Diversity heals: PTC therapeutics gives rare diseases a voice
Diversity heals: PTC therapeutics gives rare diseases a voice
In view of the German diversity tag 2025, PTC Therapeutics emphasized the importance of diversity and inclusion for health care for people with rare diseases. Kristina Kempf, Country Head for Germany, Austria and Hungary at PTC Therapeutics, emphasizes that these values are not only entrepreneurial obligations, but also crucial for success in the development of innovative therapies. PTC Therapeutics has been involved in researching and marketing solutions for rare diseases for over 25 years.The company sees the stories of patients as a source of inspiration that shape the further development of their therapies. Many of the more than 6,000 known rare diseases, of which 36 million EU citizens are affected, are often difficult to diagnose. The path to corresponding therapy can be long and stony. PTC would like to change the invisibility of these people and give them a voice to involve their perspectives in the development process.
The role of patient organizations
Patient organizations play a crucial role in the development of therapies for rare diseases. According to a study by IQVIA, these organizations are able to decisively promote the medication development process. Nitisinon is an example of the influence of committed patient organizations: Originally approved for the treatment of tyrosine anemia in 2002, it was later further developed for Alkaptonuria after a committed organization was able to finance a new study. In 2020, the EMA approved the Nitisinon treatment for Alkaptonuria.
The challenges are great. It is estimated that about half of the 30 million people in Europe have no diagnosis with a rare illness, which again underlines the need for close cooperation between pharmaceutical companies and patient organizations. In 2022, over 850 candidates for active ingredients were in a clinical test for rare diseases, but there are no approved treatments for 95% of these diseases.
trends in the pharmaceutical industry
The patient organizations worldwide represent around 2.8 million patients with rare diseases, which requires enormous needs and illustrates the urgency of their work. According to the PatientView Report of 2024, a quarter of the pharmaceutical companies decided to enter into closer cooperation with ten or more patient groups. Against this background, biotechnology companies have the best reputation, whereby 61% of patient groups assess their cooperation as "excellent" or "good".
In summary, it can be said that diversity and inclusion not only represent ethical norms, but also decisive factors for progress in therapy of rare diseases. PTC Therapeutics emphasizes the need to hear the votes of patients to develop innovative solutions and thus improve the quality of life for people with rare diseases.For more information on this topic, readers can report the reports on OTS , iqvia and read PM-Report
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