Ophthalmic Start-Up Chief Executive Officers' Perceptions of Development Hurdles.

PURPOSE: To identify current challenges facing ophthalmic pharmaceutical start-ups in developing new products. METHODS: Surveys were distributed to the chief executive officer (CEO) or president of ophthalmic start-ups. RESULTS: The survey attracted 24 responses from 78 surveys distributed (31%). The CEOs stated that a lack of financial capital (n = 18, 75%), FDA regulations (n = 6, 25%), and failure to meet clinical endpoints (n = 6, 25%) were their greatest development hurdles. Risk aversion to medicines in early development (n = 18, 75%), mergers and acquisitions reducing corporate choice for licensing agreements (n = 7, 29%), the emergence of large pharmaceutical-based venture capital funding groups (n = 12, 50%), and the failure of many large pharmaceutical companies to develop their own medicines (n = 10, 42%) were noted as recent prominent trends affecting fundraising. CONCLUSION: The study suggests that development funding, regulatory burden, and meeting clinical endpoints are the greatest development challenges faced by ophthalmic start-up CEOs.

What data sources do ophthalmologists trust?

To survey ophthalmologists regarding sources they trust when incorporating new medical knowledge into their practice. The survey was distributed primarily to US-based ophthalmologists. Questions were derived based on the lead author's research experience from congresses and discussions and from mentions in the medical literature. In total, 77 physicians completed the survey of 1886 sent (4% response rate). Regarding study design, physicians preferred a well-controlled, randomised, double-masked trial (99%) with multicentred investigational site across a wide geographical area (80%). Authorship of a research article was most desired from a well-known key opinion leader (KOL) (75%) or any KOL leader at a university (75%). The most selected journal type was a subspecialty publication (86%) and second a multispecialty high impact journal (77%). Study sponsorship was most desired from the NIH or other government agencies (71%) or a university (71%). Doctors preferred clinical opinions from an ophthalmic medical society (75%). For the source of new clinical data, physicians indicated an unsponsored peer-reviewed journal article (77%) or a lecture at a large ophthalmic congress (74%) as the preferred source. Ophthalmologists generally desire sponsors, study designs and opinions that appear free of bias on which to base their clinical practice decisions.

Description of Ophthalmic Pharmaceutical and Device Start-Up Companies.

AIMS: To describe the number, type and location of ophthalmic companies and their associated product areas and indications. METHODS: A retrospective, non-patient-based, observational review of ophthalmic pharmaceutical and device companies with a new product in development. Data was compiled by Internet searches. RESULTS: We identified 190 companies currently developing ophthalmic products: 134 (71%) were privately held and 56 (29%) publicly held, while 136 (72%) were in the United States and 53 (28%) were outside the United States. There were 436 total products of which 338 (78%) were pharmaceuticals and 98 (22%) devices. With pharmaceuticals we identified 46 separate indications with age-related macular degeneration (n = 75), glaucoma (n = 52) and dry eye (n = 46) as most common; anti-vascular endothelial growth factor, hormone therapy and anti-inflammatory products were also common classes. With devices there were 30 indications with glaucoma (n = 26), age-related macular degeneration (n = 19) and dry eye (n = 6) as most common; drug delivery, ocular implants and prostheses were less common classes. CONCLUSIONS: Ophthalmology as a specialty is benefited by a wide effort in new medicine and device development. However, a concentration of effort into relatively few indications suggests a potential lack of market analysis and possible difficulty for many companies in commercializing their product.

Long-term cost and efficacy analysis of latanoprost versus timolol in glaucoma patients in Germany.

AIM: To evaluate 5-year effectiveness and cost between latanoprost or timolol monotherapy in a pilot trial. METHODS: A retrospective, multi-center trial performed at 6 sites in Germany of patients who had a diagnosis of primary open-angle or pigmentary glaucoma, in at least one eye, initiated on monotherapy with latanoprost or timolol maleate. Qualified consecutive charts were reviewed in which 5-year efficacy, safety and cost data was abstracted. RESULTS: Seventy-seoen latanoprost and 49 timolol patients were included, at the final visit no difference existed between the two groups in disc parameters including: rim area, rim area/disc area ratio, cup volume or vertical cup/disc ratio (P>0.05). There was no difference in intraocular pressure (IOP) between the initial latanoprost (17.4±2.6) and timolol (16.3±2.8mmHg) groups. There was less change in medicines over the follow-up period (0.1 vs 0.8) and fewer medications at the final visit (1.2 vs 1.8) with latanoprost compared to timolol. No patient treated with latanoprost discontinued therapy during follow-up, while 12% discontinued timolol mostly due to inadequate IOP control. Cost/year was less with initial timolol ($458±236) as compared to latanoprost ($552±202). CONCLUSION: Patients begun on latanoprost or timolol and followed over 5 years may have similar clinical outcomes. However, timolol patients may require more medicines and medicine changes to control IOP for long-term, but at a lower cost.

Long-term outcomes of prostaglandin analog versus timolol maleate in ocular hypertensive or primary open-angle glaucoma patients in Europe.

PURPOSE: To determine the direct costs of therapy over 5 years of a European monotherapy cohort begun on a prostaglandin (PTG) versus timolol in patients with primary open-angle glaucoma or ocular hypertension. METHODS: A retrospective, multicenter, active-controlled, observational study. Data were abstracted for European patients treated as initial monotherapy in 1996 or afterward, with 5 years of available records. RESULTS: This study included 271 patients (166 on a PTG and 105 on timolol at baseline). The average cost/month/patient over 5 years was $45.47±12.61 for PTG and $31.50±15.47 for timolol (P<0.001, based on German prices). After 5 years, although there was no difference in number of glaucoma medicines prescribed between groups (1.0 PTGs and 1.1 timolol, P=0.41), the timolol group demonstrated a higher intraocular pressure (17.7±2.9 vs. 16.5±3.0 mm Hg, P<0.001), more medication changes (P=0.01), greater incidence of glaucomatous progression (P=0.04), and less patients persistent on original monotherapy (P<0.001) than the PTG cohort. CONCLUSIONS: Patients originally on timolol monotherapy have a lower cost of care over 5 years than those started on a PTG. However, timolol patients during follow-up may demonstrate a higher intraocular pressure, more progression, more medication changes, and lower persistency of the original monotherapy.

Cost-effectiveness of treating ocular hypertension.

PURPOSE: To assess the cost-effectiveness of treating ocular hypertension (OHT) in the United States. DESIGN: A Markov model was constructed to perform a cost-effectiveness analysis. PARTICIPANTS: Patients with OHT. METHODS: The health states considered were stable OHT and glaucoma. Practice patterns for the model were derived from the Ocular Hypertension Treatment Study (OHTS), and transition probabilities were derived from previous literature. Specific unit costs used for medications, patient visits, and diagnostic and therapeutic procedures were obtained from Blue Cross/Blue Shield. The time horizon was 5 years. Costs were discounted at 3% per annum. MAIN OUTCOME MEASURE: Long-term cost effectiveness of treating OHT to prevent the development of glaucoma. RESULTS: The incremental cost-effectiveness ratio (ICER) for all OHT patients to prevent 1 case from progressing to primary open-angle glaucoma was $89,072. However, the minimally cost-effective ICER level after adjustment for risk factors identified by multivariate analysis in the OHTS were: 20 years above the average of 56 years, ICER of $45,155; 4 mmHg above the average pressure of 25 mmHg, ICER of $46,748; 40 microm less than the average central corneal thickness of 573 mum, ICER of $36,683; and a vertical cup-to-disc ratio of 0.2 wider than the average of 0.4, ICER of $35,633. CONCLUSIONS: Based on the results and practice patterns of the OHTS, treating all OHT patients seems not to be cost-effective. However, treating selective OHT patients with risk factors identified in the OHTS, for example, advancing age, higher pressures, thinner central corneal thickness, and wider vertical cup-to-disc ratios, does seem to be cost-effective for preventing the onset of glaucomatous damage.