(@mtothbsf)

Goal 3: Advance Translational Research

Substituting scientific-medical insight before profit in drug development

Since the Pure Food and Drug Act of 1906 and the rise of the FDA, the US federal government has directly inserted itself into medical research, primarily from a business perspective. The Orphan Drug Act of 1983 monetarily incentivized the process for rare diseases, but for ultra-rare diseases of < 1,000 patients, it does not work. Successful drugs for rare diseases have enormous price tags to compensate their development... more »

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6 net votes
11 up votes
5 down votes
Active
(@nhlbiforumadministrator)

Goal 4: Develop Workforce and Resources

Education in glycobiology is nearly non-existent

Aside from a handful of locations around the country, there are no local opportunities for the vast majority of PhD students to learn about glycobiology. A search on NIH Reporter for T32 grants with "glycobiology” reveals two grants (one NCI for cancer cell biology, one NIGMS for pharmacology). Neither of these grants are focused upon glycobiology. In fact, glycobiology is essentially never integrated into any standard... more »

Voting

2 net votes
2 up votes
0 down votes
Active
(@sicklecellwarrior)

Goal 2: Reduce Human Disease

Mental health and wellness in sickle cell disease

A growing concern among the sickle cell community surrounds the lack of mental health and wellness services. Many in the community deal with anxiety and depression. It is well known how intricately connected mental and physical health are. So if we know that stress can trigger a psychological crisis which in turn triggers a physical pain crisis, why do we not automatically include mental health services within patient... more »

Voting

25 net votes
38 up votes
13 down votes
Active
(@katherinek)

Goal 2: Reduce Human Disease

How can we increase the pharmaceutical clinical research of targeted therapies in pediatric PAH patients, including encouraging

Clinical research, especially randomized pharmaceutical clinical trials, poses many unique challenges compared to research in adult subjects. In pulmonary arterial hypertension, a disease characterized by high blood pressure of the lungs with increased pulmonary vascular resistance leading to right ventricular failure, there are 12 FDA-approved PAH-targeted therapies for adults. None of these medications are currently... more »

Voting

66 net votes
76 up votes
10 down votes
Active
(@skrenrich)

Goal 4: Develop Workforce and Resources

Retaining Young Investigators

To ensure that young investigators remain in the field, the National Institutes of Health should consider strategies for assisting these investigators in making the career and funding move from mentored career development awards and career development awards for independent investigators (K awards) to research awards (including but not limited to R01 awards).

Voting

14 net votes
15 up votes
1 down votes
Active
(@fblei0)

Goal 4: Develop Workforce and Resources

Develop Vascular Anomalies Medical Training and Research Programs

Patients with vascular anomalies frequently see many physicians and undergo extraneous tests with incorrect diagnoses. A major reason for this is due to the fact that medical training does not include Vascular Anomalies in the syllabus. Thus, many specialties erroneously use the term "hemangioma" for any vascular diagnosis. Over the past 2 decades, there have been major breakthroughs in basic and genetic research, as... more »

Voting

-1 net votes
7 up votes
8 down votes
Active
(@ronald.d.scott)

Goal 2: Reduce Human Disease

Optimizing Cardiovasular (CV) Prevention Medicine Use

Heart attacks and strokes cause substantial morbidity and mortality, while implementation of cholesterol and other CV prevention guidelines remain low. Proposed NCQA on-statin in the last year among those with DM was 46% in national field testing, and about 75% in Kaiser Permanente (KP). KP has had some success overcoming barriers to statin, aspirin, and blood pressure medicine adherence. If the nation as a whole is... more »

Voting

-11 net votes
3 up votes
14 down votes
Active