What are orphan drugs?
The term orphan drug refers to any drug or biological product intended for treatment, prevention or diagnosis of a rare disease. The definition of rare disease varies across countries but typically considers the prevalence of the disease, severity and existence of alternative therapies. As per WHO, a disease is considered rare if it affects ≤ 1 in 1000 population. In Unites states, a condition is rare if it affects fewer than 200,000 people in the country while in European union, it’s for < 1 in 2000 people.
Examples of rare disease include genetic disease, rare cancers., infectious tropical disease and degenerative disease. Oncology products account for greatest number of orphan drug designations.
Considering the high cost of drug development and lower patient pool, it becomes unviable for pharmaceutical companies to invest in these rare diseases, thus naming them orphan. In lack of proper diagnostic tools and treatment options, the patient’s suffering widens and poses a challenge for the doctor, society and the Government. To overcome this, the government and several NGOs provide incentives to drug companies to develop and market such drugs. The support is extended by the federal legislature through various research grants, market exclusivity, access to centralized facilities, free scientific advice, tax credits and fee reduction.
Adoption of Orphan drugs
Orphan designation may be granted at any stage of drug development if proper scientific justification is provided for its intended use. Efficacy, safety and quality of the drug should be satisfied to receive market authorization.
USA was the first country to pass orphan drug act in 1983 followed by Japan in 1993, Australia in 1997 and EU in 2000.
It is difficult to understand that countries like India and China being two of the largest in terms of population size still lack the designation of orphan drugs and national legislation for them. It creates a substantial negative impact on patients affected with such diseases.
The rare diseases are estimated to affect 72-96 million people in India. Though rare disease policy was drafted by Indian Government in 2017, it has yet to see the light of the day. The policy is the first major step by the government which recommends genetic counselling, as well as pre-natal and antenatal screening. Indian Council of Medical Research (ICMR) has launched a registry for Indian rare diseases to collect data for policy framing and guide future research. Beyond funding, a more holistic approach and faster implementation is the need of the hour to facilitate the adoption of orphan drugs.
Turacoz Healthcare Solutions takes an initiative to sensitize general population and pharmaceutical companies on the challenges faced by people affected with rare disease and take adequate steps via regular screening to limit such conditions. Turacoz is a medical communication company that provides scientific/medical/ regulatory writing support to the industry and is serving their clients in developing new drug applications, protocol writing for research, and medico marketing support. To get the assistance you can get in touch with us at firstname.lastname@example.org