requires more predictability to drive greater access to appropriate use of AA for more rare disease treatments that might not be developed otherwise.This white paper proposes a scientific framework for assessing biomarker endpoints to enhance the development of novel therapeutics for rare and devastating diseases currently without adequate treatment and is based on the opinions of experts in drug development and rare disease patient groups. Specific recommendations include: 1) Establishing regulatory rationale for increased AA access in rare disease programs; 2) Implementing a Biomarker Qualification Request Process to provide the opportunity for an early determination of biomarker acceptance; and 3) A proposed scientific framework for qualifying biomarkers as primary endpoints. The paper’s final section highlights case studies of successful examples that have incorporated biomarker endpoints into FDA approvals for rare disease therapies. The focus of this paper is on the situation in the Unites States, but the recommendations are reasonably applicable to any jurisdiction.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-014-0195-4) contains supplementary material, which is available to authorized users.

show abstract

Building A Stronger Foundation For A New Century Of Treatments And Cures

Cited by 4 publications

References 1 publication

Seven Ways to Preserve the Miracle of Antibiotics

Seven Ways to Preserve the Miracle of Antibiotics

Expanding research to provide an evidence base for nutritional interventions for the management of inborn errors of metabolism

Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints

Contact Info

Product

Resources

About