NEW DELHI: The government may fix trade margins on so-called orphan drugs used to treat rare diseases, a health ministry official said, promising partial relief for patients burdened with exorbitant treatment costs. The move, if successful, could hurt multinational pharmaceutical companies such as Sanofi Genzyme, Pfizer and Shire USA, who make these drugs.

The health ministry has identified drugs to treat 21 rare diseases and 39 cancer drugs, the official cited above said on condition of anonymity, adding it has asked the department of pharmaceuticals (DoP) to examine the possibility of fixing their trade margins.

“The DoP had asked to shortlist the drugs on which trade margins can be applied. The list has been sent to them. Now, the DoP will have to examine the individual drugs and take a call," the official cited above said.

Some of the diseases mentioned in the health ministry communication to the DoP are Gaucher Disease, Hunter Syndrome, Pompe Disease, Fabry Disease, Lysosomal acid lipase deficiency, Type 2 cerliponase, Duchenne muscular dystrophy, spinal muscular atrophy and cystic fibrosis.

The health ministry has been raising concerns over the exorbitant cost of life-saving drugs for a long while. On 29 November, health secretary Preeti Sudan wrote to DoP secretary Jai Priye Prakash “to explore the feasibility of capping the prices of drugs used for treatment of rare diseases so that their prices become responsible and affordable to the patients." Mint has seen a copy of the letter.

However, in amendments to the Drug Price Control Order (DPCO) released on 3 January, the chemicals ministry exempted producers of new drugs patented under the Indian Patent Act, 1970, (39 of 1970) from price regulation for five years from the date of commencement of its commercial marketing. The matter reached the Prime Minister’s Office (PMO) where it was decided that the health ministry will prepare a list of orphan drugs and cancer drugs for fixing their trade margins.

According to a second official, who also spoke on condition of anonymity, the idea is to fix the maximum retail price (MRP) of the drug which will be decided by adding the trade margin to the price at the first point of sale (stockist). The trade margin is the difference between the price at which the manufacturers/importers sell to stockists and the price charged to consumers.

The ministry list also includes drugs to treat last-stage cancers like lymphocytic lymphoma, advanced metastatic non small cell lung cancer, non-lymphocytic leukemia of adults and children, chronic myeloid leukemia in chronic phase, breast cancer and malignant lymphomas soft tissue among others.

Experts working with people with rare diseases say that treatment cost run into crores of rupees, adding these steps do not serve any purpose. “Both the steps —fixing the trade margins and giving five-year exclusivity to the manufacturers of innovative drugs are not going to help patients in India. The only cure so far for Spinal Muscular Atrophy (SMA) costs $750,000 per child per year. Who can afford to get this treatment? The government has to create a corpus for people with rare diseases. I don’t understand why government doesn’t call enough stakeholders before taking such decisions," asked Archana Panda, co-founder, Cure SMA foundation of India, a non-government organization (NGO) that represents 300 families in India with people with SMA. SMA is a life-threatening neuro-muscular genetic disorder.

Malini Aisola, co-convenor of All India Drugs Action Network (AIDAN) said that “The discussion of trade margin capping for cancer and rare diseases is to distract from the disastrous amendments to DPCO recently notified. Margin caps of these medicines is misconceived, will not address the root of the high prices and will not increase affordability".

When contacted, Pfizer said its product Taliglucerase for Gauchers Disease is not commercially launched in India.

Sanofi India spokesperson said, as very few people suffer from these rare diseases, as such, there is no ‘market share’ to speak of. Furthermore, as each individual patient is distinct from another, the treatment becomes highly personalized and in turn, the cost to patients varies depending on the weight of the patient.

The India Charitable Access Program (INCAP) is Sanofi Genzyme’s humanitarian program through which we provide free therapy for patients here in India, who suffer from Lysosomal Storage Disorders (LSDs), namely Gaucher, Pompe, Fabry and MPS I. Currently, over 130 patients are receiving free treatment under our humanitarian program. There are a small number who are reimbursed the cost of their treatment by State run institutions.

The balance though are mostly reaching out to Central and State governments, seeking access to therapy. Until they are heard, these patients will have no access to treatment. Given this scenario, we should all really focus on urging the Government to expedite the revision of the rare disease policy so that more patients have access to therapy.

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