The role of a Research Consulting Organisation in educating healthcare professionals about new drug developments in rare diseases

A rare disease is defined as a condition that affects fewer than 1 in 2,000 people within the general population. However, over 3.5 million people in the UK are expected to be affected by a rare disease in their lifetime. With over 6,000 rare diseases already known globally, new conditions are being discovered on a regular basis.

Despite the frequency of new rare diseases being discovered and the number of people who are affected by them, there remains a lack of understanding among healthcare professionals about the drugs available to treat certain diseases. This is where research consulting organisations (RCO) must step in. 

An RCO, unlike the standard CRO counterpart, is a new type of research consulting and clinical service for biotech, pharma, and device innovators that delivers bespoke solutions which bring life-saving drugs to market sooner and more cost effectively.

An RCO plays a critical role in educating healthcare professionals about developments in rare disease drugs. By setting the stage for bringing new drugs to market through clinical trial management, they have a better understanding than most of available life-changing treatments. 

RCOs are responsible for managing the clinical trial process from start to finish, including developing the study protocol, recruiting patients, overseeing data collection and analysis.

They  have unrivalled expertise in regulatory, quality and compliance, pharmacovigilance, and medical information and their work with smaller programmes - for example, those developing molecules for rare and orphan diseases, innovative new devices, or cell therapies, who need bespoke solutions and have a wealth of data - puts an RCO in a position to educate healthcare professionals in a variety of ways.

Clinical trial participation 

One of the primary ways that an RCO can share knowledge about rare disease drug development is through clinical trial participation. RCOs must work closely with healthcare professionals to help to identify patients who may be eligible to participate in a clinical trial. This includes providing information about the design of the trial, such as the study protocol and the inclusion and exclusion data for participants.

This information is crucial for healthcare professionals, who are then able to determine if their patients are eligible for trial participation.

Sharing resources and learnings 

In addition to facilitating clinical trial participation, RCOs can also provide education and training to healthcare professionals regarding the latest developments in rare disease research.

By offering webinars, workshops, and other training programmes about new treatments, diagnostic tools, and clinical trial methodologies, RCOs are allowing healthcare professionals to stay up-to-date on the latest advancements.

This is particularly valuable for those who may not have extensive experience in treating patients with rare diseases.

The value of involving the broader healthcare community

An RCO also plays a critical role in disseminating information about rare diseases and drug developments to the broader healthcare community.

By working with patient advocacy groups and professional organisations, an RCO is able to raise awareness of rare diseases through the organisation of conferences and events, educational materials such as white papers, and engaging in PR activity. 

Patient registries 

Patient registries are databases that contain information about patients with a particular disease or condition where the data is often used in rare disease research to help identify potential study participants, track disease progression, and monitor treatment outcomes.

Healthcare professionals who have access to these databases are able to utilise the information to learn more about a particular disease and its treatment. 

Drug monitoring 

Finally, an RCO can work with healthcare professionals to monitor new drugs for rare diseases which are already in mainstream use for other conditions. These types of studies are designed to monitor the safety and effectiveness of the drug in a larger patient population and can provide valuable information about the long-term effects of treatment. Through collaboration with healthcare professionals in designing and executing these studies, RCOs can do trials with existing drugs signed off for use in larger cohorts to see if these can be utilised to treat rare diseases. 

Through their efforts, RCOs can therefore help to ensure that patients with rare diseases have access to the latest treatments and that healthcare professionals have the knowledge and tools they need to provide the best possible care.