The drug development landscape has been evolving over the years, from an earlier emphasis on blockbuster drugs developed in-house by large pharmaceutical companies, to a more inclusive, patient-centered paradigm that includes personalized medicine, orphan drugs for rare diseases, new clinical trial models, and the growing use of contract research organizations (CROs). These trends have revitalized drug development, opened new markets, made cutting-edge treatments accessible to more patients, and enabled the inclusion of a more diversified range of study participants to allow for targeted therapies that address the individual needs of each patient. Let’s take a closer look at these trends.
1. Personalized Medicine
A growing awareness of the limitations of a one-size-fits-all model of medicine, along with new technologies, has made the burgeoning field of personalized medicine both sought after and increasingly attainable. A customized approach to drug development allows medications and dosing to be more finely adapted to each patient’s genome, environment, and prognosis, with the benefits of greater efficacy and fewer adverse drug events. These benefits extend to both post-market patients and clinical trial participants. Early examples of personalized drug applications include the development of trastuzamab for the treatment of HER2-positive breast cancers, and more refined dosing of the anticoagulant warfarin.[i]
The ability to do the DNA sequencing needed for this kind of approach is fairly recent. While the Human Genome Project was started in 1990, the mapping of the complete genome was only announced in May 2021. Scientists can now explore which of the millions of genetic variations between people are implicated in various diseases, then potentially treat and prevent those diseases by addressing lifestyle and medication risks. Information obtained from DNA sequencing will also enable clinical trials to be conducted in a more targeted way by choosing only those participants likely to benefit, leading to faster testing, shorter regulatory review times, smaller trials, and lower costs. The trend toward a targeted approach allows more drugs to reach the market more quickly for that subset of patients.
2. Orphan Drugs for Rare Diseases
In the U.S., a rare disease is defined as one that affects fewer than 200,000 people domestically (approximately one in every 1,655 people), while the European Union puts the number at one in 2,000 people. While each disease affects a small number of people, the large number of different rare disease conditions – around 6,000 to 8,000 – means that about one in every 12 people is affected by rare diseases overall. In the past, this huge need was left largely unserved, given the low market potential for each individual treatment. Since the 1983 Orphan Drug Act was passed in the U.S., giving pharmaceutical companies incentives in the form of tax credits for the sake of public health, there has been growing activity in this area. Positive public opinion and recent advances in information technology that allows researchers to link to hospitals to access patient data has enabled even greater progress. With increased need and more refined technologies, this is a trend that can be expected to continue into the future.
3. Decentralized Clinical Trials
Physical distancing requirements during the COVID-19 pandemic have driven an already-existing trend toward decentralized clinical trials to the forefront for long-term, low-risk patient trials requiring a limited number of clinic visits. Recent advances in digital technologies, like wearable devices, have helped make it possible for data to be collected from patients in their homes or doctor’s offices without the commuting time and other inconveniences of participating on-site. These factors have lowered barriers to participation, resulting in greater patient interest and retention, and giving researchers access to a larger, more diversified population of participants.
4. Growing Use of CROs
As both drug development studies and regulatory processes have grown in complexity, the specialized knowledge, expertise, and resources of CROs have become increasingly important, with a 2018 survey finding that 61% of clinical trials are outsourced worldwide.[ii] Specialized CROs serve as an extension of the drug development team, providing guidance and expertise on the design and conduct of drug trials. In addition, many of the most innovative technological achievements have come from the emerging biopharma sector currently experiencing a high growth rate. These emerging biopharma companies are relying on outsourcing to complement their lean, entrepreneurial organizational models. The growing rate of clinical trials in general has also contributed to this trend, aided by the globalization of research: as more countries have built better infrastructures and acquired more advanced technologies, research can be conducted in a broader range of geographical areas. In fact, the website clinicaltrials.gov provided by the U.S. National Library of Medicine lists 220 countries, with 63% of clinical studies taking place outside of the U.S.[iii] The flexibility and agility of CROs offers faster turnaround times, with the COVID-19 pandemic having played a part in driving demand given the need to respond quickly to regulatory complexities and evolving research requirements.
Within the context of technological advances and an ongoing demand for patient-centered research and medical care, the further growth of personalized medicine, orphan drugs for rare diseases, new clinical trial models, and CROs holds promise for more targeted therapies, greater inclusiveness in drug trials, and shorter drug development timelines.