Clinical Development & Medical Affairs

10/3/2021 0 Comments

Clinical Trials in Rare Diseases: Challenges and Opportunities

In addition to disease-specific barriers, therapeutic developments in rare diseases face many challenges, such as incomplete understanding of natural histories to inform trial designs, need for alternatives to randomized controlled clinical trials, requirement for more sensitive outcome measures to quantify diseases, limited access to resources – including funding – required to set up clinical trials, and difficulties of recruiting small patient samples to participate in trials.
Among the above barriers, patient recruitment and enrollment is probably the most challenging, as enrolling enough patients in order to generate statistically meaningful results from a trial can turn to be highly time, energy and money consuming. Such a challenge may be overcome by good trial planning and design from the outset. Involving patient groups at as an early stage as study design could be rewarding as this can help suit patient needs and ensure those that join a study do not withdraw prematurely.
Planning, design and delivery of clinical trials is rare diseases may also be very challenging in small, dispersed populations, from both logistical and statistical perspectives. Here are some insights that could help on conducting a clinical trial in a rare disease:

Engage with regulatory authorities early so that an appropriate strategy to achieve approval can be agreed.
Investigate retrospective studies that can be used to better understand the disease’s natural history, define study endpoints and identify patient groups who are likely to show treatment effects while tolerating the treatment.
Maximize the information from the fewest patients – it is desirable that all patients receive the active drug. When patient numbers are very small, a single-arm design may be unavoidable. However, where larger patient numbers are feasible, the use of two-stage parallel designs can gather more information whilst still having all patients receive active drug.
It is important to perform a formal sample size calculation – Wherein the expectations of treatment effect size and variability of the primary endpoint are incorporated with adequate control of type 1 error and suitable statistical power given the expected efficacy.
Plan your data collection processes carefully – The small number of patients makes it even more important to ensure that the data collected in rare disease studies is of the highest quality so that you can maximize usage of all data available.

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19/1/2021 0 Comments

Medical Affairs: Back to Basics

In the setting of increasing challenges for the Pharma industry, medical affairs teams have wide opportunities to take the lead to improve their companies’ positions for a quality drug development, while better addressing needs from patient, physicians, regulators and stakeholders.

Tips to make medical affairs on the top could be summarized to the following:

Deeply know the product and understand the disease
Excel at communicating scientific evidence
Link scientific and clinical results to patient outcomes (and do not only focus on product)
Build long-term relationships with a variety of stakeholders (HCP, payers, patients, KOL, providers) allowing a better understanding of the market and patient needs
Actively participate to development strategy of new drugs by providing market-based input on unmet medical needs and clinical requirements
Develop a strategy for market education on a newly launched product
Design and implement data generation strategies

To do so effectively, medical value teams need to be an integral part of cross-functional product development and product management teams. By working with all pharma functions, they can help increase the company’s understanding of unmet medical needs, and attractiveness of treatment pathways, thus valuating clinical trial protocols options, speeding regulatory approvals and increasing launch success rates.

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19/1/2021 0 Comments

Pilar Role of Medical Affairs

Never before have Pharma companies developed data and scientific insights. But information channels are changing, and many companies have difficulty communicating science effectively with the growing array of stakeholders that influence purchasing decisions.

Equipped with deep product knowledge and disease understanding, medical affairs teams can become a strategic ace in an era of Big Data. They are well positioned to generate and present high-quality scientific knowledge to the market and educate stakeholders about next-generation products.

In one hand, a huge amount of data is flooding the market, leaving physicians, payers and healthcare providers struggling with data overload. On the other hand, physicians are reducing their reliance on sales representatives and are keener to seek scientific sources of information. Medical affairs have to be that bridge between stakeholders and pharmaceutical companies.

An experienced medical affairs team can link scientific and clinical results to patient outcomes, adding value at every stage of a drug’s development. When discussing a potential new compound with physicians, payers and opinion leaders, for instance, medical affairs teams gather vital feedback on the drug’s market potential and patient needs at the earliest stages of the drug development process. The insights they glean over time can improve return on investment and create a strong competitive advantage by helping companies design more effective clinical programs and launches.

The winners in an era of Big Data will transform medical affairs teams into medical value teams with three strategic roles: communicating scientific evidence, providing market-based strategic input to drug development and portfolio management, and overseeing the effort to produce Big Data and real-world evidence.

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