A variety of factors can make it difficult to conduct traditional full-scale clinical trials for new treatments of rare cancers. Consequently, because so little information is available, treatments for rare oncology patients are inadequate or nonexistent.
Luckily for both patients and researchers, the FDA and the European Medicines Agency (EMA) have introduced regulations that expedite review and approval of certain investigative drugs.
Expedited FDA Programs
It’s an exciting time for research in the US, thanks in part to regulatory efforts to speed up the treatment approval process. In fact, more than half the novel drugs to gain FDA approval in 2015 used at least one of the following expedited pathways:
- Fast Track
- Increases level of communication with FDA
- Allows for rolling review
- Only expedited review program that allows the review of non-clinical data to support the designation
- Designation can be rescinded if the drug no longer meets the qualifying criteria
- Breakthrough Therapy
- For drugs with preliminary clinical evidence of substantial improvement in at least one clinically significant endpoint over existing therapies
- Designation can also be rescinded if the drug no longer meets the qualifying criteria
- Accelerated Approval
- Early approval of a drug that is an improvement over existing treatments based on surrogate endpoints or other clinical measures
- Drug must undergo post-marketing confirmatory trials following approval
- Priority Review
- Calls for review within six months in contrast to the usual ten months
- Reserved for situations where the Center for Drug Evaluation and Research has determined that the drug has the potential to provide a significant advance in medical care
Options for Faster EMA Review
Like its American counterpart, the EMA also offers a number of programs in the European Union:
- Accelerated Assessment
- Product is expected to be of major public health interest
- Reduces review time from 210 to 150 days
- Conditional Marketing Approval
- For drugs that address seriously debilitating or life-threatening diseases, emergency threats, or orphan indications
- The potential benefit of immediate availability to public health must outweigh the risk that additional data is still required
- Like Accelerated Approval in the U.S., requires post-marketing confirmatory trials
- Valid for one year and must be renewed annually until full approval is granted
- Exceptional Circumstances
- Cases where applicants are unable to provide comprehensive clinical data due to rarity of the disease, present state of scientific knowledge, and/or ethical concerns
- Must include specific procedures to monitor safety
- Approval is for five years but also requires annual review by the Committee for Medicinal Products for Human Use
- Orphan drugs must still meet the additional criteria for orphan designation
- Priority Medicines
- New option that went into effect in early 2016
- Drugs that promise major therapeutic advantage over existing therapies or benefit patients without treatment options
- Based on early clinical data
- Researchers in the academic sector, as well as small- and medium-sized businesses, can apply for this status earlier on the basis of compelling non-clinical data, as well as tolerability data from clinical trials
- Like Accelerated Assessment, can reduce review time from 210 to 150 days
The Future of Rare Oncology
Pending legislation promises to continue the trend of increasingly progressive regulatory policies. If passed, such changes may boost the amount of rare oncology research globally. If you’re curious about what’s on the horizon for the discipline, be sure to check out our recent webinar.