Rare disease research has come a long way thanks in part to the passage of the Orphan Drug Act (ODA) of 1983. In the 40 years since, the biopharmaceutical industry has steadily increased its research and development efforts. Consider that 31% of drugs currently in development aim to target rare diseases, according to the Tufts Center for Drug Development, which is a significant increase from just 2010 when only 18% of drugs in development were for rare diseases. 

Even so, challenges remain. Rare diseases affect fewer than 200,000 people worldwide, so recruiting patients for clinical trials presents obstacles. Furthermore, with the lack of available participants, the data collected is often inconclusive, raising more questions than answers. 

Financial obstacles are another primary concern. Because of the risks involved and limited access to patients, many companies aren’t willing to invest the millions of dollars necessary to complete clinical trials. Even if a successful drug is identified, the potential population who could benefit is so small, there’s little room to make a profit. 

Despite these barriers to success, clinical research into rare diseases is vitally important. And new technologies are making it easier for biopharmaceutical companies to benefit financially. Case in point: Clinical data cloud platforms.

In a recent guest column for Clinical Leader, Venu Mallarapu, a business technology leader with more than 25 years of industry experience, discusses how cloud technology can make a positive impact on rare disease research, particularly in terms of data collection. More specifically, Mallarapu argues that clinical data cloud platforms can address common data-related headaches, including but not limited to:

• Poor quality data
• Privacy concerns
• Lack of interoperability
• Lack of robust data storage, analysis, and management tools
• Missing data across medical records
• Siloing of crucial patient data

Although these impediments will likely remain for the foreseeable future, clinical cloud data platforms may be able to improve things significantly. Here’s a closer look at 5 of the benefits they offer:

1) Data aggregation

One of the biggest challenges that rare disease researchers face is the compartmentalization of data. Essential patient information is often split up across institutions, registries, and public databases. Tracking it down and analyzing it all can be a Herculean task. Clinical cloud data platforms aggregate information from these disparate sources, making it easy to collect and interpret.

2) Standardization and harmonization

Rare disease-related data is hard to collect because of the small patient populations involved. Standardizing that information can be even more difficult because of varying collection protocols and approaches to data storage. A clinical cloud data platform makes it easier to secure consistency and comparability across different sources. It can even be used to align with specific industry standards, including CDASH, OMOP, and SDTM.

3) Data validation and curation

Many biopharmas use multiple software programs to collect, track, and analyze clinical data. While this approach can be effective in traditional pharmaceutical research, the lack of quality data in rare disease research increases the risk of missing data sets, not to mention inaccurate findings. Mallarapu argues that a clinical cloud data platform can serve as a home base for all of your team’s data inputs. With everything in one place, it’s easier to maintain accurate and reliable data sets. 

4) Transparent data governance

Patient privacy is a major concern for biopharma researchers, but many of the data-related headaches we mentioned earlier can put research participants at risk. The patchwork system that many companies use (for example, storing various sets of data across multiple silos,) increases the likelihood of data breaches and privacy concerns. Clinical cloud data platforms have transparent policies and clear data governance structures. In other words, they help protect patient privacy and promote ethical data sharing.

5) Collaborative research environment

Cloud platforms allow decision-makers, like researchers, research institutions, and governments to collaborate on rare disease research. By acting as a central information repository, they also facilitate communication and collaboration all over the world.  Collaboration is key for the development of new drugs, particularly when it comes to rare diseases. Research partnerships increase statistical accuracy, help eliminate bias, and further knowledge in the medical field. 

The bottom line

No technology can provide a single solution for the challenges that rare disease researchers face. But by embracing clinical cloud data platforms, it’s possible to address many of the data concerns that the industry is currently grappling with. Hopefully the increasing prevalence of unified clinical data cloud platforms can help lay the foundation for quicker drug development, and in turn, hope for rare disease patients and their families.