Perhaps nothing triggers frustration and hope in patients like prescription drugs.
Consider the hepatitis C drug , infamous for its $84,000 retail price tag for a 12-week course.
Despite the price, the drug embodies significant hope. It, and other , have vastly improved treatment of the disease.
potential was confirmed during clinical trials when it was tested on humans as part of the drug . Many hepatitis B patients probably wish they could have participated.
Today, I鈥檒l explain how you can find of drugs that could treat your condition 鈥� but access isn鈥檛 a sure thing. The trial could take a year or more of your time. And more often than not, the experimental drug doesn鈥檛 work.
of the medicines that enter clinical trials are approved by the U.S. Food and Drug Administration, according to the Pharmaceutical Research and Manufacturers of America, known as .
鈥淭here is hope,鈥� says , a pharmacy professor at University of the Pacific who conducts clinical trials himself. 鈥淏ut there is no guarantee.鈥�
Q: I have a rare muscle disorder and have been looking for clinical trials for five years, but most of the stuff I find is in St. Louis or Atlanta or elsewhere. Do you have any tips on finding one I can participate in?
A: This question comes from Michael Urbach, 59, who has , which has progressively weakened muscles in his arms, legs and throat. There is neither a cure nor a standard treatment for the disease.
Urbach, a resident of Oakley in Contra Costa County, tries to remain positive, but is 鈥渁nxious to try something,鈥� he says.
So he did what experts suggest you do first: He asked his doctor about clinical trials.
鈥淗e told me, 鈥榊ou have to go online and find something鈥�,鈥� Urbach says.
Like Urbach, you鈥檒l probably end up doing a lot of your own research to find a clinical trial if your doctor can鈥檛 help. But beware 鈥斅爐he volume of information available online can be daunting, and the quality questionable.
So rely primarily on sites that end in .org, .edu and .gov, says Danielle Lavieri, a spokeswoman for the (CISCRP). Those domains tend to be less commercial.
The most comprehensive online compilation of clinical trials is at , a government-run website that allows you to search by disease and location.
But the language on the site can be technical and the site difficult to navigate.
鈥淵ou鈥檙e asking people to search a non-user-friendly site and then to understand what they鈥檙e reading,鈥� says Danielle Hicks of the , based in California.
Hicks suggests contacting a patient advocacy group specific to your disease. Many offer personalized searches for clinical trials, usually for free, she says.
CISCRP, too, performs custom searches and suggests trials that meet your criteria. Visit or call 877-MED-HERO.
allows researchers to contact you directly after you鈥檝e registered if they think you鈥檙e a good match for their study.
You can also get in touch with a drug company directly if you know it is developing a medication that may help your condition, Shah says.
And don鈥檛 discount your own network. , who was diagnosed with stage IV melanoma in August, found leads on a Facebook support group.
鈥淚鈥檝e learned a lot from that group of people,鈥� says the 37-year-old Santa Cruz resident.
She participated in one clinical trial and is now searching for another.
If you鈥檝e found a trial that you think is a good fit, reach out to the researchers. The CISCRP website (CISCRP.org) offers a list of important questions to ask in its online 鈥�.鈥�
You can also find a 鈥溾�� there. Read it.
For instance, did you know that you can leave a trial before it鈥檚 over? Let鈥檚 say you鈥檙e having unpleasant side effects, Lavieri says. 鈥淎 lot of people may think they鈥檙e stuck,鈥� she says. 鈥淵ou have the right to drop out at any time.鈥�
Even if you find a trial that may work for you, you still may face obstacles:
鈥淥f all the clinical trials I have found, none of them are here,鈥� Urbach says. 鈥淓verything is out of state or in a different country.鈥�
If you find a trial that requires travel, ask if you will be reimbursed for travel costs, Lavieri suggests.
There are groups that help cover costs associated with clinical trials, such as (lazarex.org).
Beyond travel costs, consider the support system of family and friends you鈥檒l need nearby, Soderlund advises.
鈥淵ou have to think about, 鈥榃ho do I know in the city where I鈥檓 going to get treatment?鈥欌�� she says. 鈥淲hat kind of support am I going to be able to get if I get sick after treatment?鈥�
PLACEBO: In rare cases, all participants in a trial receive the experimental drug, says Jocelyn Ulrich of PhRMA.
More commonly, some participants receive the trial drug and others get a placebo 鈥� a substance or treatment that looks like the experimental treatment but has no therapeutic effect.
If a disease is life-threatening or dangerous, however, participants will never聽simply receive a placebo, Shah says. 鈥淵ou cannot not treat someone,鈥� he says.
Instead, all participants will receive at least the 鈥渟tandard of care,鈥� the best treatment for a specific condition that鈥檚 already approved and available, Ulrich says.
In these trials, you may receive only the standard of care, or you may receive the standard of care plus the investigative drug, she says.
鈥淵ou鈥檙e not guaranteed to be on the study drug,鈥� Hicks says.
PREVIOUS TREATMENT: Your previous medical treatments, even routine chemotherapy, may disqualify you from participation.
鈥淢ost adults don鈥檛 look at clinical trials until they鈥檙e in desperate mode,鈥� Hicks says. 鈥淏y that time, they may have had treatment that will disqualify them.鈥�
EFFECTIVENESS: Remember, the trial drug might not work, it might not work for you, or it might cause unintended side-effects that require the trial to be shut down.
鈥淚t may work. It may not work,鈥� Shah says, 鈥渂ut you have a chance you didn鈥檛 have before.鈥�
This story was produced by , which publishes , a service of the .