Will we be able to get stem cell therapy under the new rules?

barbara

Pioneer Founding member
HMMMMMMMM Now that the FDA has declared our own stem cells to be drugs, shouldn't we be allowed to have treatment with them if we are diagnosed with a terminal disease? I am sure that there is some loophole that will exclude autologous stem cell treatment.

By Lisa Richwine

WASHINGTON (Reuters) - The U.S. government finalized rules on Wednesday meant to make it easier for seriously ill patients to gain access to unapproved medicines when they have run out of other options.

The regulations spell out more clearly which patients are eligible for special access to experimental medicines, the Food and Drug Administration said. They also clarify when manufacturers can charge patients for the drugs.

The move aims to "achieve a balance between the need to gather information to demonstrate whether drugs work and the fact that critically ill patients urgently want access," FDA Commissioner Margaret Hamburg told reporters.

Under the rules, companies may obtain FDA permission to charge patients in order to recover their costs under certain circumstances. The goal is to encourage more companies to provide the medicines to appropriate patients, said Dr. Janet Woodcock, head of the FDA's Center for Drug Evaluation and Research.

Patients can try experimental drugs through clinical trials, but enrollment in the studies is limited, creating a push for other options.

Experimental medicines have been available to patients outside clinical trials under certain circumstances since the 1970s. Tens of thousands of patients with AIDS, cancer and other conditions have used medicines through those programs, the FDA said.

Patient groups have long been pushing for the FDA to expand access for desperate patients.

The Abigail Alliance for Better Access to Developmental Drugs, an advocacy group, said the FDA needed to go further to significantly boost access.

The new rules are "just codifying the status quo," said Steve Walker, the alliance's co-founder. Companies often see providing a drug to a patient outside of clinical trials as "an economic burden and a regulatory risk. We don't expect there to be much of a change in availability."

In 2008, the Supreme Court rejected an appeal by the Abigail Alliance and another group arguing that dying patients have a constitutional right to access experimental drugs that are not FDA approved.

Lynda Dee, a founding member of the AIDS Treatment Activist Coalition, said the FDA's effort would be "helpful to doctors and patients alike" by giving a clear picture of their options. Speaking on an FDA conference call with reporters, Dee said she was working with a group of AIDS patients developing a bid to try an experimental drug.

FDA spokeswoman Karen Riley said the agency estimated the new rules would prompt a 50 percent increase in individual patient applications for experimental drugs, which now average 659 a year.

Applications for between 10 and 100 patients are expected to jump 25 percent to 50 percent. About 55 applications of that size were received between 2000 and 2002, Riley added.

Details were posted on the FDA website here

(Reporting by Lisa Richwine; editing by Andre Grenon)
 

barbara

Pioneer Founding member
The slightest bit of manipulation makes your own stem cells prescription drugs according to the FDA. Please join the International Cell Medicine Society (ICMS) www.safestemcells.org We are fighting this ruling and need everyone to join and contact the FDA. There are instructions at the link I just gave you with the e-mail for the FDA. You should continue to contact the FDA on a regular basis. It cannot be accomplished with just a few of us. We need a lot of people to make this grassroots effort successful.
 

barbara

Pioneer Founding member
Thank you to Dr. Young for sending this.


8/14/2009
FDA Finalizes Rules on Access to Investigational Drugs
Legal News Alert: Life Sciences


On August 13, 2009, the U.S. Food and Drug Administration (FDA) published two final rules concerning access to investigational drugs and charging patients for investigational new drugs. The first rule, which addresses access to investigational drugs, does not change the methods by which seriously ill patients who are unable to participate in clinical trials can gain access to investigational therapies, but rather clarifies existing rules that have allowed some patients to access investigational drugs for several years and expands the class of patients that may have access. The second rule clarifies the conditions under which it is appropriate for drug sponsors to charge patients participating in clinical trials for investigational new drugs. Both rules take effect on October 13, 2009.

The first rule, entitled ?Expanded Access to Investigational Drugs for Treatment Use,? proposed at the end of 2006, clarifies the criteria for access to investigational drugs, enumerates the requirements for access submissions, establishes safeguards to protect patients from adverse side-effects, and implements mechanisms for maintaining meaningful data about treatment use and results. Per the rule, those who may be granted access to investigational drugs include individuals with a serious or immediately life-threatening disease and for whom there is no comparable satisfactory alternative therapy; intermediate-size patient populations comprising individuals who are ineligible to participate in clinical trials or whose disease is so rare that a drug is not being developed; and larger populations under a treatment protocol or in a trial conducted as part of an Investigational New Drug (IND) application.

The expanded access rule specifies that drug sponsors are responsible for submitting IND safety reports (and annual reports when the protocol continues for one year or more) and for providing treating physicians with necessary information to maximize the benefits and minimize the risks of treatment. Physicians who administer the treatments, who are considered ?investigators? for purposes of this rule, must report adverse drug events to the sponsor, ensure that informed consent requirements are met, and maintain accurate case histories and drug disposition records. In support of the effort to help seriously ill patients gain access to these therapies, the FDA has launched a Web site (http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/
AccesstoInvestigationalDrugs/ucm176098.htm) where patients and their physicians can learn about options for access to investigational drugs.

The second rule, entitled ?Charging for Investigational Drugs Under an Investigational New Drug Application,? amends the existing rules concerning when drug sponsors may charge patients for investigational drugs. Though drug sponsors for years have been able to apply for permission to charge clinical trial participants for drugs, the rules were unclear about the circumstances under which it was appropriate and the types of costs that could be recovered. This rule specifies that a sponsor who wishes to charge a clinical trial participant for a drug must show that the drug may provide a significant advantage over other available treatments (as demonstrated by the trial), that the data from the trial is essential to demonstrating the drug's safety and efficacy, and that charging participants is essential because the cost of the drug is ?extraordinary to the sponsor.?

This second rule also specifies that sponsors may charge clinical trial participants to recover only the direct costs of making an investigational drug available, which include those costs per unit to manufacture the drug for the trial or to acquire the drug from another manufacturer and shipping and handling costs. If the investigational drug is used to treat intermediate-sized patient populations or large populations under a treatment protocol or IND, as authorized in the companion regulation, sponsors also may charge enough to recover the costs of monitoring the expanded-access protocol or IND. Finally, the rule also states that the FDA will withdraw authorization to charge for the investigational drug if the agency determines that charging is interfering with the development of the drug for marketing approval or if the criteria for the authorization are no longer being met.

In a press release issued by the FDA, Dr. Janet Woodcock, the director of the FDA's Center for Drug Evaluation and Research (CDER), said, ?[T]he final rules balance access to promising new therapies against the need to protect patient safety and seek to ensure that expanded drug access does not discourage participation in clinical trials or otherwise interfere with the drug development process.?

The final rules can be accessed at http://www.accessdata.fda.gov/scripts/
oc/ohrms/dailylist.cfm?yr=2009&mn=8&dy=13. Foley will continue to provide updates on important FDA-related topics.
 
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