In November 2021, Takeda Pharmaceutical Company Limited, a global values-based, R&D-driven biopharmaceutical leader, together with United Nations Global Compact Network of India (UNGCNI)  launched a national initiative for improving early access to treatment for Rare Disease Patients in India. The joint rare disease initiative took charge of addressing the challenges to ensure access to treatment for rare disease patients waiting on the crowdfunding portal in an integrated manner. Through the initiative, 300+ PSUs and Corporate leaders were reached, which has helped to generate a pool of insights and recommendations that will help in shaping the future of access to rare disease treatment in the country.

The 6-months intensive initiative has been able to bring together stakeholder groups (private sector, PSUs, corporate giants, etc.) that can support mobilizing and operationalizing funding for the rare disease under CSR grants and donations for strengthening the existing crowdfunding mechanism and pathways set in the India Rare Disease Policy. Furthermore, it has created the roadmap for addressing rare disease funding requirements in alignment with the Government policies and guidelines Ministry of Corporate Affairs, Department of Public Enterprises, and Ministry of Health and Family Welfare, Govt of India.

The eminent experts from public and private sector organizations participated in the initiatives including MoHFW, AIIMS (New Delhi), Kerala State Legal Services Authority (KELSA), CoEs (SGPGI, Lucknow, KEM, Mumbai, SSKM, Kolkata), and 24 PSUs and Corporate Leaders (e.g., BHEL, Bharat Petroleum, ONGC etc).

According to  Mr. Aseem Kumar, Officiating Executive Director, UNGCNI, “Rare diseases, underpin a situation where health lies at an intersection of several Sustainable Development Goals, going well beyond only SDG 3 (Good Health and Well-being). The need to address rare diseases on priority was made a reality in 2019 when UN Member States adopted the historical Political Declaration on Universal Health Coverage. This included a commitment to strengthen efforts to address rare diseases as it was for the first-time that rare diseases were included within a UN declaration. This   helped in channelizing collaborative efforts with multi-stakeholders to strengthen the national rare disease policy execution”.

Ms. Serina Fischer, General Manager, Takeda India says, “Patients remain at the core of decision making at Takeda and we are fully committed to the fight against rare diseases by advancing treatments and addressing diagnostic delays and gaps in access to treatment. The need of the hour is to bring all stakeholders together for rare to strengthen capacity-building efforts and find sustainable funding mechanisms that ensure the treatment of people living with rare diseases. We are pleased that our association with UNGCNI has successfully generated insights and mobilized support which in the long run will be important in solving issues related to access of treatment for rare disease patients.”

“Rare disease presents itself as one of the most challenging health areas to be tackled. The first step to handling rare diseases for India was the release of NRDP in 2021 which has given direction to the efforts of rare disease players to start with. But, as in any health area, policy implementation faces its set of challenges, especially when policy provisions are there but awareness about those provisions is absent or blurred by perceived challenges. Therefore, for inspiring change in the rare disease ecosystem, it is important for policymakers, payors, providers, and patients to come together as one unit and work using the continuum of care approach for applying integrated solutions”, Dr. Ruchi Sogarwal, Head-Public Affairs & Patient Advocacy, Takeda India.

 Mr. Ajeet Kumar Sharma, AGM, Bharat Heavy Electricals Ltd. & Secretary, UNGCNI expressed, “CPSEs are key players in CSR domain, working relentlessly for equitable wellbeing of society through their CSR initiatives. They have the needed capacity of extending help to the needy patients suffering from rare diseases. However, there is a need for enhancing awareness and understating of rare diseases, issuing of appropriate guidelines & directions by concerned government agencies and enhanced connect between Centers of Excellence and CPSEs. This initiative has immensely helped in clarifying the perceived barriers in flow of CSR fund from CPSEs towards this cause.”

“The Government of India’s support to rare diseases in form of the National Policy for Rare disease-2021 is laudable and it has made rare diseases a national priority. The step has been vital in showcasing India’s commitment not only to addressing the needs of the rare disease patients but also to achieving universal health coverage (UHC). The onus now lies on us as citizens of the country, be it individuals, large or small organizations, institutions or the general public to support the Government Notified Centre of Excellences in the smooth implementation of the policy. UN-GCNI is dedicated to Improving Early Access to Treatment for Rare Disease Patients by encouraging the stakeholders such as PSUs, CSR mandated organizations, corporates, and experts to come together in support of underserved patients, with the firm belief that UHC will never be completely realized if people living with rare illnesses are left behind”, Dr. Somnath Singh, Programme Manager, UNGCNI.  

Also Read: Rare disease-right to hope

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Rare disease-right to hope. All that needed is to work towards common Goal in the best interest of Indian Rare Disease community. Some of the recent changes in the Indian scenario is giving HOPE for self and for society.

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

16/2, Sri Krishna Krupa, 19th Cross,
8th Main, kashimutt Road, Malleshwaram,
Bengaluru – 560055 Ph: +91 9980133300
E-mail: prasannashirol@gmail.com

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).

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