Over 7000 rare diseases have been identified globally, only 5% of these have identified treatments.
450 rare diseases have been reported in India affecting over 70 million people.
To call for effective dialogue and decisive action against the alarming challenges of rare diseases in India, People to People Health Foundation (PPHF), a public health organization working towards better healthcare and Niti Aayog, Co-hosted a Global Rare Disease Conference under the initiative ‘Blueprint for Rare Diseases in India – Together for Rare‘ on the International Rare Disease Day (28 February). The virtual conference was co-powered by Takeda Pharmaceutical and was supported by Japan Embassy.
Over 7000 rare diseases have been identified globally and experts opine that only 5% of these have identified treatments. The conference witnessed engaging dialogues and discussions from eminent guests who underlined the need for increasing collaboration and commitment to tackle rare diseases out of which 450 have been reported in India affecting more than 70 million people.
Speakers in the conference stressed on the urgent need for drafting a roadmap to collect disease prevalence data and guidelines on patients’ registry at the local level along with the timely release of statistics. Dr. Laxmikant Palo, CEO, PPHF, said, “Coordinated actions across key stakeholders are key to successful implementations. We can address rare disease challenges by investing more and allocating more to leave no one behind. We must change our ways and make sure that our commitments are tangible.“
The online event also focused on chalking out policies for evidence-based knowledge, innovation in therapies, better access to real-world data, funding of research for development for new medicines, among others. “The draft policy has proposed a high quantum of assistance for rare diseases. India has committed to provide health for all,” said Rajender Kumar, Director, Prime Minister’s Office, Government of India.
Speakers discussed learning from cross country models and executing tailor-made solutions that work for the nation via active involvement of all the key stakeholders simultaneously. Dr. Rakesh Sarwal, Additional Secretary, Niti Aayog said, “It is my suggestion to build a network among caregivers, healthcare providers and patients to raise awareness on rare diseases in India. Connecting them through teleconsultation or telemedicine can give access to doctors to access data on rare diseases.”
The conference witnessed participation from dignitaries including Dr. Laxmikant Palo (CEO, PPHF), Dr. Archisman Mohapatra (Executive Director, GRID Council), Dr. Rakesh Sarwal (Additional Secretary, Niti Aayog), Mr. Rajender Kumar (Director, Prime Minister’s Office, Government of India), Prof. Rajendra Pratap Gupta, (Chair – Dynamic Coalition on ‘Internet & Jobs‘, Internet Governance Forum United Nations, Mumbai Metropolitan Region) and Dr. Mahender Nayak (Area Senior Vice-President, ICMEA, Takeda), Susanne Weissbaecker (Head Access to Medicine, GEM, Takeda), Mr. Koki Sato General Manager, Takeda, Dr. Amandeep Kang, MD NHM Chandigarh, Dr. Tanushree Mondal Deputy Director of Medical Education, Govt. of West Bengal, Mr. Blaine Penny, Treasurer & Co-Chair, CORD, Ms. Durhane Wong-Reiger, President & CEO, CORD, Dr. Bobby John, Managing Director, Aequitas Consulting Pvt. Ltd. among others.
Underlining the role of pharmaceutical companies in this mission, Mahendra Nayak, Area Senior Vice-President, ICMEA, Takeda, said, “Takeda is a value-driven patient-centric organization and is committed to the cause of rare diseases. We understand the challenges posed by the unmet needs in diagnosing and managing rare diseases and closely engage with multiple stakeholders at every stage of the patients progress. Our vision is to ensure seamless access of our innovative therapies to patients and improve the standard of care through focused initiatives.“
The Global Conference on Rare Disease will bring rare diseases to the forefront of healthcare discussions to raise awareness and build partnerships for better outcomes. “Nobody should have to choose between paying for treatment of rare diseases or feeding and schooling of their children,” underlined Susanne Weissbaecker, Head Access to Medicine, GEM, Takeda.