HYBRID EVENT: You can participate in person at London, UK or Virtually from your home or work.

International Conference On Orphan Drugs and Rare Diseases

August 21-23, 2023

August 21 -23, 2023 | London, UK

Medical Aspects and Regulatory issues

Medical Aspects and Regulatory issues

Diseases classified as rare (orphan) are those with a very low prevalence. Children are disproportionately affected by rare or orphan diseases, which are frequently inherited. Many of these diseases are incurable and have terrible consequences for individuals and their families. Patients and health-care systems alike face numerous challenges as a result of rare diseases. They are rarely explored since they are unusual, and there is less understanding about the condition and fewer doctors who specialise in it. Furthermore, because most diseases do not have a specific therapy, diagnosis is not necessary. Patients must get safe and effective treatments, according to regulatory standards for drug approval for rare diseases. The challenge for regulators, patient groups, pharmaceutical companies, politicians, and payers is to ensure that novel therapies are available to everyone, regardless of nationality or economic status. Despite improvements, there is still a need for investment in the field of rare diseases. There is no financial incentive for industry to invest in discovering medicines for rare diseases because the market potential is small. All of this prompted patients, researchers, and governments all over the world to pass a bill encouraging and incentivizing researchers and the pharmaceutical industry to produce scientific and clinical understanding, as well as potential remedies, for these disorders.

  • Global Regulatory Status
  • Clinical Trial Design
  • Novel Statistical Analysis Approach
  • Methodological and Statistical Challenges
  • Legal Aspects
  • Healthcare Impacts
Committee Members
Speaker at Orphan Drugs and Rare Diseases 2023 -  Daniel Scherman

Daniel Scherman

Fondation Maladies Rares, France
Speaker at Orphan Drugs and Rare Diseases 2023 -  Jiao Fuyong

Jiao Fuyong

Children’s Hospital of Shaanxi Provincial People’s Hospital, China
Speaker at Orphan Drugs and Rare Diseases 2023 -  Victor Shengkan jin

Victor Shengkan jin

Rutgers University, United States
Orphan Drugs 2023 Speakers
Speaker at Orphan Drugs and Rare Diseases 2023 - Candida Fratazzi

Candida Fratazzi

BBCR Consulting - Massachusetts, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Philip Bielefeld

Philip Bielefeld

Avignon Hospital, France
Speaker at Orphan Drugs and Rare Diseases 2023 - Harsha Rajasimha

Harsha Rajasimha

Jeeva Informatics Solutions Inc, United States

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