BBC News says we are a step closer to microchips that can be “implanted under a patient’s skin to control the release of drugs”.
The news was based on a study that tested the use of advanced microchips containing tiny drug reservoirs that can be remotely triggered to release medication into the body. Creating workable drug-release chips has long been a goal of researchers, as it could help people take the correct dose of vital medicines such as insulin.
In this particular trial, reported to be the first of its kind, eight women were given the chips filled with a drug to combat osteoporosis. The drug, teriparatide, is normally delivered by daily injection, but researchers found that using the chips produced similar physical results to injections. Also, there were no toxic or adverse events, due to either the microchip or the drug, and all the patients reported that it did not impact on their quality of life.
This study throws up a range of possible uses for microchip-based drug delivery, which could one day be used for the treatment of wider conditions that require frequent, scheduled dosing, particularly where standard treatment is through injection.
However, much more testing of the technology will be needed to firmly establish its safety, and to see whether there could be wider applications. One key consideration though, would be whether the use of this advanced technology can actually prove better or cheaper than the use of injections.
Where did the story come from?
The study was carried out by researchers from MicroCHIPS, Inc, (a private company producing medical microchips ; the Harvard Medical School; Case Western Reserve University; On Demand Therapeutics, Inc, and the Massachusetts Institute of Technology. It was funded by MicroCHIPs, Inc.
The study was published in the peer-reviewed scientific journal Science Translational Medicine.
The results of this study have also been presented at the annual meeting of the American Association for the Advancement of Science (AAAS .
The story appeared on the BBC and a number of newspapers, including the Daily Mail, the Daily Mirror and The Independent.
Most of the coverage of the story was good. However, alongside The Independent’s main article the newspaper featured an opinion-based section discussing potential uses of the device, including allowing psychiatrists to trigger doses in schizophrenic patients when they resist injections of medication. There is a distinct difference between using medical devices to structure the delivery of medication and using them to force people to take medication against their will.
It seems unlikely that medical groups would find this theoretical use to be ethically acceptable, and it should be noted that the treatment of mental health problems was not assessed in the study or in other coverage.
The Independent also used a photograph of a distressed man huddled on the floor wearing no shoes, intended to illustrate schizophrenia. While the condition can certainly involve periods of acute problems and distress, it seems to a rather extreme and particularly negative depiction of someone with schizophrenia.
What kind of research was this?
This was a small cohort study of a drug delivery microchip, implanted under the skin. The microchip contains tiny drug reservoirs and can be programmed to wirelessly release discrete doses of a medication.
This particular study used the drug teriparatide, prescribed by specialists only for the treatment of severe osteoporosis (bone weakening . It is normally delivered by daily injection and given for a maximum treatment period of two years only.
The researchers aimed to see whether the drug released from the device had similar ‘pharmacokinetics’ (adsorption, distribution, metabolism and excretion and biological effect to the drug administered by standard injection. They also monitored how reliable and reproducible drug release from the microchip was, and if there were any side effects of the implant.
This was the first clinical trial of this microchip. As the researchers state, further development is required to ensure proper operation of implanted devices, and devices containing more reservoirs will be needed if the device were to provide regular doses over one or more years. In addition, before this technology becomes available, it will have to be tested in larger, controlled trials.
What did the research involve?
Eight women with osteoporosis, aged between 65 and 70, were recruited for the study. The drug delivery microchip was implanted under the skin, just under the waistline. The devices were implanted for four months. Eight weeks after implantation, the microchip started releasing daily doses of teriparatide for a period of 20 days. Blood samples were drawn regularly to monitor the pharmacokinetics and to determine levels of bone markers. A safety assessment was also performed.
After the 20 days of drug release from the device, the researchers administered the osteoporosis drug by injection, and again took blood samples, so that release from the microchip and from the injection could be compared.
What were the basic results?
In one patient, feedback from chip indicated that the drug was not being released. The results from this patient were excluded.
Drug released from the microchip in the seven other patients had similar pharmacokinetics to drug administered by injection, and bone markers indicated that drug released from the microchip increased bone formation as expected. However, the effectiveness of medication released from the microchip was not compared to the effectiveness when given by injection.
There were no toxic or adverse events due to the device or drug. Patient response to the implant was also favourable, stating that it did not impact upon their quality of life.
How did the researchers interpret the results?
The researchers concluded that the programmable implant was able to deliver teriparatide at scheduled intervals, with pharmacokinetics similar to injections ‘without the pain and burden of daily injections’.
Conclusion
This study was a small clinical trial, performed in eight women, of an implantable microchip-based drug delivery device. It found that the microchip could deliver the osteoporosis drug teriparatide with similar pharmaceutical properties to injections, including adsorption, distribution, excretion and metabolism by the body. There were no toxic or adverse events due to either the microchip or the drug, and the patients all responded favourably to the implant, stating it did not affect quality of life.
Larger controlled trials comparing this device with conventional injected teriparatide would be needed to confirm the safety and efficacy findings. Furthermore, trials may need to assess use of the chip over a longer period - on prescription, teriparatide may be administered by daily injection for up to two years.
The findings also suggest that this microchip-based drug delivery device may have the potential to be used for the treatment of wider conditions that require frequent, scheduled dosing, particularly where standard treatment is through injection. However, much more testing of the technology will be needed to see whether there could be wider applications.
Analysis by Bazian
Links To The Headlines
Dawn of the age of wireless medicine. The Independent, February 17 2012
'Wireless medicine' helps solve one of doctors' biggest problems - getting patients to take drugs. The Independent, February 17 2012
New microchip will let doctor administer drugs into your body over the phone. Daily Mirror, February 17 2012
'Pharmacy on a chip' gets closer. BBC News, February 17 2012
Links To Science
Farra R, Sheppard NF, McCabe L, et al. First-in-Human Testing of a Wirelessly Controlled Drug Delivery Microchip. Science Translational Medicine. Published online February 16 2012
Devices Are the Future of DrugsBy: Norbert Sparrow 30 January, 2012
Last week, I posted an article on medtech insider that listed five reasons why medical device manufacturers should consider attending Pharmapack Europe in Paris on 15 and 16 February 2012. One of those reasons was a conference session titled, Why Medical Devices Are the Future of Drugs. The topic intrigued me and I wanted to know more, so I called up Dr. Yves Tillet, CEO of Paris-based consultancy White-Tillet, who will be addressing this subject on 16 February. Here’s a preview of the case he will make to Pharmapack Europe conference attendees.
Most drugs are administered via a systemic route, says Tillet, and to ensure that the medication reaches the target site, it is administered in large concentrations. “If you are able to target just the tumour, for example, you can use much smaller quantities of the drug, and thus improve the risk-benefit ratio,” explains Tillet. Moreover, drugs that did not get past phase 2 of the drug development process “because of toxicity issues can be rehabilitated by being administered locally,” he adds. Introduced into the body in much smaller quantities and precisely delivered to the target site, the drug no longer poses a threat to healthy cells.
During his presentation, Tillet will describe various types of innovative drug-delivery systems that are transforming the practice of healthcare. I asked him if there were any that he finds especially promising. After some thought, he pointed to microsphere technology and phototherapy.
“For cancer treatments, the microsphere carries the drug to the target site and isolates the tumour by preventing blood from getting to it. The cytotoxic agent acts on the tumour, which can’t spread,” says Tillet. Phototherapy is also very promising, he adds, because a laser is used to activate the drug only after it has reached the target site.
Traditionally, drugs have been formulated for delivery via a systemic route, notes Tillet, and this can cause a number of issues. At the Pharmapack Europe conference, Tillet will explain in some detail how combination products can eliminate these problems and fundamentally change the way in which serious conditions are treated.
FDA NEWS RELEASE
For Immediate Release: Feb. 17, 2012
Media Inquiries: Morgan Liscinsky, 301-796-0397;
morgan.liscinsky@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA
FDA approves Korlym for patients with endogenous Cushing’s syndrome
Today, Korlym (mifepristone was approved by the U.S. Food and Drug Administration to control high blood sugar levels (hyperglycemia in adults with endogenous Cushing’s syndrome. This drug was approved for use in patients with endogenous Cushing’s syndrome who have type 2 diabetes or glucose intolerance and are not candidates for surgery or who have not responded to prior surgery. Korlym should never be used (contraindicated by pregnant women.
Prior to FDA’s approval of Korlym, there were no approved medical therapies for the treatment of endogenous Cushing’s syndrome.
Endogenous Cushing’s syndrome is a serious, debilitating and rare multisystem disorder. It is caused by the overproduction of cortisol (a steroid hormone that increases blood sugar levels by the adrenal glands. This syndrome most commonly affects adults between the ages of 25 and 40. About 5,000 patients will be eligible for Korlym treatment, which received an orphan drug designation by the FDA in 2007.
Korlym blocks the binding of cortisol to its receptor. It does not decrease cortisol production but reduces the effects of excess cortisol, such as high blood sugar levels.
The safety and efficacy of Korlym in patients with endogenous Cushing’s syndrome was evaluated in a clinical trial with 50 patients. A separate open-label extension of this trial is ongoing. Additional evidence supporting the agency’s approval included several safety pharmacology studies, drug-drug interaction studies and published scientific literature. Patients experienced significant improvement in blood sugar control during Korlym treatment, including some patients who had marked reductions in their insulin requirements. Improvements in clinical signs and symptoms were reported by some patients.
The most common side effects experienced by endogenous Cushing’s syndrome patients treated with Korlym in clinical trials were nausea, fatigue, headache, arthralgia, vomiting, swelling of the extremities, dizziness and decreased appetite. Other side effects of Korlym include adrenal insufficiency, low potassium levels, vaginal bleeding and a potential for heart conduction abnormalities. Certain drugs used in combination with Korlym may increase its drug level. Health care professionals must be aware of the potential for drug-drug interactions and adjust dosing or avoid using certain drugs with Korlym.
Korlym should never be used by pregnant women. Although pregnancy is an extremely rare occurrence in Cushing’s syndrome patients because of the suppressive effect of excess cortisol on female reproductive function, Korlym will carry a Boxed Warning advising health care professionals and patients that the therapy will terminate a pregnancy.
The FDA has determined that a Risk Evaluation and Mitigation Strategy (REMS is not necessary for Korlym to ensure that the benefits outweigh the risks for patients with endogenous Cushing’s syndrome. Several factors were considered in this determination including the following:
- There are no other approved medical therapies for this debilitating form of Cushing’s syndrome and very sick patients would suffer if impediments to access were imposed.
- The number of Cushing’s syndrome patients who will require treatment with Korlym is small, with an estimated 5,000 patients being eligible for treatment.
- The number of health care professionals in the United States who would potentially prescribe Korlym is very small and highly specialized. They are familiar with the risks of Korlym treatment in the endogenous Cushing’s syndrome population and frequently monitor patient status.
- The risks of Korlym treatment in the intended population can be managed through physician and patient labeling. The risks associated with Korlym will be outlined in a medication guide for patients.
The company has voluntarily proposed distributing Korlym through a central pharmacy to ensure the timely, convenient and appropriate delivery of the drug to Cushing’s patients or to the health care institutions where this therapy may be initiated. Most retail pharmacies are unlikely to keep adequate supplies of the drug for this rare condition and central distribution will give patients with Cushing’s syndrome better access to Korlym.
Korlym is manufactured by Corcept Therapeutics of Menlo Park, Calif.
For more information:
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
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A Tasmanian initiative to prevent the abuse of controlled drugs such as painkilling prescription drugs will be rolled out nationally from July.
The Federal Government has announced plans to establish a $5 million national electronic records system that will enable pharmacists, doctors and other health authorities to monitor the real time prescribing and dispensing of addictive drugs.
“Abuse of these drugs can cause enormous harm and is a growing problem in the community,” she said.
Ms Plibersek said health professionals will be able to immediately detect people suspected of trafficking in painkillers, forging prescriptions and ‘doctor-shopping’.
“The new records system will be able to flag patients in real time who have repeatedly sought controlled drugs, helping to prevent people from inappropriately using the drugs or selling them to others.
“If a pharmacist determines it is not clinically appropriate to dispense a medicine to a patient, it is their duty of care to restrict access to that patient.” The system will replace paper-based prescription records still operating in some Australian states and enable pharmacists to check on prescription records from across the country.
The national scheme comes as figures show the amount of prescription opioids used in Australia is on the rise.
The total value of Pharmaceutical Benefits Scheme opioid prescriptions increased from $2 million in 1992 to $7 million in 2007, according to the Internal Medicine Journal.
MENLO PARK, Calif. - February 17, 2012
Corcept Therapeutics (NASDAQ:CORT announced today that the U.S. Food and Drug Administration (FDA has approved Korlym™ (mifepristone 300 mg Tablets as a once-daily oral medicine to control hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing's syndrome who have diabetes mellitus type 2 or glucose intolerance and have failed surgery or are not candidates for surgery.
"We appreciate the FDA's diligent attention to our NDA and its grant of approval on the PDUFA date," said Joseph K. Belanoff, M.D., the company's Chief Executive Officer. "We plan to make Korlym available to patients by May 1 through a distribution system designed to support both patients and prescribers."
Corcept will be the sole marketer of Korlym. "A relatively small number of endocrinologists regularly treat patients with Cushing's syndrome," added Dr. Belanoff. "These doctors can be reached without a large sales and marketing infrastructure." The company has begun hiring Medical Science Liaisons to inform practitioners about the drug, which will be dispensed by the leading specialty pharmacy company CuraScript SP, a subsidiary of Express Scripts.
"Korlym is a significant advance in the treatment of patients suffering from the debilitating symptoms of Cushing's syndrome," said Robert L. Roe, M.D., Corcept's President. "For the first time, these patients have access to an approved therapy when surgery has failed or is not an option."
Korlym clinical trial investigator Amir Hamrahian, M.D., Department of Endocrinology, Diabetes and Metabolism at the Cleveland Clinic said, "There are not many effective treatment options for patients with Cushing's syndrome. Although surgery is standard first line treatment for the disease, it is not always successful and not all patients are candidates. As part of the clinical trial, I have used Korlym successfully and my patients continue to do well on the medicine. I'm excited to be able to continue using Korlym in these patients and others who need it. This medicine's approval gives me a much needed tool to better treat patients."
Dr. Hamrahian's comments were seconded by Maureen V., a patient in Corcept's Phase 3 clinical trial: "I had pituitary surgery to treat my Cushing's syndrome. Unfortunately, my surgery wasn't successful. I was lucky to get into the study and get Korlym treatment. I have been taking the medicine successfully for over a year, and I am extremely happy that it was approved by the FDA. Now I know I'll be able to keep taking it. It has made a big difference in my life."
Clinical Trial Results Supporting FDA Approval
The clinical data supporting the FDA approval of Korlym resulted from an uncontrolled, open-label, multi-center, 24-week phase III study of 50 patients who had endogenous Cushing's syndrome and were either not eligible for or had relapsed from surgery and were either glucose intolerant (29 patients or had hypertension (21 patients . Within the glucose intolerant group, 60 percent of patients had a greater than 25 percent reduction from baseline in the area under the curve in the oral glucose tolerance test. In this group, mean hemoglobin A1C (HbA1C was reduced from 7.4 percent to 6.3 percent. All 14 patients with above-normal HbA1C levels at baseline experienced reductions. Eight of these 14 normalized their HbA1C. Antidiabetic medications were reduced in seven of the 15 patients with diabetes mellitus type 2 and remained constant in the others.
Patients who responded to therapy were allowed enrollment in an extension trial. Eighty-eight percent of the patients who completed the trial chose to do so.
A peer-reviewed analysis of the study results will soon be published in a leading journal.
Patients in the study started Korlym treatment on a dose of 300 mg administered once daily. Their dose was then titrated to maximum clinical effect. As indicated in the medicine's label, physicians prescribing Korlym may determine the appropriate dose for each patient by assessing tolerability and degree of improvement in Cushing's syndrome manifestations. In the first six weeks, these manifestations may include changes in glucose control, anti-diabetic medication requirements, insulin levels and psychiatric symptoms. After two months, assessment may also be based on improvements in cushingoid appearance, acne, hirsutism, striae, decreased body weight, along with further changes in glucose control.
About Korlym™ (mifepristone 300 mg Tablets
Korlym is a once-daily oral medication that blocks the glucocorticoid receptor type II (GR-II to which cortisol normally binds. By blocking this receptor, Korlym inhibits the effects of excess cortisol in Cushing's syndrome patients.
The FDA has designated Korlym as an Orphan Drug for treatment of the clinical manifestations of endogenous Cushing's syndrome. Orphan Drug designation is a special status designed to encourage the development of medicines for rare diseases and conditions. Because Korlym is an Orphan Drug, Corcept will have marketing exclusivity consistent with the FDA's designation until February 2019.
About Cushing's Syndrome
Endogenous Cushing's syndrome is a rare and life-threatening endocrine disorder that results from long-term exposure to excess levels of the hormone cortisol. This excess is caused by tumors that usually occur in the pituitary or adrenal glands that over-produce, or prompt the over-production of, cortisol.
Although cortisol at normal levels is essential to health, in excess it causes a variety of problems, including hyperglycemia, upper body obesity, a rounded face, stretch marks on the skin, an accumulation of fat on the back, thin and easily bruised skin, muscle weakness, bone weakness, persistent infections, high blood pressure, fatigue, irritability, anxiety, psychosis and depression. Women may have menstrual irregularities and facial hair growth, while men may have decreased fertility or erectile dysfunction. More than 70 percent of Cushing's syndrome patients suffer from glucose intolerance or diabetes.
The treatment of an endogenous Cushing's syndrome patient depends on the cause. The first-line approach is surgery to remove the tumor. If surgery is not successful or is not an option, radiation may be used, but that therapy can take up to ten years to achieve full effect. Surgery and radiation are successful in only approximately one-half of all cases.
If left untreated, Cushing's syndrome has a five-year mortality rate of 50 percent.
An orphan disease, Cushing's syndrome occurs in about 20,000 people in the United States, mostly women between the ages of 20 and 50.
Conference Call Information
Corcept will hold a conference call on Tuesday, February 21, 2012 at 9:00 a.m. Eastern Time (6:00 a.m. Pacific Time to discuss this announcement. To participate in the live call please dial 1-800-264-7882 from the United States or +1-847-413-3708 internationally. The pass code is 31838602. Please dial in approximately 10 minutes before the start of the call.
A replay of the conference call will be available through March 6, 2012 at 1-888-843-7419 from the United States and +1-630-652-3042 internationally. The pass code is 31838602.
IMPORTANT SAFETY INFORMATION
WARNING: TERMINATION OF PREGNANCY
See full prescribing information for complete boxed warning.
has potent antiprogestational effects and will result in the termination of pregnancy. Pregnancy must therefore be excluded before the initiation of treatment with Korlym, or if treatment is interrupted for more than 14 days in females of reproductive potential.
Contraindications
- Pregnancy
- Use of simvastatin or lovastatin and CYP 3A substrates with narrow therapeutic range
- Concurrent long-term corticosteroid use
- Women with history of unexplained vaginal bleeding
- Women with endometrial hyperplasia with atypia or endometrial carcinoma
Warnings and Precautions
Adrenal insufficiency
: Patients should be closely monitored for signs and symptoms of adrenal insufficiency.
Hypokalemia
: Hypokalemia should be corrected prior to treatment and monitored for during treatment.
Vaginal bleeding and endometrial changes
: Women may experience endometrial thickening or unexpected vaginal bleeding. Use with caution if patient also has a hemorrhagic disorder or is on anti-coagulant therapy.
QT interval prolongation
: Avoid use with QT interval-prolonging drugs, or in patients with potassium channel variants resulting in a long QT interval.
Use of Strong CYP3A Inhibitors
: Concomitant use can increase plasma levels significantly. Use only when necessary and limit dose to 300 mg.
Adverse Reactions
Most common adverse reactions in Cushing's syndrome (≥ 20% : nausea, fatigue, headache, decreased blood potassium, arthralgia, vomiting, peripheral edema, hypertension, dizziness, decreased appetite, endometrial hypertrophy.
To report suspected adverse reactions, contact Corcept Therapeutics at 1-855-844-3270 or FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
Drug Interactions
- Drugs metabolized by CYP3A: Administer drugs that are metabolized by CYP3A at the lowest dose when used with Korlym
- CYP3A inhibitors: Caution should be used when Korlym is used with strong CYP3A inhibitors. Limit mifepristone dose to 300 mg per day when used with strong CYP3A inhibitors.
- CYP3A inducers: Do not use Korlym with CYP3A inducers.
- Drugs metabolized by CYP2C8/2C9: Use the lowest dose of CYP2C8/2C9 substrates when used with Korlym.
- Drugs metabolized by CYP2B6: Use of Korlym should be done with caution with bupropion and efavirenz.
- Hormonal contraceptives: Do not use with Korlym.
Use in Specific Populations
- Nursing mothers: Discontinue drug or discontinue nursing.
Please see the accompanying full Prescribing Information including boxed warning at
www.corcept.com/prescribinginfo.pdf
Please see the accompanying Medication Guide at
www.corcept.com/medicationguide.pdf
About Corcept Therapeutics Incorporated
Corcept is a pharmaceutical company engaged in the discovery, development and commercialization of drugs for the treatment of severe metabolic and psychiatric disorders. Korlym, a first generation GR-II antagonist, is the company's first FDA-approved medication. The company has a portfolio of new selective GR-II antagonists that block the effects of cortisol but not progesterone. Corcept also owns an extensive intellectual property portfolio covering the use of GR-II antagonists, including mifepristone, in the treatment of a wide variety of psychiatric and metabolic disorders. The company also holds composition of matter patents for its selective GR-II antagonists.
Statements made in this news release, other than statements of historical fact, are forward-looking statements. Forward-looking statements are subject to a number of known and unknown risks and uncertainties that might cause actual results to differ materially from those expressed or implied by such statements. For example, there can be no assurances that clinical results will be predictive of real-world use, or regarding the pace of Korlym's acceptance by physicians and patients, the reimbursement decisions of government or private insurance payers, the effects of rapid technological change and competition, the protections afforded by Korlym's Orphan Drug Designation or by Corcept's other intellectual property rights, and the cost, pace and success of Corcept's other product development efforts. These and other risks are set forth in the Company's SEC filings, all of which are available from our website (www.corcept.com or from the SEC's website (
www.sec.gov . We disclaim any intention or duty to update any forward-looking statement made in this news release.
CONTACTS:
Invvestor Contact
Charles Robb
Chief Financial Officer
Corcept Therapeutics
650-688-8783
Media Contact
Alissa Maupin
Communications Strategies, Inc.
973-635-6669
Health and Human Services Secretary Kathleen Sebelius said Thursday in Philadelphia that the federal government is doing all it can legally to alleviate drug shortages, but that manufacturers and market forces play the biggest role.
"Right now, there is a gap between demand and manufacturing capacity, which we cannot fix," Sebelius said after leading a panel discussion at the African American Museum that focused on health insurance access for women. "It really is market demand, and, frankly, drug companies are making decisions each and every day about which lines of drugs to run."
By David Sell
Inquirer Staff Writer
ORONTO - First Nations leaders say a health crisis is about to be unleashed on northern Ontario reserves because thousands of residents addicted to OxyContin will soon be cut off from the prescription opiate.
The maker of OxyContin, which is up to twice as strong as morphine, will stop manufacturing the drug in Canada at the end of the month. As of March 1, Purdue Pharma Canada will replace OxyContin with a new formulation called OxyNEO.
OxyContin, taken orally in pill form, is a long-acting form of the highly addictive opioid oxycodone. But when the pill is chewed or crushed, then injected or inhaled, it produces a "heroin-like euphoria," Health Canada says.
OxyNEO will also be made with oxycodone, but it's formulated to make abuse more difficult: the tablet is hard to crush and when added to liquid, it forms a thick gel that stops oxycodone from being extracted for injection.
Leaders of Ontario's Nishnawbe Aski Nation, or NAN, said that with no OxyContin available, those addicted to the drug will go into withdrawal.
"It scares me. It's going to be a catastrophe," said NAN Chief Stan Beardy, stressing that there is potential for a "mass involuntary opiate withdrawal" on the horizon.
"I don't think governments understand the severity of addictions we're talking about here," Beardy said in an interview Thursday.
Source: MicroChips
Coming soon, whether we like it or not! The
Financial Times reports:
A wirelessly controlled implant, which delivers precise drug doses into the patient’s body, has had a successful first clinical trial, bringing the possibility of the “pharmacy on a chip” that could transform drug delivery closer.
Researchers used the microchip device to give seven women with osteoporosis daily doses of a bone-strengthening hormone that was normally injected. The results were announced at the start of the American Association for the Advancement of Science annual meeting on Thursday.
The device could transform drug delivery and help usher in a new era of telemedicine – delivering healthcare over a distance – said Robert Langer, a professor at the Massachusetts Institute of Technology where the project started 15 years ago.
“You could literally have a pharmacy on a chip,” he said. “You can do remote control delivery, you can do pulsatile drug delivery, and you can…
Leerink Swann's Global Healthcare Conference 2012 featured two presentations and several more private discussions featuring Center for Drug Evaluation and Research ("CDER" Director Janet Woodcock, M.D., as well as her colleagues, Steven Kozlowski, M.D., CDER's Director of Office of Biotechnology Products, and Keith Own Webber, Ph.D., CDER's Deputy Director, Office of Pharmaceutical Science, Acting Director, Office of Generic Drugs. The trio presented a discussion "Biosimilars Take the Stage" followed by Woodcock's Keynote Address on "New Trends in Drug Regulation and Innovation", and several smaller question-and-answer-format meetings regarding these topics and more. This is part one of a two-part series and concerns the biosimilars presentations. Part two will be posted on Tuesday.
Leerink's biosimilars session was moderated by Joshua Schimmer, M.D. and Jason Gerberg, J.D./M.B.A. with co-moderators Seamus Fernandez and Joseph Schwartz. Kozlowski provided some preliminary remarks followed by a question-and-answer format by the moderators. Kozlowski explained that the purpose of FDA's first set of biosimilars guidances, which we initially reported on
here, was to set the scientific framework for approaching biosimilar product development and provide some interpretations of the new statutory terminology. Kozlowski explained that FDA decided to take a "totality of the evidence" and "stepwise approach", where the structural similarity of the biosimilar, as demonstrated by the sponsor, drives the development of the clinical program to support approval.
Woodock added that FDA has had a challenge to retrain their reviewers to think of biosimilar review as an "inverted pyramid." Typical drug review includes pre-clinical studies followed by increasingly large clinical studies, culminating in pivotal, larger-scale studies to demonstrate the safety and efficacy of the product that become the focus of the approval, i.e., the regular pyramid (pre-clinical on top, phase III clinical trials on the bottom . In contrast, biosimilar product development is envisioned to be characterized as spending a bulk of the resources up front, characterizing the innovator's product and the biosimilar (i.e., the top of the pyramid to lead to smaller clinical studies to demonstrate similarity or test for immunogenicity or other elements that require testing, not duplicating the safety and efficacy testing that was already conducted by the innovator.
JUDY WOODRUFF: House Minority Leader Nancy Pelosi, thank you for talking with us.
REP. NANCY PELOSI, D-Calif.: Thank you for having me.
JUDY WOODRUFF: This coming, evidently, agreement on the payroll tax cut. Speaker Boehner is saying today that this is necessary because of the president's failed economic policies.
REP. NANCY PELOSI: Well, how sad. This is necessary because our economy and our people need this boost. For individual families, 160 million American families, this will be a boost. From a macro economic standpoint the demand that is injected into the economy as people spend this money will be a job creator. Again, a temporary measure to help give us a boost. The same as the unemployment insurance extension. Economists tell us that that's an immediate jumpstart to the economy because of the macroeconomics of getting money, spending it immediately, injecting demand, creating jobs.
Does he wants us to get into a discussion of how we got here before, the Bush meltdown of our financial institutions, two unpaid for wars, a prescription drug bill that gave away the store for the pharmaceutical industry, a tax cut for the wealthiest people in our country, unpaid for.
I don't think that we want to go down that path, the American people have been there, seen it, they know. Whatever it is. there is a ground truth in our country that we have to help the middle class.
JUDY WOODRUFF: Do you worry though that this does take money out of the Social Security trust fund and that it may never be fully be refunded, repaid?
REP. NANCY PELOSI: No, I don't worry about that. I think that this should be the last year for it.
I do believe that other factors in economic growth are weighing in now and we see an improvement in our growth possibilities but I think one or two years, no, the trust fund can handle that.
JUDY WOODRUFF: If this, this is now off the table assuming the vote goes as I think you and the speaker are suggesting it will. What else can Congress get done this year?
REP. NANCY PELOSI: I don't know, you'd have to ask him because so far any initiative the president has put on the table for job creation they have said no to.
What's really sad is as we are meeting this week, they have a highway bill on the floor of the House. Traditionally, highway bills have been a bipartisan initiative for job creation. This is a bill, that has been, for the first time in people say 50 years, has not been put together in a bipartisan way, it is not a job creator, but a job killer for over half a million jobs will be lost if this bill were to become law and it's dangerous to the public safety. For many reasons, this would have been the wrong path to go down and this would have been an important initiative for job creation.
JUDY WOODRUFF: I know that there's a real disagreement about that. What about the president's budget, even there are bi-partisan groups out there applauding the President for getting a start tackling the debt, but in fact the president promised he was going to cut the deficit in half by this point in his presidency. This is not a budget that goes after entitlement reform and it uses what it calls ‘savings' from ending the wars in Iraq and Afghanistan that, again, bi-partisan outside groups are saying are really a gimmick, that isn't money that can really be counted on as savings.
REP. NANCY PELOSI: Well, you put several aspects of the budget on the table. Let me say that I'm proud of what the president's budget it. It's a statement of our national values, as to what is important to us as a country being reflected into our investment priorities in a budget. It talks about investments in our future, it talks about job creation, it talks about deficit reduction. It talks about lowering taxes for the middle class as an ongoing theme in his agenda. Many of these outside groups did not say the same thing when the Republicans put the savings from the overseas war in their budgets, so this is a legitimate place. The savings are there and can be used to offset other investments. But it is, again, a continuation of what the president started when he came into office and he made that statement, reduce the deficit, create jobs, lower taxes for the middle class, do so by investments in education and innovation into healthcare and to energy innovations for which he is in the lead. What's also important about it though and is important to note, is that when the new president made that statement he was basing his figures on those given to him by the Commerce Department of the previous administration which either didn't know or didn't tell what the actual fiscal situation was.
JUDY WOODRUFF: We are launching into an election year, in the last campaign Madam Minority Leader you became a negative symbol for the Democrats and the Republicans went after you with a vengeance, do you think they are going to do the same thing this year?
REP. NANCY PELOSI: Well it doesn't matter but what's important about that is they used money to win the election. They used money. Undisclosed, unlimited, special interest money, and that's why our focus is on disclose. We want to know where that money's coming from, we want people to stand by their ad. You have something to say? You're going to spend tens of millions of dollars to misrepresent the president's position on something, going to say and mischaracterize the record of a member of Congress, stand by your ad. So we want to disclose. We want to reform the system, after we win. We want to amend the Constitution to get rid of this Citizens United unlimited money in the system.
JUDY WOODRUFF: Well, what do you think about the fact the Obama campaign -- Democrats have embraced the system, the Super PACs, just as the Republicans have?
REP. NANCY PELOSI: No, I want to make the distinction. The Republicans' money was undisclosed. Undisclosed. The president is, I think, doing the right thing to win this election by disclosing what the source of the money is. That makes all the difference in the world when people know where the money is coming from, because if people have to stand by their ad they may not make such outrageous charges or they may not even put the money up. So, I think they president has to win, he can't go to a baseball game without a bat, and there is so much at stake for the American people in this election. But his is different from theirs in that his is disclosed, theirs was not, to the tune of about $150 million, as you said, directed at me.
JUDY WOODRUFF: You, I was mentioning that the Republicans used you, you became a negative symbol to the part of the Republicans in the last election, should Democrats do the same with Speaker Boehner or another Republican?
REP. NANCY PELOSI: I think the Democrats are running about the future. The Republicans have always got to attack sombody else because they're frankly bankrupt in terms of ideas for the future. We are talking about reigniting the American dream. The president will go out there and talk about what is at stake for the future. It isn't about attacking an individual because if you don't have the issues you attack the person. He has the issues. He has the values. Reignite the American dream, build ladders of opportunity for all who want to work hard, play by the rules, take responsibility. But we have work to do to do that, and we would hope to do it in a bipartisan way. I think that's what you'll see on the Democratic side. And winning the election to change the system to diminish the role of money in the campaigns so the people's voices and votes will decide the election, not the bankrolls of a very few people.
JUDY WOODRUFF: Another topic, the challenge to the healthcare reform law, the Affordable Care Act, coming up before the Supreme Court next month, are you worried about that?
REP. NANCY PELOSI: Well I think we're in pretty good shape constitutionally. I mean we wrote a bill understanding our responsibilities to the Constitution of the United States. You never know what happens in a court, but in terms of the substance and the constitutionality of it, we believe that we're on solid ground.
JUDY WOODRUFF: And if it were to be in part or fully overturned, what would that mean for the Congress?
REP. NANCY PELOSI: Well, let's be on the positive side. Even some of the most conservative groups, the Heritage Foundation for example, supported the individual mandate and that's really the essence of the case going to the Supreme Court. The individual mandate. That has had support from the right to the left on the political spectrum. But you know what, it's the third branch of government. They will act on their responsibilities and the substance before them. In terms of what we legislated, we feel very strongly and believe that its on strong constitutional ground.
JUDY WOODRUFF: A question, Madam Leader about the war in Afghanistan, there was a report in the New York Times last week, about an exhaustive and a really devastating report written by an Army colonel who went over to Afghanistan. His assessment is that this war has essentially been a disaster, the military leaders of the United States have not leveled with the American people saying the situation on the ground is much worse than what the American people have been lead to believe. My question is, first of all, are you familiar with the report and if you are, do you agree with his assessment, you've spent time looking at this?
REP. NANCY PELOSI: I've gone to Afghanistan many times in the course of our involvement there and my last trip was most optimistic. It was bearing fruit from the changed approach that President Obama has taken. You have to remember, that when the president took office, there had been no plan for Afghanistan for the full time, for the seven years before he took office. It's a very sad tale to say we went in, we routed the Taliban we did not defeat them, and we went to Iraq. The president came in with a new approach for how to transfer the responsibility for security in Afghanistan to the Afghan forces and that I see much more progress in the past year than I had in the ten years before.
JUDY WOODRUFF: Do you have confidence in what the military leadership is saying about how the war is going?
REP. NANCY PELOSI: From what I have seen there. Now let me say this, I think we will be honoring the president's announcement that we will be reducing our number of troops or changing their mission in this year, in 2013 the year to come, in order for us to leave in 2014.
JUDY WOODRUFF: Minority Leader Nancy Pelosi, thank you very much for talking with us.
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