What are the Trends in Small Molecule Pharmaceutical Development?
There are several factors driving trends in small molecule pharmaceutical development. We have identified some which are referred to as the 6 “Ps”:
1. Potents
Specialised drugs for various cancers represent the largest area of R&D meaning that approximately 30% of drugs in development remain classified as highly potent throughout their lifecycle. Pressure to accelerate drug candidates in all therapeutic areas through to Proof-of-Concept stage of clinical development as quickly as possible can also mean starting formulation development work earlier and before important toxicological data on the drugs are available. This results in the assumption that these drugs are potent to maintain employee safety. Ensuring safe handling is paramount therefore the installation of well-engineered high containment systems around development and manufacturing processes is essential.
2. Paediatrics
Many factors need to be considered for paediatric development and manufacture. Children’s medication is required to span large age and developmental stages. Also, it must be palatable meaning that taste masking becomes a key factor. Unfortunately, there is limited clinical trial data available for paediatric treatments which results in adult studies being utilised and reformulated. Regulatory standards regarding these medications are very stringent and must be adhered to. Creating dosage forms specifically for children is key. These include mini tablets and granule formulations which are filled into stick packs, sachets and sometimes capsules. These measured quantities protect against overdosing and enable easier administration at point-of-care such as ingestion with food or reconstitution.
3. Personalised Medicines
Accelerated Approval and Fast Track designation create opportunities for pharma companies who are developing drugs for rare diseases. At the manufacturing stage of the supply chain, the emergence of so many niche drugs with small patient populations have impacted batch sizes and production volumes. More and more frequently we see drugs which remain at what would have historically been “clinical scale” right through to registration and the product commercialisation phase. This impacts a company’s strategic capital investments. Often the most important element of production is the efficient conversion of a limited supply of expensive API into a good quality drug product. GMP manufacturing capabilities and supporting systems must therefore be agile enough to simultaneously support both clinical and commercial manufacturing.
4. Poorly Soluble Drugs
Recent reporting indicates that 40% of approved drugs and 90% of molecules in the discovery pipeline are poorly water soluble. This creates a challenge to develop and manufacture formulations with good bioavailability. There are many aspects to consider when formulating a poorly soluble API.
5. Project Timelines/Project Management
Many Pharma companies have expressed a need to compress the overall clinical supply chain and accelerate the development of products from drug substance through to formulated drug product and onward to commercialisation. Managing development activities to strict timelines, identification and timely resolution of development challenges, alignment of the various elements in the supply chain and clear and concise communication has never been more important. Successful project managers identify and mitigate risks, effectively managing the resources available to both themselves, the project team, and ensure stakeholders are well informed throughout the project lifecycle.
6. People
Having the best facilities and equipment may be advantageous, but without a strong technical team success is not guaranteed. Partnering with a highly skilled team who can support products from the early stages of pharmaceutical development through to commercial supply is key to success.
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