Rare diseases: a problem that should not be underestimated
Rare diseases (or “orphan” because of the lack of interest in their study) represent a large group of diseases in the United States and consequently a major problem to be solved for national Healthcare.
It is estimated to be around 7000 and of these more than 4000 of genetic origin , often appearing in the first years of life and are seriously debilitating. Taken singularly they seem little common but all together they represent around 10% of the chronic illnesses.
An orphan disease is defined as a condition that affects less than 200,000 people in a given population: according to the Kakkis EveryLife Foundation in the United States they affect 1 in 10 people (about 25 million) and half of these are children. People with these rare diseases depend on researchers to develop new drugs (“orphan drugs”) that can improve the quality of life.
Challenges for the development of orphan disease treatment
Considering the thousands of drugs developed for more common diseases, we see that researchers are only affecting the surface of the world of orphan diseases. Until the last 5-10 years, pharmaceutical companies have first defined the need for a drug based on the prevalence of a condition, and then developed a drug treatment program based on that need.
Historically, given the nature and lack of understanding of some rare diseases, pharmaceutical companies were reluctant to develop drugs for these diseases but in 1983, the Orphan Drug Act created government incentives for pharmaceutical companies to pursue treatment developments in the pharmaceutical industry. the scope of the orphan disease. Since then, around 600 new drugs for orphan diseases have been approved by the Food and Drug Administration (FDA) and introduced to the market.
However, almost 95% of orphan diseases do not have FDA approved treatment and the need for new treatments is often defined on a patient-patient basis.
One of the biggest challenges with studying the orphan disease is finding patients who participate in clinical trials. Patients who have an orphan disease often have feelings of alienation, loneliness, loss of hope or defeat due to failed diagnosis or treatment and loss of power. These feelings can sometimes be attributed to misdiagnosis, be ignored by health professionals or geographically isolated from resources and information on potential treatments.
Social media’s popularity gains have, however, helped overcome some of these challenges. Previously, the development of orphan drugs depended on awareness groups and associations to offer patients a platform to express their concerns. The rise of social media in recent years has expanded and amplified the voice of patients carrying these rare diseases.
How can social media help healthcare?
The intersection between clinical studies and the use of social media has been a much debated topic due to the dramatic increase in Digital Marketing in clinical research. Social media communities offer a unique opportunity to address some of the concerns about recruiting and accessing information. There are three main ways in which social media can specifically help patients with orphan diseases:
Given the common nature of social media, the transmission of information can be almost instantaneous. People suffering from orphan diseases may feel lonely because they know something is wrong, but they know nothing about the symptoms of their illness or where to learn more about it. Social media can serve as a useful tool for sharing new advances in understanding and continuing education of a disease.
Never as in recent decades have patients become active consumers within the health field and what differentiates the experience between the two categories are the emotions that come into play in communication . In no other industry, you have to deal with such a wide spectrum of emotions, from indifference to preventive activities to the extreme, when fear is triggered. On the other hand, restrictions in advertising and health marketing make it more difficult to combat medical lies.
Providing patients with orphan diseases a platform where they can interact with others who have had similar experiences can create a sense of community. This can happen on one or more social media platforms (Facebook, Twitter, Instagram, etc.), through support groups, patient awareness campaigns or direct advertising.
Furthermore, the use of social media to connect an orphan disease population with others can enable early awareness of the disease, improve patient experiences when interacting with healthcare professionals, reveal potential recruitment opportunities for clinical trials and improve collaboration in the search for accurate diagnoses.
An important aspect of the spread of digital is that it can enhance the role of patients with the rare disease. This empowerment can come through testimonials geared towards physicians, raising funds for awareness of the disease, a feeling of self-esteem by participating in research, or knowing that there are others out there looking for information.
In particular, thanks to technologies, doctors improve:
- Patient care techniques;
- Disease prevention strategies;
- Health communication with patients, more direct;
- The organization of work activities;
- The possibilities of growth of one’s business.
As for the patient, instead, they improve:
- Contact with the attending physician, who is more empathetic;
- The assistance service, faster and multi-channel;
- Greater confidence built also on the testimonies of other patients;
- Waiting times, as the doctor could answer questions via online connection or, for example, prescribe a digital recipe rather than a paper one.