A Global Perspective is Crucial to Improve Rare Disease Care, Part 2

This is Part 2 of a two part story. Check out Part 1 here.

The loss of his child through Edwards Syndrome helped Dr. Harsha Rajasimha understand the suffering and pain of families affected by a rare disease. Becoming an advocate for rare diseases was a natural career change. According to a recent article in Genetic Engineering and Biotechnology News, Dr. Rajasimha saw an urgent need to fill the unmet needs of rare disease patients.

After his experience as head of the Organization for Rare Disease (ORDI USA) he founded IndiaUSrare with the goal of bridging the United States with the Indian subcontinent.

IndoUSrare’s goal is even more ambitious. The company is focused on educating and training patient advocates to provide current information to patients and their families that will aid clinical research and the discovery of treatments.

The doctor created Jeevatrials at a later date. It is being used in gene and cell therapy trials in India and the U.S. as well as for natural history studies and certain rare disease patient registries.

Dr. Rajasimha also founded Jeeva Informatics Solutions a genomics and Bioinformatics services company.

Advocating For Rare Diseases

Dr. Rajasimha noted that India lacked an organization working on behalf of people with rare diseases. He co-founded ORDI and actively encouraged a policy comparable to the Orphan Drug Act in the United States. After ten years of advocating, a national policy was established in India for rare diseases.

Patient Registries: Recruiting Indian Patients for U.S. Clinical Trials

Patient registries are essential for clinical development programs in connection with rare disease therapies. Companies that sponsor clinical trials rely on public databases usually obtained from the NIH or patient advocacy foundations. Although there is an awareness of the importance of global representation, almost 90% of patient registries carry entries from the United States and Europe and are primarily Caucasian.

The NIH program All of Us is a research initiative designed to correct the lack of true diversity. Dr. Rajasimha encourages people on the Indian subcontinent to participate in natural history studies and patient registries.

When recruitment begins for clinical trials, preference is given to patients who participated in natural history studies. These patients have developed baseline data to be compared against a new treatment that is being assessed for efficacy and safety.

Barriers in Recruiting

Language is a deterrent to recruiting rare disease patients around the globe. Information about clinical trials that are provided online is mostly presented in English.

Dr. Rajasimha emphasized that there is a need for information pertaining to healthcare and clinical trials to be in local Indian languages in order to create awareness in urban and rural populations. These changes should reach geneticists, medical doctors, and other professionals.

The doctor added that software platforms should also be aligned with the culture, as the bulk of software management has been developed for the English-speaking population.

About Jeevatrials

Dr. Rajasimha explained that it is also necessary to increase Indian and Asian influence by promoting cultural awareness in clinical trials. As an example, he noted the different social and religious holidays that require calendar scheduling. Therefore, the software has to have features that will recruit and retain patients in clinical studies.

Jeevatrials was established by Dr. Rajasimha to fulfill these requirements.

IndoUSrare’s Five Programs

Currently, IndoUSrare is focused on five programs.

  • The first program provides information that has been requested from the FDA, NIH, and other global databases.
  • Secondly, a patient foundation program involves patients in India with rare diseases. These patients will be included in registries in the United States. At present, the program includes thirty-three advocacy organizations, five hundred rare diseases, and 25,000 patients.
  • The corporate alliance is their third program. The alliance encourages treatment options where currently none exist. This is true of 93% of rare diseases. Patient groups must interact with the industry and motivate them to develop therapies. At present, the alliance has five industry members hoping to expand to over twenty next year.
  • The fourth program creates a network of geneticists who are its research core. As each new member organization joins, the network is accessed and several doctors usually reply. The doctors are introduced to the patient groups. When there is no patient organization for a specific disease in India, IndoUSrare contacts Global Genes or other organizations to create a new entity that will expedite the program.
  • The fifth program involves IndoUSrare, the EveryLife Foundation, and members of the patient alliance communicating with congressmen and senators in the United States on behalf of rare disease patients.

While in India, members of IndoUSrare issue comments on draft policies by the Ministry of Health and other departments.

Dr. Rajasimha noted that the FDA’s draft policies combine Pacific Islanders and Asians. This group adds up to half the worldwide population. He suggests that the number of people who are not represented sets limits on the diversity of the clinical trials.

About Funding

IndoUSrare’s primary source of funding is provided by industry collaborators and corporate partners in connection with the membership program of their corporate alliance.

Dr. Rajasimha believes rare diseases with no approved treatment will continue to shrink. He sees a growing number of viable options and positive trends.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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