QR Group analyzes the regulatory impacts of ANVISA’s new guidelines and the challenges for the pharmaceutical industry

In recent years, the regulation of medicines for rare diseases in Brazil has undergone significant advances. ANVISA has been working to make registration processes more flexible and faster, especially for innovative drugs aimed at treating conditions with a high burden of suffering and few therapeutic options available.

QR Group, a consulting firm specializing in health product regulation, analyzes the current scenario, the impacts of the measures adopted by ANVISA, and what companies need to consider when entering this highly technical and sensitive segment.

What changes in the regulation of medicines for rare diseases?

Historically, patients with rare diseases have faced difficulties in accessing treatments due to delays in health registrations, absence of regulatory incentives, and high research and production costs. To face this scenario, ANVISA has implemented measures such as:

• Prioritization of analysis for drugs intended for rare diseases;

• Adoption of conditional registration in cases with robust preliminary evidence;

• Possibility of using international data, especially from FDA and EMA;

• Strengthening expanded access and compassionate use policies.

These changes aim to balance the need for safety with therapeutic urgency, accelerating access to innovative treatments without compromising technical evaluation.

Opportunities and challenges for the pharmaceutical industry

Despite the advance, the sector still faces important challenges:

• Requirement of consistent clinical dossiers, even in conditions with low prevalence;

• Need for regulatory strategies adapted to the specificities of the disease;

• Rigorous post-market follow-up, including with real-world data;

• Constant technical dialogue with ANVISA.

Companies interested in operating in this field should invest in customized regulatory strategies and rely on experts to navigate a regulatory landscape that is still in continuous development.

How QR Group supports companies in this strategic segment

QR Group offers complete regulatory advice to companies operating or looking to enter the rare disease drug market. Our services include:

• Development of strategic regulatory routes, based on ANVISA guides and international benchmarks;

• Assembly of clinical and non-clinical dossiers, even with limited data;

• Formal technical consultations with ANVISA for early clarification;

• Support in conducting compassionate use or expanded access programs.

With a technical, safe and personalized approach, QR Group helps its customers transform innovations into solutions accessible to the population.

Talk to QR Group

Does your company develop medicines for rare diseases?
Count on QR Group’s technical support to safely navigate ANVISA’s requirements and ensure the access of your products to the Brazilian market.

Source: QR Group