Speedy Drug Approvals Have Become the Rule, Not the Exception

By Margot Sanger-Katz

Congress has over the past few decades passed a series of special approval pathways for important drugs that treat life-threatening or rare diseases. This week, a new bill introduced in the House could add two more.

You might expect these existing special programs to represent a small fraction of new and unusual drugs. But data from the Food and Drug Administration show that a majority of recent drug development has been in therapies that qualify for at least one of these programs. About a third of recently approved drugs qualify for two or more of five special approval programs.

Dr. Kesselheim, an associate professor of medicine at Harvard Medical School, said the popularity of the expedited programs could be worrisome; the expedited pathways typically require less evidence of a drug’s efficacy or safety than the standard process. “These pathways were intended for very urgent circumstances of serious life-threatening conditions, or no other reasonable clinical alternatives,” he said. “They were originally intended as the exceptions, but now the exceptions are beginning to swallow the rule.”

The most recent approval category was created by legislation in 2012. So-called “breakthrough therapies,” like the blockbuster hepatitis C treatment Sovaldi, represented nine of the 41 drugs approved last year. Over 80 drugs in development have earned the designation and will go through the breakthrough approval process. Drugs that qualify can be approved through trials that are smaller or if they show changes in a biological marker of disease — like the size of a tumor or the results of a blood test — instead of the standard measures of symptoms or survival. The details are different, but three other expedited review paths share somewhat similar features.

The first such program, called Fast Track, was passed in the late 1980s to help speed the approval of treatments for H.I.V./AIDS. There’s also a program called Priority Review, and another, Accelerated Approval.

There’s also the Orphan Drug Act, passed in 1983 to encourage drugmakers to develop drugs to treat rare diseases. Such diseases were rarely a target of drug development before that because small patient populations made it hard for drugmakers to make back their investment. But the law made it possible for drugmakers to test the drugs in smaller patient populations and to obtain guaranteed market exclusivity. The path has become enormously popular, resulting in the development of (often extremely expensive) drugs for very rare genetic diseases, and also, increasingly,cancer treatments for select patient groups. Keytruda, a lung cancer drug produced by Merck, for example, was approved through the program last year.

The legislation introduced this week, the 21st Century Cures Act, is a wide-reaching bill aimed at improving medical innovation and reforming the F.D.A.’s approval process for drugs and medical devices. (My colleague Sabrina Tavernese has an article describing its ambitions and main features.) It includes two new special categories for drugs that can qualify for special status. One, an approval path for “dormant therapies,” is still uncertain.

The current legislation leaves a hole where language describing the process may go. A discussion document released earlier this year indicated that drugs qualifying for the program could get as many as 15 years of guaranteed patent exclusivity. A smaller program, for new antibiotic drugs, would give a speedier review for such drugs if they are marketed only for limited populations. The Senate is considering some similar measures.

The F.D.A. has published a detailed review of its special programs, which it says has helped the agency speed up review of new drugs. The F.D.A. now approves new drugs, on average, more quickly than European and Japanese authorities, with about 60 percent of new drugs approved by the F.D.A. first.

The popularity of the programs is no sign that drug companies are doing anything wrong. The F.D.A. determines which compounds qualify for the various programs, and it has been somewhat expansive in its definitions. Industry analysts also say that the companies have shifted their research priorities toward the special categories — an outcome members of Congress sought by giving such drugs enhanced incentives. And the bipartisan passage of additional approval routes suggests that members of Congress think it is still too hard for some important drugs to be developed and approved.

Senator Michael Bennet of Colorado, a Democrat who helped write the breakthrough therapy provision, says he has been surprised by the popularity of the program, but pleasantly so. “The Breakthrough Therapy pathway has been more successful than we anticipated,” he said. “That’s good news.”


B Vitamin Is Found to Help People Prone to Some Types of Skin Cancer

An inexpensive vitamin can help reduce the occurrence of common skin cancers in people prone to that disease, researchers reported on Wednesday.

In a clinical trial, people who took two pills a day of nicotinamide, a form of vitamin B3 available as a nutritional supplement, had a 23 percent lower risk of developing non-melanoma skin cancer than those who took placebo pills.

“It’s safe, it’s almost obscenely inexpensive and it’s widely available,” Dr. Diona Damian, the lead investigator of the study, said in a news conference organized by the American Society of Clinical Oncology, who said the findings could be put into practice right away.

“This one’s ready to go straight into the clinic,” said Dr. Damian, a dermatology professor at the University of Sydney in Australia. However, she said the vitamin should be used only by people who get frequent skin cancers, not by everyone.

The study will be presented at the annual meeting of the oncology society, which begins May 29 in Chicago. Abstracts for most of the nearly 5,000 studies were released late Wednesday, and a few results were discussed in a news conference organized by the society.

Investors and Wall Street analysts began poring over the abstracts late on Wednesday, looking for information that could affect the stocks of pharmaceutical companies.

In another study, an experimental drug for relapsed multiple myeloma being developed by Bristol-Myers Squibb and AbbVie reduced the risk of cancer progression by about 30 percent when added to two existing drugs.

Those who received the experimental drug, called elotuzumab, had a median of 19.4 months before their cancer worsened or they died, compared with 14.9 months for those who received only the two existing drugs, Revlimid and dexamethasone.

A spokeswoman for Bristol-Myers said it was premature to say when the company would apply for approval of the drug. Another trial is in progress.

The skin cancer prevention study focused on non-melanoma skin cancer, particularly basal cell carcinomas and squamous cell carcinomas. Together, these are the most common form of cancer in the United States, with the most recent study estimating that in 2006 there were 3.5 million cases in 2.2 million Americans. Most of these are curable through surgery or other techniques, though the surgery can leave scars and in some cases the cancers do become more serious.

The study involved 386 people in Australia who had had two or more such skin cancers in the previous five years. The average for the patients in the trial was eight cancers over the past five years; one patient had 52 cases.

Those who were randomly assigned to take the vitamin had an average of 1.77 new cancers in the year of the study compared with 2.42 cases for those in the placebo group, a risk reduction of 23 percent, after certain statistical adjustments. Those getting nicotinamide also had fewer cases of actinic keratosis, thick patches of skin that may become cancerous.

Dr. Damian said the effect of the vitamin seemed to disappear once people stopped taking it. She said that nicotinamide did not have the side effects, like headache and flushing, of niacin, another form of vitamin B3.

Protecting oneself from the sun is perhaps the most effective way to prevent non-melanoma skin cancers. Drugs called retinoids, which are related to vitamin A, can also be used, but they can cause birth defects and other side effects. Some studies have suggested that aspirin or other non-steroidal anti-inflammatory drugs can also be preventative.

Two experts not involved in the study said the results were “interesting and potentially important,” in part because nicotinamide had fewer side effects than the pharmaceutical alternatives for skin cancer prevention.

The experts, Dr. Clara Curiel-Lewandrowski, clinical director of the Skin Cancer Institute at the University of Arizona, and Steven Stratton, a pharmacologist there, said in a joint email that patients should nonetheless talk to their doctors before taking the vitamin.


What most moms want for Mother’s Day is to spend quality time with people they love. Whatever you do doesn’t need to be complicated or expensive.

To help you celebrate, we rounded up 14 Mother’s Day activities for seniors. Even if none of these ideas are perfect for your mom, this list is sure to get your creative juices flowing and help you come up with something she’ll love.

7 activities for seniors who like to go out

These activities are great for those who enjoy getting out of the house. They can be done together with lots of family and friends or one-on-one for some quiet time together.

1)Share a nice meal at their favorite restaurant. It doesn’t have to be fancy, just a place they really enjoy.

2)If the weather is nice, go to the park for a picnic or a walk.

3)Visit a botanical garden to take in the beautiful flowers. You could also bring a picnic lunch!

4)Take your sports fan to a ball game – whether it’s a local or national team, it’s sure to be a good time.

5)Walk leisurely through a nice indoor or outdoor shopping mall. Indoor malls are great during bad weather, there’s plenty of seating for taking breaks, places to get snacks and drinks, and lots of bathrooms. Plus, it’s fun to window shop!

6)Art lovers may enjoy visiting a nearby museum. A good conversation starter is to ask them about the pieces they like best.

7)For someone who is really excited about a hobby (examples: music, fishing, dancing), enjoy that activity with them or join in a class they take.
7 activities for seniors who like to stay in

These activities are perfect for those who enjoy staying home. Many are good for including family and friends and others are better one-on-one.

1) Cook their favorite meal or get takeout from their favorite restaurant. Arrange the food on nice plates and decorate the table a bit. You could even go all out with candles, flowers, and fancy china!

2) Relax together while watching a favorite movie or TV program.

3)For avid readers, consider reading aloud to them from a book of their choice. You could even take turns reading.

4)Bake cookies or cook a favorite dish together. Their participation can range from working side-by-side with you to helping mix a few ingredients to keeping you company in the kitchen and being your taste tester.

5)Enjoy a sparkling non-alcoholic “cocktail” as way to fancy-up afternoon snack time. Just add sparkling juice or bubbly water to lemonade, juice, or iced tea and put it in a pretty cup!

6)Play cards or a board game together.

7)Throw a casual potluck party where everyone brings a dish and spends the afternoon relaxing, chatting, and eating together.