Issue link: https://www.e-digitaleditions.com/i/1073928
communicate (for instance, www.PatientsLikeMe. com, www.COPDFoundation.org, www.CFF.org and www.NTMir.org). Contact and volunteer for patient advocacy groups such as the Alpha-1 Foun- dation, COPD Foundation, Cystic Fibrosis Foun- dation, NTM Info and Research Inc. and the PCD Foundation in the US, and similar organizations in other countries. At conferences, go to sessions where patients are speakers. Find physicians who treat these patients, ask them to introduce you, then invite those patients to your institution to tell their stories. Use all that knowledge and emotional energy for bold assessment of critical attributes of new products, then share that information with your colleagues in the regulatory bodies, compen- dial and professional organizations such as the United States Pharmacopeia, European Pharmaco- poeia, the American Association of Pharmaceutical Scientists (AAPS), the International Society of Aerosol in Medicine (ISAM), the American Tho- racic Society (ATS) and the European Respiratory Society (ERS), as well as with those in cross-indus- try groups like the International Pharmaceutical Aerosol Consortium on Regulation and Science (IPAC-RS), the European Pharmaceutical Aerosol Group (EPAG), etc. With regard to regulations, have an open mind and examine existing draft and final guidelines to see if they are still appropriate, or if changes should be made in line with our current scientific knowledge, including the nature of the target diseases and criti- cal safety and efficacy issues for patients. You can also offer to serve on standards and pharmacopeial committees, where public expert participation is usually welcomed. Determining aerodynamic particle size distribution An "impactful" contribution can be made in this area, which is unique to pharmaceutical aerosols because aerodynamic size distribution is the key determinant of their biological impact. As you will see, there are significant opportunities to advance this field for the benefits of many. Size fractionation by cascade impactor Prior to the use of impactors or impingers, the "regula- tory" method was optical microscopy. However, it did not provide information about "aerodynamic" prop- erties of inhaled medications that related to their deposition and biological effects in the respiratory tract. Often it could not distinguish between the drug and the non-volatile excipients and, in the absence of electron microscopy, it could not quantify sub-visible particles. Today of course, electron microscopy is much more accessible and in conjunction, for instance with single particle spectroscopic methods, can be a powerful tool in inhalation formulation develop- for other indications or administered by other routes. Such drugs may be able to treat severe respira- tory diseases via inhalation while reducing the risks and timelines required to accumulate knowledge about new chemical entities. 3 There are excellent examples of success with this approach, including inhaled antibiotics for cystic fibrosis, where drugs previously approved as injec- tions for acute infection treatment were developed as chronic inhaled prophylactic and palliative treat- ments (e.g., tobramycin, aztreonam and colistin). e most recent success is the US approval of inhaled liposomal amikacin (ALIS, Insmed) for the treat- ment of patients who have refractory lung infections with Mycobacterium avium. But beware, as the former CEO of Genentech, Kirk Raab, remarked, "The longest path to get a drug approved is a shortcut." 4 e repurposing approach still requires thorough safety studies focused on the inhaled route of administration, proof of efficacy in the new indication and demonstration of well-con- trolled quality of the product. Getting involved with patients A constructive, collaborative approach among industry, healthcare professionals, regulators and payors—with patient advocacy groups at the center of the dialogue—would be the most promising and impactful way to develop and supply the correct medications for the appropriate indications at an affordable cost to society. e intensive participation of patients in clinical trials must be matched with their presence in making decisions about develop- ment of new products. Getting them involved early, for example, through participation in focus groups, could help identify product attributes that are important for patients using a particular therapy. It is very encouraging that the United States Food and Drug Administration (FDA) has been actively involved in this process. 5 The ultimate goal should be about improving patients' quality of life; therefore, their voices need to be reflected in setting targets for the balance of safety and efficacy. I believe this approach can profoundly accelerate the process of making accessible, precise and individualized therapies. An example of this type of collaboration and a "step in the right direction" are the recent European Respiratory Society Guidelines on management of bronchiectasis, which represent the collective views of patient representatives and key opinion leaders. 6 So, budding OINDP scientists, get on with research in the most challenging areas. And get to know patients and their aspirations to have better lives. You will be infused with motivation like you have never felt before. ere are many ways to learn and get involved. Read blogs on the web where patients 18 February 2019 Inhalation