The war between the science and the bureaucracy, between truth and lie, passion and negligence. This is the war of the despairing families against the government. The war against time. Against death. The war for life. Children's life. Goodness will conquer corruption. And justice will prevail.
A hopeful tone of the presentation. FDA will respond positively in March. Partnership is desirable for all venues, but DMD. Next week SP will go up.
It's Faster... with 3D printing, the delay between design and fabrication is now a few hours instead of a few weeks. Motorcycle maker Ducati cut 20 months (or 70%) off its usual development time for a new racing engine.
It's Cheaper... companies that use 3D printing don't need to tie up capital investing in expensive tools and dies. They can also eliminate shipping costs. A small supplier to Nike called Union Footwear got a leg up on its bigger competitors by faxing its design bid in 3D.
It Encourages Innovation... by eliminating the delays and costs created by traditional "manufacturability" constraints, 3D printers allow designers to change their minds and implement a new idea at any time. An Ohio company that supplies crash test dummies to automakers is using the technology to produce new shapes and sizes that respond to client needs that can evolve by the day.
It's Less Wasteful... traditional manufacturing is "subtractive," discarding up to 90% of the initial blocks of raw material. 3D printing is an "additive" process that builds objects from the ground up, layer by layer. One architect is even using it to make entire buildings.
It's More Durable... designers can tweak these "additive" printers to produce denser (but lighter) layers of material than would usually be possible. The Smithsonian is using 3D printers to digitize their entire collection; the statue of Thomas Jefferson you see in the National Museum of American History in Washington, DC is actually a 3D replica of the original brass statue.
It's Customer Friendly... 3D printing allows for a level of product customization and personalization that has never before been possible. Shoppers are already ordering lamps, jewelry, sunglasses, and even bikini swimsuits, all made to their exact specifications. (I know I won't miss having to try on shoes at the mall.)
Assume - I'm FDA. I"ve made a deadly mistake. Hid it for a while, playing in a dark, but my victim turned all the lights up. I am exposed and now I am in troubles. The truth is taking over... Shell I ignore all that? Aren't I Almighty? The hell - I am FDA, and I am above the common sense. Yet, Instinct tells me that this time my usually unquestioned judgement may not be excepted. I am loosing... No! ...Eureka! Play along. Give them what they want... Aren't I wise?
Sentiment: Strong Buy
You are a paid schmuck with scripted by shorts' pseudo-meaningful statements to which the price is ZERO. Get lost, moron.
Summary of Recommendations
• Recommendation 1:
The FDA should expand the use of accelerated
approval for therapies intended to treat rare
diseases, including Duchenne muscular dystrophy.
• Recommendation 2:
The FDA should expand the scope of acceptable
endpoints, including novel surrogate and
intermediate clinical endpoints, used to approve
drugs for serious or life-threatening diseases with
unmet medical need, such as Duchenne.
• Recommendation 3:
Using existing authority under current law, the
FDA should pilot the use of adaptive approval
for serious and life-threatening disorders with
significant unmet medical need.
• Recommendation 4:
The FDA should give greater weight to the
demonstrated benefit/risk preferences of patients,
as well as caregivers in the case of pediatric illness,
when making benefit/risk determinations.
The need for strong, reliable clinical data
demonstrating substantial evidence of safety and
efficacy is of critical importance for the review and
approval of new drugs. In weighing these data during
the evaluation of new drugs, the FDA must determine
in each case whether the benefits appear to outweigh
the known risks. Striking the correct balance between
the need for more data and access to new, potentially
beneficial treatments is the constant challenge set
before the Agency. To successfully navigate this
challenge, it is imperative that the FDA adhere to
its own guidance calling for the consideration of the
relative lethality and the seriousness of the disorder the
drug is designed to treat, as well as the availability or
lack of alternative treatments. These considerations will
vary substantially from one disorder to another, and it is
often the case that patients’ risk tolerance increases in
relation to the morbidity and mortality associated with
a particular illness.31 To date, however, there has been
little indication that the FDA is inclined to utilize the
significant discretion at its disposal in reviewing drugs
The passage of FDASIA and its implementation over
the next several years have created new opportunities
for the FDA to speed responsible access to new
therapies for Duchenne and other rare, serious and
life-threatening disorders. The FDA must seize this
opportunity to issue guidance providing clear and
accessible standards for the application of accelerated
approval to therapies for Duchenne, and it should
move immediately to better incorporate the patient
perspective into its assessment of an appropriate
benefit/risk ratio. PPMD and the distinguished panel of
advisors who contributed to this report stand ready to
work alongside the FDA to strike a more appropriate
balance between clinical certainty and patient access
to potentially life-saving treatments. Patients and
their families, frustrated by the slow pace of progress
and desperate for access to new treatments for this
devastating illness, deserve nothing less.
Sentiment: Strong Buy