ORPHAN DRUGS AND DISEASE: REGULATORY AND LEGAL FRAMEWORK
Orphan Drugs and Disease : Regulatory and Legal Framework
History and Background
The healthcare system in India and across the globe has been over-burdened with diseases and discovering ways to regulate and control them. In such dire circumstances, addressing legislative and regulatory challenges for “Rare/Orphan Diseases” becomes quite difficult. “The principle of proportionality” would always argue that more attention and resources should be allocated to common and/or widespread health problems that affect bigger populations than to disorders that afflict a smaller number of individuals
Because of factors such as a lack of epidemiological data, a scarcity of relevant research, the lack or inaccessibility of drugs and treatments, and complex tertiary level management involving long-term care and rehabilitation; the rarer the disease, the greater the investment and resource allocation is required. The sheer logic of lesser numbers and incidence, on the other hand, can never justify the governmental attention paid to “Rare/Orphan Diseases”. The reality is that a single person's suffering, or worse, death, as a consequence of a “Rare disease is a public health issue” and must be addressed.
A Rare Disease has no commonly accepted definition. “Rare Disease is defined by the World Health Organization as a debilitating lifelong disease or disorder condition with a prevalence of 1 or fewer per 1000 people.” Different nations, on the other hand, have their respective definitions to fit their set of requirements and in the context of their population, resources, and healthcare system.
Position in India
India, like many other developing nations, lacks a standardized definition of rare diseases as well as data on their prevalence. If we use the international estimate of 6% to 8% of the population being afflicted by rare diseases, it can be estimated that 72 to 96 million individuals in India are impacted by rare diseases, which is a considerable figure. However, this is only a rough estimate, and India will need to come up with its own estimate and definition of rare diseases.
Legislative Developments
Realizing the dire need of the situation, the government in recent years has addressed the issue of Rare Diseases in India. The first initiative began with the National Health Policy 2017 which dealt with the management of Rare Diseases to resolve the deficiencies in public service through a public-private partnership. Thereafter, the National Policy for the Treatment of Rare Diseases (NPTRD) was drafted in July 2017 which then was subsequently reviewed by an Expert Committee in 2018 owing to implementation issues, among other things.
In March 2021, the Delhi High Court in the case of Master Arnesh Shaw vs UOI, directed the Centre to set up a “Rare Diseases Committee, a Rare Diseases Fund and to finalize and notify the National Health Policy for Rare Diseases” on or before 31st March 2021. Pursuant to this directive, the Ministry of Health and Family Welfare approved a comprehensive “National Policy for Rare Diseases 2021”. Additionally, “the Indian Council of Medical Research (ICMR) launched a hospital-based “National Registry for Rare Diseases”, incorporating facilities around the country that specialize in the detection and treatment of rare diseases.
Under “the New Drugs and Clinical Trial Rules 2019”, Orphan Drugs have been defined as a “drug intended to treat a condition which affects not more than five lakh (500,000) persons in India”. The regulatory framework for an Orphan Drug clinical trial is identical to that of other drugs, with the exemptions provided to Orphan Drugs:
India's highest drug regulatory organization, “the Central Drugs Control Standards Organization (CDSCO)”, can waive the necessity of performing local clinical trials in cases of Orphan drugs.
The sponsor of a clinical trial for an orphan drug might urge the CDSCO to speed up the approval procedure for that drug.
A clinical trial of an Orphan Drug does not need payment of an application fee.
The National Rare Disease Policy 2021
The Ministry of Health and Family Welfare approved the “National Rare Disease Policy 2021” with an aim:
The policy intends to enhance emphasis on indigenous research and local manufacture of medications, as well as lowering the costs of treating rare diseases.
Those suffering from rare diseases (diseases specified in Group 1 of the rare disease policy) who require one-time treatment would be eligible for “financial assistance of up to Rs20 lakh under the Rashtriya Arogya Nidhi umbrella plan”. It will cover “around 40% of those who are qualified under the Pradhan Mantri Jan Arogya Yojana”.
To finance the expense of treating uncommon diseases, the policy will employ a crowdfunding system. Corporates and individuals would be encouraged to contribute financially through a comprehensive IT platform.
A national hospital-based registry for rare diseases will be developed to ensure that researchers and developers have access to enough data and thorough information about such ailments.
The strategy intends to test and discover rare disorders at an early stage, which will aid in their prevention, with the support of Health and Wellness Centres, District Early Intervention Centres, and counselling.
Latest Developments
The Union Health Ministry issued an office memorandum on May 19 2022 increasing the financial aid to the patients suffering from rare diseases under “the National Policy of Rare Diseases, 2021 from Rs 20 lakhs to Rs 50 lakhs under the umbrella scheme of Rashtriya Arogya Nidhi (RAN).” The earlier grant of twenty lakhs was limited to only a few conditions in Group 1 but the new grant can be availed for all rare diseases, including lung transplants. Such an initiative comes as a relief to patients suffering from rare diseases and will substantially raise access to treatment for thousands and thousands of patients with rare diseases in India. The hike in the grant also comes as a major boon to the stressed families of patients suffering from rare diseases in India
Many rare disease drugs are unavailable in India, and even if they are, they have been "exorbitantly expensive, putting enormous strain on resources." Only a few drugs for rare disorders are manufactured in India, thus all of these drugs are eventually imported. The majority of orphan drugs are palliative rather than curative, and they need to be given on a regular basis. Some people with rare diseases may require lifetime treatments that are either unavailable or prohibitively expensive in India. Hence, reform in the policy is a blessing not just for patients, but also for pharmaceutical companies seeking to launch rare drug treatments or therapies in India. The pharmaceutical firms can now launch advanced therapies for rare diseases within and for India.
Conclusion
The new policy reform grants financial aid of fifty lakhs to patients suffering from rare diseases for the treatment only in the Centres of Excellence (CoEs) mentioned in the NPRD 2021. States such as Kerala don’t have hospitals that have been declared as the Centre of Excellence. As a consequence of this, most of the patients suffering from rare diseases in such states are unable to avail of the benefits under the Scheme as it is near impossible for such patients to travel to the neighbouring states in order to access treatment in the CoE and thereby avail the benefits of the Scheme. Hence, Centres of Excellence must be established in each State to ensure the successful implementation of the Scheme.
In recent years, 96 per cent of Rashtriya Arogya Nidhi's funding for treating Group 1 Disorders has gone unused. This clearly reveals the lack of effective implementation to treat these patients. Several patients have already died as a result of a lack of institutional financing, and many more are at risk.
Without a proper definition of rare diseases and their specifications; an effective policy and its successful implementation will be difficult. The use of terms which are not defined leads to ambiguity and inconsistencies, which has ramifications for treatment access as well as research and development. Hence, we need to allocate additional resources and personnel to the National Registry for Rare Diseases to speed up the collection of epidemiological data. This would aid us in coming up with a proper definition and other related specifications in our own country.
Early and accurate diagnosis would not only reduce long-term expenses, for both the patient and the government but also prove to be lifesaving. Hence, it is a necessity to increase the allocation of funds and divert them to related infrastructure and equipment such that they are readily available and easily accessible in our hospitals and clinics.