Clinical trials must to curb rare diseases
As a person with beta-Thalassemia, I have experienced the difference that a robust clinical research programme can make towards improving therapeutics and our quality of life. My late paternal grandfather M A Sethu Rao, spent almost 15 years writing to clinicians and researchers on the possibility of a gene therapy clinical trial for Thalassemia.
From the late 80s, the thalassemia community has been awaiting human trials on gene therapy. This has also been the goal of clinician-researchers for more than three decades (Nienhuis & Persons, 2012). In 2005, plans for the first clinical trials in beta-Thalassemia using lentiviral vectors for successful normal gene delivery were announced (Nienhuis & Persons, 2012).
I have been actively following the progress of the trials since 2010 and am happy to note that after the successful completion of Phase I and II which evaluated safety and efficacy of the therapy, it is now moving into Phase III. Results from Phase I and II have given hope to the community. Besides this cure, other drugs are also being tested in the Phase III trials. These drugs work on reducing dependence on blood transfusion in patients.
I am hopeful that the clinical trials scenario in India will soon become as seamless and robust as that in the United States and Europe. We did see setbacks in 2013 when the government changed certain guidelines pertaining to clinical trials, citing concerns of safety, reporting timelines and compensations related to serious adverse events (SAE), injuries and deaths during drug studies.
These changes created hurdles for investigators, agencies, institutions and organisations conducting clinical trials in India. The number of approvals for trials drastically dropped from more than 500 prior to 2011 to around 25 in 2013-14.
However, following a number of discussions in July 2014, facilitated and guided by the pharma and biotech industries, the department of biotechnology (DBT), government agencies and other stakeholders like clinicians, researchers and patient organisations, it has become easier to conduct clinical trials in India in accordance with global standards of compliance because two contentious guidelines have been amended to create a more positive climate.
In keeping with global guidelines, an order issued in September 2014, indicated three key parameters for evaluating global clinical trials in India. These were: Gap in medical needs in the country. Assessing risk versus benefit for patients. And innovation in drugs or therapeutic procedures with regard to currently available options.
These parameters will bring more transparency into the process, because now the authorities will have to justify the advantages of conducting clinical trials in India to the citizens.
The policy changes governing clinical trials made by the Central Drugs Standard Control Organisation (CDSCO) bring even more hope to people with rare diseases in the country. One change concerns the waiver of clinical trials for orphan diseases for which there is no known cure or therapy.
Most rare diseases have no cure and have a high mortality rate, because there is no available treatment for them. Any step to accelerate access to new therapies is most welcome, even a trial drug or therapy may be the right intervention to save the patient’s life. Most patients and families with rare diseases are aware of the importance of participating in clinical trials and providing access to personal medical data for research purposes.
Time is of critical importance for people with rare diseases. The amended policy in the clinical trials guidelines has given the rare disease community in India a new hope.
We anticipate that more clinical research programmes will be undertaken now which will give us faster access to new and urgently needed therapies for the rare diseases.
(The writer is research Director, Centre for Health Ecologies and Technology, Bengaluru)