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What Are Orphan Drugs Doing for Rare Cancers?

Lab technicians develop orphan drugs

When you boil it down, drug companies exist for two main reasons: to provide medication to those who are sick, and to make money for their owners or shareholders.

If somebody has a common condition, the drug company stands to earn large amounts of money selling their products. This means there is large incentive to bring these drugs into market through the U.S. Food & Drug Administration (FDA).

On the other hand, with rare conditions, diseases, or disorders, drug companies have a much smaller pool of people to whom they can sell their medications. For these drugs, there is little incentive to go through the rigors of FDA testing, which can be a years-long process and cost a lot of money. Even in those rare instances when such drugs are brought to market, they are often prohibitively costly, as the pharmaceutical company looks to recoup expenses and make a profit from that small group of people.

However, people with rare diseases need medication too, especially diseases like mesothelioma, which is an uncommon, life-threatening form of cancer that has an extremely poor prognosis. Fortunately, there is an alternative route pharmaceutical companies can take when they do not pursue FDA approval: orphan drugs.

What Are Orphan Drugs?

Orphan drugs are medications for those with serious, often life-threatening, rare diseases. Since there is not enough demand for real profitability in a rare disease, drug companies cannot afford to or do not want to bring their drug to market.

However, for those actually suffering from the rare disorders, the ability to access these drugs is paramount. That’s why President Ronald Reagan provided the FDA with another classification beyond traditional approval.

In 1983, the Orphan Drug Act was passed to help facilitate necessary drugs to the small numbers of people who suffer from rare conditions. This law allows drug companies to avoid the long and expensive approval process normally required – giving individuals with rare diseases a better opportunity to get treatments in a timely manner.

More recently, the 21st Century Cures Act passed in December 2016 allows grants to be used for orphan drugs during observational studies. These types of studies are often used to help researchers understand how a rare disease develops, giving them more information from which to come up with potential cures. The ability to use research grants in this manner increases the likelihood of finding an effective treatment that was not previously available.

Orphan Drugs vs. Other Drugs

As may be expected, the FDA treats traditional drugs and orphan drugs very differently.

For a traditional FDA approval, a drug must go through numerous steps of scientific testing, animal testing, and human trials. All of these trials are intended to demonstrate the drug’s safety and effectiveness. This entire process can take up to a dozen years and cost an average of $350 million per drug. Note that these time and costs add up regardless of whether the drug is ultimately approved or not, meaning that companies can rack up billions of dollars in research, development, and testing for drugs that never even make it to market.

In contrast, an orphan drug needs only to be shown to safe and effective in treating or preventing rare diseases or disorders. In essence, the company can skip some of the more tedious administrative steps of the process when seeking approval for a drug under the FDA’s Orphan Drug Designation program.

Going through the orphan drug process does not mean a drug is less safe. There is still a set of official approval criteria that drugs need to meet, as outlined in the Orphan Drug Act and the FDA’s regulations overseeing the program. Evidence of an orphan drug’s safety must be shown through “well-controlled studies,” and the evidence is still weighed by regulators before receiving final approval.

The expedited process provided through the Orphan Drug Designation program provides a huge incentive for drug companies to develop and test drugs for rare conditions and diseases. In many cases, these are drugs that might never even be pursued under the standard FDA approval process. However, by reducing the amount of administrative red-tape involved, the costs of producing these drugs is greatly reduced, making it easier for patients to access new treatments while still giving pharmaceutical firms a chance to earn a profit. It’s a win-win situation.

While there could still be some work done in this area, the process does seem to be working as intended. In 2015, the FDA approved 21 new orphan drugs, and the agency approved nine more in 2016. These may not seem like a lot of new drugs, but for the individuals whose lives literally depend on finding a cure for their rare disease, they are in some respects miraculous.

Orphan Drugs to Treat Rare Cancers

“Cancer” is not a singular disease; rather, it is a class of many different diseases that have some broad similarities. Some types of cancer are well known, like lung cancer and breast cancer, because they affect a lot of people. However there are a significant number of lesser known rare cancers that affect relatively small numbers of people. Mesothelioma is one of these.

Some orphan drugs currently being tested and/or used as cancer treatments, including as treatment for mesothelioma, are as follows:

  • Alimta®(Pemetrexed): A form of chemotherapy approved for pleural mesothelioma and non-small cell lung cancer. It is the most common chemotherapy drug used with mesothelioma.
  • Nintedanib: An oral medication being used for the treatment of mesothelioma. Given orphan status in December 2016.
  • Nexavar®: A chemotherapy for advanced renal cell carcinoma, hepatocellular carcinoma, and progressive differentiated thyroid carcinoma.
  • Sprycel®: A form of chemotherapy that treats acute lymphoblastic leukemia and chronic myeloid leukemia.
  • Velcade®: Another form of chemotherapy that can treat mantle cell lymphoma and multiple myeloma.

Of course, this is only a small sample. Over 400 orphan drugs have been approved since the program’s start in the 1980s. While some deal with other conditions like multiple sclerosis or hemophilia, rare cancer treatments are well represented among the full list.

Expanding the Orphan Drug Program

As hopeful as it is that there is an FDA-backed option for those with rare cancers to receiving medications, there is a downside.

Orphan drugs have a reputation for being extremely expensive. As The Washington Post investigated in 2016, orphan drugs get exclusivity in the market for seven years. The lack of competition makes prices soar.

In fact, the prices of orphan drugs have been doubling every five years. Out of the 10 top-selling drugs for 2015, seven were orphan drugs.

The good news is that those with rare diseases/rare cancers can have access to the medicine they need to potentially save their lives. Those medications may be expensive, but they could also be covered by insurance or other options. Ultimately, it’s better to have drugs for rare cancers be available than for not. The next step is expanding the program to include even more life-saving drugs, and hopefully making them more affordable for those who need them!