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Understanding the FDA's Drug Approval Process: A Primer for Deep Tech Startups | Deep Tech Catalyst

A chat with Daniela Drago, Regulatory Expert, Partner @ NDA Partners

Welcome back to Deep Tech Catalyst, the channel where science meets venture!

We're thrilled to welcome Daniela Drago, Regulatory Expert, and Partner at NDA Partners!

Today, we'll dive into the complexities of new drug approval and marketing, exploring the essential criteria required by the United States Food and Drug Administration (FDA) and tackling the challenges and risks associated with IND submissions.

We'll also discuss emerging regulatory trends for 2024 and share valuable advice for those interested in pursuing a career in regulatory affairs.

Let's dive into it!

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💊 FDA 101 for Aspiring Deep Tech Founders

The Food and Drug Administration, or FDA, is the authoritative body in the United States responsible for the approval and marketing of new drugs.

It establishes regulations, guidelines, and standards throughout the drug development process. This regulatory framework combines legal, administrative, and scientific measures.

There are three essential criteria for a sponsor seeking approval for a new drug in the U.S.:

  • the drug must be safe;

  • it must be effective in treating the targeted disease;

  • it must be manufactured and controlled to ensure quality.

While this may sound straightforward, it actually involves years of research and substantial resources.

The process of bringing a new drug to market is lengthy and costly. Indeed, it's a complex, expensive, and risky journey to achieve that one success story, where many candidates do not succeed.

🤔 Why is Bringing a New Drug to the Market so Hard?

According to published data, it typically takes between 10 to 15 years and between $1.3 to $2.6 billion to transition a new molecule from the laboratory bench to the patient's bedside.

So, a well-defined roadmap and understanding of these steps are crucial.

For example, the development process for small molecule drugs is generally divided into 6 main phases: drug discovery, preclinical testing, and then phases [1-4] of clinical trials.

In the U.S., the two key regulatory milestones are the submission of an Investigational New Drug (IND) application before beginning clinical trials and the submission of a New Drug Application (NDA).

Approval of an NDA allows a company to market its product in the U.S.

What Factors Indicate Whether a Drug Candidate Will Advance to the Next Stage?

Focusing on the early phases, if you're a startup thinking about potential outcomes and risks, it's essential to understand the factors that indicate whether a drug candidate will advance to the next stage.

The first step in drug development is discovering targets.

Researchers examine tens of thousands of compounds, and through a rigorous selection process, identify a viable drug candidate. Statistically, out of every 5,000 to 10,000 compounds in the discovery phase, only about 250 advance to preclinical testing.

At this early stage, several factors must be assessed. For instance, it's crucial to evaluate the drug's formulation to ensure it's suitable for the intended indication, disease severity, and patient population.

The drug's half-life, which is the time it takes for its concentration in the body to reduce by half, must also be determined to ensure it meets the proposed dosing frequency and treatment duration.

Additionally, the toxicity study duration supporting the planned clinical trial must be scrutinized. The material used in preclinical studies must be comparable to that proposed for clinical trials.

How to Mitigate Risks in IND Submissions (and Consequently in Startup Operations) Before Seeking FDA Approval

Some companies engage with the FDA early on, requesting meetings to discuss these and other factors influencing the probability of moving to the next stage.

For a sponsor aiming to enter a clinical trial in the US through an IND (Investigational New Drug), engaging with the FDA is crucial for de-risking your IND submission. You want to ensure that, upon submitting your IND, the agency permits you to proceed with the trial.

During pre-meeting discussions, it's advisable to present your plans and inquire whether the agency concurs with your proposed activities. This approach helps de-risk the submission, not because the FDA acts as a consultant, but rather as a partner, ensuring your submission is not halted by a clinical hold.

🧗 Common Challenges for Founders in IND Submissions

When navigating FDA regulations, startups face several pitfalls and challenges, particularly in the early stages. Many organizations lack the experience needed to compile an IND submission—a task requiring a skilled team. Trusting a team with the capability to administer products to human subjects underlines the importance of safety for human subjects.

Startups sometimes underestimate this, resulting in clinical holds.

Following an IND submission, the FDA has 30 days to review it, with its primary objective being to protect study participants from unreasonable risks. If the agency identifies any issues, it can impose a “clinical hold”, preventing the drug from being administered to study participants.

4 Key Suggestions for Avoiding a Clinical Hold

Here are 4 key recommendations to avoid a clinical hold:

  1. Select the Right Animal Model: Non-safety studies must be adequate and comprehensive, allowing the FDA to assess the investigational treatment's risk to humans.

  2. Focus on Chemistry, Manufacturing, and Control (CMC): It's vital to show that the drug product is adequately controlled and that manufacturing processes do not introduce impurities that could harm patients.

  3. Document Safety Expectations in the Informed Consent Form: Ensuring that participants are fully aware of the potential risks is essential.

  4. Engage the Right Experts: Emerging biotech firms often lack subject matter experts in all necessary disciplines to compile a robust IND. Consulting with external experts can be invaluable.

These steps underscore the importance of thorough preparation and expert consultation in successfully navigating the FDA's regulatory landscape.

How to De-risk the Manufacturing Process?

To ensure the manufacturing process does not introduce risks into your drug development project, especially from a hardware perspective transitioning into biotech and drug discovery, a multifaceted approach is required.

This involves a combination of chemistry assessments and both in vivo and in vitro evaluations to understand, particularly for small molecules, the potential impurities that could arise from the synthesis process.

Additionally, toxicology studies play a crucial role, combining with the chemistry assessment to ensure the investigational drug has an acceptable impurity profile. Seeking expertise in these specific areas is crucial to navigate these complexities effectively.

🚀 Emerging Regulatory Trends to Watch in 2024

Looking ahead to 2024, several regulatory trends worth noting include the advancement of digital health, the expansion and democratization of gene therapies, and addressing the regulatory talent shortage.

  1. Advancing Digital Health: The integration of digital health technologies is transforming healthcare by improving remote patient monitoring, data collection, sharing, and analysis. These technologies aim to reduce inefficiencies and costs while increasing access and flexibility in care. Ongoing efforts to define standards and best practices in digital health are expected to continue, further shaping the landscape.

  2. Gene Therapies: Recent years have seen significant excitement in the gene therapy field, with regulatory frameworks evolving in the US, Europe, and other regions to keep pace with scientific advancements. However, patient access barriers, particularly in certain geographies, remain a concern. This area is likely to see continued focus and development.

  3. Regulatory Talent Shortage: The life sciences industry has experienced significant growth, leading to a high demand for regulatory talent. Overcoming the image problem associated with regulatory professions and attracting more individuals to this field will be essential. Efforts to enhance the perception and appeal of regulatory roles are crucial to address this shortage.

💼 Off-Topic: How to Start a Career in Regulatory Affairs?

For scientists interested in pursuing a career in regulatory affairs post-PhD in chemistry, biotechnology, or medicine, the field has evolved significantly, becoming more professionalized and specialized.

Regulatory professionals may specialize in areas such as strategy, and operations, or focus on specific product categories like drugs versus devices.

Key attributes for success in this field include the ability to understand and communicate science effectively, inquisitive nature, diplomacy, negotiation skills, and the ability to manage projects efficiently.

In summary, transitioning into a regulatory role involves not just a deep understanding of the science but also the skills to navigate the complex interface between innovation, regulatory standards, and market demand.

This is all for today!

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