Right to HOPE                                                                      Blog No 2

Life is all about changes and change is part of life. While only change is constant, one cannot imagine the effects of change immediately and may not see. Only the end result of any activity or event decides whether, it was a good  or bad CHANGE. Change is also perceptional and perception depends upon ones general knowledge and domain knowledge, It also depends  on  once  involvement in the activity and passion in the field, it also depends on analytical capacity and factors considered  for analytics  from all side. Grass is not always green on the other side and one has to see closely to come to conclusion on anything in Life.

We (Rare Disease Patients Support Group)  being in the field of Rare Disease and we are responsible for expecting  and delivering  HOPE for self and  for society. Recently there are many good changes in the field of Rare Disease in India and globally. Let me talk about India.

If you look at recent changes in the Indian scenario from all the stakeholders, I personally see lot of HOPE and optimism on our combined work and I am sure you also appreciate these changes.  Let me attempt to  list few of them to recap

Central Govt. –  Recognizing Rare disease by Govt. in many departments and drafting Rare Disease policy, Schemes like UMMID, Inclusion of Rare Disease in RAN scheme, Questions and responses to our letter from MOH and other department, FSSAI guidelines on Diet  etc

DCGA/CDSCO – Orphan Drug Policy, Clinical Trial Guidelines, Rare Disease definition etc

 ICMR-  Patient Registry initiative, Gene therapy Guidelines, Bio ethics guideline, DNAR – Do not attempt Resuscitation  discussion etc

IRDA – Beginning of  Some thought process on genetic diseases and basic guidelines in Insurance sector,

Loksabha -Increased Questions in Loksabha by MPS and Govt responses,

ESI ( employee State Insurance)  – Relief to some patients based on the court cases filed by patients.

State Govts – 8 State Govt  forming State Technical Committee,  Karnataka Govt confirmation of 40% on its share towards the treatment for Rare diseases officially.

Judiciary – Favorable judgments by many High courts in India  favorable judgment upholds the Human Rights and Right to Health. Treatment in Karnataka for some children affected with Rare Diseases as a result.

Medical Fraternity– I see a lot of changes in some doctors, hospitals and cities towards Rare diseases and as a result increased diagnosis , care, Increased discussion in CME and conference.

Genetic Testing Companies:  Maturity among Genetic testing companies on reliability of results and confidence from medical fraternity on these tests.

PAG Advocacy- Many individual disease support group have increased their level of activity or intend to do some work.

PAG interest in finding a cure: We see many patients groups now have started raising funds and investing / supporting  in R&D to find a cure for their children or groups condition.

Public Sector : Some public sector extending treatment support to their employees and families

The list is endless, All these are the result of all of our combined efforts. Yes I agree we are still not able to save all our children diagnosed and suffering from Rare Diseases. Rare Disease being a global challenges and exorbitant cost of Diagnosis, treatment and supportive care. It may take some time. We need to have HOPE

I feel now in India the timing is right. We need to work towards common goal  of advocating for Rare Disease policy for India collectively. Individual disease specific support group need to continue towards supporting their members. Only this can be a long-term , sustainable approach to save  children’s suffering from Rare and Genetic diseases. Please note there is no shortcut.

The current speed and momentum gathered is a definitely a path to HOPE in India.

All that needed is every individual, Patients, Families, Patient Support Group  need to work towards common Goal and objective in the best interest of Indian Rare Disease community.

Hope this New Year brings another chance of “GETTING IT RIGHT”, another chance  of HOPE.

I wish all those working and supporting selflessly towards this RARE DISEASE CAUSE, All those mother’s and fathers’s suffering in silence, All those RARE WARRIORS , All those in Companies and  employees, Policy makers, Researchers, Doctors, Nurses, and billions of people directly or indirectly working for the benefit of Rare Disease community

 Happy, Healthy, Hopeful and Prosperous  New Year 2020.

Prasanna Shirol

Father of a daughter affected with Rare Disease called Pompe  *Ashoka Fellow  *Rare Disease Advocate *Social Entrepreneur  *Rare Champion of Hope Awardee 2019 from Global Gene  *Co founder and Director – ORDI -Organization for Rare Diseases India  * President – Pompe Foundation  *Adviser – Inclusive In India *Co founder, Former Founding President – LSDSS (Lysosomal Storage Disorders  Support Society).