Orphan drugs and Orphan Diseases
Dr K K Aggarwal, National President IMA
An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.
Delhi High Court Judgment: Mohd. Ahmed (Minor) vs Union Of India & Ors. on 17 April, 2014, Manmohan, in the High Court of Delhi at New Delhi, WPC 7279/2013
· On account of lack of Government planning, there is 'pricing out' of orphan drugs for rare and chronic diseases, like Gaucher. The enzyme replacement therapy is so expensive that there is a breach of constitutional obligation of the Government to provide medical aid on fair, reasonable, equitable and affordable basis. By their inaction, the Central and the State Governments have violated Articles 14 and 21 of the Constitution.
· Just because someone is poor, the State cannot allow him to die.
· In fact, Government is bound to ensure that poor and vulnerable sections of society have access to treatment for rare and chronic diseases, like Gaucher especially when the prognosis is good and there is a likelihood of the patient leading a normal life. After all, health is not a luxury and should not be the sole possession of a privileged few.
· Although obligations under Article 21 are generally understood to be progressively realizable depending on maximum available resources, yet certain obligations are considered core and non-derogable irrespective of resource constraints. Providing access to essential medicines at affordable prices is one such core obligation.
· The Court is under a duty to ensure that effective relief is granted. The nature of the right infringed and the nature of the infringement provides guidance as to the appropriate relief in a particular case.
· As health is a State subject, the present petition is disposed of with a direction to the Government of NCT of Delhi, to discharge its constitutional obligation and provide the petitioner with enzyme replacement therapy at AIIMS free of charge as and when he requires it.”
Suggestions by the court
Both the Central and State Governments should consider the following suggestions:
· All government hospitals could have a separate CSR/ Charitable entity/account wherein donations can be received. The donations could be subject to an audit.
· Each hospital could have a designated officer, to whom applications for assistance can be made by patients in need. The decision to whom financial assistance could be provided, be left to the Medical Superintendent/CEO of the Hospital along with Head of the Departments. Delhi could be adopted as the first model state.
· The Ministries of Corporate Affairs and Finance could consider providing extra credit (for instance increased credit) for donations in certain sectors, such as health.
· The Government could adopt a holistic approach to facilitate donations, so that the tax regime supports the said efforts.
· All donations in cash and kind must be accounted for, with complete transparency to ensure no misuse or misappropriation of donations.
· Government hospitals could put up list on the State Department of Health website of the drugs, implants and devices they require for EWS/BPL patients. This way people would donate as per the need of each hospital. This could be revised on a monthly basis.
· The State Government may put up a list of drugs, implants and devices, which are excluded from its budget for which donations would be welcome.
· Both the Central and State Governments could create a revolving fund to take care of recurring expenditure of patients suffering from chronic and rare diseases.
· The Government could constitute a High Powered Inter-disciplinary Committee to: Develop and update a list of guiding principles/best practices in the area of donations in healthcare; develop a policy for tackling rare diseases and promoting the development of orphan drugs; evolve new and innovative methods for attracting spending in the area of healthcare. This Committee could have representatives from various State and Central Government departments, private and government hospitals, non-governmental organizations working in the area of healthcare, representatives of patients-rights groups, representatives of pharmaceutical and other companies in the healthcare sector.
· However, as the concept of CSR is still at a nascent stage and there is no mechanism in place which popularizes and facilitates donation, this Court is of the view that State must bear the burden of the treatment.
Another Delhi High Court Judgment: Amit Ahuja vs Union Of India & Ors on 1 April, 2014: Manmohan: In the High Court of Delhi at New Delhi, W.P.(C) 1507/2014 & CM APPL. 3144/2014
· Facility to provide AHF for haemophilia patients shall be provided in three hospitals of this Government of Delhi namely, Lok Nayak, DDU and GTB hospital.
· Treatment in these three hospitals would be given along the following lines:-
o All BPL families will be supplied AHF free of cost.
o Free treatment will be given to all Hemophilia patients who came to designated hospitals i.e. LNH, GTB and DDU in an emergency.
o In respect of APL patients with minimum three years domicile in Delhi, the following graded payment system will be adopted.
a. Family income up to Rs. 2 lakh per annum-20% of cost of AHF (concerned MS would be competent to relax 20% charge in exceptional cases for reasons to be recorded in writing).
b. Family income between Rs.2-5 lakh per annum-50% of cost of AHF.
c. Family income above Rs.5 lakh per annum-full cost of AHF.
All haemophilia patients irrespective of the medication that they require have to be given treatment in accordance with paragraph 1 of the said letter.
Another example of rare diseases
"Spinal Muscular Atrophy (SMA)" is a rare genetic disease. In India, there are families whose children are suffering from this progressive disease. Due to SMA, children cannot walk, and, the nightmare does not end here as it is a progressive degenerative disease which means the kids may potentially see deterioration in function of their other limbs - hands, spines and even lungs. An investigational drug “Nusinersen” from Biogen is under development, which has shown promising results after undergoing clinical trials. The drug is not yet launched in India. The estimated cost is more than $100,000 per year. No Health Insurance Company in India covers such disease or costs
· All Insurance companies should cover all diseases including rare diseases.
· There should be a centralized CSR fund, which should sanction a percentage of such treatment costs.
· PM fund should have a fixed sanction of percentage of this cost. State CM should match the PM fund grant.
· MOH should have a separate budget allocated for rare drugs and rare diseases treatment.
· All hospitals should contribute by subsidizing the cost of treatment.
· Patient, relatives and friends of the patient must also contribute a part of the treatment.
· IMA is also launching a centralized transparent fund for this purpose.
· All NGOs should also pool their resources in a centralized manner for this purpose.
· Special schemes are also available for BPL, EWS, Arogya Nidhi, Arogya Kosh, RSBY etc.
· NRIs can also be involved for donations.
· International funding agencies can also be tapped.
· Pharma companies can also donate. A pharmaceutical company donating medicines/drugs within section 135 (read with Schedule VII to the Act) is a CSR Activity, as the same is not an activity undertaken in pursuance of its normal course of business which is relatable to health care or any other entry in Schedule VII.
If each of the above can pitch in, no patient will die in the country just because he or she cannot afford the treatment.