Parents of baby Rajiv (name is changed) were wondering why their precious little baby was so floppy. When they finally learned the diagnosis – that of spinal muscular atrophy (SMA type 1), they were devastated. “Yes, we were heart broken when we learned that our little one had a life-limiting disease,” said the father, “but the most upsetting fact was that there was a medicine available for this condition. Being the costliest medicine on planet earth, there was simply no way we could ever afford it. We would have been better off not knowing about the availability of this remedy.”
The father was referring to Zolgensma (generic name Onasemnogene abeparvovec), a pioneering gene therapy. Zolgensma uses a harmless virus with some of its DNA overwritten by a copy of the human SMN1 gene, which is absent or faulty in the affected babies. Given to less than two years of age with SMA, the treatment is the only potential cure available in the world right now. Good news! (Or is it?). The problem is that the price tag is out of reach for most of us – `18 crore for one single injection. In Bangalore Baptist Hospital (BBH), the pediatric neuromuscular disease unit has been able to treat 13 such babies over the last few months with Onasemnogene. Only one family among the 13 treated at BBH bought the medicine out of their own resources, supplemented by some crowd funding effort. The other 12 received it through a compassionate access program, decided by an international forum through a blinded lottery system.
But treatment options like these bring up difficult ethical questions. It is like saying to a drowning man, “There is a certain rope available to pull you out; but then, you cannot afford it.” The same applies to technology and advanced equipment, which are out of reach of the common man. We cannot wish away the problem of limited resources. In a country like ours, we cannot and should not pour our money and resources into ivory towers, from which very few benefit. Equity and social medicine do go hand in hand.
The other side of the coin is that the cost of advanced technology and certain medicines will come down when their usage goes up. Take the example of the motor car. Henry Ford’s dream was to make the Model T a common household item. The cost came down drastically with mass production. Could this be repeated in the case of medical gadgets and medicines? Will Onasemnogene become more affordable? How about medical equipment? We know by experience that the cost of medical instruments and implements keep going up.
The wants of the clinician and the needs of the patient keep ascending northwards. Much of it is indicated and has resulted in a perceptible increase in patient safety, and has affected improved patient experience in the healthcare facility. It is also to be remembered that the pay back has to be good enough to inspire adequate research and development.
When it comes to the medical equipment industry, it is imperative for all concerned that we are made cognizant of the fact that at the other end of the stick is a vulnerable human being. Illness and disease are not welcome intruders; they cause much pain. Insurance penetration is still much less in our country. One can never be totally prepared for a sudden illness. There is a role for tempering economics with compassion. A touch of empathy is the flavor one needs when deciding on issues of such significant human interest.
Ultimately, the viable model is one where there is an ongoing and growing demand for the implement or medicine as the medicine’s usage increases and price comes down. May the modern medical gadgets and medicine become the Model T in the days to come.
Above all, let there be a compassionate response from all of us stakeholders. Human innovation and industry must go hand in hand with our commitment to our fellow human beings. May that be our guiding principle in all that we think and do.