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6 Tips About Breakthrough Drugs: For Example, Should Breast Cancer Patients Try to Get Palbociclib?

Breakthrough drugs are widely covered in the media. After hearing about these new drugs, patients and their families are always asking me how they can get access to the new medicine or participate in a clinical trial of the new drug. Let's look at a current example.
08/12/2014 12:38pm ET | Updated October 12, 2014
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Breakthrough drugs are widely covered in the media. After hearing about these new drugs, patients and their families are always asking me how they can get access to the new medicine or participate in a clinical trial of the new drug. Let's look at a current example.

Widespread breast cancer is a serious incurable problem. But with hormonal therapy such as tamoxifen or aromatase inhibitors (like letrozole), it has turned into a chronic disease that patients can live with for several years. However, patients become resistant to tamoxifen or aromatase inhibitors and must then proceed on to other treatments including hormonal medications, chemotherapy or radiation therapy.

Any new advance that can lead to longer and better life for these women surely must represent a breakthrough. Now new results suggest that a new pill, palbociclib, might be an important advance.

Because of very impressive results, this drug has been given breakthrough designation by the FDA. If the encouraging results continue, will the drug be a breakthrough, or will it be just a small improvement?

In the most recent study reports, women who took either the daily oral standard drug letrozole plus a palbociclib pill for three weeks out of every monthly cycle had control of the cancer for twice as long as patients on letrozole alone! However, survival of the patients was not significantly longer on palbociclib (but the final results are not yet in).

Were there any side effects? Thirteen percent of the women taking the new drug with letrozole dropped out of the study, compared to only 2 percent of women on letrozole. A side effect fatigue may have accounted for this difference. Fatigue is one of the worst side effects because it is so difficult to manage and reduces a person's quality of life.

Another factor to consider is: What will this drug cost patients? If it is approved by the FDA (and I hope the results continue to be this good or better and that it will be approved), and if it were to cost about $10,000 per month (like so many of the new cancer fighting drugs are costing), many of my patients would not be able to afford this medicine or might be poorly compliant with taking it.

What are my tips for you about breakthrough drugs?

• When you hear about a new drug for your condition, write down the name, search for it on the Internet to learn more, and then ask your physician about it.

• Ask your physician about access to clinical trials. These studies can provide patients with access to a new drug before the FDA approves it and before it becomes widely available. For more information about clinical trials and how to get into them, see my book Surviving American Medicine.

• If a new drug is approved, search the Internet to learn about side effects and patients' comments about using the drug. Then discuss them with your doctor to get the risks and benefits as they apply to you and your condition.

• If you want to take a new drug, but the price is very high, ask your doctor and your pharmacist if they know of discounts or free drug that can be made available to you by the drug manufacturer, or by foundations that can help patients. Do not be embarrassed to ask! Some companies provide help or free drug even if your income is over $100,000 per year. If they do not know, contact the drug manufacturer or look up information on their website.

• Continue to be encouraged and optimistic about new breakthrough drugs. I will highlight many of them in my blog, but your awareness of them can help you and friends or family challenged by serious disease to get up-to-date treatments with better outcomes.

So I truly hope that with these tips you can have access to any new drug that can help your condition. I hope palbociclib and many other new anti-cancer drugs will continue to give good results, be approved by the FDA, and be affordable to all patients who need them.

Dr. Presant has no relationship with any company that manufactures any drug mentioned in the article.