Obtain the data you need to make the most informed decisions by accessing our extensive portfolio of information, analytics, and expertise. Sign in to the product or service center of your choice.
The Indian government has implemented new clinical trial
regulations intended to expedite the registration of
orphan-designated and innovative medicines in the country. While
the measures constitute part of a wider policy drive to entice
market entry of advanced new treatments, IHS Markit expects the
affordability barrier to remain a major obstacle to patient
access.
India's Central Drugs Standard Control Organization (CDSCO) last
month implemented its New Drugs and Clinical Trials Rules, 2019.
The changes encompass a new conditional approval pathway for
orphan-designated medicines for rare diseases, and for new
innovative medicines that have already been approved and licensed
by regulators in the European Union, the United States, Australia,
Canada or Japan. Qualifying products can now be launched in Indian
without companies needing to conduct phase III clinical trials to
test for efficacy and safety in the Indian population, provided the
associated global studies included Indian patients. Companies will
need to conduct post-marketing phase IV trials to evaluate
long-term effects, but ultimately, the new rules should speed up
the approval process and the availability of new medicines in
India.
The new regulations define orphan drugs for the first time - as
medicines to treat conditions that affect fewer than 500,000 people
in India. Under the new rules, Indian regulators now have the
authority to exempt orphan drugs from both phase III and IV
clinical trials. Manufacturers or sponsors of orphan drugs can
apply to India's CDSCO for an expedited review process, waiving the
requirement for local clinical trials in recognition of significant
unmet medical need.
Other measures aim to reduce timelines for granting approvals
for clinical trials. For example, application processing has been
reduced to a maximum of 90 days. In the case of an investigational
new drug subject to discovery, research, and manufacture in India,
the application will be processed within 30 days. Additionally,
application fees for orphan drug trials in India will be waived. By
streamlining the application process for clinical trials, these new
provisions aim to encourage more indigenous research and
development (R&D) for rare diseases affecting patients in
India.
The need for reform in this domain could hardly be doubted; to
date, few orphan drugs have been registered in India, with some
patients resorting to importing these medicines directly.
Similarly, only a small fraction of the innovative new oncology
therapies that have been launched globally in recent years have
reached the Indian market, despite an estimated 10 million cancer
patients in the country. The new regulations, which also clarify
the conditions for bioavailability and bioequivalence studies, have
been welcomed by industry players and by the Indian Society for
Clinical Research (ISCR). The ISCR states that the changes are
"well balanced", will "further the conduct of ethical and quality
clinical trials", and will speed up access to more effective
treatments.
The new clinical trial rules were developed in collaboration
with all stakeholders. By reducing costs, trimming approval
timelines and cutting bureaucratic red tape, they create a more
streamlined legal framework for biopharmaceutical market entry. By
bringing greater clarity to clinical trial rules - and maintaining
robust rules for safety reporting, patient protection and
requirements for compensation payouts - the government hopes the
new rules will also increase clinical research in India. The aim is
to boost investment in domestic R&D of lower-cost innovative
medicines that address the health needs and epidemiological profile
of the Indian population. Currently, India hosts just 1.2% of the
world's clinical trials, despite the country's high disease
burden.
The new measures come as part of a wider policy drive to entice
market entry by reducing regulatory disincentives that have long
been linked by industry players to the lag in availability in India
of the most effective new medicines. Implementation of the CDSCO's
new clinical trial rules comes close on the heels of a new
exemption for all new in-patent drugs from price controls for a
period of five years. Under the same new policy, orphan drugs can
also now circumvent all forms of price control, irrespective of
their patent or new drug status, subject to application for an
exemption.
However, despite the clear rationale behind these measures - to
ease regulatory barriers and thereby encourage market entry - the
apparent fillip to research-based pharmaceutical companies is
deeply mired by the steep challenge of financing advanced and rare
disease treatments in a country where out-of-pocket payments (OPP)
still account for most expenditure on medicines. In short, the
registration of advanced new treatments and orphan drugs is not
synonymous with accessibility for all target patient populations,
least not in a country where public health expenditure accounts for
just 1% of gross domestic product (GDP) and GDP per capita stands
at just USD2,024.68 (2018, IHS Forecasts).
Despite being the second most populous country in the world,
India generates modest sales volumes for premium-priced medicines,
irrespective of product value due to added clinical and economic
benefit. This is due to the absence of a mandatory universal health
insurance system and a comprehensive drug reimbursement mechanism.
For now, access to new patented medicines - for the vast majority
of the population - is either not possible, or it constitutes a
catastrophic health expenditure. For orphan drugs, the
affordability barrier is prohibitively high for all but an almost
negligible fraction of the wealthiest patients in India. The launch
last year of Indian Prime Minister Narendra Modi's flagship
Ayushman Bharat - National Health Protection Mission (AB-NHPM)
initiative, informally known as 'Modicare', has significantly
improved prospects for affordable healthcare. However, there are
several caveats that prevent it from being an immediate game
changer with respect to access to medicines. For example, although
the scheme targets approximately 500 million low-income and
vulnerable beneficiaries, a per family annual ceiling on benefit
cover of INR500,000 (USD7,210) is indicative of the scheme's
constrained purchasing power.
By defining orphan drugs for first time, the new clinical trial
rules do deliver a significant positive for the treatment of rare
diseases in India. This should provide impetus for greater
engagement in R&D initiatives that target rare diseases
affecting Indian patients. However, the ongoing absence of any
dedicated mechanism of public funding for the treatment of rare
conditions blunts the benefit to patients in the immediate term.
Furthermore, patient advocacy groups in India fear that the
exemption of orphan drugs from price controls - without a
pre-existing regulatory framework to formally govern and fund
patient access programs - could in fact put these treatments even
further out of reach for the majority of patients. The new clinical
trial rules come after the government retracted its 2017 National
Policy for Treatment of Rare Diseases (NPTRD), which had been drawn
up to create a permanent funding mechanism for orphan drugs, due to
implementation challenges and financial constraints. The health
ministry has reportedly started to convene meetings with
stakeholder groups as part of fresh efforts to formulate a
comprehensive rare disease policy, but plans for funding access to
orphan medicines still sit in limbo.
By reducing costs and time to market, the new clinical trial
rules will help to entice earlier market entry of effective new
drugs. They represent a step in the right direction and they
signify recognition from the Indian authorities that availability
of innovative medicines requires support for industry. However, it
is unlikely that significant improvements in patient access will be
achieved without higher public expenditure and more comprehensive
reforms; in particular, further policy reforms are needed to
establish more robust pharmaco-economic capabilities for evaluating
a product's budgetary impact and economic benefits, in order to
optimize rational use of resources. Additionally, the health system
needs legal frameworks to formally govern risk-sharing agreements,
patient access schemes and various alternative and flexible
reimbursement models for higher cost medicines that deliver added
value.
Posted 17 April 2019 by Sacha Baggili, Research Analyst, Life Sciences