Politics Bipartisan measure can expedite development of therapies for rare diseases
RAD Model: Advantages And Disadvantages
RAD model is an application development process where each project is built from prototypes, and without considerable planning. The model therefore pays more attention to development as opposed to planning. Rapid Application Development implements a life-cycle methodology that commonly covers four phases.
These days there are not many issues which garner widespread bipartisan, bicameral agreement within the corridors of the United States Capitol; yet the urgent need to encourage the development of better drugs and treatments for patients with rare diseases has proven to be such an issue. After all, we all know someone impacted by a rare medical condition-because rare diseases are not a rare problem. A rare disease isAn estimated 30 million Americans suffer with one of the 7,000 known rare diseases, and 95 percent of these conditions currently have no FDA approved treatment or cure. Traditional regulatory processes have become more complex involving combinations of therapies, genomics, novel diagnostic tests, multi-systemic diseases, small patient populations, and precision medicine. As a result, numerous parts of the regulatory system need to cohesively work together. The development process for a rare disease drug can take an average of 15 years, which leaves rare disease patients and their families waiting without access to life-changing or even life-saving treatments. Further complicating the situation is that when new therapies for rare diseases are finally approved under the current structure, patients often face unnecessary delays and barriers to access, resulting in avoidable health deterioration. Tragically, many of these patients cannot afford to wait decades for help.
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FRANKFORT, Ky. (AP) — Gov. Andy Beshear signed legislation Wednesday expanding early voting in Kentucky, a rare display of bipartisan cooperation in the heart of Trump country at a time of national conflict over restrictive election measures. The Democratic governor called it “a good day for democracy.” The bill's GOP sponsors and Republican Secretary of State Michael Adams joined him at the signing ceremony. “This new law represents anThe Democratic governor called it “a good day for democracy.” The bill's GOP sponsors and Republican Secretary of State Michael Adams joined him at the signing ceremony.
In 2016, Congress came together in near unison to enact theto accelerate development and access to innovative treatments and cures for those with rare diseases. Now, nearly five years later, leaders from both parties in the House and Senate are once again working feverishly together to pass the of 2021. This important legislation will enact targeted, impactful, and attainable policy reforms at the FDA to accelerate development of therapies across the spectrum of rare diseases and disorders and facilitate patient access to such therapies. Specifically, the STAT Act will:
Biden advisors say bipartisan support doesn't mean GOP lawmakers
White House officials, as they push President Joe Biden's agenda, are giving new meaning to the phrase 'bipartisan support,' arguing it doesn't mean winning Republican votes on Capitol Hill. 'If you looked up 'bipartisan' in the dictionary, I think it would say support from Republicans and Democrats,' Anita Dunn, a senior Biden adviser, told The Washington Post. 'It doesn't say the Republicans have to be in Congress.' And Rahm Emanuel, the former Chicago mayor who served as Barack Obama's chief of staff, argued Biden redefined bipartisanship.
- Improve rare disease coordination, stakeholder engagement, and policy development within FDA
- Inform rare disease policies and actions by creating a Rare Disease and Condition Drug Advisory Committee,
- Fund regulatory science and related activities to support development of therapies to treat very small rare disease populations, and
- Strengthen rare disease patient access to FDA-approved therapies in both public and commercial plans through enhanced FDA and Centers for Medicare and Medicaid Services coordination, proactive engagement of payers, and specific actions intended to strengthen Medicare and Medicaid beneficiary access to novel therapies.
These objectives are accomplished through the creation of a Center of Excellence within the FDA to improve coordination and expedite approval of rare disease drugs. First authorized under the 21st Century Cures Act, the Center of Excellence model has proven effective for rapidly stimulating the development of drugs to treat cancer within the past three years. Last year, the FDA announced a second Center of Excellence focused on digital health, further building on its success. The goal of the STAT Act is to replicate the Agency's success with this model for the rare disease community. Most importantly, the FDA reforms contained in this legislation will provide hope to the millions of patients and families who are coping with a rare disease. As co-chair of the Congressional Rare Disease Caucus in the House, I am proud to be leading this patient-centered, bipartisan initiative, and encourage my colleagues to join us in this fight.
Bilirakis represents the 12th District of Florida.
Modernization Committee eyes accountability and new proposals .
Reps. Derek Kilmer, a Democrat from Washington, and William R. Timmons IV, a Republican from South Carolina, talk over each other on a joint phone call for just a moment before recognizing that they are in agreement and trying to say the same thing. The pair are at the helm of the House Select Committee […] The post Modernization Committee eyes accountability and new proposals appeared first on Roll Call.