FDA, Me and Maybe the Mafia—A True Story
May 14th, 2012In honor of FDA Matters’ third anniversary, I am sharing a personal story. It reflects FDA’s history as a struggle of competing interests—where sometimes reasonable people disagree, often vehemently, while at other times it is obvious that indifference or greed are the driving forces.
Both are a fact of everyday life at FDA and in the FDA-regulated world. Here is my own little story and I still can’t say for sure whether it involved reasonable people or dark forces.
Read the rest of this entrySafety and Efficacy Standards: Innovative Approaches to Radical Ideas
May 7th, 2012FDA is in the midst of its quinquennial* (five year) review from Congress as part of the user fee reauthorization cycle. Lots of proposals are on the table and FDA Matters agrees with some and disagrees with others. So far though, there doesn’t seem to be anything that would pull FDA apart or create an agency that cannot act with integrity.
In contrast, two key opinion leaders are talking about potentially radical changes in FDA’s safety and efficacy standards. While neither has seen their specific proposals become part of the Hill debate, there are redeeming qualities to what both of them are suggesting.
Read the rest of this entryOMB, HHS, or FDA: Who Really Makes FDA’s Decisions?
April 23rd, 2012OMB, White House staff, and the Secretary of HHS review many FDA decisions and their oversight sometimes alters FDA’s positions. This was chronicled in a recent NY Times article and generated a number of editorial comments criticizing anyone tampering with FDA’s integrity. However, the individuals named in the article have the authority (on behalf of the President) to question FDA’s judgments.
More importantly, FDA Matters observes that the vast majority of FDA-related decisions–and virtually all of the science-related decisions–are made by the FDA. The key is the strong public health and scientific expertise of FDA staff and the credibility this brings to any scientifically-based agency decision.
Read the rest of this entryMedical Innovation: The Dream of More Cures and More Industry Success
April 16th, 2012Can we, as a society, stimulate medical innovation? If so, how? These are key questions facing Congress as it considers amendments to the FDA user fee reauthorization legislation. The answers are of central importance to FDA, patients and industry. So far, most of the “solutions” being considered by Congress (legislatively) and FDA (administratively) are worthwhile and likely to have a positive impact over time.
However, in FDA Matters’ view, the challenge of stimulating medical innovation mostly lies outside the policy sphere. Instead, achieving more cures and more industry success requires substantive and attitudinal changes inside the research and development process itself.
Read the rest of this entryProposals to Speed-Up Drug Approvals: Not Created Equal
April 3rd, 2012An important part of the 2012 user fee reauthorization cycle is Congressional efforts to push FDA toward approving drugs and biologics more rapidly. Most of industry and a large number of patient groups agree. Proposals to speed up FDA are already in play.
Since these proposals have a common purpose, it is easy to think of them as alike. However, most are different from each other—in focus, intent, and likely impact on the agency’s existing decisionmaking process. This is FDA Matters’ analysis of why proposals to speed up drug approvals can’t be lumped together and why FDA may support some, but not others.
Read the rest of this entryFDA Progress “At Risk:” An Update on Funding and a Call to Action
March 25th, 2012FDA Matters’ State of the FDA—January 2012 identified agency funding as the greatest threat to the FDA’s future. The agency received a very small increase in FY 12 appropriated funding, reflecting the severe budgetary pressure on all U.S. federal agencies. That pressure continues and FDA faces potential cuts in FY 13 (starting October 1, 2012).
Advocacy by the Alliance for a Stronger FDA and other stakeholder groups is critical to sustaining the agency’s appropriated funding and pressing Congress for increases to meet the growing demands on FDA. This column explains the budget situation and calls upon all stakeholders to support increased FDA funding.
Read the rest of this entryThe FDA Matters “Guide to the User Fee Reauthorization Process”
March 19th, 2012The prescription drug (PDUFA) and medical device (MDUFA) user fee programs, which run for 5 years, must be renewed by September 30 of this year (last day of the current fiscal year). House committee staff has just released a 205-page first draft of reauthorization legislation. The Senate has starting releasing drafts on specific issues and has a March 29 hearing scheduled.
Because the PDUFA and MDUFA provisions are pre-negotiated by FDA with industry and patient groups, they are likely to change little. Congress’ focus will be on the backlog of FDA-related legislative proposals that have accumulated while awaiting a “must pass” FDA legislative vehicle. This is FDA Matters’ guide to the process and likely amendments.
Read the rest of this entryBiological Complexity and the Myth of the Low-Hanging Fruit
March 12th, 2012Two events persuaded FDA Matters to write another column on biological complexity and its implications for medical research, drug discovery, and personalized medicine. First was the release of a remarkable study on gene mutations in cancer tumors. It is a stellar and sobering example of how biological complexity confounds our expectations that rapid advances in science will quickly lead to cures.
Second was the multiple comments from readers of last week’s column suggesting that drug discovery (and biomedical advances generally) are so hard because “the low-hanging fruit has been picked.” This is a persistent and dangerous myth that devalues past breakthroughs and distorts the challenges bio-medicine faces over the next decade.
Read the rest of this entryWhy is Drug Discovery So Hard (and Expensive)?
March 5th, 2012Notwithstanding herbal medicines and the re-emergence of leeches as therapy, it is only in the last 70 years that physicians have had proven medical science to support “the healing arts.” Potions and procedures of dubious value have been replaced by powerful medicines that treat infections, heart disease, diabetes and mental illness.
While the benefits are clear, the difficulties and cost of creating new medicines are a source of widespread frustration to patients and industry alike. FDA Matters is impressed that current efforts to speed up drug discovery are gaining momentum. At the same time, the nature of human biology dictates that the creation of new therapies will never be easy or inexpensive.
Read the rest of this entryQuality and Safety “Just Don’t Get No Respect”
February 27th, 2012In every successful company, the glittery careers and the recognizable names belong to people who develop new products that meet consumer and patient needs. Innovation in new products (and careful husbanding of intellectual property and market share) is what brings in the revenue and determines corporate success.
By comparison, there is little recognition and often sparse resources for the people devoted to making sure those products (new and old) are safe and of high-quality. The best product ever developed is worthless, and possibly harmful, if standards are not maintained and manufacture and supply carefully monitored. The stakes are so much higher for FDA-regulated products.
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