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MACRO Trials: Optimizing trials for rare diseases

MACRO Trials is building a digital contract research organization (CRO) to run distributed clinical trials in the fast-growing and operationally-complex rare disease space. MACRO partners with biopharmaceutical companies and CROs to help them design, plan and execute complex trials, bolstered with a breadth of digital offerings to enable decentralized trials.

A textbook Healthy Ventures investment

  • Identifies a lucrative market gap - in this case, the intersection between rare disease and clinical trial site optimization 
  • Sells to life sciences, a non-cyclical customer base that is a core focus of our portfolio and where our relationships run deep
  • Quickly tractioned in the market, now running 4 clinical trials and generating ~$2m in revenue for 2021
  • Marquee customer roster including 8 of the largest 20 CROs and several major pharma companies 
  • Powerhouse founding & advisory team with decades of experience running clinical trials
  • We preempted a competitive process through our relationship with a prior investment 

Rare diseases represent an enormous market opportunity

The market for drugs focused on rare diseases (so called ‘orphan drugs’) is exploding. Over the past 20 years, approvals for orphan drugs have more than quadrupled to account for 44% of all new drug applications. Orphan drug sales are growing at twice the rate of non-orphan drugs and are expected to be $242B by 2024, capturing 20% of worldwide prescription sales.

Yet, while rare diseases are rapidly becoming the most important area of new drug development, they are fundamentally underserved by traditional clinical research infrastructure. Because rare diseases are uncommon, the pool of patients eligible for a trial is scarce, heterogenous and widely dispersed. Additionally, pediatric patients make up about half of the rare disease population, further complicating clinical trials. While recruitment and retention are one of the most challenging aspects of any clinical trial, it is exponentially more difficult in rare disease.

The market for performing clinical trials in rare diseases is bimodal: wanting to participate in such a large and growing market, big CROs try to facilitate these trials but often fail because the logistics and operations are so different from their bread-and-butter mainstream patient trials. There is also a long tail of specialized CROs that typically focus on only a few areas and have a very difficult time scaling their operations to meet demand. CRO performance in rare diseases has been so poor that many biopharmaceutical companies try to run their own trials.   

MACRO Trials steps into this lucrative market gap: they specialize in rare disease trials and in optimizing clinical trial sites. Although early, they’ve already delivered incredible results to their partners, exceeding 100% of enrollment targets, 2x faster enrollment, and 90% retention across therapeutic areas.


The key leader at MACRO is CEO Daniel Perez who has spent his entire career in clinical trials. Prior to MACRO, he spent ten years running all aspects of study design and implementation at Cedars-Sinai and Children’s Hospital Los Angeles.

Daniel is flanked by the Company’s two co-founders, Dr. Azizzadeh and Dr. Hamilton, who are physicians as well as well-regarded principal investigators in clinical trials, bringing real-world understanding of what sites and physicians need to run a successful trial in rare diseases. They recognized early on the revolution from clinical trials being run exclusively at academic or professional study sites to hybrid or distributed trials.

Hot M&A Market with Many Routes to Exit

Clinical trial technology is in high demand among several categories of buyers, many of which are already MACRO customers. CROs are snapping up clinical trial solutions as they look for ways to accelerate recruitment timelines and bolster their own capabilities, while pharma sponsors have been equally active, hoping to realize efficiencies by bringing more of the trial operations in-house. Consolidated services groups such as IQVIA could similarly be candidates as they look to technology to scale. 

In just the last year, IQVIA (a consolidated services group) acquired trial lab services provider Q2 Solutions for $760m in April 2021, as well as virtual trial provider Obvio Health in March 2021. On the tech side, Google subsidiary Verily acquired trial site software provider SignalFire in August 2021.

MACRO Trials is a portfolio company of Initiate Studios, in which we are an investor. This allowed us to get an early look at MACRO and pre-empt competitors who also wanted to invest in this round. 

We invested $500k in an uncapped note at a 20% discount, and secured an allocation for 20% of the upcoming Series A round in early 2022. 


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