From a puddle of potential amalgams, a limited select leads that validate a association between chemical structure & target-based action in a biochemical or cell-based assay are produced. The present drug development tunnel is multi-layered and intricate, consist of higher risk that a medicine will not be fruitful even after many resources have been capitalized. Drug development faces two challenges: rising costs and pressure on pricing. The lack of action by experts to reduce current health needs has not been proven, as pharmaceutical companies and many others are debating ways to maximize the benefits of research and development.
“When something is important enough, you do it even if the odds are not in your favor.”
For patients with chronic disease, the new treatment should have a significant improvement in efficacy and / or superiority. Small improvements are not innovative and therefore do not cover the cost of tags. On the other hand, although successful treatment does not yet lead to many diseases, there are reasons. Some Disease treatment is difficult, such as schizophrenia or Alzheimer’s disease, or unnatural or unsafe medications such as antibiotics.
- Drug development is a long, difficult and costly process, rooted with a high grade of ambiguity that a medicine will actually thrive.
- Determining unknown pathophysiology is difficult in many diseases
- Most animals model do not recap an entire disease.
- Heterogeneity challenges in clinical patients can be addressed through clinical phenotype and endotype.
- Greater prominence over-human data can improve target identification & validation.
- Valid and Therapeutic biomarkers are not available to detect and resist biological agents.
- Existing regulatory processes for INDs application will not be resolved in pre-IND meetings.
The combination of increased drug development cost and disproportionate investment in drugs, challenges the current business model of the pharmaceutical industry. Therefore, a new design is necessary to develop and reduce drug cost. Some models use non-clinical or translated information to improve efficacy and drug safety.
Though more new drugs were approved in 2019 by the US FDA or the EMA, than in several preceding decades, the burden between cumulative development budgets per launched medicine & largely capped societal expenditure remnants to challenge the prevailing business prototype of major pharma companies.
The term “adaptive trial design” is used to designate a method that allows modifications to the design model based on (adaptive trial design) data visualization inside the clinical trial and concurrently monitoring the inclusive type I error level. The Adaptive trial design should be pre-selected in the protocol and should not be added while running clinical trials. “Adaptive trial design” rarely refer to amendments to protocol that involve minor modification of inclusion or exclusion criteria. Adaptive trial design should not be muddled with adaptive licensing While considering adaptive design, team must be conscious of risks for trial veracity. Therefore, if investigator and patients know interim results, the results of the interim analysis may possibly threaten trial integrity & acquaint with bias. If the sponsor or advisory board changes the characteristics of the clinical study, this will also affect the trial integrity.
Adaptive trial design can improve the effectiveness of drug production by reducing time. However, there are three important things to keep in mind:
- The complete type I error should be sturdily controlled.
- Prospects for adaptation should be redefined prospectively.
- The timeline of the interim assessment should be premeditated prospectively.
Increasing drug development cost adds to the current market trend in the pharmaceutical industry to increase in drug prices. Public-private partnerships, innovative adaptive designs, and the widespread use of clinical data provide greater opportunities for drug development. Which of these services is usually effective can only be determined in the future. Today, we believe that all process offered to competitors expect better and efficient research and development. However, the benefits of these options are only realized when we understand the basic limitations of each. Given all these areas, it can conclude that the pharmaceutical industry will not be able to offer new drugs in isolated area. However, we advise companies to provide new drugs for the existing health needs with mutual collaboration between scientists, regulators, academic investigators, and most importantly patients.