Cancer is not a single disease, but rather hundreds of different diseases. Thus, there will never be a single cure to cancer. As our knowledge of oncology science advances and expands, a growing number of malignancy types and subtypes are constantly being identified, breaking down target patient populations into ever-smaller fractions. This trend progressively reduces the attractiveness of engaging in the early stages of oncology therapy development in the eyes of Big Pharma and opens the space for small pharma and biotech players.

Oncology therapies typically have a comparatively shorter lab-to-clinic turnaround time than therapeutics targeting other diseases. Sponsors can select the patient population and leverage companion diagnostics, and nonclinical study requirements such as genotoxicity and carcinogenicity studies can often be waived or delayed to later stages of development, especially for end-stage cancer therapies. Biotech companies developing oncology assets thus tend to reach the clinical-stage value inflection point faster than sponsors of other therapies do.

From the clinical stage onwards, oncology therapies’ development key performance indications (KPIs) – such as clinical success rate, average cost, and time to market – are consistent with the average values of general therapeutics development. The investment level in oncology and projected total therapeutics market share by 2025 are, however, head and shoulders above all other therapeutic areas’ indicators.* With this in mind, representatives from Premier Consulting recently attended the FDA’s Oncology Therapy Development Workshop and has compiled several key takeaways to help startup oncology sponsors entering this evolving landscape.

Become Fluent in the Drug Development “Languages.”

As anyone who has ever operated in a start-up environment will tell you, wearing multiple hats simultaneously is the daily norm. The key to success is speaking the language associated with each of these hats:

• The language of business, to attract investors and funds that will take forward the development programs
• The language of accounting, to optimally allocate capital to maximize results
• The language of people, to hire the right people at the right time in the right places
• The language of science, with all its verticals (clinical, nonclinical, CMC, regulatory)

Build a Pitch and Team That Appeal to Investors.

Attracting investors early on is certainly the key to any start-up venture’s success. Investors’ ideal scenario for investing in a company developing an oncology product typically contains the following four elements:

• A drug candidate or technology addressing an unmet need
• A strong IP position
• A comprehensive, efficient development pathway and plan, including a commercial assessment and strategy
• An experienced and collaborative team in place

An investor’s due diligence process is lengthy and includes deep dives and difficult questions around the development path and commercial strategy for the therapeutic asset(s). Investors like to bet on the jockey, not the horse, and will thus look at the team first, the opportunity second, and the science third when making investment decisions. It is therefore very important to assemble a team with appropriate talent and synergy and to plan for the evolving needs of a fledgling venture.

Maximize Output from Interactions with the FDA.

Development of oncology therapeutics is becoming increasingly complex, and regulatory requirements are constantly evolving. The FDA’s authorization of a new drug or device, however, still rests on three core pillars: safety for human use, efficacy in the targeted indication, and purity and potency of the drug product.

Sponsors sometimes mistakenly believe that if they meet with the FDA in advance, the Agency will require them to do studies they do not want to do. However, interacting with the FDA with a non-adversarial approach can go a long way. The FDA is available to guide sponsors and ensure their products meet the requirements for approval, providing various opportunities for interaction at key milestones of a development program.

Sponsors can engage the FDA in formal Pre-IND, End-of Phase, Type A or C meetings, and Pre-NDA/BLA meetings, as well as the more informal INTERACT meetings (INitial Targeted Engagement for Regulatory Advice on CBER producTs, formerly known as “Pre-Pre-IND meetings”) and CATT meetings (CBER Advanced Technologies Team). The latter are both highly recommended for getting early Agency buy-in on the development path of complex biologics or novel drug-device combination therapies at very early stages of development. In addition, sponsors can engage the FDA’s Oncology Center of Excellence to discuss pediatric requirements when developing an oncology product with a pediatric molecular target (per updated FDARA requirements) with a Pediatric Oncology Product Development Early Advice meeting.

The FDA typically only grants one shot at a Pre-IND meeting, so the interaction needs to be carefully planned and scheduled primarily according to development status, though other factors such as fundraising needs are often taken into account in as well. Although a Pre-IND meeting can be requested at any time, having the items below established ahead of the meeting can be very useful in maximizing the value of the interaction:

• Identification of the clinical candidate
• The manufacturing process, set and reviewed by a GMP-compliant manufacturer
• A proposed strategy and planned nonclinical program, including pharmacology and toxicology studies
• A plan for the clinical trial – patient population, proposed dosing regimen, combination with any other therapies, etc.

Secure the Right Development and Regulatory Partners.

It is important to get the FDA’s buy-in early and to keep in mind that the Agency holds everyone to the same standard. Through the various interactions available, FDA experts advise on whether they agree with a sponsor’s development approach and if the proposed program meets regulatory requirements, as well as what information and data the Agency will require to support authorizing approval of a drug candidate for human administration.

However, the FDA is not able to provide consultancy advice and cannot stop development of a program unless it identifies a safety risk to patients outweighing the benefits of the candidate therapy. Engaging with the appropriate consultants early on can ensure that a development program is on the right path toward approval and a successful launch from the start. The right consultant can ensure that your development plan is streamlined and meets regulatory requirements and that interactions with the Agency are leveraged to gain as much insight as possible into the full drug development program.

---

In developing oncology therapeutics, seeking external advice early and often can give you key access to relevant experience, up-to-date perspectives, and unbiased assessments. Premier Consulting’s cross-functional teams can deliver value across all disciplines, providing an end-to-end holistic approach to development planning and regulatory interaction, as well as commercial assessment and strategy. Contact us to find out how we can support your oncology program today.

*Source: EvaluatePharma – Calculating Risk and Return in Oncology Development