MedPath

DCGI Expedites Approval Process for Rare Disease Drugs in India

• The DCGI is fast-tracking approvals for rare disease drugs, aiming to improve patient access and comply with a Delhi High Court order. • State and Union Territory drug controllers are directed to adhere to a 90-day timeline for processing rare disease drug applications. • Clinical trial waivers will be considered for gene and cellular therapies, pandemic-related drugs, and those for special defense purposes. • Priority testing of rare disease drug samples in government laboratories and expedited import processes are also mandated.

The Drug Controller General of India (DCGI) is accelerating the approval process for drugs targeting rare diseases, prioritizing patient access in response to a Delhi High Court order issued on October 4. This order clarified that the import of rare disease drugs should be facilitated in the interest of patients.

Implementation Directives

In a letter dated December 9, 2024, DCGI Rajeev Singh Raghuvanshi urged all state and Union Territory drug controllers to ensure compliance with a 90-day timeline for the approval of rare disease drugs and devices. The directive emphasizes the expeditious disposal of registration certificate (RC) issuance and import processes for these critical medications.
"State drug controllers are requested to monitor compliance with the directions regarding the timeline for the approval of all applications for rare disease drugs which should be processed within 90 days from the date of receiving," the letter stated.
The DCGI has also instructed divisional heads to proactively monitor global and local clinical trials for rare diseases, ensuring that these files are processed swiftly. This includes fast-tracking approvals for clinical trial applications, including post-approval changes such as increasing the number of subjects to facilitate early enrollment.

Clinical Trial Waivers and Special Provisions

To further expedite the availability of treatments, the DCGI will consider clinical trial waivers for gene and cellular therapy products, new drugs used in pandemic situations, and new drugs intended for special defense purposes. These measures aim to reduce regulatory hurdles and accelerate the introduction of innovative therapies.
The regulatory body is also streamlining the import process for rare disease drugs to address urgent patient needs. This includes prioritizing the testing of rare disease drug samples at government laboratories and expediting the issuance of registration certificates.

Existing Regulatory Framework

The DCGI reminded states and UTs that the New Drugs and Clinical Trials Rules, 2019, and Medical Device Rules, 2017, already include provisions under Chapter X for waiving local clinical trials for new drugs and medical devices, particularly those targeting rare diseases. The letter emphasized that these provisions should be actively considered to expedite approvals.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

Capricor Therapeutics Completes FDA Submission for Deramiocel in DMD Cardiomyopathy

Capricor Therapeutics has completed its Biologics License Application (BLA) submission to the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy.
The BLA is supported by data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, showing attenuation of cardiac implications of DMD.

FDA Clarifies Confirmatory Trial Requirements for Accelerated Drug Approvals

The FDA issued draft guidance clarifying requirements for confirmatory trials needed to verify clinical benefit of drugs receiving accelerated approval.
The guidance emphasizes early consultation with the FDA on trial design and the need for sponsors to commit sufficient resources for timely trial completion.

FDA Grants Fast Track and Rare Pediatric Disease Designations to Gildeuretinol for Stargardt Disease

The FDA has granted rare pediatric disease and fast track designations to gildeuretinol, an oral therapy being developed by Alkeus Pharmaceuticals for Stargardt disease.
Clinical data from the TEASE-1 study showed gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to placebo over 24 months.

FDA Approves PTC Therapeutics' KEBILIDI, First Gene Therapy Delivered Directly to the Brain for AADC Deficiency

The FDA has granted accelerated approval to PTC Therapeutics' KEBILIDI, a gene therapy for AADC deficiency, marking the first such therapy delivered directly to the brain.
KEBILIDI works by using an adeno-associated virus to deliver a functional DOPA decarboxylase gene, restoring dopamine production in patients with AADC deficiency.

FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Six Gene Therapies

The FDA has granted Rare Pediatric Disease (RPD) and Orphan Drug Designations (ODD) to six investigational gene therapies developed under the Accelerating Medicines Partnership (AMP) Bespoke Gene Therapy Consortium (BGTC).
These designations aim to incentivize the development of gene therapies for rare diseases affecting children, offering financial benefits such as Priority Review Vouchers and tax credits.

FDA's Rare Disease Innovation Hub Aims to Accelerate Treatment Development

The FDA has launched a Rare Disease Innovation Hub to foster collaboration and accelerate the development of therapies for rare diseases.
The hub seeks to streamline the treatment-approval process, making it more accessible for patients and their families.

Diamyd Medical Pursues Accelerated Approval for Diamyd® in Type 1 Diabetes

Diamyd Medical is set to pursue an accelerated approval pathway in the U.S. for Diamyd® for Stage 3 Type 1 Diabetes.
The FDA has acknowledged C-peptide levels as a surrogate endpoint, potentially expediting the approval process for Diamyd®.

India Waives Local Clinical Trial Requirements for Approved Drugs in Major Markets

The CDSCO's waiver of local clinical trial requirements for drugs approved in well-regulated markets aims to expedite access to essential medications for Indian patients.
This decision is particularly beneficial for patients with rare diseases and those requiring cell and gene therapies, addressing previously unmet needs.

FDA Expands Access to Investigational Drugs for Treatment Use

The FDA has finalized a rule to expand access to investigational drugs for patients with serious or immediately life-threatening diseases or conditions who have no comparable or satisfactory alternative therapy. This rule aims to increase awareness and knowledge of expanded access programs, detailing criteria, submission requirements, and safeguards for different types of expanded access.

Reference News

[1]
DCGI Pushes Fast-Track Approval for Rare Disease Drugs, Prioritizes Patient Access
medicaldialogues.in · Dec 13, 2024

Delhi HC order facilitates import & expeditious disposal of rare disease drugs; DCGI directs 90-day approval timeline, p...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy