Patients make their voices heard in drug approval process

When federal regulators rejected Genzyme’s experimental multiple sclerosis drug late last year, disappointed patients — who were counting on the powerful new medicine — quickly mobilized.

They filed petitions with the Food and Drug Administration demanding patients be given permission to take the drug, called Lemtrada, so long as regulators and their doctors explained the risk of side effects.

“I was running out of treatments, so I really wanted another option,” said Melissa J. Burdick, who has tried several other drugs since being diagnosed with the autoimmune disorder 14 years ago.

“This is the closest thing to a cure for MS that’s out there,” said the Waterford, Conn., resident, who noted Lemtrada was available in Canada and Europe.

Burdick and others who objected to the FDA’s ruling joined a growing movement: newly empowered patients campaigning to influence drug approvals — something that used to be left to regulators and manufacturers.

“People want to be able to make their own decisions,” said Lorraine Heidke-McCartin, a Hanson breast cancer patient who has lobbied for state “right to try” laws, which give patients greater access to drugs in early stage clinical trials. “You need to have patients at the table.”

This kind of patient push is happening throughout the drug industry. And it’s making a difference.

Leukemia patients began phoning and writing letters to regulators hours after Cambridge’s Ariad Pharmaceuticals Inc. halted sales of its drug Iclusig last year because of potentially lethal side effects. The patients, and a group of doctors, wanted assurances that people suffering from leukemia would still be able to get Iclusig while the FDA reviewed new clinical data.

Parents of boys stricken with Duchenne muscular dystrophy, a devastating genetic disorder that causes progressive muscle weakness, have pressed the FDA to speed approval of experimental treatments being developed by Sarepta Therapeutics Inc. of Cambridge and other drug makers.

“We’re seeing a trend of people getting more involved in their health care decisions,” said Michael Ringel, a partner and managing director at Boston Consulting Group who focuses on health care business. “Because of the Internet, it’s a lot easier for groups to self-organize. That is helping to drive the increased involvement of patient groups.”

Their efforts are being rewarded in many cases, though FDA officials will not say what role, if any, such advocacy plays in deliberations.

Last month, the agency reversed the decision on Lemtrada and approved it for US sale to patients who had not responded well to two or more MS drugs. Like other MS medicines, Lemtrada’s label will include a warning that it may increase the risk of malignancies such as thyroid cancer.

The Lemtrada ruling hinged partly on a dispute over how Genzyme designed its clinical studies. The FDA changed its ruling only after the Cambridge biotech submitted additional information sought by the agency on why MS patients in its trials were told which of two drugs they were taking. Often, the FDA prefers patients don’t know what they’re taking in trials.

“The company provided additional information that addressed the concerns we communicated to them in our response to the initial application,” FDA spokeswoman Sandy Walsh wrote in an e-mail. “This additional information contributed to our conclusion that the application provided substantial evidence of effectiveness for Lemtrada.”

Separately, the FDA last year started a program that allows leukemia patients who had been buying Iclusig commercially to continue receiving it — with their doctors’ permission — after the drug was pulled from the market. Sales were eventually allowed to resume for a narrower set of patients. The FDA has also stepped up programs for fast-tracking reviews of designated life-saving drugs for Duchenne and other diseases for which there are few, if any, treatments.

“Patients are more informed, and I think [the FDA] feels like they have to listen,” said Heidke-McCartin. “People are making it known that they think things are taking way too long and they’re starting to say ‘enough is enough.’ These are desperate people, but they aren’t crazy.”

Patients and their advocates have also been playing a bigger role in everything from funding drug discovery to shaping how clinical studies are conducted.

The Cystic Fibrosis Foundation parlayed a $150 million investment in Kalydeco, a drug developed by Boston’s Vertex Pharmaceuticals Inc., into a payout exceeding $3 billion for selling its royalty rights. Patient advocates, meanwhile, have been pushing for greater participation by women and minorities in clinical trials, especially for studies of heart drugs and devices.

But patient involvement in drug approval decisions — sometimes aligned with investors with a financial stake in the outcome — represents the most important, and potentially most controversial, arena.

“The concern of the FDA is to make sure the drugs are safe for patients,” said life sciences adviser Leora Schiff, principal at Altius Strategy Consulting in Somerville. “For patients, the issue is whether they’re willing to assume the risk of taking a drug before it’s approved. With a lot of these diseases . . . in order to address the underlying condition, you’re dealing with drugs that have toxicity.” Put another way — they may work, but not without terrible side effects.

While companies sometimes allow patients to take unapproved drugs under compassionate use programs when there are no other options, they are not the norm. Manufacturers also worry that if side effects kill even one patient, an experimental drug’s reputation could be ruined before it is submitted for regulatory review.

Patient lobbying has a potential upside for drug makers, but companies have to be careful to keep their distance. If a manufacturer were to coordinate a drug application with patient efforts, it could antagonize regulators. Investors, however, have fewer qualms about getting involved in the approval process. Some are becoming more vocal on behalf of patients.

“We were in a position to bring many of these voices together in support of” Lemtrada, said Chris DeMuth Jr., a partner at Rangeley Capital in New Canaan, Conn., who invested in Genzyme and worked with patients who submitted petitions to the FDA. “We are pleased that the FDA reversed their decision, and believe that Lemtrada will be an invaluable tool to help those suffering from MS.”

Robert Weisman can be reached at robert.weisman@globe.com. Follow him on Twitter @GlobeRobW.